Drug Repurposing News



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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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)


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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)


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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).


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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."


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

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.


News Methods Funding Collaborations

Network-based analysis of transcriptional profiles from chemical perturbations experiments

April 11th 2017, Posted by: Drug Repurposing Portal

A pipeline for analyzing transcriptional network inference and comparison for grouping chemicals with similar functions and carcinogenicity / genotoxicity profiles. In the context of drug discovery or drug repositioning, discussed method could help assign new functions to novel or existing drugs, based on the similarity of their associated network with those built for other known compounds. Additionally, the method has broad applicability beyond the uses here described and could be used as an alternative or as a complement to standard approaches of differential gene expression analysis.


News Methods Funding Collaborations

A one-two punch hits pancreatic cancer where it hurts

April 10th 2017, Posted by: Drug Repurposing Portal

Australian scientists have uncovered a promising new approach to treating pancreatic cancer, by targeting the tissue around the tumour to make it 'softer' and more responsive to chemotherapy with a three-day course of Fasudil, which is an inhibitor of the protein ROCK and approved for the treatment of cerebral vasospasm. The findings are recently published in Science Translational Medicine.


News Methods Funding Collaborations

Systematic drug repositioning through mining adverse event data in ClinicalTrials.gov

April 6th 2017, Posted by: Drug Repurposing Portal

Drug repositioning (i.e., drug repurposing) is the process of discovering new uses for marketed drugs. Historically, such discoveries were serendipitous. However, the rapid growth in electronic clinical data and text mining tools makes it feasible to systematically identify drugs with the potential to be repurposed. Described here is a novel method of drug repositioning by mining ClinicalTrials.gov. The text mining tools I2E (Linguamatics) and PolyAnalyst (Megaputer) were utilized. An I2E query extracts "Serious Adverse Events" (SAE) data from randomized trials in ClinicalTrials.gov


News Methods Funding Collaborations

Sh100 malaria drug could be used to manage colon cancer

April 6th 2017, Posted by: Drug Repurposing Portal

A team of researchers from two institutions in the United Kingdom, St George's University of London and St George's Hospital, have demonstrated that artesunate, a common and cheap oral malaria drug that costs less than Sh100, reduces the multiplication of tumour cells and the chance of colorectal cancer spreading or recurring after surgery.


News Methods Funding Collaborations

Senicapoc: Repurposing a Drug to Target Microglia KCa3.1 in Stroke

April 6th 2017, Posted by: Drug Repurposing Portal

Stroke is the leading cause of serious long-term disability and the fifth leading cause of death in the United States. Treatment options for stroke are few in number and limited in efficacy. Neuroinflammation mediated by microglia and infiltrating peripheral immune cells is a major component of stroke pathophysiology. Interfering with the inflammation cascade after stroke holds the promise to modulate stroke outcome. The calcium activated potassium channel KCa3.1 is expressed selectively in the injured CNS by microglia. KCa3.1 function has been implicated in pro-inflammatory activation of microglia and there is recent literature suggesting that this channel is important in the pathophysiology of ischemia/reperfusion (stroke) related brain injury. Here we describe the potential of repurposing Senicapoc, a KCa3.1 inhibitor, to intervene in the inflammation cascade that follows ischemia/reperfusion.


News Methods Funding Collaborations

Laying in silico pipelines for drug repositioning: a paradigm in ensemble analysis for neurodegenera

April 6th 2017, Posted by: Drug Repurposing Portal

When faced with time- and money-consuming problems, new practices in pharmaceutical R&D arose when trying to alleviate them. Drug repositioning has great promise and when combined with today's computational power and intelligence it becomes more precise and potent. This work showcases current approaches of creating a computational pipeline for drug repositioning, along with an extensive example of how researchers can influence therapeutic approaches and further understanding, through either single or multiple disease studies. This paradigm is based on three neurodegenerative diseases with pathophysiological similarities. It is our goal to provide the readers with all the information needed to enrich their research and note expectations along the way.


