Drug Repurposing News



Martin Pharmaceuticals Receives FDA Orphan Drug Designation for LIVANTRA

March 30th 2018, Posted by: Drug Repurposing Portal

Martin Pharmaceuticals, a clinical stage pharmaceutical company focused on repurposing already-approved drugs to offer life-changing advances to patients afflicted with rare (orphan) diseases or challenging medical conditions, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for LIVANTRA in the treatment of Acute on Chronic Liver Failure.


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Anticancer Agent LENVIMA (lenvatinib mesylate) Approved for Additional Indication of Unresectable Hepatocellular Carcinoma

March 30th 2018, Posted by: Drug Repurposing Portal

LENVIMA, used to treat certain types of thyroid cancers has been approved in Japan for unresectable hepatocellular carcinoma (HCC).This approval was based on a phase 3 clinical study (Study 304/REFLECT study) conducted by Eisai investigating LENVIMA as a first-line treatment in patients with unresectable HCC.


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AI Is Being Put to Work to Treat Parkinson's Disease in the UK

March 29th 2018, Posted by: Drug Repurposing Portal

Europe's largest AI company; Benevolent AI announces its collaboration with two UK charities; Parkinson's UK and The Cure Parkinson's Trust for identifying two novel drug targets for Parkinson's disease treatment by repurposing at least three existing drugs employing five-layer neural network to develop models that can predict the blood brain barrier penetration and other properties of potential drug candidates.


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Findings From Comprehensive Drug Repurposing Screening To Treat Epileptic Encephalopathy Published In Epilepsia

March 28th 2018, Posted by: Drug Repurposing Portal

Pairnomix, LLC published the results from a comprehensive drug repurposing screen performed for a patient with SCN8A epileptic encephalopathy in Epilepsia using a high-throughput screen of generic & marketed drugs and identified 90 drugs with therapeutic benefit against excess sodium influx in in vitro cell models.


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New drug candidates for treatment of atypical meningiomas: An integrated approach using gene expression signatures for drug repurposing

March 23rd 2018, Posted by: Drug Repurposing Portal

Atypical meningiomas are one of the most common cerebral neoplasms with poor prognosis and extremely high recurrence rate compared to their grade I subtypes. The only available therapeutic routes are surgery and radiotherapy, with limited efficacy. In a recent study published in PLoS One, researchers have identified novel drug candidates such as emetine, verteporfin, phenoxybenzamine and trazodone using a computational drug repurposing approach based on gene expression signatures of atypical meningioma tissues, with subsequent ingenuity pathway analysis (IPA) of drug-generated expression profiles. IPA revealed that these drugs target signal cascades such as ERK/MAPK signaling which potentially drive pathogenesis of meningiomas, and warrant further in vivo experimental validation. Beyond proposing already approved drug candidates in the management of atypical meningioma the study highlights the potential of computational techniques in improving disease management strategies.


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Parasite Drug Shows Effectiveness Against Mesothelioma

March 23rd 2018, Posted by: Drug Repurposing Portal

Dr. Antonio Giordano believes his team has cleared the path to the next breakthrough in the treatment of malignant pleural mesothelioma. The advancement involves a new use for an old drug, pyrvinium pamoate, which doctors have successfully used for more than 50 years to treat infections of pinworm parasite. This drug that no one expected to be involved with mesothelioma is potentially a new weapon against this aggressive type of tumor, Giordano told Asbestos.com. Our findings are exciting, very exciting. This one can move forward.


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Identification of small-molecule inhibitors of human Golgi mannosidase via a drug repositioning screen

March 23rd 2018, Posted by: Drug Repurposing Portal

Golgi mannosidases (GM) inhibitor-based therapies have gained significant attention for cancer treatment since they regulate several biological events such as cell-cell communication, differentiation, and apoptosis in cancer cells. However, no GM inhibitor has been approved or is in clinical development for anti-cancer treatment till date. A new study by researchers from Nihon University in Japan performed a drug repositioning screen of a compound and approved drugs library, and found that the estrogen receptor antagonists tamoxifen and raloxifene inhibited human GMs at the cellular level. They also found that the NSAID Sulindac could inhibit GMs.The study thus highlights the importance of drug repositioning and drug screening strategies for identifying new lead compounds for anti-cancer treatment.


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Combination of pyrvinium and paclitaxel could be potential treatment strategy for Renal Cell Carcinoma

March 23rd 2018, Posted by: Drug Repurposing Portal

This study evaluated anti-cancer functions of FDA approved anti-helminthic drug pyrvinium in clear cell renal cell carcinoma (RCC). Pyrvinium inhibited the growth and induced apoptosis in various RCC cell lines. In line, pyrvinium treatment decreased tumor growth and ?-catenin levels clear cell RCC xenograft mouse model. Additionally, combination of pyrvinium and paclitaxel resulted in greater efficacy in in vitro and in vivo. These findings suggest that pyrvinium is a potential addition to the treatment armamentarium for clear cell RCC


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Diabetes Medicine Reduces Liver Fat in Nonalcoholic Fatty Liver Disease

March 22nd 2018, Posted by: Drug Repurposing Portal

In people with type 2 diabetes, nonalcoholic fatty liver disease (NAFLD) is common and can progress to a severe liver disease known as nonalcoholic steatohepatitis (NASH). Now a study has found that empagliflozin, a newer treatment for type 2 diabetes, reduces liver fat in patients with NAFLD and diabetes.


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he prescribable drugs with efficacy in experimental epilepsies (PDE3) database for drug repurposing research in epilepsy

March 20th 2018, Posted by: Drug Repurposing Portal

This study aim was to create a database of ?prescribable? drugs, approved for other conditions, with published evidence of efficacy in animal models of epilepsy, and to collate data that would assist in choosing the most promising candidates for drug repurposing. The database was created by the following: (1) computational literature?mining using novel software that identifies Medline abstracts containing the name of a prescribable drug, a rodent model of epilepsy, and a phrase indicating seizure reduction; then (2) crowdsourced manual curation of the identified abstracts.The final database includes 173 drugs and 500 abstracts. It is made freely available at www.liverpool.ac.uk/D3RE/PDE3. The database is reliable: 94% of the included drugs have corroborative evidence of efficacy in animal models (for example, evidence from multiple independent studies).


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Quinacrine, an Anti-protozoan Drug, is a plausible drug candidate for p53-negative malignancies

March 19th 2018, Posted by: Drug Repurposing Portal

Quinacrine an approved drug with several medical applications. It is commonly used as an antiprotozoal, anthelmintic and in the treatment of giardiasis. Recent studies reported anti-tumor effects of Quinacrine through suppression of NF-?B and activation of p53. In line, the present study demonstrated the anti-cancer effect of Quinacrine via a novel pathway through the elimination of check point kinase 1/2 (Chk1/2) under p53 inactivated conditions both in vitro and in vivo suggesting the potential of Quinacrine repurposing for relapsed cancers, small-cell lung cancer, breast cancer as well as various p53-inactivated human malignancies.


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Calcium channel blockers as drug repurposing candidates for gestational diabetes: Mining large scale genomic and electronic health records data to repurpose medications.

March 17th 2018, Posted by: Drug Repurposing Portal

New therapeutic approaches are needed for gestational diabetes mellitus (GDM). In this study they investigated associations between electronic medical record (EMR) phenotypes and genetic variants to uncover drugs currently considered safe in pregnancy that could treat or prevent GDM. The study identified 129 systemically active drugs considered safe in pregnancy targeting the proteins produced from 196 genes. They also tested for associations between GDM and/or type 2 diabetes (DM2) and 306 SNPs in 130 genes represented on the Illumina Infinium Human Exome Bead Chip (DM2 was included due to shared pathophysiological features with GDM). On the other hand, they tested the association between drugs and glucose tolerance during pregnancy as measured by the glucose recorded during a routine 50-g glucose tolerance test (GTT). They found an association between GDM/DM2 and the genes targeted by 11 drug classes. Thus, CCBs were identified as a class of drugs considered safe in pre gnancy could have efficacy in treating or preventing GDM. 5HT-3 antagonists may be associated with worse glucose tolerance.


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Simvastatin therapy for drug repositioning to reduce the risk of prostate cancer mortality in patients with hyperlipidemia

March 17th 2018, Posted by: Drug Repurposing Portal

Prostate cancer (PCa) is one of the most commonly diagnosed cancers in the western world, and the mortality rate from PCa in Asia has been increasing recently. Statins are potent inhibitors of 3-hydroxy-3-methyl glutaryl coenzyme A (HMG-CoA) reductase and are commonly used for treating hyperlipidemia, with beneficial effects for cardiovascular disease and they also exhibit anti-cancer activity. However, the protective effects of statins against PCa are controversial. In this study, we investigated the effect of two types of statins (simvastatin and lovastatin) and the mortality rate of PCa patients by using the Taiwan National Health Insurance Research Database (NHIRD). A total of 15,264 PCa patients with hyperlipidemia records and medical claims from the Registry of Catastrophic Illness were enrolled. The patients were divided into two cohorts based on their statin use before the diagnosis of PCa: statin users (n = 1,827) and non-statin users (n = 1,826).


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The multitargeted drug ivermectin: from an antiparasitic agent to a repositioned cancer drug

March 17th 2018, Posted by: Drug Repurposing Portal

Drug repositioning is a highly studied alternative strategy to discover and develop anticancer drugs. This drug development approach identifies new indications for existing compounds. Ivermectin belongs to the group of avermectins (AVM), a series of 16-membered macrocyclic lactone compounds discovered in 1967, and FDA-approved for human use in 1987. It has been used by millions of people around the world exhibiting a wide margin of clinical safety. In this review, we summarize the in vitro and in vivo evidences demonstrating that ivermectin exerts antitumor effects in different types of cancer. Ivermectin interacts with several targets including the multidrug resistance protein (MDR), the Akt/mTOR and WNT-TCF pathways, the purinergic receptors, PAK-1 protein, certain cancer-related epigenetic deregulators such as SIN3A and SIN3B, RNA helicase, chloride channel receptors and preferentially target cancer stem-cell like population. Importantly, the in vitro and in vivo antitumor activities of ivermectin are achieved at concentrations that can be clinically reachable based on the human pharmacokinetic studies done in healthy and parasited patients. Thus, existing information on ivermectin could allow its rapid move into clinical trials for cancer patients.


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BTI files for $69M IPO to run trials in Alzheimer's and cancer

March 17th 2018, Posted by: Drug Repurposing Portal

BioXcel Therapeutics (BTI) has filed to raise $69 million through a Nasdaq IPO. BTI will spend the cash on phase 2 trials of a once-failed cancer asset and an old drug repurposed for use in Alzheimer?s and schizophrenia.


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Repurposing of Kinase Inhibitors as Broad-Spectrum Antiviral Drugs

March 16th 2018, Posted by: Drug Repurposing Portal

There is an urgent need for antiviral strategies to combat hundreds of human disease-causing viruses. Currently approved antiviral drugs treat fewer than ten viral infections. A majority of these drugs are direct-acting antivirals (DAAs) that target proteins encoded by individual viruses. As such, this approach provides a narrow spectrum of coverage and therefore cannot address the large clinical need. Targeting host proteins required by multiple viruses can thus provide a broad-spectrum coverage with a possible added benefit of a high genetic barrier to resistance. Moreover, a broad-spectrum therapeutic could be administered even before a viral threat has been accurately diagnosed, thereby increasing protection. Host kinase inhibitors represent one category of compounds with a great potential to be repurposed as broad-spectrum antivirals. Viruses hijack a large number of host kinases at distinct steps of their life cycle (Supekova et al., 2008; Li et al., 2009; Keating a nd Striker, 2012; Jiang et al., 2014). Some of these host kinases are broadly required and thus represent attractive targets for broad-spectrum therapy. These findings, combined with the development and approval of a large number of kinase inhibitors for the treatment of cancer (Gross et al., 2015) and inflammatory conditions (Ott and Adams, 2011) have sparked efforts aimed to determine the therapeutic potential of such drugs to combat viral infections.


