Drug repurposing: progress, challenges and recommendations

Phosphatidylinositol 3-Kinase α–Selective Inhibition With Alpelisib (BYL719) in PIK3CA-Altered Solid Tumors: Results From the First-in-Human Study

(2018) Dejan Juric et al.   JOURNAL OF CLINICAL ONCOLOGY

Market watch: Top drugs and companies by sales in 2017

(2018) Lisa Urquhart   NATURE REVIEWS DRUG DISCOVERY

Reverse Screening Methods to Search for the Protein Targets of Chemopreventive Compounds

(2018) Hongbin Huang et al.   Frontiers in Chemistry

New human gene tally reignites debate

(2018) Cassandra Willyard   NATURE

Targeted therapy in patients with PIK3CA-related overgrowth syndrome

(2018) Quitterie Venot et al.   NATURE

Genetic regulation of gene expression in the epileptic human hippocampus

(2017) Nasir Mirza et al.   HUMAN MOLECULAR GENETICS

Neurokinin 3 receptor antagonism as a novel treatment for menopausal hot flushes: a phase 2, randomised, double-blind, placebo-controlled trial

(2017) Julia K Prague et al.   LANCET

Association of genetic variation in the tachykinin receptor 3 locus with hot flashes and night sweats in the Womenʼs Health Initiative Study

(2017) Carolyn J. Crandall et al.   MENOPAUSE-THE JOURNAL OF THE NORTH AMERICAN MENOPAUSE SOCIETY

Non-kinase targets of protein kinase inhibitors

(2017) Lenka Munoz   NATURE REVIEWS DRUG DISCOVERY

Deal watch: Neurokinin 3 receptor antagonist revival heats up with Astellas acquisition

(2017) Megan Cully   NATURE REVIEWS DRUG DISCOVERY

Opportunities and challenges in phenotypic drug discovery: an industry perspective

(2017) John G. Moffat et al.   NATURE REVIEWS DRUG DISCOVERY

A network integration approach for drug-target interaction prediction and computational drug repositioning from heterogeneous information

(2017) Yunan Luo et al.   Nature Communications

Rho Kinase Inhibition with Fasudil in the SOD1G93A Mouse Model of Amyotrophic Lateral Sclerosis—Symptomatic Treatment Potential after Disease Onset

(2017) René Günther et al.   Frontiers in Pharmacology

Discovery of Novel Small Molecules that Activate Satellite Cell Proliferation and Enhance Repair of Damaged Muscle

(2016) Andrew N. Billin et al.   ACS Chemical Biology

Chemical Proteomics Reveals Ferrochelatase as a Common Off-target of Kinase Inhibitors

(2016) Susan Klaeger et al.   ACS Chemical Biology

A Biomarker Harvest from One Thousand Cancer Cell Lines

(2016) Yu-Han Huang et al.   CELL

A Landscape of Pharmacogenomic Interactions in Cancer

(2016) Francesco Iorio et al.   CELL

IBM Watson: How Cognitive Computing Can Be Applied to Big Data Challenges in Life Sciences Research

(2016) Ying Chen et al.   CLINICAL THERAPEUTICS

The hVps34‐SGK3 pathway alleviates sustained PI3K/Akt inhibition by stimulating mTORC1 and tumour growth

(2016) Ruzica Bago et al.   EMBO JOURNAL

Pathway and network-based strategies to translate genetic discoveries into effective therapies

(2016) Casey S. Greene et al.   HUMAN MOLECULAR GENETICS

A phenome-wide association study of a lipoprotein-associated phospholipase A2loss-of-function variant in 90 000 Chinese adults

(2016) Iona Y Millwood et al.   INTERNATIONAL JOURNAL OF EPIDEMIOLOGY

Effects of Src-kinase inhibition in cancer-induced bone pain

(2016) Milena De Felice et al.   Molecular Pain

China embraces precision medicine on a massive scale

(2016) David Cyranoski   NATURE

Can you teach old drugs new tricks?