News Methods Funding Collaborations

Repurposed Diabetes Drug Shows Promise as Breast Cancer Treatment

April 6th 2017, Posted by: Drug Repurposing Portal

According to a study published by the journal of experimental medicine, an approved diabetes drug, epalrestat, which is an AKR1B1 inhibitor, may be able to prevent metastasis in patients with aggressive, basal-like triple-negative breast cancer. In this study, authors discovered that basal-like triple-negative breast cancers had high levels of AKR1B1 expression, which was linked to high rates of metastasis and poor patient outcomes.


News Methods Funding Collaborations

Repurposing 2 autoimmune drugs for chikungunya virus

April 5th 2017, Posted by: Drug Repurposing Portal

It has been demonstrated by Teck-Hui Teo and team that chikungunya virus infected mice given multiple sclerosis therapy Fingolimid, which prevents T cells from leaving the lymph nodes, experienced less joint inflammation. Corroborating these results further, Jonathan Miner and colleagues found that the rheumatoid arthritis drug Abatacept, which hinders T cell activation in the lymph nodes, decreased inflammation in the joints of infected mice, and abolished arthritis completely when given in combination with an antibody targeting the virus.


News Methods Funding Collaborations

IntelGenx Presents at the 13th International Conference on Alzheimer's and Parkinson's Diseases

April 5th 2017, Posted by: Drug Repurposing Portal

IntelGenx Technologies Corp, a leading oral drug delivery company presented its current research findings on repurposing Montelukast; an FDA approved Asthma drug for the treatment of Alzheimer's disease. IntelGenx?s proprietary Versafilm Technology platform improves the bioavailability of Montelukast rendering an enhanced blood brain barrier penetrance in a Phase I study conducted. The drug with its leukotriene receptor antagonistic action, might be effective in restoring brain cell function via reducing neuro-inflammation and the efficacy needs to be further demonstrated in a Phase II trial.


News Methods Funding Collaborations

Drug repurposing for castration resistant prostate cancer based on disease-disease rel

April 5th 2017, Posted by: Drug Repurposing Portal

Prostate cancer (PCa) ranks second in terms of cancer-related deaths among men in the United States. The primary cause is the emergence of castration resistant prostate cancer (CRPC) and subsequent metastasis and chemoresistance. There is no known cure for CRPC with a median survival rate of about 18 months. CRPC tumors are either intrinsically resistant or rapidly develop resistance to chemotherapy. Traditional approaches to drug discovery are highly specific to single targets (molecules and indications) and are time-consuming trial-and-error approaches, which are often ineffective and unsafe for humans. This approach has resulted in a dearth of novel drugs (currently less than thirty are approved each year) and the time and costs to develop and bring one to market are prohibitive ($2.6 billion in 2013). A solution is to repurpose existing drugs with known safety profiles that interact with therapeutic targets and can be rapidly deployed for use in mono and multi-drug therapies. However, there is a problem in consistently identifying high-confidence drugs for CRPC. Many computational tools have been developed for analyses of high-throughput genomics data, and these methods have led to improved understanding of cancer biology. However, despite the generation of enormous amounts of data, improved treatments and diagnostics take a long time, and a substantial amount of research is needed to translate a genomic discovery to the bedside. We have developed an integrated drug discovery/repurposing platform that analyzes compound-protein structural interaction signatures across multiple proteomes to determine drug behavior, in contrast to traditional single (or few) target approaches. The platform implements a modeling pipeline that generates an interaction between "all" (currently 3,733) human approved drugs and "all" (currently 48,278) proteins using our interactome docking with dynamics program to compute ~ 1 billion proteome-compound interactions. We have prospectively validated our predictions with in vitro and in vivo preclinical studies for more than 10 different diseases including immunological, metabolic, infectious and genetic indications. Recently, we applied our integrated drug discovery pipeline and found strong relationship between CRPC with breast cancer, hypertension and inflammation to repurpose human approved drugs for CRPC. We have newly identified an FDA approved drug with IC50 less than 10nM activity on LnCaP and C4-2 cell proliferation. The repurposed lead is tissue specific and its toxicity is very less compared to other chemotherapeutic agents used for cancer. Using our computational design approach of taking into account target and anti-target binding profile we are modifying and synthesizing new analogues of this potent anti-cancer human approved drug for immunomodulation without effecting anti-cancer potency. We conclude that compared to traditional single target drug discovery that is slow and error prone, interactome based d