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Repurposed arthritis drug could become a non-opioid pain reliever

March 16th 2018, Posted by: Drug Repurposing Portal

Researchers from the Indiana University in the US have found that the CB2 cannabinoid receptor agonist LY2828360, once studied as a possible treatment option for osteoarthritis, might have the potential to be used as a non-opioid pain reliever. In the study performed on mice, the researchers observed that when combined with morphine, the cannabinoid agonist was able to block chemotherapy-induced neuropathic pain with sustained efficacy, and concomitantly attenuated tolerance and dependence towards morphine. Thus, LY2828360 could be repurposed to serve as a first-line treatment in chemotherapy-induced neuropathic pain, and also hold therapeutic promise in other opioid analgesic-refractory neuropathic pains.


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Chondroprotective Effects and Mechanisms of Dextromethorphan: Repurposing Antitussive Medication for Osteoarthritis Treatment.

March 16th 2018, Posted by: Drug Repurposing Portal

Dextromethorphan (DXM), an antitussive medication has been found to be protective in pro-inflammatory cytokine-stimulated chondrocytes and in a collagen-induced arthritis (CIA) animal model. DXM protected the CIA mice from severe inflammation and cartilage destruction by reducing the inflammation-mediated matrix degradation.


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Software Helps Find Drugs That Can be Repurposed to Treat Rare Disease

March 16th 2018, Posted by: Drug Repurposing Portal

Researchers at the LSU Computational Systems Biology group have developed a sophisticated and systematic way to identify existing drugs that can be repositioned to treat a rare disease or condition. They have fine-tuned a computer-assisted drug repositioning process that can save time and money in helping these patients receive effective treatment.


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Healthcare Veterans Launch Martin Pharmaceuticals to Repurpose Drugs for Orphan Indications; Seed Round Oversubscribed

March 16th 2018, Posted by: Drug Repurposing Portal

Two accomplished healthcare executives, David Martin Geliebter and Sven Martin Jacobson, founders of Remedy Pharmaceuticals, announced the launch of Martin Pharmaceuticals, with the mission of repurposing already-approved drugs in order to offer life-changing advances to patients afflicted with rare (orphan) diseases or challenging medical conditions.


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An ancient remedial repurposing: synthesis of new pinocembrin fatty acid acyl derivatives as potential antimicrobial/anti-inflammatory agents

March 16th 2018, Posted by: Drug Repurposing Portal

Five new pinocembrin derivatives (MC1-MC5) synthesized by Steglich reaction were investigated for their antimicrobial, antioxidant, and anti-inflammatory activity in this study. MC2 (oleoyl derivative) and MC3 (linoleoyl derivative) have shown the highest inhibitory effects on bacterial proliferation against Staphylococcus aureus. The docosahexaenoyl derivative MC5 displays the highest anti-inflammatory activity, decreasing nitric oxide production in LPS-stimulated. The introduction of fatty acid substituents improves the biological profile of pinocembrin. Moreover, the chemical nature of substituents significantly affects the bioactivity. These preliminary results outline the importance to investigate the synthesis of pinocembrin fatty acids derivatives as new and safe anti-microbial/anti-inflammatory agents.


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DNA methylation-based classification of central nervous system tumours

March 15th 2018, Posted by: Drug Repurposing Portal

Accurate pathological diagnosis is crucial for optimal management of patients with cancer. For the approximately 100 known tumour types of the central nervous system, standardization of the diagnostic process has been shown to be particularly challenging with substantial inter-observer variability in the histopathological diagnosis of many tumour types. In the article the authors report a comprehensive approach for the DNA methylation-based classification of central nervous system tumours across all entities and age groups, and demonstrate its application in a routine diagnostic setting. They demostrate that the availability of this method may have a substantial impact on diagnostic precision compared to standard methods, resulting in a change of diagnosis in up to 12% of prospective cases. The results provide a blueprint for the generation of machine-learning-based tumour classifiers across other cancer entities, with the potential to fundamentally transform tumour pathology.


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Inventing new therapies without reinventing the wheel: the power of drug repurposing

March 15th 2018, Posted by: Drug Repurposing Portal

Current drug development strategies devote extensive effort to identifying the molecular mechanism of action of new drug candidates. Often, if more than one unrelated enzyme or protein is affected by a certain agent at similar concentrations, the agent is considered to be ?non-specific? (or not specific enough) and thus of questionable therapeutic utility. However, specificity for drugs is surprisingly not only a function of concentration, as thought in pharmacology books, but also a function of time: the more a drug is studied, the more likely it is that additional targets or indications will be discovered. In 1993, the Nobel Laureate Sir John Vane ? who spent his career studying drugs and their modes of action ? said that ?The specificity of drugs decreases over time? (Sir John Vane ? while making this comment to a group of young investigators including one of the authors of this Editorial (C.S.) ? also stated that he believes that this quote originally belongs to another prominent pharmacologist, Sir James Black (also a Nobel Laureate). In spite of extensive internet searches, we were unable to verify the origin of the original quote.). On the one hand, additional targets might mean trouble for a drug candidate under development. On the other hand, new molecular targets and new pharmacological actions may present new therapeutic opportunities for clinically used drugs ? in the framework of therapeutic repurposing.


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A diseasome cluster-based drug repurposing of soluble guanylate cyclase activators from smooth muscle relaxation to direct neuroprotection

March 15th 2018, Posted by: Drug Repurposing Portal

Network medicine utilizes common genetic origins, markers and co-morbidities to uncover mechanistic links between diseases. These links can be summarized in the diseasome, a comprehensive network of disease?disease relationships and clusters. The diseasome has been influential during the past decade, although most of its links are not followed up experimentally. Here, the authors' team investigate a high prevalence unmet medical need cluster of disease phenotypes linked to cyclic GMP. Hitherto, the central cGMP-forming enzyme, soluble guanylate cyclase (sGC), has been targeted pharmacologically exclusively for smooth muscle modulation in cardiology and pulmonology. Here, we examine the disease associations of sGC in a non-hypothesis based manner in order to identify possibly previously unrecognized clinical indications. Surprisingly, they found that sGC, is closest linked to neurological disorders, an application that has so far not been explored clinically. Indeed, when investigating the neurological indication of this cluster with the highest unmet medical need, ischemic stroke, pre-clinically they found that sGC activity is virtually absent post-stroke. Conversely, a heme-free form of sGC, apo-sGC, was now the predominant isoform suggesting it may be a mechanism-based target in stroke. Indeed, this repurposing hypothesis could be validated experimentally in vivo as specific activators of apo-sGC were directly neuroprotective, reduced infarct size and increased survival. Thus, common mechanism clusters of the diseasome allow direct drug repurposing across previously unrelated disease phenotypes redefining them in a mechanism-based manner. Specifically, our example of repurposing apo-sGC activators for ischemic stroke should be urgently validated clinically as a possible first-in-class neuroprotective therapy.


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High-throughput screen of drug repurposing library identifies inhibitors of Sarcocystis neurona growth

March 15th 2018, Posted by: Drug Repurposing Portal

The apicomplexan parasite Sarcocystis neurona is the primary etiologic agent of equine protozoal myeloencephalitis (EPM), a serious neurologic disease of horses. Many horses in the U.S. are at risk of developing EPM; approximately 50% of all horses in the U.S. have been exposed to S. neurona and treatments for EPM are 60?70% effective. Advancement of treatment requires new technology to identify new drugs for EPM. To address this critical need, the researchers developed, validated, and implemented a high-throughput screen to test 725 FDA-approved compounds from the NIH clinical collections library for anti-S. neurona activity. Their screen identified 18 compounds with confirmed inhibitory activity against S. neurona growth, including compounds active in the nM concentration range. Many identified inhibitory compounds have well-defined mechanisms of action, making them useful tools to study parasite biology in addition to being potential therapeutic agents. In comparing the activity of inhibitory compounds identified by their screen to that of other screens against other apicomplexan parasites, we found that most compounds (15/18; 83%) have activity against one or more related apicomplexans. Interestingly, nearly half (44%; 8/18) of the inhibitory compounds have reported activity against dopamine receptors. They also found that dantrolene, a compound already formulated for horses with a peak plasma concentration of 37.8???12.8?ng/ml after 500?mg dose, inhibits S. neurona parasites at low concentrations (0.065??M [0.036?0.12; 95% CI] or 21.9?ng/ml [12.1?40.3; 95% CI]). These studies demonstrate the use of a new tool for discovering new chemotherapeutic agents for EPM and potentially providing new reagents to elucidate biologic pathways required for successful S. neurona infection.


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Use of Computational Functional Genomics in Drug Discovery and Repurposing for Analgesic Indications

March 15th 2018, Posted by: Drug Repurposing Portal

The novel research area of functional genomics investigates biochemical, cellular, or physiological properties of gene products with the goal of understanding the relationship between the genome and the phenotype. These developments have made analgesic drug research a data-rich discipline mastered only by making use of parallel developments in computer science, including the establishment of knowledge bases, mining methods for big data, machine-learning, and artificial intelligence.


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A novel computational approach for drug repurposing using systems biology

March 15th 2018, Posted by: Drug Repurposing Portal

Identification of novel therapeutic effects for existing U.S. Food and Drug Administration (FDA)-approved drugs, drug repurposing, is an approach aimed to dramatically shorten the drug discovery process, which is costly, slow and risky. Several computational approaches use transcriptional data to find potential repurposing candidates. The main hypothesis of such approaches is that if gene expression signature of a particular drug is opposite to the gene expression signature of a disease, that drug may have a potential therapeutic effect on the disease. However, this may not be optimal since it fails to consider the different roles of genes and their dependencies at the system level.


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Drug repurposing: In-vitro anti-glycation properties of 18 common drugs

March 15th 2018, Posted by: Drug Repurposing Portal

Article Authors Metrics Comments Related Content Abstract Introduction Materials and methods Results and discussion Conclusion Supporting information Acknowledgments References Reader Comments (0) Media Coverage (0) Figures Abstract Drug repositioning or repurposing, i.e. identifying new indications for existing drugs, has gained increasing attention in the recent years. This approach enables the scientists to discover ?new targets? for known drugs in a cost and time efficient manner. Glycation, the non-enzymatic reaction of sugars with proteins or nucleic acids to form early glycation (Amadori or fructosamine) products, is a key molecular basis of diabetic complications. Inhibiting the process of non-enzymatic protein glycation is one of the key strategies to prevent glycation-mediated diabetic complications. The present study focuses on the anti-glycation activity of 18 drugs, commonly used for the treatment of gastrointestinal, central nervous system, inflammatory diseases, bacterial infections, and gout.


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Ibandronate metal complexes: solution behavior and antiparasitic activity

March 14th 2018, Posted by: Drug Repurposing Portal

Bisphosphonates that are clinically used for bone diseases have been studied as agents against Trypanosoma cruzi, causative parasite of Chagas disease.


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Chemotherapeutic potential of 17-AAG against cutaneous leishmaniasis caused by Leishmania (Viannia) braziliensis.

March 14th 2018, Posted by: Drug Repurposing Portal

Previously, we showed that 17-(allylamino)-17-demethoxygeldanamycin (17-AAG), a Heat Shock Protein 90 (HSP90)-specific inhibitor, reduces L. (L.) amazonensis infection in vitro. Herein, we expand the current knowledge on the leishmanicidal activity of 17-AAG against cutaneous leishmaniasis, employing an experimental model of infection with L. (V.) braziliensis.


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The existing drug vorinostat as a new lead against cryptosporidiosis by targeting the parasite histone deacetylases.

March 14th 2018, Posted by: Drug Repurposing Portal

Fifteen (15) compounds exhibited anti-cryptosporidial activity at nanomolar level in vitro. Among them, the histone deacetylase (HDAC) inhibitor vorinostat retained outstanding efficacy in vitro (EC50 = 203 nM) and in IL-12 knockout mouse model (ID50 = 7.5 mg/kg). Vorinostat was effective on various parasite developmental stages, and could irreversibly kill the parasite. Vorinostat was highly effective against the parasite native HDAC enzymes (IC50 = 90.0 nM) and a recombinant C. parvum HDAC (Ki = 123.0 nM).