(2016) Nicola Nosengo   NATURE

Identification of small-molecule inhibitors of Zika virus infection and induced neural cell death via a drug repurposing screen

(2016) Miao Xu et al.   NATURE MEDICINE

Connectivity mapping (ssCMap) to predict A20-inducing drugs and their antiinflammatory action in cystic fibrosis

(2016) Beth Malcomson et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Integrative methods for analyzing big data in precision medicine

(2016) Vladimir Gligorijević et al.   PROTEOMICS

Extraction and analysis of signatures from the Gene Expression Omnibus by the crowd

(2016) Zichen Wang et al.   Nature Communications

Crizotinib inhibits NF2-associated schwannoma through inhibition of focal adhesion kinase 1

(2016) Scott Troutman et al.   Oncotarget

The use of cellular thermal shift assay (CETSA) to study Crizotinib resistance in ALK-expressing human cancers

(2016) Abdulraheem Alshareef et al.   Scientific Reports

The 100 000 Genomes Project

(2016) Mark Peplow   BMJ-British Medical Journal

The 100 000 Genomes Project

(2016) Mark Peplow   BMJ-British Medical Journal

Rapid antimicrobial susceptibility test for identification of new therapeutics and drug combinations against multidrug-resistant bacteria

(2016) Wei Sun et al.   Emerging Microbes & Infections

Identify potential drugs for cardiovascular diseases caused by stress-induced genes in vascular smooth muscle cells

(2016) Chien-Hung Huang et al.   PeerJ

Fyn inhibition rescues established memory and synapse loss in Alzheimer mice

(2015) Adam C. Kaufman et al.   ANNALS OF NEUROLOGY

Role of autophagy in the pathogenesis of amyotrophic lateral sclerosis

(2015) Jae Keun Lee et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Novel therapeutics for coronary artery disease from genome-wide association study data

(2015) Mani P Grover et al.   BMC Medical Genomics

Structures of G protein-coupled receptors reveal new opportunities for drug discovery

(2015) Robert M. Cooke et al.   DRUG DISCOVERY TODAY

Drugs that reverse disease transcriptomic signatures are more effective in a mouse model of dyslipidemia

(2015) A. Wagner et al.   Molecular Systems Biology

Big data: The power of petabytes

(2015) Michael Eisenstein   NATURE

Comprehensive characterization of the Published Kinase Inhibitor Set

(2015) Jonathan M Elkins et al.   NATURE BIOTECHNOLOGY

Understanding multicellular function and disease with human tissue-specific networks

(2015) Casey S Greene et al.   NATURE GENETICS

Pioneering government-sponsored drug repositioning collaborations: progress and learning

(2015) Donald E. Frail et al.   NATURE REVIEWS DRUG DISCOVERY

An analysis of the attrition of drug candidates from four major pharmaceutical companies

(2015) Michael J. Waring et al.   NATURE REVIEWS DRUG DISCOVERY

Methods of integrating data to uncover genotype–phenotype interactions

(2015) Marylyn D. Ritchie et al.   NATURE REVIEWS GENETICS

A New Initiative on Precision Medicine

(2015) Francis S. Collins et al.   NEW ENGLAND JOURNAL OF MEDICINE

GABA and GABA receptors in the gastrointestinal tract: from motility to inflammation

(2015) Michelangelo Auteri et al.   PHARMACOLOGICAL RESEARCH

Harnessing Connectivity in a Large-Scale Small-Molecule Sensitivity Dataset

(2015) B. Seashore-Ludlow et al.   Cancer Discovery

Drug Signature-based Finding of Additional Clinical Use of LC28-0126 for Neutrophilic Bronchial Asthma

(2015) Eunji Shin et al.   Scientific Reports

Repurpose terbutaline sulfate for amyotrophic lateral sclerosis using electronic medical records

(2015) Hyojung Paik et al.   Scientific Reports

A phase Ib multiple ascending dose study of the safety, tolerability, and central nervous system availability of AZD0530 (saracatinib) in Alzheimer’s disease

(2015) Haakon B Nygaard et al.   Alzheimers Research & Therapy

Extracting research-quality phenotypes from electronic health records to support precision medicine

(2015) Wei-Qi Wei et al.   Genome Medicine

Unravelling druggable signalling networks that control F508del-CFTR proteostasis

(2015) Ramanath Narayana Hegde et al.   eLife

A Semi-Supervised Approach for Refining Transcriptional Signatures of Drug Response and Repositioning Predictions

(2015) Francesco Iorio et al.   PLoS One

Neurokinin B Administration Induces Hot Flushes in Women

(2015) Channa N. Jayasena et al.   Scientific Reports

Src Kinase Is a Novel Therapeutic Target in Lymphangioleiomyomatosis

(2014) A. Tyryshkin et al.   CANCER RESEARCH

Seeding Collaborations to Advance Kinase Science with the GSK Published Kinase Inhibitor Set (PKIS)