News Methods Funding Collaborations

Mendelian randomization: a novel approach for the prediction of adverse drug events and drug repurposing

April 5th 2017, Posted by: Drug Repurposing Portal

Identification of unintended drug effects, specifically drug repurposing opportunities and adverse drug events, maximizes the benefit of a drug and protects the health of patients. However, current observational research methods are subject to several biases. These include confounding by indication, reverse causality, and missing data. We propose that Mendelian randomization (MR) offers a novel approach for the prediction of unintended drug effects. In particular, we advocate the synthesis of evidence from this method and other approaches, in the spirit of triangulation, to improve causal inferences concerning drug effects. MR overcomes some of the limitations associated with the existing methods in this field. Furthermore, it can be applied either pre- or post-approval of the drug and could therefore prevent the potentially harmful exposure of patients in clinical trials and beyond. The potential of MR as a pharmacovigilance and drug repurposing tool is yet to be realized and could both help prevent adverse drug events and identify novel indications for existing drugs in the future.


News Methods Funding Collaborations

Interferon Drug Shows Promise in Treating Ebola

April 4th 2017, Posted by: Drug Repurposing Portal

Interferon β-1a, used to treat hepatitis and some forms of multiple sclerosis has been shown for the first time to ease symptoms of Ebola patients in a pilot study conducted in 9 individuals with Ebola virus. The results were compared retrospectively with a matched cohort of 21 infected individuals receiving standardized supportive care only during the same time period at the same treatment centre in Guinea. 67 per cent of the interferon-treated patients were still alive at 21 days in contrast to 19 per cent of the former patients. Additionally, the viral blood clearance was faster in those patients treated with Interferon β-1a. Many clinical symptoms such as abdominal pain, vomiting, nausea and diarrhea were also relieved earlier in the interferon-treated patients.


News Methods Funding Collaborations

Computational Discovery of Niclosamide Ethanolamine, A Repurposed Drug Candidate That Reduces Growth

April 3rd 2017, Posted by: Drug Repurposing Portal

Drug repositioning offers a shorter approval process than new drug development. The article describes large public datasets search for drug-induced gene expression signatures to identify agents that might be effective against hepatocellular carcinoma (HCC).


News Methods Funding Collaborations

On the Integration of In Silico Drug Design Methods for Drug Repurposing

March 23rd 2017, Posted by: Drug Repurposing Portal

Drug repurposing has become an important branch of drug discovery. Several computational approaches that help to uncover new repurposing opportunities and aid the discovery process have been put forward, or adapted from previous applications. A number of successful examples are now available. Overall, future developments will greatly benefit from integration of different methods, approaches and disciplines. Steps forward in this direction are expected to help to clarify, and therefore to rationally predict, new drug-target, target-disease, and ultimately drug-disease associations.


News Methods Funding Collaborations

Biomedical Catalyst: Developmental Pathway Funding Scheme (DPFS): Mar 2017

March 16th 2017, Posted by: Drug Repurposing Portal

The DPFS scheme is a key part of our Translational Research Strategy and supports the translation of fundamental discoveries toward benefits to human health. It funds the pre-clinical development and early clinical testing of novel therapeutics, devices and diagnostics, including ?repurposing? of existing therapies. The scheme supports academically led projects whose goals are to improve prevention, diagnosis, prognosis, or treatment of significant health needs, or that focus on developing research tools that increase the efficiency of developing interventions.