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Repurposed Leukemia Drugs Could Ward Off Melanoma Metastasis

March 14th 2018, Posted by: Drug Repurposing Portal

Study published in Science Signaling has showed new evidence linking the activation of ABL kinases - cancer-promoting genes - to the secretion of pro-metastatic cathepsins in melanoma. The Cathepsins are enzymes that degrade proteins and are highly expressed in cancer cells, resulting in their release into the environment between the cells. These enzymes "chew up" the fibrous matrix around tumors, which allows them to get into the blood stream and lymphatic system and spread around the body. Their work showed that ABL kinases induce cathepsin expression and secretion by increasing the activity of key transcription factors that upregulate numerous proteins involved in metastasis. Transcription factors bind to the regulatory part of genes and induce their expression. This study is the first to demonstrate that ABL kinases not only increase the abundance of the transcription factors, but also regulate the ability of these transcription factors to bind to the promoters and induce gene expression. The researchers found that ABL kinases inhibitors already approved by the Food & Drug Administration (FDA) for treating leukemia also prevented metastasis induced by secreted cathepsins in animal models of metastatic melanoma


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Autism: Anti-cancer drug may improve social behavior

March 14th 2018, Posted by: Drug Repurposing Portal

Investigators at the State University of New York at Buffalo reveal that Romidepsin an anti-cancer drug may be able to reverse social impairments associated with autism.


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FoxO3a mediates the inhibitory effects of the antiepileptic drug Lamotrigine on breast cancer growth

March 14th 2018, Posted by: Drug Repurposing Portal

Researchers at the University of Calabria show that Lamotrigine (LTG), a broadly used anticonvulsivant, could be "repurposed" as an antitumoral drug in Breast Cancer. They show that LTG inhibits the proliferation, the anchorage-dependent and independent cell growth in BC cells (BCCs), including hormone-resistant cell models.


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Using a novel computational drug-repositioning approach (DrugPredict) to rapidly identify potent drug candidates for cancer treatment

March 9th 2018, Posted by: Drug Repurposing Portal

Computation-based drug-repurposing/repositioning approaches can greatly speed up the traditional drug discovery process. To date, systematic and comprehensive computation-based approaches to identify and validate drug-repositioning candidates for epithelial ovarian cancer (EOC) have not been undertaken. Here, we present a novel drug discovery strategy that combines a computational drug-repositioning system (DrugPredict) with biological testing in cell lines in order to rapidly identify novel drug candidates for EOC.


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Computational drug repositioning using low-rank matrix approximation and randomized algorithms

March 9th 2018, Posted by: Drug Repurposing Portal

Computational drug repositioning is an important and efficient approach towards identifying novel treatments for diseases in drug discovery. The emergence of large-scale, heterogeneous biological and biomedical datasets has provided an unprecedented opportunity for developing computational drug repositioning methods. The drug repositioning problem can be modeled as a recommendation system that recommends novel treatments based on known drug?disease associations. The formulation under this recommendation system is matrix completion, assuming that the hidden factors contributing to drug?disease associations are highly correlated and thus the corresponding data matrix is low-rank.


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Indian scientists find asthma drug to be useful against tuberculosis as well

March 9th 2018, Posted by: Drug Repurposing Portal

Scientists at the Indian Institute of Science in Bangalore have found that an existing anti-asthma drug is effective against tuberculosis and can help address the problem of drug resistance as well. The drug targets a unique arginine biosynthesis enzyme which is exclusive to Mtb, thereby impeding its arginine production. It also targets the pathogen pro-survival pathways in the host, directly reducing intracellular survival of the TB bacteria.


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Repurposed Parasite Drug New Weapon Against Mesothelioma

March 9th 2018, Posted by: Drug Repurposing Portal

Anthelmintic drug pyrvinium pamoate, already approved to treat infections of pinworm parasite was shown to effectively impair both mesothelioma cell growth and migration. Researchers found that the drug affected the expression of downstream genes in the WNT signaling pathway, which are implicated in mesothelioma aggressiveness and its resistance to conventional therapy.


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LifeArc Announces ?5M Support for Rare Disease Research

March 9th 2018, Posted by: Drug Repurposing Portal

LifeArc, the medical research charity, today announced its Philanthropic Fund will award ?5M in grants to support medical research projects focused on rare diseases. The grants scheme was announced at the Drug Repurposing for Rare Diseases Conference 2018.


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gene2drug: a computational tool for pathway-based rational drug repositioning

February 26th 2018, Posted by: Drug Repurposing Portal

Drug repositioning has been proposed as an effective shortcut to drug discovery. The availability of large collections of transcriptional responses to drugs enables computational approaches to drug repositioning directly based on measured molecular effects. We introduce a novel computational methodology for rational drug repositioning, which exploits the transcriptional responses following treatment with small molecule. Specifically, given a therapeutic target gene, a prioritisation of potential effective drugs is obtained by assessing their impact on the transcription of genes in the pathway(s) including the target. We performed in silico validation and comparison with a state-of-art technique based on similar principles.


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Repurposing probenecid for the Treatment of Heart Failure

February 23rd 2018, Posted by: Drug Repurposing Portal

Probenecid, an FDA approved drug for the treatment of gout and hyperuricemia is recently been in phase II clinical trials for the treatment of heart failure. In their recent study, Robbins and coworkers revealed that probenecid increased the calcium sensitivity of cardiomyocytes likely through the stimulation of cardiac TRPV2 channels. In line, probenecid also improved the heart-pumping function in heart failure patients with reduced ejection fraction


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Calcium channel blockers as potential repurposing candidates for gestational diabetes mellitus

February 23rd 2018, Posted by: Drug Repurposing Portal

The incidence of gestational diabetes mellitus (GDM) continues to rise suggesting for development of novel therapeutic strategies. However, the usual target-driven drug discovery is complicated in pregnancy due to associated risk factors. To overcome this drawback, the authors of the present study, proposed a new treatment strategy from the associations between electronic medical record (EMR) phenotypes and GDM genetic variants. Drugs considered safe in pregnancy are sorted from EMR and corresponding target genes associated with GDM and type 2 diabetes are identified. In a parallel analysis, authors have shown that L-type calcium channel blocking antihypertensives (CCBs) were associated with a decrease in glucose during glucose tolerance test. These findings demonstrate the repurposing potential of calcium channel blockers for gestational diabetes mellitus treatment.


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Cambridge biotech startup seeking to repurpose Vioxx raises $5M

February 21st 2018, Posted by: Drug Repurposing Portal

Tremeau Pharmaceuticals, a Cambridge biotech startup that is seeking to repurpose the controversial Merck & Co. arthritis drug Vioxx as a treatment for joint pain caused by hemophilia, has closed its first equity round for USD 5Mn.\nTremeau has already received so-called orphan drug designation from the FDA, which reduces the cost of developing treatments for rare diseases that affect fewer than 200,000 people in the U.S.


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Repurposed Drug Hydroxyurea May Improve Glioblastoma's Response to Chemotherapy

January 24th 2018, Posted by: Drug Repurposing Portal

Glioblastoma (GBM) is the most common and most aggressive type of brain tumors in adults. Over the last two decades, the major improvement in the treatment for GBM has been the addition of the chemotherapeutic temozolomide (TMZ) to the standard of care (surgery and radiation), however, despite this aggressive therapy, over 90% of patients die within five years after diagnosis. Combining FDA-approved drug hydroxyurea with TMZ for the treatment of GBM could be highly beneficial for these patients, which could lead to an increase in their survival rate.


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One gene links two different diseases: A stretch of DNA is implicated in both Crohn's and Parkinson's diseases

January 24th 2018, Posted by: Drug Repurposing Portal

Inga Peter of the Icahn School of Medicine at Mount Sinai in New York City and her colleagues analysed gene sequences in more than 2,000 people with Crohn's disease and 3,600 individuals without the condition, all of whom were of Ashkenazi Jewish descent. The team found a link between Crohn's disease and a particular DNA sequence in a gene called LRRK2. The variant DNA causes the LRRK2 protein to become more active than the typical protein — as does a Parkinson's disease-associated variant in the same part of the protein.


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Protein Analysis Enables Treatment of Eye-Disease Symptoms with Existing Drugs

January 9th 2018, Posted by: Drug Repurposing Portal

University School of Medicine has matched existing drugs to errant proteins expressed by patients with a rare eye disease. “Analyzing fluid samples from the eye can totally change how we treat patients,” said Vinit Mahajan, MD, PhD, associate professor of ophthalmology. The team employed proteomics, the large-scale study of proteins, in identifying four on-the-market drugs that successfully quelled symptoms triggered by several of the overabundant proteins.


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Anti-cryptosporidial activity of anti-neoplastic drug vorinostat

January 8th 2018, Posted by: Drug Repurposing Portal

Cryptosporidiosis is an intestinal disease caused by the microsopic parasite Cryptosporidium that can cause life-threatening disease in individuals with weak immune system. The existing therapeutic options for cryptosporidiosis are very limited and symptomatic. Recently published study exhibited anti-cryptosporidial activity of histone deacetylase (HDAC) inhibitor vorinostat at nanomolar level in vitro. The study also demonstrated irreversible killing of the parasite by vorinostat by inhibiting the parasite at different developmental stages via targeting parasite?s HDAC enzymes. These data suggest the potential for repurposing of vorinostat to treat cryptosporidiosis.


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Anti-cryptosporidial activity of anti-neoplastic drug vorinostat

January 8th 2018, Posted by: Drug Repurposing Portal

Cryptosporidiosis is an intestinal disease caused by the microsopic parasite Cryptosporidium that can cause life-threatening disease in individuals with weak immune system. The existing therapeutic options for cryptosporidiosis are very limited and symptomatic. Recently published study exhibited anti-cryptosporidial activity of histone deacetylase (HDAC) inhibitor vorinostat at nanomolar level in vitro. The study also demonstrated irreversible killing of the parasite by vorinostat by inhibiting the parasite at different developmental stages via targeting parasite’s HDAC enzymes. These data suggest the potential for repurposing of vorinostat to treat cryptosporidiosis.


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Scancell buddies up with 'new immuno-oncology powerhouse'

January 8th 2018, Posted by: Drug Repurposing Portal

UK-based oncology specialist Scancell Holdings (LSE: SCLP) has announced a collaboration with Europe's largest privately-held biopharmaceutical company BioNTech. The collaboration will focus on the potential development of innovative, T-cell receptor based therapeutics for the treatment of cancer. Scancell and BioNTech will seek to discover and characterize T-cell receptors specific for citrullinated epitopes from vimentin and enolase. The technology overcomes the immune suppression induced by tumors themselves without the need for checkpoint blockade inhibitors, thereby allowing activated T-cells to seek out and kill tumor cells that would otherwise be hidden from the immune system. Pre-clinical data from this collaboration has shown unprecedented anti-tumor effects can be delivered without the need for checkpoint inhibition.


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Novel diabetes drugs sensitize cancer cells to chemotherapy agents

January 4th 2018, Posted by: Drug Repurposing Portal

A recent study published in Proceedings of the National Academy of Sciences by scientists at the Dana-Farber Cancer Institute highlights the efficacy of experimental anti-diabetic drugs, similar to thiazolidinediones (TZDs), in sensitizing lung cancer cells to conventional chemotherapeutic agents such as carboplatin. This effect was also reproducible in triple-negative breast cancer cells, and resulted in their self-destruction. The molecular mechanism of action involved phosphorylation of PPAR-gamma, a receptor essential for fat cell development, and a target of the TZD class of anti-diabetic agents. Coincidentally, PPAR-gamma is dysregulated in various cancers such as lung, triple-negative breast, colorectal, and pancreatic cancers. Thus, repurposing diabetes drugs in combination with traditional chemotherapeutic agents for various malignancies could potentially improve clinical outcomes in cancer patients.