(2014) David Drewry et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Topiramate Use Does Not Reduce Flares of Inflammatory Bowel Disease

(2014) Seth D. Crockett et al.   DIGESTIVE DISEASES AND SCIENCES

Reverse docking: a powerful tool for drug repositioning and drug rescue

(2014) Prashant S Kharkar et al.   Future Medicinal Chemistry

Effect of Darapladib on Major Coronary Events After an Acute Coronary Syndrome

(2014) Michelle L. O’Donoghue et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Safety and efficacy of ceftriaxone for amyotrophic lateral sclerosis: a multi-stage, randomised, double-blind, placebo-controlled trial

(2014) Merit E Cudkowicz et al.   LANCET NEUROLOGY

Rituximab

(2014) Ulrich Storz   mAbs

NQO2 Is a Reactive Oxygen Species Generating Off-Target for Acetaminophen

(2014) Teemu P. Miettinen et al.   MOLECULAR PHARMACEUTICS

Darapladib for Preventing Ischemic Events in Stable Coronary Heart Disease

(2014)   NEW ENGLAND JOURNAL OF MEDICINE

Larval Zebrafish Model for FDA-Approved Drug Repositioning for Tobacco Dependence Treatment

(2014) Margot A. Cousin et al.   PLoS One

Network based elucidation of drug response: from modulators to targets

(2013) Francesco Iorio et al.   BMC Systems Biology

Fasudil, a rho kinase inhibitor, limits motor neuron loss in experimental models of amyotrophic lateral sclerosis

(2013) M Takata et al.   BRITISH JOURNAL OF PHARMACOLOGY

An Interactive Resource to Identify Cancer Genetic and Lineage Dependencies Targeted by Small Molecules

(2013) Amrita Basu et al.   CELL

Computational Drug Repositioning: From Data to Therapeutics

(2013) M R Hurle et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Rho kinase inhibition modulates microglia activation and improves survival in a model of amyotrophic lateral sclerosis

(2013) Lars Tönges et al.   GLIA

Rational drug repositioning by medical genetics

(2013) Zhong-Yi Wang et al.   NATURE BIOTECHNOLOGY

Monitoring Drug Target Engagement in Cells and Tissues Using the Cellular Thermal Shift Assay

(2013) D. M. Molina et al.   SCIENCE

Drug Repositioning: A Machine-Learning Approach through Data Integration

(2013) Francesco Napolitano et al.   Journal of Cheminformatics

DvD: An R/Cytoscape pipeline for drug repurposing using public repositories of gene expression data

(2012) Clare Pacini et al.   BIOINFORMATICS

Dynamic Reprogramming of the Kinome in Response to Targeted MEK Inhibition in Triple-Negative Breast Cancer

(2012) James S. Duncan et al.   CELL

Transcriptional data: a new gateway to drug repositioning?

(2012) Francesco Iorio et al.   DRUG DISCOVERY TODAY

Phase II study of the Src kinase inhibitor saracatinib (AZD0530) in metastatic melanoma

(2012) Tara C. Gangadhar et al.   INVESTIGATIONAL NEW DRUGS

Predicting New Indications for Approved Drugs Using a Proteochemometric Method

(2012) Sivanesan Dakshanamurthy et al.   JOURNAL OF MEDICINAL CHEMISTRY

The Cancer Cell Line Encyclopedia enables predictive modelling of anticancer drug sensitivity

(2012) Jordi Barretina et al.   NATURE

Cell lines battle cancer

(2012) John N. Weinstein   NATURE

NCATS launches drug repurposing program

(2012) Malorye Allison   NATURE BIOTECHNOLOGY

Use of genome-wide association studies for drug repositioning

(2012) Philippe Sanseau et al.   NATURE BIOTECHNOLOGY

A public-private partnership to unlock the untargeted kinome

(2012) Stefan Knapp et al.   Nature Chemical Biology

Diagnosing the decline in pharmaceutical R&D efficiency

(2012) Jack W. Scannell et al.   NATURE REVIEWS DRUG DISCOVERY

Mining electronic health records: towards better research applications and clinical care

(2012) Peter B. Jensen et al.   NATURE REVIEWS GENETICS

RASMutations in Cutaneous Squamous-Cell Carcinomas in Patients Treated with BRAF Inhibitors

(2012) Fei Su et al.   NEW ENGLAND JOURNAL OF MEDICINE

Identification of Common Biological Pathways and Drug Targets Across Multiple Respiratory Viruses Based on Human Host Gene Expression Analysis