News Methods Funding Collaborations

Drug repurposing screen identifies lestaurtinib amplifies the ability of the poly (ADP-ribose) polymerase 1 inhibitor AG14361 to kill breast cancer associated gene-1 mutant and wild type breast cancer cells

March 16th 2017, Posted by: Drug Repurposing Portal

Lestaurtinib, which is an orally bioavailable multikinase inhibitor that has been used in clinical trials for myeloproliferative disorders and acute myelogenous leukemia, enhanced the activity of the potent PARPi AG14361 on breast cancer cell growth both in vitro and in vivo conditions. Lestaurtinib amplifies the ability of the PARP1 inhibitor AG14361 to kill BRCA1 mutant and wild-type breast cancer cells, at least in part, by inhibiting NF-?B signaling. Each of these drugs has been approved for several different cancers, and their combination treatment should be applicable for a breast cancer trial in the future.


News Methods Funding Collaborations

Bowel cancer medication could help combat early-onset Parkinson's disease

March 14th 2017, Posted by: Drug Repurposing Portal

A study, which has been published in Science Matters, suggests that folinic acid, which is used in medications to treat bowel cancer, can also protect neurons associated with Parkinson's disease in fruit flies.


News Methods Funding Collaborations

A standard database for drug repositioning

March 14th 2017, Posted by: Drug Repurposing Portal

A gold standard database, repoDB, that consists of both true positives (approved drugs), and true negatives (failed drugs) developed by Adam Brown and colleagues at Biomedical Department of Harvard Medical School. The team has made the full database and all code used to prepare it publicly available, and have developed a web application that allows users to browse subsets of the data. This could be accessed at- http://apps.chiragjpgroup.org/repoDB.


News Methods Funding Collaborations

Deep-Learning-Based Drug-Target Interaction Prediction

March 13th 2017, Posted by: Drug Repurposing Portal

Wen and team from Central South University & Chinese Academy of Tropical Agricultural Sciences-China, have found a method to accurately predict new drug-target interactions (DTIs) between approved drugs and targets without separating the targets into different classes. The group has developed a deep-learning-based algorithmic framework named DeepDTIs which can be further used to predict whether a new drug targets to some existing targets or whether a new target interacts with some existing drugs.


News Methods Funding Collaborations

Non-kinase targets of protein kinase inhibitors

March 10th 2017, Posted by: Drug Repurposing Portal

Kinome-wide profiling platforms have comprehensively identified the relevant kinases that are targeted by numerous protein kinase inhibitors. However, recent projects have begun to discover non-kinase targets of kinase inhibitors. These non-kinase targets can contribute to the desired or undesired activities of inhibitors, or act as silent bystanders. As a full awareness of a drug's mechanism of action is crucial for the interpretation of results and for successful preclinical and clinical drug development, these discoveries highlight the importance of understanding the pharmacology of kinase inhibitors beyond the kinome. In this Review, I discuss kinase inhibitors for which non-kinase targets have been identified and the application of emerging techniques to validate drug?target engagement in intact cells.


News Methods Funding Collaborations

In vitro screening of an FDA-Approved Library against ESKAPE pathogens

March 9th 2017, Posted by: Drug Repurposing Portal

In an effort to repurpose drugs and explore new leads in the field of antimicrobial drug discovery, Younis et al at Purdue University College of Veterinary Medicine performed a whole-cell screening assay of 1,600 Food and Drug Administration (FDA) approved drugs against Enterococcus faecium, Staphylococcus aureus, Klebsiella pneumoniae, Acinetobacter baumannii, Pseudomonas aeruginosa, and Enterobacter cloacae (ESKAPE) pathogens. The in vitro screening identified 49 non-antimicrobial drugs that were active against at least one species of ESKAPE pathogen. Although some of these drugs were known to have antibacterial activity, many have never been reported before and Sulfonamide based structures were proposed for further investigation.


News Methods Funding Collaborations

Arthritis Drug Could Give Chemotherapy More Punch Against Triple-Negative Breast Cancer

March 8th 2017, Posted by: Drug Repurposing Portal

A combination of FDA approved drug for rheumatoid arthritis; leflunomide and Adriamycin effectively reduced the tumor volume in TNBC mice models compared to the treatment with both the drugs alone in a study conducted by Toker from Cancer Center at Beth Israel Deaconess Medical Center in Boston. A clinical trial is planned to test the efficacy and safety of these two drugs, suggesting a new drug repurposing treatment strategy for TNBC patients.