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Repurposing anticonvulsants for treatment of injury-induced osteoarthritis

January 4th 2018, Posted by: Drug Repurposing Portal

Drugs designed for the treatment of central nervous system diseases could also be used to prevent the development of injury-induced arthritis, potentially saving the NHS around half a billion pounds a year


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KAIST team develops technology to find optimum drug target for cancer

January 4th 2018, Posted by: Drug Repurposing Portal

A technology using systems biology approach for identifying the optimum drug target based on cancer cell type was developed by KAIST research team led by Professor Kwang-Hyun Cho of the Department of Bio and Brain Engineering. Large-scale cancer cell genomic data from The Cancer Cell Line Encyclopedia (CCLE) was used to construct different molecular networks specific to the characteristics of genetic variations that aid in predicting the drug resistance in cancer cells. The team suggest that the new technology can be used in drug repositioning via identifying optimum drug targets.


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Diabetes Drug 'Significantly Reverses' Memory Loss in Mice with Alzheimer's

January 3rd 2018, Posted by: Drug Repurposing Portal

A drug developed for diabetes could be used to treat Alzheimer's after scientists found it "significantly reversed memory loss" in mice through a triple method of action.This is the first time that a triple receptor drug has been used which acts in multiple ways to protect the brain from degeneration. It combines GLP-1, GIP and Glucagon which are all growth factors.The study used APP/PS1 mice, which are transgenic mice that express human mutated genes that cause Alzheimer's. Those genes have been found in people who have a form of Alzheimer's that can be inherited. Aged transgenic mice in the advanced stages of neurodegeneration were treated.Although the benefits of these 'triple agonist' drugs have so far only been found in mice, other studies with existing diabetes drugs such as liraglutide have shown real promise for people with Alzheimer's, so further development of this work is crucial.


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Drug repurposing for the treatment of glioblastoma multiforme

January 3rd 2018, Posted by: Drug Repurposing Portal

Glioblastoma Multiforme is the deadliest type of brain tumor and is characterized by very poor prognosis with a limited overall survival. Current optimal therapeutic approach has essentially remained unchanged for more than a decade, consisting in maximal surgical resection followed by radiotherapy plus temozolomide.Needless to say, in order to efficiently repurpose drugs that are already approved for human use, a careful selection is required, followed by thorough demonstration of their effectiveness in other biological contexts. We will now discuss some methods useful for selection of effective testing and repurposing of drugs in cancer therapy.


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An appeal for help in the fight against MS

December 20th 2017, Posted by: Drug Repurposing Portal

This Christmas, the MS Society?s annual appeal is seeking to raise ?250,000 and fund three landmark trials designed to turn existing drugs into treatments for MS. We do this because, by repurposing existing drugs, we can speed up the clinical trials process and get new MS treatments out there much faster.


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Drug Discovery Could Accelerate Hugely with Machine Learning

December 20th 2017, Posted by: Drug Repurposing Portal

The algorithm?partly devised by Dr. James Kermode from Warwick?s School of Engineering?can accurately predict the interactions between a protein and a drug molecule based on a handful of reference experiments or simulations.\nUsing just a few training references, it can predict whether or not a candidate drug molecule will bind to a target protein with 99 percent accuracy. The algorithm can also tackle materials-science problems such as modeling the subtle properties of silicon surfaces, and promises to revolutionize materials and chemical modeling?giving insight into the nature of intermolecular forces. The design of this algorithm, which combines local information from the neighborhood of each atom in a structure, makes it applicable across many different classes of chemical, materials science, and biochemical problems. The research illustrates how chemical and materials discovery is now benefitting from the Machine Learning and Artificial Intelligence approache s that already underlie technologies from self-driving cars to go-playing bots and automated medical diagnostics.\nNew algorithms allow us to predict the behavior of new materials and molecules with great accuracy and little computational effort, saving time and money in the process.


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Tapeworm Drug Could Lead the Fight Against Parkinson\'s Disease

December 19th 2017, Posted by: Drug Repurposing Portal

Researchers at Cardiff University, in collaboration with the University of Dundee, have identified a drug molecule (Niclosamide) used to treat tapeworm infections which could lead to new treatments for patients with Parkinson\'s disease.Several studies have suggested that discovering a drug which is capable of enhancing the function of PINK1 could be a significant step in halting neurodegeneration and therefore slow down or even treat Parkinson\'s disease.Researchers at Cardiff and Dundee Universities have discovered that is an effective activator of the PINK1 protein.Now these findings to the next level by evaluating the ability of Niclosamide to treat Parkinson\'s disease in disease models.


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Large-Scale Off-Target Identification Using Fast and Accurate Dual Regularized One-Class Collaborative Filtering and Its Application to Drug Repurposing

December 18th 2017, Posted by: Drug Repurposing Portal

Target-based screening is one of the major approaches in drug discovery. Besides the intended target, unexpected drug off-target interactions often occur, and many of them have not been recognized and characterized. The off-target interactions can be responsible for either therapeutic or side effects. Thus, identifying the genome-wide off-targets of lead compounds or existing drugs will be critical for designing effective and safe drugs, and providing new opportunities for drug repurposing. Although many computational methods have been developed to predict drug-target interactions, they are either less accurate than the one that we are proposing here or computationally too intensive, thereby limiting their capability for large-scale off-target identification. In addition, the performances of most machine learning based algorithms have been mainly evaluated to predict off-target interactions in the same gene family for hundreds of chemicals. It is not clear how these algor ithms perform in terms of detecting off-targets across gene families on a proteome scale. Here, we are presenting a fast and accurate off-target prediction method, REMAP, which is based on a dual regularized one-class collaborative filtering algorithm, to explore continuous chemical space, protein space, and their interactome on a large scale. When tested in a reliable, extensive, and cross-gene family benchmark, REMAP outperforms the state-of-the-art methods. Furthermore, REMAP is highly scalable. It can screen a dataset of 200 thousands chemicals against 20 thousands proteins within 2 hours. Using the reconstructed genome-wide target profile as the fingerprint of a chemical compound, we predicted that seven FDA-approved drugs can be repurposed as novel anti-cancer therapies. The anti-cancer activity of six of them is supported by experimental evidences. Thus, REMAP is a valuable addition to the existing in silico toolbox for drug target identification, drug repurposing, ph enotypic screening, and side effect prediction.


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Structure-based identification of a NEDD8-activating enzyme inhibitor via drug repurposing

December 18th 2017, Posted by: Drug Repurposing Portal

NEDD8-activating enzyme (NAE) is an important part of the NEDD8 conjugation pathway which regulates protein degradation. Meanwhile, drug repurposing is one such technique which is cost-efficient to identify new therapeutic uses for existing scaffolds. In this article, mitoxantrone (1) was repurposed as an inhibitor of NAE by virtual screening of an FDA-approved drug database. Compound 1 inhibited NAE activity in cell-free and cell-based systems with high selectivity and was competitive with ATP. Furthermore, compound 1 induced apoptosis of colorectal adenocarcinoma cancer cells through inhibiting the degradation of the neddylation substrate p53.


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Repurposing of the anti-malaria drug chloroquine for Zika Virus treatment and prophylaxis

December 18th 2017, Posted by: Drug Repurposing Portal

One of the major challenges of the current Zika virus (ZIKV) epidemic is to stop congenital foetal abnormalities, including microcephaly, following ZIKV infection of pregnant women. Given the urgent need for ZIKV prophylaxis and treatment, repurposing of approved drugs appears to be a viable and immediate solution. We demonstrate that the common anti-malaria drug chloroquine (CQ) increases the lifespan of ZIKV-infected interferon signalling-deficient AG129 mice. However, the severity of ZIKV infection in these mice precludes the study of foetal (vertical) viral transmission. Here, we show that interferon signalling-competent SJL mice support chronic ZIKV infection. Infected dams and sires are both able to transmit ZIKV to the offspring, making this an ideal model for in vivo validation of compounds shown to suppress ZIKV in cell culture. Administration of CQ to ZIKV-infected pregnant SJL mice during mid-late gestation significantly attenuated vertical transmission, reduci ng the ZIKV load in the foetal brain more than 20-fold. Given the limited side effects of CQ, its lack of contraindications in pregnant women, and its worldwide availability and low cost, we suggest that CQ could be considered for the treatment and prophylaxis of ZIKV.


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gene2drug: a Computational Tool for Pathway-based Rational Drug Repositioning.

December 15th 2017, Posted by: Drug Repurposing Portal

A novel computational methodology for rational drug repositioning, which exploits the transcriptional responses following treatment with small molecule. Specifically, given a therapeutic target gene, a prioritization of potential effective drugs is obtained by assessing their impact on the transcription of genes in the pathway(s) including the target. (Available @ http://gene2drug.tigem.it)


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Repositioning CEP-1347, a chemical agent originally developed for the treatment of Parkinson's disease, as an anti-cancer stem cell drug.

December 15th 2017, Posted by: Drug Repurposing Portal

CEP-1347 is a promising candidate for cancer stem cell-targeting therapy. In vitro, CEP-1347 efficiently induced differentiation and inhibited the self-renewal and tumor-initiating capacities of human cancer stem cells from glioblastoma as well as from pancreatic and ovarian cancers at clinically-relevant concentrations, without impairing the viability of normal fibroblasts and neural stem cells. CEP-1347 is a mixed lineage kinase inhibitor tested in a large-scale phase 2/3 clinical trial in early Parkinson's disease.


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DrugPredict - a novel computational drug-repositioning approach to identify new drug candidates

December 15th 2017, Posted by: Drug Repurposing Portal

Researchers at the Case Western Reserve University have developed a new online drug repositioning, discovery and development tool that aids a user in browsing and in rapidly identifying a database of available FDA-approved small molecule drugs for new therapeutic applications. The system uses a predictive analysis approach to provide calculated probability values of the known mechanisms of action, clinical efficacy, and side effects of existing drugs in specific diseases. Applying the functionality of this tool to real-world issues, the researchers published their findings in Oncogene (PMID: 28967908) where they showed that common non-steroidal anti-inflammatory drugs (NSAIDs) such as aspirin were effective in eliminating patient-derived epithelial ovarian cancer cells. DrugPredict might hence be one among several drug repurposing tools that will aid in the quick identification of new medical uses of old drugs.


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Repurposing Drugs in Oncology (ReDO)-chloroquine and hydroxychloroquine as anti-cancer agents.

December 15th 2017, Posted by: Drug Repurposing Portal

Chloroquine (CQ) and hydroxychloroquine (HCQ) are well-known 4-aminoquinoline antimalarial agents. Scientific evidence also supports the use of CQ and HCQ in the treatment of cancer. Preclinical studies support CQ and HCQ use in anti-cancer therapy, especially in combination with conventional anti-cancer treatments since they are able to sensitize tumour cells to a variety of drugs, potentiating the therapeutic activity. Interestingly, CQ and HCQ exert effects both on cancer cells and on the tumour microenvironment.


News Methods Funding Collaborations

Structure-based identification of a NEDD8-activating enzyme inhibitor via drug repurposing.

December 15th 2017, Posted by: Drug Repurposing Portal

NEDD8-activating enzyme (NAE) is an essential player of the NEDD8 conjugation pathway that regulates protein degradation. Authors reported that Mitoxantrone was repurposed as an inhibitor of NAE by virtual screening of an FDA-approved drug database and inhibited NAE activity in cell-free and cell-based systems with high selectivity. Furthermore Mitoxantrone also induced apoptosis of colorectal adenocarcinoma cancer cells.


News Methods Funding Collaborations

Anti-parasite drug, nitazoxanide could be potential therapy for prostate cancer

December 14th 2017, Posted by: Drug Repurposing Portal

Researchers at the University of Bergen found anti-cancer activity of the parasitic drug nitazoxanide during multidrug screening. They demonstrated that nitazoxanide inhibited cancer cell growth by degradation of the protein beta-catenin. These data suggest for the possibility of repurposing the anti-parasite drug nitazoxanide for prostate cancer treatment.