(2012) Steven B. Smith et al.   PLoS One

Role for kisspeptin/neurokinin B/dynorphin (KNDy) neurons in cutaneous vasodilatation and the estrogen modulation of body temperature

(2012) M. A. Mittelman-Smith et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases

(2011) K. Xu et al.   BRIEFINGS IN BIOINFORMATICS

Exploiting drug-disease relationships for computational drug repositioning

(2011) J. T. Dudley et al.   BRIEFINGS IN BIOINFORMATICS

mRNA Expression Signatures of Human Skeletal Muscle Atrophy Identify a Natural Compound that Increases Muscle Mass

(2011) Steven D. Kunkel et al.   Cell Metabolism

Retrospective clinical analysis for drug rescue: for new indications or stratified patient groups

(2011) David Cavalla et al.   DRUG DISCOVERY TODAY

Activation of Neurokinin 3 Receptors in the Median Preoptic Nucleus Decreases Core Temperature in the Rat

(2011) Penny A. Dacks et al.   ENDOCRINOLOGY

China Kadoorie Biobank of 0.5 million people: survey methods, baseline characteristics and long-term follow-up

(2011) Z. Chen et al.   INTERNATIONAL JOURNAL OF EPIDEMIOLOGY

Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm

(2011) Paul Wicks et al.   NATURE BIOTECHNOLOGY

Comprehensive analysis of kinase inhibitor selectivity

(2011) Mindy I Davis et al.   NATURE BIOTECHNOLOGY

The productivity crisis in pharmaceutical R&D

(2011) Fabio Pammolli et al.   NATURE REVIEWS DRUG DISCOVERY

Is there still a future for neurokinin 3 receptor antagonists as potential drugs for the treatment of psychiatric diseases?

(2011) Guy Griebel et al.   PHARMACOLOGY & THERAPEUTICS

Systematic Drug Repositioning Based on Clinical Side-Effects

(2011) Lun Yang et al.   PLoS One

Computational Repositioning of the Anticonvulsant Topiramate for Inflammatory Bowel Disease

(2011) J. T. Dudley et al.   Science Translational Medicine

Discovery and Preclinical Validation of Drug Indications Using Compendia of Public Gene Expression Data

(2011) M. Sirota et al.   Science Translational Medicine

Drug-Resistant Aurora A Mutants for Cellular Target Validation of the Small Molecule Kinase Inhibitors MLN8054 and MLN8237

(2010) Dominic A. Sloane et al.   ACS Chemical Biology

Identification of small molecules enhancing autophagic function from drug network analysis

(2010) Francesco Iorio et al.   Autophagy

Genome-wide meta-analysis increases to 71 the number of confirmed Crohn's disease susceptibility loci

(2010) Andre Franke et al.   NATURE GENETICS

Fingolimod (FTY720): discovery and development of an oral drug to treat multiple sclerosis

(2010) Volker Brinkmann et al.   NATURE REVIEWS DRUG DISCOVERY

Discovery of drug mode of action and drug repositioning from transcriptional responses

(2010) F. Iorio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Molecular targeting of the oncogene eIF4E in acute myeloid leukemia (AML): a proof-of-principle clinical trial with ribavirin

(2009) S. Assouline et al.   BLOOD

sscMap: An extensible Java application for connecting small-molecule drugs using gene-expression signatures

(2009) Shu-Dong Zhang et al.   BMC BIOINFORMATICS

High-Throughput Cell-Based Screening of 4910 Known Drugs and Drug-like Small Molecules Identifies Disulfiram as an Inhibitor of Prostate Cancer Cell Growth

(2009) K. Iljin et al.   CLINICAL CANCER RESEARCH

Systematic Evaluation of Drug–Disease Relationships to Identify Leads for Novel Drug Uses

(2009) A P Chiang et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Predicting new molecular targets for known drugs

(2009) Michael J. Keiser et al.   NATURE

Long-Term Results From a Randomized Phase II Trial of Standard- Versus Higher-Dose Imatinib Mesylate for Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors Expressing KIT

(2008) Charles D. Blanke et al.   JOURNAL OF CLINICAL ONCOLOGY

A quantitative analysis of kinase inhibitor selectivity

(2008) Mazen W Karaman et al.   NATURE BIOTECHNOLOGY

Drug Target Identification Using Side-Effect Similarity

(2008) M. Campillos et al.   SCIENCE