The FULL agenda for the 7th Annual Drug Repositioning, Repurposing and Rescue Conference has not yet been announced. Please check back in the following weeks for updates or CLICK HERE to request a conference brochure.
Open Innovation - A Driver for Drug Repositioning
In this presentation, Dr. Hayes will discuss the rational for repositioning. He will also delve into the various features of AstraZeneca's Open Innovation program which supports repositioning efforts. Lastly, he will discuss ongoing internal and collaborative case studies.
David Hayes, Ph.D., Director, Translational Research Science, AstraZeneca
Repositioning is Easy - Or Is It? The impact of AI on Future Progress
There are several easy and quick and cheap ways to do repositioning. They generate results of variable quality and confidence, as the compromise for being quick/easy/cheap. Big healthcare data has the potential to help do repositioning very well, but not all approaches, including different AIs, are the same. Examples of matching the right repositioning problem with the right AI solution will be shared, including Biovista's Project Prodigy AI.
Aris Persidis, Ph.D., President, Biovista
Leveraging Human Genetics to Repurpose Drugs: Lessons Learned
Last year Dr. Lavieri presented an overview of how we are using the data housed within BioVU, a DNA biobank linked to a de-identified version of Vanderbilt University Medical Center’s electronic health record system, to generate hypotheses for drug repurposing projects. This year, he will share information on the four phase II studies their team is launching this year, and he also will also share lessons learned from several projects that did not advance to clinical development.
Robert R. Lavieri, Ph.D., Project Manager, Vanderbilt Institute for Clinical and Translational Research, Vanderbilt University Medical Center
The Drug Repurposing Hub: A Community Resource for Indication Discovery
A longstanding obstacle to drug repurposing is the lack of a comprehensive library of clinical compounds suitable for testing. To address this challenge, we developed the Drug Repurposing Hub, a hand-curated physical and online collection of more than 5,000 compounds. This talk will describe the development of this new resource and current efforts to test drugs across multiple cellular assays.
Steven Corsello, MD, Postdoctoral Fellow, Broad Institute, Instructor in Medicine, Dana-Farber Cancer Institute
Using Artificial Intelligence to Identify Potential Drug Repurposing Opportunities
BenevolentBio is focused on AI technology in the bioscience industries. The initial focus has been on human health – generating new ideas that have the potential to improve the lives of millions and deliver better medicines to patients faster in currently overlooked areas such as orphan diseases and rare cancers. We apply our AI to radically improve the efficiency of drug development, produce better target selection, optimize compounds and draw previously impossible insights from hundreds of millions of associations between biologically meaningful entities and unstructured text. We have developed a significant pipeline of drug candidate programs ranging from early-stage discovery assets to Phase IIb clinical development assets and are working with major pharmaceutical groups to license compounds and develop drugs. We also work closely with charities and other funders, especially in rare disease areas.
Bryn Williams Jones, Director of Exploratory Research, Benevolent AI
Application of In Silico Proteome Screening to Discover a Drug’s Polypharmacology to Facilitate Drug Repurposing
In this presentation, Dr. Shahani will discuss the following:
♦ Polypharmacology and its importance in drug discovery and particularly drug repositioning
♦ Technical overview of Cyclica’s in silico Proteome Screening approach and the data it generates
♦ Data from repurposing study of FDA approved drugs for treatment of Systemic scleroderma
♦ Additional data from separate study exploring the repurposing of an anti-clotting agent for treatment of cancer in combination with statin drugs
Vijay Shahani, Ph.D., Senior Solution Scientist, CyclicaRx
Nutraceuticals as Repositioning Agents: Rapid Acceleration from Bench to Clinical Trial
Nutraceuticals or dietary supplements are regulated compounds with demonstrated bioactivity. Leveraging existing government regulations and data highlighting safety and efficacy, nutraceuticals can be repurposed for several clinical indications. This talk will focus on strategies to rapidly move nutraceuticals from bench to bedside and will discuss how a novel avocado-derived lipid was quickly moved into a Phase I trail.
Paul Spagnuolo, Ph.D., Associate Professor, Department of Food Science, University of Guelph
A Systems Biology Driven Repositioning Strategy Identifies Digoxin for Treating Groups 3 and 4 Medulloblastoma
Medulloblastoma is the most common malignant brain tumor of childhood, treatments are sorely needed both to improve survival and reduce treatment related complications. Its sub-types, Groups 3 and 4, an unmet medical need, represent a particular challenge due to their intra-group heterogeneity, which limits the options for "rational" targeted therapies. We report a systems biology driven strategy that combines data from multiple genomic and epigenomic sources to construct novel driver signaling networks to accelerate the discovery of new treatments for cancers. From over 1,300 known drug candidates studied, we identified five members of the cardiac glycoside family potentially inhibit the growth of Groups 3 and 4 medulloblastoma, and subsequently confirmed digoxin, one of the five cardiac glycosides, with in vitro and in vivo studies.