News Methods Funding Collaborations

Antimalarial drug chloroquine could be repurposed to treat Zika virus infection

December 14th 2017, Posted by: Drug Repurposing Portal

Researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) and UC San Diego School of Medicine during their investigations treated Zika infected pregnant mice with chloroquine and found that chloroquine markedly decreased the viral load in maternal blood and neural progenitor cells in the fetal brain. These data demonstrate a new use for the long standing drug chloroquine for targeting Zika virus infection.


News Methods Funding Collaborations

Anti-diabetic drug Metformin can reverse and prevent the onset of multi-drug resistance in breast cancer

December 14th 2017, Posted by: Drug Repurposing Portal

A new study published in PLOS One indicates that the diabetes wonder drug Metformin can potentially inhibit the development of multi-drug resistance (MDR) in the breast cancer cells. Recent clinical findings underscore the importance of the anti-proliferation effect of metformin in multiple cancer sub-types. The authors observed that pretreatment of MCF7 breast cancer cells with metformin prevented or delayed the development of drug resistance. Moreover, metformin was also found to reverse MDR-associated protein markers after the onset of drug resistance in aggressive breast cancer in mouse models. More research is required to assess the short- and long-term sustained effects of metformin in neoplastic cells.


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Researchers Show Aspirin Added to Cancer Drug Improves Effectiveness

December 14th 2017, Posted by: Drug Repurposing Portal

UQ Diamantina Institute researcher Associate Professor Helmut Schaider said cancers driven by mutations in a group of genes, known as RAS, had a low response to treatments with currently no drug directly targeting them. They found that the addition of aspirin to a cancer inhibitor drug, Sorafenib, strongly enhanced its effectiveness against mouse models of lung cancer and melanoma with RAS mutations.scientists are hoping clinical trials could soon be underway for people with lung, pancreatic and colorectal cancers that have not responded to other therapies.


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How cancer could be treated with an old alcoholism drug

December 14th 2017, Posted by: Drug Repurposing Portal

Previous studies have demonstrated that the alcohol abuse drug disulfiram has anticancer properties. But until now, researchers had not found the mechanism by which the drug can target cancer. New research sheds light, paving the way for the repurposing of the drug


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Copaxone, an old drug for Multiple Sclerosis, is an intriguing new therapeutic tool to fight antibiotic resistance

December 14th 2017, Posted by: Drug Repurposing Portal

Giving new life to old drugs is the need of the hour to fight several diseases and infections. One of the latest breakthroughs in the battle against superbugs comes in the form of Glatiramer Acetate or Copaxone, a drug used over two decades to treat Multiple Sclerosis. A team of researchers led by Thomas Vorup-Jensen from the department of biomedicine at Aarhus University, Denmark found copaxone to have huge potential in killing antibiotic-resistant gram negative bacteria within minutes.The study published in Nature highlights that the chemical properties of copaxone's proteins are highly similar to antimicrobial peptides, as a result of which, the drug easily eliminated the infectious gram negative bacteria E.coli and Pseudomonas Aeruginosa. The researchers studied this property of copaxone in patients with Cyctic Fibrosis, whose lungs cannot clear mucus due to infestation with Pseudomonas bacteria, and found remarkable therapeutic benefits. The study hence underscored that in the frantic fight against antibiotic-resistance, repurposing old drugs such as copaxone could help in finding cures for difficult-to-treat medical ailments.


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New-age cancer drug Nivolumab has the potential to eradicate HIV infections in patients

December 14th 2017, Posted by: Drug Repurposing Portal

Nivolumab (trade name Opdivo), a new generation drug used in the targeted therapy of cancers such as metastatic melanoma, squamous non-small cell lung cancer, and renal cell carcinoma could potentially treat millions of HIV-positive patients. According to the findings of a study in the Annals of Oncology on a 51 year old HIV-positive man being treated for lung cancer, it was found that nivolumab reduced the reservoir of latent HIV-infected cells and boosted the immunity in the patient by increasing the activity of CD8 killer T-cells. While the study requires more validation in other patients, it does offer the hope that regardless of the presence of an underlying malignancy, a stand-alone cure for HIV infections and diseases is possible in the near future.


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A new hope for COPD patients - Heparin to the rescue?

December 14th 2017, Posted by: Drug Repurposing Portal

Chronic obstructive pulmonary disease (COPD) is a mounting healthcare issue due to the limited availability of new cures for it, thereby accelerating its progressive impact on proper lung functioning and causing early deaths. A preliminary study conducted at the University of Portsmouth claims that an inhaled nebulized unfractionated form of common blood thinner, heparin, can potentially improve the symptoms of several respiratory diseases like COPD and cystic fibrosis. Further clinical evaluation is thus required to prove the long-term safety and efficacy of inhaled heparin, and hence its utility as a repurposed drug in patients with moderate-to-severe COPD.


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Chloroquine and Hydroxychloroqunine can be reprofiled to treat cancers

December 14th 2017, Posted by: Drug Repurposing Portal

Chloroquine (CQ) and Hydroxychloroquine (HCQ) are generic drugs that have been used over six decades as anti-malarial agents, and have subsequently also found use in treating lupus and rheumatoid arthritis. As part of the ongoing Repurposing Drugs in Oncology (ReDO) endeavors, new scientific studies have found that both CQ and HCQ may be used as anti-cancer agents, especially in combination with multiple conventional cancer therapies. CQ and HCQ exert their manifold anti-tumorigenic effects on both the tumor and its microenvironment, and deserve further clinical evaluation to be acknowledged as repurposed drugs.


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Bexarotene, an existing cancer drug, can potentially treat Huntington’s disease

December 12th 2017, Posted by: Drug Repurposing Portal

Huntington's disease is a fatal inherited disease that progressively results in neuronal degeneration in the brain, with no sure-shot medication to slow or stop its symptoms. There may however be hope in the form of Bexarotene, a US-FDA approved anti-neoplastic agent used for treating certain forms of lung cancer, breast cancer, cutaneous T-cell lymphoma, and Kaposi's sarcoma. As per the study published in the latest issue of Science Translational Medicine, Bexarotene and a drug similar in action and function, KD3010, activate the transcription factor PPARγ, which in turn improves mitochondrial health in neurons and enhances removal of damaged misfolded proteins as observed in a mouse model of Huntington's disease. Further validation is required in patients with Huntington's to determine and optimize the efficacy, dosage, and neuroprotective role(s) of Bexarotene.


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The gastric acid suppressant Lansoprazole may target Tuberculosis

December 12th 2017, Posted by: Drug Repurposing Portal

The proton pump inhibitor (PPI) Lansoprazole, also called Prevacid, is an oral molecule commonly used for suppressing stomach acidity, ulcers, and gastroesophageal reflux disease (GERD). Recent findings published in PLOS Medicine suggest that it may work against and eliminate the Tuberculosis (TB) causing Mycobacterium tuberculosis. People on lansoprazole were less likely to develop TB when compared to usage of other PPIs such as omeprazole or pantoprazole according to the results of the study based on analysis of laboratory, animal, and epidemiological data. While it is too early to say if lansoprazole can treat and eradicate TB, further clinical investigation in this direction is certainly warranted.


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Repurposing amitriptyline and phenytoin as topiceuticals in neuropathic pain: how to do it?

December 1st 2017, Posted by: Drug Repurposing Portal

Topical formulations of amitriptyline might help to ease pain in neuropathic pain patients, but only if the concentration selected is high enough and the selected vehicle is optimal. In case of transdermal formulations where sufficient blood levels are required, one should systematically analyze plasma levels after application. This has never been properly documented. If one selects a topical, dermal formulation and wants to reach targets in the skin, for instance in cases of burning pain in diabetes or small fiber neuropathy, one needs to select a different formulation [17]. Amitriptyline has clearly a potential as a topical analgesic but to date has never been properly investigated.


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New Drug Repurposing e-learning from ecancer and ReDO

November 30th 2017, Posted by: Drug Repurposing Portal

ecancer.org and the Repurposing Drugs in Oncology (ReDO) Project are delighted to announce the launch of a new free e-learning module designed to teach clinical researchers and health care professionals about the key issues involved in drug repurposing.


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Nippon Chemiphar and SOM Biotech to Cooperate on New, Orphan Disease Indications for bevantolol (Calvan)

October 23rd 2017, Posted by: Drug Repurposing Portal

SOM Biotech (SOM), a leading drug repurposing company based in Barcelona, Spain, has determined and validated an entirely new usage for Nippon Chemiphar’s marketed hypertension therapy bevantolol (Calvan). After using a proprietary ligand analysis computational algorithm and completing the preclinical assays, SOM determined that bevantolol (coded SOM3355 by SOM) is a highly effective Vesicular Monoamine Transporter Type 2 (VMAT2) inhibitor, a class of compounds that has demonstrated to have success in treating Central Nervous System movement disorders such as Huntington’s Chorea, Tardive Dyskinesia, and Tourette’s syndrome.


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An Old Blood Pressure Drug Shows Potential to Cure Blood Cancer

October 21st 2017, Posted by: Drug Repurposing Portal

Irena Misiewicz-Krzeminska and Norma C. Gutierrez of the Cancer Research Center-IBMCC in Salamanca, Spain recently identified cancer-killing ability of an old blood pressure lowering drug, amiloride. Amiloride is an old diuretic drug which has been used to treat high blood pressure, congestive heart failure, swelling in different parts of the body like feet, and low potassium level in the blood. Study showed that amiloride interfered with a significant feature of cancer cells called alternative splicing and killed human multiple myeloma (a type of blood cancer) cells in mice without any toxicity. The antimyeloma activity of amiloride and open a new route for its use as an alternative treatment for multiple myeloma in patients whose cancer returned.


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Moffitt Team Uses Proteomic Profiling to ID New Ceritinib Targets in Lung Cancer

October 21st 2017, Posted by: Drug Repurposing Portal

Researchers at the H. Lee Moffitt Cancer Center and Research Institute have used a chemical proteomic and phosphoproteomics-based approach to identify new targets for approved cancer drug ceritinib. The researchers identified that the drug-ceritinib binds in addition to its intended target ALK to kinases including FAK1, RSK1/2, ERK1/2, CAMKK2, and FER, with FAK1 and RSK1/2.


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Cancer drug found to offer promising results in treating sepsis in test mice

October 21st 2017, Posted by: Drug Repurposing Portal

A combined team of researchers from China and the U.S. has found that a drug commonly used to treat lung cancer in humans offers a degree of protection against sepsis in test mice. Researchers found that a cancer drug Ceritinib could be used for the treatment of Sepsis condition. Ceritinib, does its work by suppressing a protein called ALK, which serves as a signal to set off a STING response. A stimulator of interferon genes (STING), which activates prior to the onset of sepsis and which is believed to be among the agents that cause sepsis to kick off. (Ref: DOI: 10.1126/scitranslmed.aan5689)


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FDA-approved antabuse drug disulfiram can be used to treat chemo resistant lung cancer

October 13th 2017, Posted by: Drug Repurposing Portal

The FDA-approved alcohol aversion drug disulfiram functions by restricting ALDH activity. Research scientists in Dublin found that high levels of ALDH activity in lung cancer stem cells causes resistance to chemotherapy. In parallel, they have also demonstrate that inhibition of ALDH activity resulted in decreased tumor cell growth and increased killing of lung cancer stem cells.


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Daiichi Sankyo, Astellas and Mitsubishi Tanabe partner to develop new therapeutics

October 13th 2017, Posted by: Drug Repurposing Portal

Japan-based companies Daiichi Sankyo, Astellas Pharma and Mitsubishi Tanabe Pharma have collaborated to jointly carry out a programme to discover new therapeutic drugs using drug-repositioning compound libraries.