News Methods Funding Collaborations

Teaching old drugs new tricks - drug repurposing for rare disease

February 28th 2017, Posted by: Drug Repurposing Portal

Rick Thompson at FindACure provides a 360 degree overview on the drug repurposing space for Rare Disease. Topics covered include, how challenging it is to find a treatment for a given rare disease, what exists out there in the space in terms of medical options for patients, how drug repurposing helps here to identify opportunities to treat such devastating disease conditions and some success stories that the organization (FindACure) has identified.


News Methods Funding Collaborations

Identifying New Antiepileptic Drugs Through Genomics-Based Drug Repurposing

February 28th 2017, Posted by: Drug Repurposing Portal

Dr. Nasir Mirza, et al at the University of Liverpool has been able to identify new leads as potential treatment options for Epilepsy. The team is conducting a webinar on the results from their study where they have been able to implement GBR (genomics based drug repurposing) and identify 36 lead compounds to control seizures in patients.


News Methods Funding Collaborations

Construction of an miRNA-regulated drug-pathway network reveals drug repurposing candidates for myasthenia gravis

February 28th 2017, Posted by: Drug Repurposing Portal

Myasthenia gravis (MG) is a rare debilitating autoimmune neuromuscular disorder. Many studies have focused on the mechanism and treatment strategies of MG. However, the exact pathogenesis of MG and effective treatment strategies remain unclear. Recent studies have indicated that microRNAs (miRNAs or miRs) can regulate the pathological pathways of MG, suggesting their potential role in novel treatments. In the present study, the authors created a comprehensive catalog of experimentally confirmed MG risk genes and miRNAs by manually mining published literature and public databases. Based on these genes and miRNAs, they identified 41?MG risk pathways and 105?approved drugs that can affect these pathways. Some important MG-related pathways, such as hsa04060 (cytokine-cytokine receptor interaction) and hsa05200 (pathway in cancer), were found to be regulated by MG risk miRNAs and drugs. Furthermore, they constructed an miRNA-regulated drug-pathway network and identified miRNAs and drugs that synergistically regulate key MG pathways and biological processes. They developed a drug repurposing strategy to identify 25?drug repurposing candidates for MG; several of these drugs, such as rituximab, adalimumab, sunitinib, and muromonab, have the potential to be novel MG treatment drugs. This study provides novel insight into the pathogenesis of MG and potential drug candidates for MG were identified.


News Methods Funding Collaborations

Drug voyager: a computational platform for exploring unintended drug action

February 28th 2017, Posted by: Drug Repurposing Portal

This study addresses how we can computationally represent drug-signaling pathways to understand unintended drug actions and to facilitate drug discovery and screening. This is ?a novel platform to construct a drug-specific pathway in which a molecular-level mechanism of action is formulated based on pharmacologic, pharmacogenomic, transcriptomic, and phenotypic data related to drug response ( http://databio.gachon.ac.kr/tools/ )


News Methods Funding Collaborations

Computational Multitarget Drug Design

February 23rd 2017, Posted by: Drug Repurposing Portal

An overview of recent progress on finding drugs that can hit multiple targets for a complex disease. Such approach takes in to account features of both ligand and its different receptors. On successful application, these drugs have the potential to replace or reduce the requirements of multi-drug therapy.