Stephen Wong, Ph.D., P.E., Chief Research Information Officer and Professor and Chair, Department of Systems Medicine and Bioengineering, Houston Methodist
A Systems Medicine Approach to Drug Repurposing
The REPO-TRIAL investigates an unconventional strategy to improve the efficacy and precision of drug repurposing trials. Instead of applying classical disease definitions based on symptoms or organs, REPO-TRIAL uses a systems medicine based in-silico approach that identifies mechanistically related disease phenotypes and, as a result, a virtual patient cohort. Research animal use will be kept to a minimum by applying a preclinical randomised confirmatory trial (pRCTs) concept, preclinical systematic reviews, and meta-analyses facilitated by our open access pre-clinicaltrials.org platform. Selected results will be validated via high precision clinical trials in patients with cerebro-cardiovascular phenotypes stratified using an exclusive mechanistic biomarker panel. We thus innovate two biomedical product classes, drugs and diagnostics. REPO-TRIAL commenced in February 2018, and will conclude in January 2023.
Hermann Mucke, Ph.D., Principal, H.M. Pharma Consultancy
Systematic Drug (Re)Purposing by Triangulating Insights from Unstructured and Structured Data Using Machine Learning
Through a case study, Qrativ (joint venture between Mayo Clinic and nference) will describe how its software platform Darwin.ai can be used to extract and triangulate insights from unstructured data (e.g. text...) and structured data (e.g. genome sequencing, real world evidence...) in order to systematically identify drug (re)purposing opportunities.
Agustin J. Lopez Marquez, Vice President, Business Development and Marketing, nference, Qrativ
Expanding the Impact of Repurposing Research
Repurposing research is a growing and critical part of therapy discovery. Repurposing research can quickly provide affordable "new" therapies, with comparably lower research costs. Many repurposing research successes are patient driven and/or physician discovered, and new bioinformatics, phenotypic screening and global information sharing are further driving purposeful repurposing research opportunities. Cures Within Reach and our many industry, academic, philanthropic, clinical and advocacy partners are leading this Repurposing Research Revolution, and together we are developing new financial and collaborative initiatives to continue this growth. This presentation will focus on the future of repurposing research and how every stakeholder group must play a role.
Dr. Bruce Bloom, President & Chief Science Officer, Cures Within Reach
Clare Thibodeaux, Ph.D., Director of Scientific Affairs, Cures Within Reach
Repurposing Low-dose Methotrexate for the Treatment of Myeloproliferative Disease
We have previously found that low doses of the generic rheumatology drug methotrexate acts as a JAK/STAT pathway inhibitor. As such, MTX potentially represents a low cost treatment for diseases caused by over activation of the JAK/STAT pathway such as the myelproliferative blood cancers polycythemia vera and essential thrombocytosis. However, moving from preclinical and case study data to human clinical trials has proven to be far more difficult than originally imagined - even for the repurposing of a safe, low cost and widely available drug such as MTX. We have now partnered with a blood cancer charity to gain access to clinical trials unit expertise and a generics drug manufacturer to obtain orphan drug designation for this disease / drug combination. My talk will describe the scientific background to this story and will detail the hurdles we have faced, the solutions we have found and the prospects for repurposing success we anticipate.
Martin Zeidler, Ph.D., Reader, Cellular Signaling, Department of Biomedical Science, The University of Sheffield
From Toenail Fungus to Leukemia to Bladder Cancer – Overcoming Drug Development Obstacles to Bring a Promising New Treatment to Patients
Scott J. Weir, Pharm.D., Ph.D., Director, Institute for Advancing Medical Innovation, Associate Director, Translational Research, University of Kansas Cancer Center, Professor, Department of Pharmacology, Toxicology & Therapeutics, University of Kansas Medical Center
Multi-orthogonal Disruptive Integration (MODI) to Teach Old Drugs New Tricks
New cancer drug discovery is hampered by the persistent increase in failure rates caused by; lack of innovation, redundant targets and approaches, me too products, poor bioavailability, poor safety, limited efficacy, lengthy clinical trials and a daunting regulatory approval process costing nearly $2 billion. OncoStemyx has developed a rapid and safe multi-orthogonal disruptive integration platform that empirically identifies drug combinations for the treatment of many cancers. The application of this platform are limitless such as diabetes, heart disease, neurological disorders, age related diseases and many more. In the era of reducing prescription drug costs, innovative repositioning will precede all new drug development. This talk will outline the path to successfully reposition drugs, approval process, funding challenges, and advocacy.
Vikash Jaganath Bhagwandin, Ph.D., Founder and CEO, OncoStemyx
Big Data-based Drug Repurposing Strategy: Special Focus on Brain Ischemia
In this presentation, Dr. Casas will present her team's innovative in-silico based approach to improve the efficacy and precision of drug repurposing trials. This systems medicine-based in-silico approach identifies mechanistically related disease phenotypes followed by later validation of this in-silico drug repurposing approach via pre-clinical experimentation and clinical trials. Their approach has been validated in one of the highest prevalence unmet medical needs, brain ischemia. Surprisingly, her team finds that sGC, an hitherto exclusive cardiovascular target, is closely linked to neurological disorders, an application that has so far not been explored clinically. Indeed, we repurposed apo-sGC activators for ischemic stroke suggesting this drug class as a possible first-in-class neuroprotective therapy for stroke.
Ana Casas Guijarro, Ph.D., Department of Pharmacology and Personalised Medicine, Maastricht University