News Methods Funding Collaborations

Parkinson's drug carbidopa can be repurposed for pancreatic cancer

October 12th 2017, Posted by: Drug Repurposing Portal

Carbidopa is commonly used in combination with levodopa to treat Parkinson?s disease. Earlier studies have shown that Parkinson's patients tend to have a lower incidence of cancer. In line, a recent study demonstrated anti-cancer effects of carbidopa in mice and human pancreatic cells suggesting the potential of carbidopa to be repurposed for treating pancreatic cancer.


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Ceritinib an approved metastatic ALK-rearranged NSCLC drug is a potential candidate for repurposing

October 11th 2017, Posted by: Drug Repurposing Portal

A team of researchers at Moffitt Cancer Center performed a cellular drug screening, functional proteomics and computer-based modeling of 240 FDA approved or clinical drugs to identify novel targets. Ceritinib is FDA approved for treating the patients with ALK-rearranged metastatic non-small cell lung cancer (NSCLC) is found to inhibit the growth of lung cancer cells that do not have ALK-rearrangements. They have also demonstrated that ceritinib also inhibits other previously unknown targets that are known to cause paclitaxel resistance. These results suggest that ceritinib can be repurposed for the treatment of other cancers that do not have ALK rearrangements and/or are resistant to paclitaxel therapy.


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Recursion racks up $60m Series B funding

October 6th 2017, Posted by: Drug Repurposing Portal

US based pharma company, Recursion Pharmaceuticals obtained $60 million funding to facilitate their Drug repurposing program in various therapeutic areas and its machine-learning based drug discovery programs


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Tea Aids Weight Loss through Microbiome Alteration

October 6th 2017, Posted by: Drug Repurposing Portal

Investigators at UCLA have demonstrated that tea, and in particular black tea, may promote weight loss and other health benefits by changing bacteria within the gut. The results suggest that both green and black teas are prebiotics, substances that induce the growth of good microorganisms that contribute to a person's well-being, Black Tea Polyphenols (BTP) increased the relative proportion of Pseudobutyrivibrio, a type of bacterial metabolites that have been shown to alter the energy metabolism in the liver and intestinal formation of short-chain fatty acids (SCFA). And GTP and BTP induced a significant increase in hepatic 5'adenosylmonophosphate-activated protein kinase (AMPK) phosphorylation by 70 and 289%, respectively.


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Trinity Researchers Re-Purpose Drug to Better Treat Lung Cancer

October 5th 2017, Posted by: Drug Repurposing Portal

Recent study showed that combining Cisplatin with anti-alcohol addiction drug Antabuse is very effective in killing chemotherapy resistant Lung cancer cells. In a sub-group of lung cancer patients aldehyde dehydrogenase is very active in non-small cell lung cancer and one of the reasons for chemotherapy resistance. Antabuse (Disulfiram) targeting aldehyde dehydrogenase already approved for alcohol addiction is used in conjunction with Cisplatin and showed positive results in killing cancer cells. This is a fine example of COMBINATION THERAPY and DRUG-REPURPOSING.


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Could repurposing a depression drug help burn belly fat?

October 5th 2017, Posted by: Drug Repurposing Portal

monoamine oxidase-A or MAOA inhibition (or MAOAi) was the mechanism of action for the first major class of antidepressants, which included drugs like isocarboxazid and phenelzine. Inhibition of these enzymes restored fat metabolism and reduced obesity. So, antidepressants could be used to treat obesity.


News Methods Funding Collaborations

Cell-line dependent antiviral activity of sofosbuvir against Zika virus

September 23rd 2017, Posted by: Drug Repurposing Portal

Sofosbuvir is used for the treatment of hepatitis C virus infection. Current study assessed antiviral activity of sofosbuvir and mericitabine against Zika virus (ZIKV) using Vero, A549, and Huh7 cells. Interestingly, Mericitabine did not show any activity, while sofosbuvir inhibited ZIKV with an IC50 of 4 M, but only in Huh7 cells. This inhibition is correlated with the intracellular concentration of the active triphosphate metabolite of sofosbuvir, GS-461203 or 007-TP which was 11-342 times higher in Huh7 cells compared to Vero and A549 cells. These data demonstrate the potential of sofosbuvir as anti-ZIKV compound


News Methods Funding Collaborations

An in-silico approach to predict and exploit synthetic lethality in cancer metabolism

September 21st 2017, Posted by: Drug Repurposing Portal

Synthetic lethality is a promising concept in cancer research, potentially opening new possibilities for the development of more effective and selective treatments. Here, we present a computational method to predict and exploit synthetic lethality in cancer metabolism. Our approach relies on the concept of genetic minimal cut sets and gene expression data, demonstrating a superior performance to previous approaches predicting metabolic vulnerabilities in cancer. Our genetic minimal cut set computational framework is applied to evaluate the lethality of ribonucleotide reductase catalytic subunit M1 (RRM1) inhibition in multiple myeloma. We present a computational and experimental study of the effect of RRM1 inhibition in four multiple myeloma cell lines. In addition, using publicly available genome-scale loss-of-function screens, a possible mechanism by which the inhibition of RRM1 is effective in cancer is established. Overall, our approach shows promising results and lays the foundation to build a novel family of algorithms to target metabolism in cancer.


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Study suggests epilepsy drug can be used to treat form of dwarfism

September 20th 2017, Posted by: Drug Repurposing Portal

Carbamazepine, currently used for the treatment of epilepsy and bi-polar disease has been shown to be effective in lowering the effects of Metaphyseal chondrodysplasia type Schmid (MCDS). A three week treatment in mice at the University of Manchester, Murdoch Children's Research Institute Australia, showed a profound increase in bone growth rate along with a reduction in hip dysplasia. Repurposing Carbamazepine for a rare disease like MCDS warrants further tests in human beings, the study suggested.


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New Indication For BRIVIACT (Brivaracetam): UCB's Newest Antiepileptic Drug Approved By FDA As Monotherapy Treatment Of Partial-Onset Seizures In Adults

September 18th 2017, Posted by: Drug Repurposing Portal

U.S. Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) for BRIVIACT (brivaracetam) CV as monotherapy for partial-onset (focal) seizures (POS) in patients 16 years and older with epilepsy. This is a new indication for BRIVIACT, which is already approved in the U.S. as adjunctive treatment for POS in patients in this age group. As a result, adults and adolescents aged 16 years and older with POS in the U.S. can now be initiated on BRIVIACT as monotherapy or adjunctive therapy.


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New Indication For BRIVIACT (Brivaracetam): UCB's Newest Antiepileptic Drug Approved By FDA As Monotherapy Treatment Of Partial-Onset Seizures In Adults

September 15th 2017, Posted by: Drug Repurposing Portal

U.S. Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) for BRIVIACT (brivaracetam) CV as monotherapy for partial-onset (focal) seizures (POS) in patients 16 years and older with epilepsy. This is a new indication for BRIVIACT, which is already approved in the U.S. as adjunctive treatment for POS in patients in this age group. As a result, adults and adolescents aged 16 years and older with POS in the U.S. can now be initiated on BRIVIACT as monotherapy or adjunctive therapy.


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Aspirin in reversing the effects of tooth decay

September 11th 2017, Posted by: Drug Repurposing Portal

A preventable disease, tooth decay affects billions worldwide. Treatment involves fillings which may need to be replaced over the course of a lifetime. The researchers from Queens University Belfast, had used combined genomics and novel bioinformatics to identify aspirin as a candidate drug. It appears that aspirin?s properties stimulate existing stem cells in the tooth to promote regeneration of the damaged tooth structure.


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Submit your repurposing research proposal on CureAccelerator

September 10th 2017, Posted by: Drug Repurposing Portal

CuresWithinReach (a not-for-profit organization), is conducting an event via its platform, CureAccelerator-Live! on November 15th, 2017 to select clinical trial proposals designed to repurpose therapies. Project ideas will be selected for treatments that could be repurposed in cancer/unmet cancer needs or repurpose current cancer therapies for use in non-cancer disease. The winner of the selected project will be awarded USD 50,000.


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Purdue Researcher Receives $1.6M Grant For Repurposing Meds

September 7th 2017, Posted by: Drug Repurposing Portal

Purdue researcher, Saleem Mohamed received a $1.6 million grant from NIH for a period of five years, for testing the potential of repurposing two FDA-approved drugs in treating bacterial infections.


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Can Zika virus be re-purposed to fight Brain cancer? Scientists say may be.

September 5th 2017, Posted by: Drug Repurposing Portal

Zika virus cause microcephaly (abnormally small heads) and associated neurological problems in the babies of women who were infected while pregnant, as well as a higher rate of miscarriage. The virus does this because it can pass from blood into the brain, where it infects and kills stem cells, having severe effects on developing brains. Jeremy Rich at the University of California, San Diego, and his team have tested the Zika virus on glioblastoma, the most common kind of brain cancer. The team found that exposing samples of human glioblastoma tumours grown in a dish to the Zika virus destroyed the cancer stem cells. It is these stem cells that usually kill a person, as they can become resistant to all available treatments. When the team tested the virus on ordinary brain cells from adults without cancer, they found that it didn't infect this tissue. It is unclear how this would translate to people as the disease affects mice differently to humans.


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Study: Asthma Medicine Halves Parkinson's Risk

September 1st 2017, Posted by: Drug Repurposing Portal

Researchers at the Department of Global Health and Social Medicine (IGS) at the University of Bergen (UiB), in collaboration with researchers from Harvard University , conducted a comprehensive study, which included data from the National Prescription Drug Registry. The authors looked at the pharmaceutical history of more than 4 million Norwegians over an 11-year period and found a reduced risk of PD among those that were taking one of the β2AR agonists (salbutamol, a brain-penetrant asthma medication) for other medical problems.


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Asthma drugs may reduce Parkinson's risk

August 31st 2017, Posted by: Drug Repurposing Portal

Clemens Scherzer of and her team at Brigham & Women's Hospital and Harvard Medical School, screened about 1,126 compounds (including FDA approved drugs) to see reduced expression of the α-synuclein gene in neuroblastoma cells. Surprisingly, the team found 4 hits of which 3 were targeting β2 adrenoreceptor. Clenbuterol, reduced expression of the α-synuclein gene in part of the brain affected by Parkinson's. The drug also protected neurons typically destroyed by the disease in mice. Further studies need to be conducted to explore if this hypothesis could be applied across patient population and how much of cardiovascular side-effects are seen in these putative asthma drugs for treating PD patients.


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An inflammatory Drug for Cancer risk

August 28th 2017, Posted by: Drug Repurposing Portal

Canakinumab is a human monoclonal antibody that neutralizes interleukin-1?, a pro-inflammatory cytokine that results in increased inflammation throughout the body as well as increased levels of hsCRP. In phase III clinical trial, patients on on the higher doses of canakinumab were 15 percent less likely to suffer another major cardiac event compared to those taking a placebo. Chronic inflammation can drive cancer. Results of blinded oncology safety analyses showed a 77 percent reduction in lung cancer mortality and 67 percent reduction in lung cancer cases in patients treated with higher doses of canakinumab.


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Diabetes Drug Could Be Potential parkinson's treatment

August 9th 2017, Posted by: Drug Repurposing Portal

Exenatide, a commonly prescribed drug for type 2 diabetes, could possibly be repurposed as a modifying therapy for patients diagnosed with Parkinson's disease. Injections of exenatide showed signs of improving movement in Parkinson's patients over a one year period versus those who took placebo, suggested a new study.


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A diabetes drug to slow Parkinson's

August 5th 2017, Posted by: Drug Repurposing Portal

Replacing dopamine can improve the tremors and stiffness of Parkinson's but it doesn't stop the brain from continuing to deteriorate. In an attempt to slow this, Foltynie and his colleagues have turned to a drug typically used to treat type 2 diabetes, called exenatide. This drug comes from a class of compounds originally isolated from the venom of a lizard called the gila monster. Exenatide, seems to target the underlying cause of the condition, not just its symptoms


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Alzheimer's Drug Cuts Hallmark Inflammation Related to metabolic Syndrome by 25 Percent

July 28th 2017, Posted by: Drug Repurposing Portal

In a study by Feinstein Institute researchers found that the FDA-approved Alzheimer's drug "galantamine" cut key markers of inflammation a hallmark of metabolic syndrome by more than 25 percent, leading to reduced insulin resistance. A cluster of four risk factors increased blood pressure, a high blood sugar level, excess body fat around the waist and abnormal cholesterol levels comprise metabolic syndrome, which greatly raises risks for cardiovascular disease and type 2 diabetes.