News Methods Funding Collaborations

A Systematic Framework for Drug Repositioning from Integrated Omics and Drug Phenotype Profiles Using Pathway-Drug Network

February 23rd 2017, Posted by: Drug Repurposing Portal

Drug repositioning offers new clinical indications for old drugs. Recently, many computational approaches have been developed to repurpose marketed drugs in human diseases by mining various of biological data including disease expression profiles, pathways, drug phenotype expression profiles, and chemical structure data. However, despite encouraging results, a comprehensive and efficient computational drug repositioning approach is needed that includes the high-level integration of available resources. In this study, we propose a systematic framework employing experimental genomic knowledge and pharmaceutical knowledge to reposition drugs for a specific disease. Specifically, we first obtain experimental genomic knowledge from disease gene expression profiles and pharmaceutical knowledge from drug phenotype expression profiles and construct a pathway-drug network representing a priori known associations between drugs and pathways. To discover promising candidates for drug repositioning, we initialize node labels for the pathway-drug network using identified disease pathways and known drugs associated with the phenotype of interest and perform network propagation in a semi-supervised manner. To evaluate our method, we conducted some experiments to reposition 1309 drugs based on four different breast cancer datasets and verified the results of promising candidate drugs for breast cancer by a two-step validation procedure. Consequently, our experimental results showed that the proposed framework is quite useful approach to discover promising candidates for breast cancer treatment.


News Methods Funding Collaborations

Human enterovirus 71 protein interaction network prompts antiviral drug repositioning

February 21st 2017, Posted by: Drug Repurposing Portal

Lu et al of Beijing Institute of Radiation Medicine used a combination of experimental and computational approaches to identify interactions between EV71 proteins and host cellular proteins and confirmed the functional relationships of EV71-interacting proteins (EIPs) with virus proliferation and infection by integrating a human protein interaction network and by functional annotation. The team predicted tanespimycin as a candidate and demonstrated its antiviral efficiency in vitro. These findings provide the first systematic identification of EV71-host protein interactions, an analysis of EIP protein characteristics and a demonstration of their value in developing host-oriented antiviral therapies.


News Methods Funding Collaborations

Systematic analyses of drugs and disease indications in RepurposeDB reveal pharmacological, biological and epidemiological factors influencing drug repositioning

February 15th 2017, Posted by: Drug Repurposing Portal

Drug repositioning, the discovery of new or improved therapies by reevaluation of approved or investigational compounds, solves a significant gap in the public health setting and improves the productivity of drug development. Increase in global population and growing disease burden due to the emergence of infectious diseases (Zika virus), multidrug-resistant pathogens, drug-resistant cancers (cisplatin-resistant ovarian cancer) and chronic diseases (arterial hypertension) necessitate effective therapies to improve health outcomes. Collectively, RepurposeDB is developed as the reference database for drug repositioning investigations. The pharmacological, biological and epidemiological principles of drug repositioning identified from the meta-analyses could augment therapeutic development


News Methods Funding Collaborations

NCATS Seeks Applications to Repurpose Existing Drugs

February 15th 2017, Posted by: Drug Repurposing Portal

In collaboration with AstraZeneca and Janssen Research & Development, LLC, NCATS is seeking applications through its NIH-Industry Partnerships initiative to explore new treatments for patients, using existing experimental drugs or biologics. NCATS plans to commit an estimated $6 million to fund six to 10 awards to support partnerships between the pharmaceutical companies and the biomedical research community by making a selection of industry assets available to test ideas for new therapeutic uses.


News Methods Funding Collaborations

German Biotech gets Seed Funding to Repurpose Cancer Drugs for the Flu

February 8th 2017, Posted by: Drug Repurposing Portal

Atriva Therapeutics was founded in T?bingen, Germany in 2015 with a quite particular aim: repurposing cancer drugs to treat influenza. Two private investors, the Dutch Stichting Participatie Atriva and the German High-Tech Gr?nderfonds (HTGF) have contributed with an undisclosed amount of seed funding that will help the young biotech push its first drug candidate to the clinic. Atriva?s lead candidate, ATR-002, is expected to enter clinical trials in 2018. The drug is intended for treating influenza in high-risk patients with severe respiratory complications due to bacterial co-infections


News Methods Funding Collaborations

Brain Tumor Cells Sensitive to FDA-approved Ovarian Cancer Drug, Study Finds

February 3rd 2017, Posted by: Drug Repurposing Portal

Oaparib, a PARP inhibitor already approved for ovarian cancer treatment showed a 50 fold increased death of brain tumor cells with IDH1 and IDH2 gene mutations in a study conducted by scientists from Yale University. A clinical trial study for repurposing drugs like oaparib for the treatment of brain tumors is also planned by the team in future.