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Repurposed Asthma Drug Shows Blood Sugar Improvement Among Some Diabetics

July 10th 2017, Posted by: Drug Repurposing Portal

Amlexanox, an anti-inflammatory and anti-allergic drug used to treat asthma was developed in the 1980s in Japan. The clinical observation, by the researchers at University of California San Diego School of Medicine and University of Michigan, states that after 12 weeks of taking asthma drug, a subset of patients with type 2 diabetes showed a clinically significant reduction in blood glucose. The discovery is not ready for the clinic, but it does reveal a potential new therapeutic approach for treating type 2 diabetes.


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An agent to increase radiotherapy efficiency, has the ability to make a tumor responsive to immunotherapy

June 23rd 2017, Posted by: Drug Repurposing Portal

Nanobiotix's NBTXR3, was developed to increase the amount of radiotherapy that can be delivered to a tumor. It consists of hafnium oxide nanoparticles injected straight into the cancer. In patients with soft tissue sarcoma, or cancer of connective tissue, a combination of NBTXR3 and radiotherapy prompted three types of immune cells to infiltrate the tumor: two kinds of T-cells and dendritic cells. Patients who received radiotherapy only had no immune response.


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New Outcomes Study Planned for Diabetes Drug with Potential in Treating Kidney Disease

June 20th 2017, Posted by: Drug Repurposing Portal

The landmark EMPA-REG OUTCOME trial demonstrated that empagliflozin reduced the risk of cardiovascular death by 38 percent versus placebo in people with type 2 diabetes and established cardiovascular disease when added to standard of care (including glucose-lowering agents and cardiovascular drugs).


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Repurposing metformin in FXS

June 16th 2017, Posted by: Drug Repurposing Portal

Summary: Researchers report a common diabetes drug helped to restore protein production in the brain to normal levels and repaired brain connections in mouse models of Fragile X. Source: University of Edinburgh. A widely used diabetes medication could help people with a common inherited form of autism, research shows. Scientists found that a drug called metformin improves sociability and reduces symptomatic behaviours in adult mice with a form of Fragile X syndrome. Researchers say that metformin could be repurposed as a therapy for Fragile X syndrome within a few years ? if clinical trials prove successful. Fragile X syndrome is caused by inherited defects in a gene called FMR1, which leads to excess protein production in the brain. This results in the breakdown of connections between brain cells, leading to changes in behaviour.


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Keytruda as standalone therapy for other cancers

June 15th 2017, Posted by: Drug Repurposing Portal

Ketruda, Developed by Merck, is a potent anti-PD-1 antibody that helps activate the immune system?s response to cancer cells. U.S. Food and Drug Administration had approved for the treatment of melanoma, lung. The Phase 2 KEYNOTE-059 trial is currently testing the efficacy of this anti-PD1 therapy as standalone treatment in patients with gastric or gastroesophageal junction adenocarcinoma who have not responded to two or more chemotherapy regimens. PD-L1 positive patients had an overall response rate to treatment of 15.5%. In contrast, only 6.4% of patients whose cancers did not expressed PD-L1 (109 patients) responded to Keytruda.


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Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy

June 14th 2017, Posted by: Drug Repurposing Portal

Dr. Marugan at NCATS Chemical Genomics Center along with the team and collaborators, screened 350,000 compounds and identified SU9516 as a lead molecule that was used previously for the treatment of leukemia which could have potential utility in treatment for Duchenne muscular dystrophy (DMD). The research demonstrated that this compound improved muscle function in both laboratory and animal DMD models and the results were published recently in Journal of Molecular Therapy.


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ADA 2017: Investigational Drug for Knee Osteoarthritis Avoids Significant Rise in Blood Glucose in Type2 Diabetes Patients

June 13th 2017, Posted by: Drug Repurposing Portal

Flexion Therapeutics, Inc. presented data from a Phase II study which found its lead investigational drug candidate, FX006 (Zilretta) was associated with reduced blood glucose elevation compared with immediate release triamcinolone acetonide in crystalline suspension (TAcs) in patients with Type 2 diabetes and knee osteoarthritis (OA). FX006 was created to address the issue of the inadequate duration of the adequate pain relief associated with immediate relief steroids. Zilretta was formulated in such a way to achieve therapeutic concentrations or effective concentrations in the joint for three months and we demonstrated that we not only achieved longer relief which was the original goal, but pleasant surprise, Zilretta achieved better relief than seen with the immediate relief steroids?without the spike in blood glucose levels.


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Rituximab, re-purposed to ALS

June 8th 2017, Posted by: Drug Repurposing Portal

Rituximab is indicated to treat chronic lymphocytic leukemia, non-Hodgkin's lymphoma and rheumatoid arthritis. Scientists from Ben-Gurion University had successfully redesigned a portion of rituximab into a new molecule to treat ALS. In lab studies using mice, the therapy restored the immune cells of the central nervous system, which could potentially help extend survival in ALS patients.


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Doxycycline as repursed drug against parkinson's disease

May 23rd 2017, Posted by: Drug Repurposing Portal

In Parkinson's disease, Molecular triggers of inflammation are known to be targeted by Antibiotics. A study published in Scientific Reports found that a low dose of doxycycline was able to reduce the toxicity of &aplha;-synuclein, which aggregates to form amyloid fibrils the molecular trigger of inflammation in PD. The study concludes that, Doxycycline shows promise as repurposed drug against Parkinson's disease.


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Cancer drug Imatinib for severe asthma

May 19th 2017, Posted by: Drug Repurposing Portal

Imatinib is currently used to effectively treat cancers with specific mutation. Mechanism of action include, targeting mast cell development and stem cell factor the KIT receptor tyrosine kinase. In severe asthma, airways are infiltrated with mast cells and acts as an indicator of asthma. Researchers from Brigham and Women's Hospital had published in the New England Journal of Medicine that, targeting the mast cells with imatinib, improved airway hyperresponsiveness, a measure of the sensitivity of the airway, and decreased the number of mast cells present in the airway. Treatment also produced a small improvement in airway function. Researchers note that larger-scale studies are needed to confirm their finding and evaluate longer durations of therapy in order to definitively determine clinical efficacy.


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Multiple Sclerosis Drug Effectively Stops Mesothelioma Cells

May 13th 2017, Posted by: Drug Repurposing Portal

Research from the University of Hawaii Cancer Center shows an immune suppression drug, fingolimod (FTY720), which is already used to treat multiple sclerosis, could become an effective tool against malignant mesothelioma. The drug showed an ability to shrink mesothelioma tumors cells in the laboratory and in animal models without causing substantial side effects.


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Bavencio, An IO drug, by Pfizer and EMD Serono, gains approaval for Bladder Cancer Indication

May 12th 2017, Posted by: Drug Repurposing Portal

Bevanico gained approval in March for a rare form of skin cancer called Merkel Cell carcinoma. In less than two months, FDA had granted accelerated approval to Bavencio (avelumab) to treat patients with locally advanced or metastatic urothelial carcinoma, which is an aggressive form of bladder cancer that has a high rate of recurrence. The FDA made their decision after reviewing tumor response and response duration results from a phase 1 open-label study exploring Bavencio's safety and efficacy profiles with 242 patients diagnosed with this disease.


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One protein inhibitor could treat Chagas, leishmaniasis and sleeping sickness

May 10th 2017, Posted by: Drug Repurposing Portal

Wellcome Trust-backed scientists have found a compound that treats three neglected parasitic diseases in mice: leishmaniasis, Chagas disease and sleeping sickness. The Wellcome-funded team from the Novartis Research Foundation's Genomics Institute (GNF) focused on the genetic and biological similarities between the kinetoplastids, single-celled parasites.


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Translating GWAS Findings Into Therapies For Depression And Anxiety Disorders: Drug Repositioning Us

May 9th 2017, Posted by: Drug Repurposing Portal

Depression and anxiety disorders are the first and sixth leading cause of disability worldwide according to latest reports from the World Health Organization. Despite their high prevalence and the significant disability resulted, there are limited advances in new drug development. On the other hand, the advent of genome-wide association studies (GWAS) has greatly improved our understanding of the genetic basis underlying psychiatric disorders. In this work we employed gene-set analyses of GWAS summary statistics for drug repositioning. We explored five related GWAS datasets, including two on major depressive disorder (MDD-PGC and MDD-CONVERGE, with the latter focusing on severe depression cases), one on anxiety disorders, and two on depressive symptoms and neuroticism in the population. For example, the top repositioning hit using meta-analyzed p-values was fendiline, which was shown to produce antidepressant-like effects in mouse models by inhibition of acid sphingomyelinase and reducing ceramide levels. Taken together, our findings suggest that human genomic data such as GWAS might be useful in guiding drug discoveries for depression and anxiety disorders.


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Tofacitinib, An arthritis drug to treat moderate to severe ulcerative colitis

May 4th 2017, Posted by: Drug Repurposing Portal

Ulcerative colitis is a chronic inflammatory bowel disease. The illness causes inflammation, irritation, swelling and sores on the lining of the large intestine. New research lead by Dr. William Sandbornat finds that, Xeljanz targets certain proteins involved in the body's inflammatory and immune responses that other so-called biologic drugs don't. Thus Xeljanz, may relieve people with moderate to severe ulcerative colitis who haven't done well on other treatments.


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A structure- and chemical genomics-based approach for repositioning of drugs against VCP/p97 ATPase

April 25th 2017, Posted by: Drug Repurposing Portal

Valosin-containing protein (VCP/p97) ATPase (a.k.a. Cdc48) is a key member of the ER-associated protein degradation (ERAD) pathway. ERAD and VCP/p97 have been implicated in a multitude of human diseases, such as neurodegenerative diseases and cancer. Inhibition of VCP/p97 induces proteotoxic ER stress and cell death in cancer cells, making it an attractive target for cancer treatment. However, no drugs exist against this protein in the market. Repositioning of drugs towards new indications is an attractive alternative to the de novo drug development due to the potential for significantly shorter time to clinical translation. Here, we employed an integrative strategy for the repositioning of drugs as novel inhibitors of the VCP/p97 ATPase. We integrated structure-based virtual screening with the chemical genomics analysis of drug molecular signatures, and identified several candidate inhibitors of VCP/p97 ATPase. Importantly, experimental validation with cell-based and in vitro ATPase assays confirmed three (ebastine, astemizole and clotrimazole) out of seven tested candidates (~40% true hit rate) as direct inhibitors of VCP/p97 and ERAD. This study introduces an effective integrative strategy for drug repositioning, and identified new drugs against the VCP/p97/ERAD pathway in human diseases.


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Malaria experts set sights on single-shot cure by 2030s

April 25th 2017, Posted by: Drug Repurposing Portal

One of the objects of desire in the anti-malarial battle has been that some medicines already in use to treat other diseases could be repurposed to tackle malaria. Despite more than a decade looking for quick fixes from existing drugs, however, successes have been few and far between. The idea behind repurposing drugs had its modern-day genesis just before the turn of the millennium when the world?s armoury against malaria was bare. The most celebrated instance of a repurposed or crossover drug is the case of ivermectin, designed to kill the parasites that cause river blindness and filariasis, also known as elephantiasis. Researchers at Colorado State University in 2015 found that administering the drug en masse helped reduce malaria episodes in some areas of west Africa. A separate study by the US Army found the drug helped block development of Plasmodium vivax parasites in mosquitoes most commonly found in Asia.