News Methods Funding Collaborations

A Pinworm Medication Is Being Tested As A Potential Anti-Cancer Drug

January 30th 2017, Posted by: Drug Repurposing Portal

Cancer researchers are testing whether a generic drug that has been used for more than 40 years to treat parasitic infections may also help fight cancer. The tests of mebendazole are part of a growing effort to take a fresh look at old medicines to see if they can be repurposed for new uses. Gregory Riggins, a researcher at Johns Hopkins University, discovered that laboratory mice didn't develop cancer after being given a drug for pinworms.


News Methods Funding Collaborations

DeSigN: connecting gene expression with therapeutics for?drug repurposing?and development

January 25th 2017, Posted by: Drug Repurposing Portal

The?drug?discovery and development pipeline is a long and arduous process that inevitably hampers rapid?drug?development. Therefore, strategies to improve the efficiency of?drug?development are urgently needed to enable effective drugs to enter the clinic. Precision medicine has demonstrated that genetic features of cancer cells can be used for predicting?drug?response, and emerging evidence suggest that gene-drug?connections could be predicted more accurately by exploring the cumulative effects of many genes simultaneously.


News Methods Funding Collaborations

Pharmacophore-based screening and drug repurposing exemplified on glycogen synthase kinase-3 inhibitors

January 21st 2017, Posted by: Drug Repurposing Portal

Study by Crisan et al elaborate a novel pharmacophore model to accurately map selective glycogen synthase kinase-3 (GSK-3) inhibitors, and perform virtual screening and drug repurposing. Pharmacophore modeling was developed using PHASE on a data set of 203 maleimides. Two benchmarking validation data sets with focus on selectivity were assembled using ChEMBL and PubChem GSK-3 confirmatory assays. A drug repurposing experiment linking pharmacophore matching with drug information originating from multiple data sources was performed. A five-point pharmacophore model was built consisting of a hydrogen bond acceptor (A), hydrogen bond donor (D), hydrophobic (H), and two rings (RR). An atom-based 3D quantitative structure-activity relationship (QSAR) model showed good correlative and satisfactory predictive. The pharmacophore and 3D QSAR models can provide assistance to design novel, potential GSK-3 inhibitors with high potency and selectivity pattern, with potential application for the treatment of GSK-3-driven diseases. A class of purine nucleoside antileukemic drugs was identified as potential inhibitor of GSK-3, suggesting the reassessment of the target range of these drugs.


News Methods Funding Collaborations

Machine Vision Helps Spot New Drug Treatments

January 21st 2017, Posted by: Drug Repurposing Portal

A startup uses algorithms that understand the anatomy of cells to discover new uses for existing drugs.


News Methods Funding Collaborations

Link prediction in drug-target interactions network using similarity indices

January 21st 2017, Posted by: Drug Repurposing Portal

In silico drug-target interaction (DTI) prediction plays an integral role in drug repositioning. One popular method of drug repositioning is network-based DTI prediction, which uses complex network theory to predict DTIs from a drug-target network. DTI prediction of this algorithm makes use of only network topology information yield higher precision for high-ranking predictions than Restricted Boltzmann Machines (RBM) when no information regarding DTI types is available.


News Methods Funding Collaborations

The clinically approved antiviral drug sofosbuvir inhibits Zika virus replication

January 18th 2017, Posted by: Drug Repurposing Portal

Zika virus (ZIKV) is a member of the Flaviviridae family, along with other agents of clinical significance such as dengue (DENV) and hepatitis C (HCV) viruses. Since ZIKV causes neurological disorders during fetal development and in adulthood, antiviral drugs are necessary. Sofosbuvir is clinically approved for use against HCV and targets the protein that is most conserved among the members of the Flaviviridae family, the viral RNA polymerase. Indeed, we found that sofosbuvir inhibits ZIKV RNA polymerase, targeting conserved amino acid residues. Sofosbuvir inhibited ZIKV replication in different cellular systems, such as hepatoma (Huh-7) cells, neuroblastoma (SH-Sy5y) cells, neural stem cells (NSC) and brain organoids. In addition to the direct inhibition of the viral RNA polymerase, we observed that sofosbuvir also induced an increase in A-to-G mutations in the viral genome. Together, our data highlight a potential secondary use of sofosbuvir, an anti-HCV drug, against ZIKV.


News Methods Funding Collaborations

Increasing Niacin Intake May Benefit Parkinson's Patients

January 14th 2017, Posted by: Drug Repurposing Portal

Recent research in fruit flies from Dr. Miguel Martins, of the University of Leicester suggests eating a niacin-rich diet could benefit people with early-onset Parkinson's disease by boosting levels of the compound NAD, which is vital for keeping mitochondria healthy. Niacin, also known as vitamin B3, is made into NAD in the body and can be found in foods such as nuts and meat. Parkinson's disease occurs when dopaminergic neurons in a part of the brain called the substantia nigra are lost, said study leader Dr. Miguel Martins, of the University of Leicester, said in a statement. This can happen for a variety of reasons, but in some hereditary cases, the main problem is unhealthy mitochondria the organelles that power the cell.


News Methods Funding Collaborations

Construction of an miRNA-regulated drug-pathway network reveals drug repurposing candidates for myasthenia gravis

January 11th 2017, Posted by: Drug Repurposing Portal

Myasthenia gravis (MG) is a rare debilitating autoimmune neuromuscular disorder. Many studies have focused on the mechanism and treatment strategies of MG. However, the exact pathogenesis of MG and effective treatment strategies remain unclear. Recent studies have indicated that microRNAs (miRNAs or miRs) can regulate the pathological pathways of MG, suggesting their potential role in novel treatments. In the present study, we created a comprehensive catalog of experimentally confirmed MG risk genes and miRNAs by manually mining published literature and public databases. Based on these genes and miRNAs, we identified 41 MG risk pathways and 105 approved drugs that can affect these pathways. Some important MG-related pathways, such as hsa04060 (cytokine-cytokine receptor interaction) and hsa05200 (pathway in cancer), were found to be regulated by MG risk miRNAs and drugs. Furthermore, we constructed an miRNA-regulated drug-pathway network and identified miRNAs and drugs that synergistically regulate key MG pathways and biological processes. We developed a drug repurposing strategy to identify 25 drug repurposing candidates for MG; several of these drugs, such as rituximab, adalimumab, sunitinib, and muromonab, have the potential to be novel MG treatment drugs. This study provides novel insight into the pathogenesis of MG and potential drug candidates for MG were identified.


News Methods Funding Collaborations

Repurposing an antidandruff agent to treating cancer: zinc pyrithione inhibits tumor growth via targeting proteasome-associated deubiquitinases

January 10th 2017, Posted by: Drug Repurposing Portal

Zhao et al report that zinc pyrithione (ZnPT) targets the proteasome-associated DUBs (USP14 and UCHL5) and inhibits their activities, resulting in a rapid accumulation of protein-ubiquitin conjugates, but without inhibiting the proteolytic activities of 20S proteasomes. Furthermore, ZnPT exhibits cytotoxic effects against various cancer cell lines in vitro, selectively kills bone marrow cells from leukemia patients ex vivo, and efficiently inhibits the growth of lung adenocarcinoma cancer cell xenografts in nude mice. The ubiquitin-proteasome system (UPS) plays a central role in various cellular processes through selectively degrading proteins involved in critical cellular functions. Targeting UPS has been validated as a novel strategy for treating human cancer, as inhibitors of the 20S proteasome catalytic activity are currently in clinical use for treatment of multiple myeloma and other cancers, and the deubiquitinase activity associated with the proteasome is also a valid target for anticancer agents. This study has identified zinc pyrithione, an FDA-approved pharmacological agent with potential antitumor properties as a proteasomal DUB inhibitor.


News Methods Funding Collaborations