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Global Drug Repurposing Market to Witness a Pronounce Growth During 2017 to 2025

April 25th 2017, Posted by: Drug Repurposing Portal

Drug repurposing or re-profiling has been the hallmark to bring strong business growth and the trend is being followed by majority of the pharmaceutical and biopharmaceutical companies. Among all biologics approved in the U.S. during 2007-2009, 30-40% of them were the drugs repurposed or repositioned. National Institute of Health (NIH), U.S. Department of Health defines drug repurposing as discovering new uses for approved drugs to provide the quickest possible transition from bench to bedside. Drug repurposing opens up various opportunities to answer current unmet medical needs to come up with cost-effective solutions to expensive drug development process.


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Repurposed drugs targeting eIF2α-P-mediated translational repression prevent neurodegeneration in m

April 24th 2017, Posted by: Drug Repurposing Portal

NINDS small molecule library of 1040 drugs was used to perform phenotypic screnning and looking at anti-eIF2α-P activity suitable for clinical use. The researchers found trazodone hydrochloride and dibenzoylmethane with maximum potenital, which reversed eIF2α-P-mediated translational attenuation in vitro and in vivo. Both drugs were markedly neuroprotective in two mouse models of neurodegeneration, using clinically relevant doses over a prolonged period of time, without systemic toxicity.


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Filling the gap in CNS drug development: evaluation of the role of drug repurposing

April 24th 2017, Posted by: Drug Repurposing Portal

Drug repurposing has been considered a cost-effective and reduced-risk strategy for developing new drugs. Little is known and documented regarding the efficiency of repurposing strategies in drug development. The objective of this article is to assess the extent and meaning of this process in the CNS area.


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Scientists discover two repurposed drugs that arrest neurodegeneration in mice

April 22nd 2017, Posted by: Drug Repurposing Portal

A team of researchers tested 1,040 compounds and identified two drugs that restored protein production rates in mice, trazodone hydrochloride, a licensed antidepressant, and dibenzoylmethane, a compound being trialled as an anti-cancer drug. Both drugs prevented the emergence of signs of brain cell damage in most of the prion-diseased mice and restored memory in the FTD mice. In both mouse models, the drugs reduced brain shrinkage which is a feature of neurodegenerative disease.


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Accelerating Precision Drug Development and Drug Repurposing by Leveraging Human Genetics

April 21st 2017, Posted by: Drug Repurposing Portal

The potential impact of using human genetic data linked to longitudinal electronic medical records on drug development is extraordinary; however, the practical application of these data necessitates some organizational innovations. Vanderbilt has created resources such as an easily queried database of >2.6 million de-identified electronic health records linked to BioVU, which is a DNA biobank with more than 230,000 unique samples. To ensure these data are used to maximally benefit and accelerate both de novo drug discovery and drug repurposing efforts, we created the Accelerating Drug Development and Repurposing Incubator, a multidisciplinary think tank of experts in various therapeutic areas within both basic and clinical science as well as experts in legal, business, and other operational domains. The Incubator supports a diverse pipeline of drug indication finding projects, leveraging the natural experiment of human genetics.


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Heter-LP: A heterogeneous label propagation algorithm and its application in drug repositioning

April 20th 2017, Posted by: Drug Repurposing Portal

Drug repositioning offers an effective solution to drug discovery, saving both time and resources by finding new indications for existing drugs. Typically, a drug takes effect via its protein targets in the cell. As a result, it is necessary for drug development studies to conduct an investigation into the interrelationships of drugs, protein targets, and diseases. Although previous studies have made a strong case for the effectiveness of integrative network-based methods for predicting these interrelationships, little progress has been achieved in this regard within drug repositioning research. Moreover, the interactions of new drugs and targets (lacking any known targets and drugs, respectively) cannot be accurately predicted by most established methods. In this paper, we propose a novel semi-supervised heterogeneous label propagation algorithm named Heter-LP, which applies both local and global network features for data integration. To predict drug-target, disease-target, and drug-disease associations, we use information about drugs, diseases, and targets as collected from multiple sources at different levels. Our algorithm integrates these various types of data into a heterogeneous network and implements a label propagation algorithm to find new interactions. Statistical analyses of 10-fold cross-validation results and experimental analyses support the effectiveness of the proposed algorithm.


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MRC scientists discover two repurposed drugs that arrest neurodegeneration in mice

April 20th 2017, Posted by: Drug Repurposing Portal

In a study funded by MRC and ADDF Professor Mallucci and team screened about 1040 compounds from the National Institute for Neurological Disorders and Stroke, first in worms (C.elegans) which have a functioning nervous system and are a good experimental model for screening drugs to be used on the nervous system and then in mammalian cells. This research led to identification of two drugs that restored protein production rates in mice trazodone hydrochloride, an antidepressant, and dibenzoylmethane (DBM), a compound being trialled as an anti-cancer drug. However these compounds are safe to be tested in humans, currently they are under experimental studies.


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Mesothelioma shows promising response to existing immunotherapy drug

April 19th 2017, Posted by: Drug Repurposing Portal

Mesothelioma is a rare cancer that arises in the thin lining of tissue that covers the inside of the chest, the heart, the abdomen, and most internal organs. The main risk factor for mesothelioma is inhalation of asbestos. Dr. Evan Alley and his colleagues, University of Pennsylvania Health System in Philadelphia, has found a new class of drugs, checkpoint inhibitors. Checkpoint inhibitors are drugs designed to help the body fight cancer by defeating certain mechanisms that cancer cells use to avoid being attacked by the immune system. Pembrolizumab is one such drug. Pembrolizumab is already used to treat non-small cell lung cancer, melanoma, and some head and neck cancers. New results show that Pembrolizumab lead to tumour shrinkage in mesothelioma patients after treatment.


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Could antidepressants stop prostate cancer from spreading?

April 19th 2017, Posted by: Drug Repurposing Portal

Jason Wu, of Washington State University-Spokane, and colleagues found that a drug called clorgyline - a drug once used as an antidepressant can control the growth of prostate cancer cell's. Clorgyline is known to block the activity of MAOA, an enzyme that prompts a signaling cascade that simplifies the process by which prostate cancer cells spread to the bone. Researchers found that the drug prevented MAOA from activating the three proteins that enhance osteoclast function, thereby reducing the prostate cancer cell's ability to invade and grow in bone.


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Identifying new antiepileptic drugs through genomics-based drug repurposing

April 19th 2017, Posted by: Drug Repurposing Portal

Currently available antiepileptic drugs (AEDs) fail to control seizures in 30% of patients. Genomics-based drug repurposing (GBR) offers the potential of savings in the time and cost of developing new AEDs. In the current study, we used published data and software to identify the transcriptomic signature of chornic temporal lobe epilepsy and the drugs that reverse it. After filtering out compounds based on exclusion criteria, such as toxicity, 36 drugs were retained. 11 of the 36 drugs identified (>30%) have published evidence of the antiepileptic efficacy (for example, curcumin) or antiepileptogenic affect (for example, atorvastatin) in recognised rodent models or patients. By objectively annotating all 20,000 compounds in the LINCS database as either having published evidence of antiepileptic efficacy or lacking such evidence, we demonstrated that our set of repurposable drugs is 6-fold more enriched with drugs having published evidence of antiepileptic efficacy in animal models than expected by chance (P-value<0.006). Further, we showed that another of our GBR-identified drugs, the commonly-used well-tolerated antihyperglycemic sitagliptin, produces a dose-dependent reduction in seizures in a mouse model of pharmacoresistant epilepsy. In conclusion, GBR successfully identifies compounds with antiepileptic efficacy in animal models and, hence, it is an appealing methodology for the discovery of potential AEDs.


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Some Remarks on Prediction of Drug-Target Interaction with Network Models.

April 18th 2017, Posted by: Drug Repurposing Portal

System-level understanding of the relationships between drugs and targets is very important for enhancing drug research, especially for drug function repositioning. The experimental methods used to determine drug-target interactions are usually time-consuming, tedious and expensive, and sometimes lack reproducibility. Thus, it is highly desired to develop computational methods for efficiently and effectively analyzing and detecting new drug-target interaction pairs. With the explosive growth of different types of omics data, such as genome, pharmacology, phenotypic, and other kinds of molecular networks, numerous computational approaches have been developed to predict drug-target interactions (DTI). In this review, we make a survey on the recent advances in predicting drug-target interaction with network-based models from the following aspects: i) Available public data sources and benchmark datasets, ii) Drug/target similarity metrics, iii) Network construction, iv) Common network algorithms, v) Performance comparison of existing network-based DTI predictors.


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Use of antiparasitic drug as new treatment for brain tumors explored by researchers

April 18th 2017, Posted by: Drug Repurposing Portal

A team of researchers led by Dr. Symons from the Feinstein Institute for Medical Research's Karches Center for Oncology Research has recently found that mebendazole, a medication that is used to treat parasitic pinworms, is effective in the treatment of glioma tumors. The study results have recently been published in journal Molecular Medicine.


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Orphan Drugs Market Growing at a CAGR of 10.20% During 2017 to 2021, Says a New Report at ReportsnR

April 17th 2017, Posted by: Drug Repurposing Portal

One trend in orphan drugs market is repurposing of non-orphan drugs to orphan drugs. The orphan drugs market has witnessed many successful repositioning of drugs from non-orphan label to orphan label. Since the implementation of the orphan drug legislation in the United States, more than 69 drugs which were first approved by US FDA for the treatment of rare diseases were not entirely new and had been repurposed from the non-orphan label to the orphan label. Today, most of the pharmaceutical and biotechnological companies have adopted the strategy of drug repurposing in order to save time and money. Repurposing of an already approved drug for a different indication helps to mitigate the risk of drug failure as the drug has undergone the pharmacovigilance regulatory requirement and post-marketing survey. This reduces the risk of heavy financial loss to the manufacturer. In addition, the drug repurposing strategy also helps manufacturers to extend their product life cycle by getting orphan drug status, and also prevents their product from generic competition.


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NuMedii, Inc. Announces New Partnership To Discover And Advance New Treatments For Idiopathic Pulmon

April 13th 2017, Posted by: Drug Repurposing Portal

NuMedii & Three Lakes Partners, LLC enter into a collaboration in the pursuit of identifying therapies for idiopathic pulmonary fibrosis (IPF) based on NuMedii's Big Data intelligence technology. NuMedii's CEO, Gini Deshpande says "This unique partnership between an AI drug discovery company and a patient-centric organization further builds on our work in inflammation and showcases our progress, particularly our development capabilities in rare disease."


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The Drug Repurposing Hub: a next-generation drug library and information resource

April 11th 2017, Posted by: Drug Repurposing Portal

An online repurposing library, drug Repurposing Hub b (http:// www.broadinstitute.org/repurposing) contains hand-curated a collection of 4,707 compounds with experimentally confirmed their identities and annotated with literature-reported targets. The collection includes 3,422 drugs that are marketed around the world or that have been tested in human clinical trials.


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A Biologically-Based Computational Approach to Drug Repurposing for Anthrax Infection

April 11th 2017, Posted by: Drug Repurposing Portal

Developing drugs to treat the toxic effects of lethal toxin (LT) and edema toxin (ET) produced by B. anthracis is of global interest. We utilized a computational approach to score 474 drugs/compounds for their ability to reverse the toxic effects of anthrax toxins. For each toxin or drug/compound, we constructed an activity network by using its differentially expressed genes, molecular targets, and protein interactions. Gene expression profiles of drugs were obtained from the Connectivity Map and those of anthrax toxins in human alveolar macrophages were obtained from the Gene Expression Omnibus. Drug rankings were based on the ability of a drug/compound's mode of action in the form of a signaling network to reverse the effects of anthrax toxins; literature reports were used to verify the top 10 and bottom 10 drugs/compounds identified. Simvastatin and bepridil with reported in vitro potency for protecting cells from LT and ET toxicities were computationally ranked fourth and eighth. The other top 10 drugs were fenofibrate, dihydroergotamine, cotinine, amantadine, mephenytoin, sotalol, ifosfamide, and mefloquine; literature mining revealed their potential protective effects from LT and ET toxicities. These drugs are worthy of investigation for their therapeutic benefits and might be used in combination with antibiotics for treating B. anthracis infection.


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