Drug Repurposing and Repositioning: Workshop Summary by Sarah H. Beachy

Author:Sarah H. Beachy
Language: eng
Format: epub
Publisher: The National Academies Press
Published: 2014-08-13T00:00:00+00:00

OVERCOMING BARRIERS TO DRUG REPURPOSING AT NCATS

The mission of NCATS is “to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions,”1 said Christine Colvis, director of extramural therapeutics discovery at NCATS. As part of that mission, NCATS has launched a Therapeutic Discovery Pilot program with the goal of identifying “new therapeutic uses of proprietary compounds and biologics across a broad range of human diseases in areas of medical need,” Colvis said.

Eight pharmaceutical company partners—AbbVie (formerly Abbot), AstraZeneca, BMS, Eli Lilly, GlaxoSmithKline, Janssen, Pfizer, and Sanofi—have collectively made 58 compounds available, Colvis said. All are in early stages of development and are not approved drugs, and in most cases academic researchers would typically have been unaware of the existence of these compounds. NCATS listed the compounds with their known mechanisms of action, original indications, route of administration, penetration into the central nervous system, safety and tolerability data, and clinical trial information. The intention was to provide enough information for a researcher to determine whether a drug might be appropriate for a disease of interest.

In order to be included in the program, a company needed to have at least three compounds it could offer, and each company had different philosophies and strategies for selecting compounds, Colvis said. In sending compounds to NCATS, BMS chose only compounds that were safe because it felt uncomfortable putting compounds with significant concerns about safety into the hands of people outside the company, given that BMS would have no control over how the drugs would be used, said Taylor. The company also chose compounds with enough patent life that if a positive study occurred, BMS would have the financial incentive to continue study of that compound, which would be needed for the compound to be manufactured and distributed. Only three compounds met these criteria, Taylor said, although the company has had far more than three failures in drug development.

The intent of the NCATS program was to match candidate agents from the pharmaceutical partners with members of the research community who have innovative ideas for using shelved compounds for new indications. Each match represents a three-part interaction among NIH, industry, and academic researchers. NIH provides template collaborative research agreements, confidential disclosure agreements, mechanisms for peer review, funding, and oversight of the program. The pharmaceutical partners provide therapeutic compounds and relevant drug data along with in-kind support. Academic research partners provide disease biology knowledge, new ideas for different drug indications, and access to patients. The program is designed to be a collaborative process that should ultimately benefit patients, Colvis emphasized, and it does not involve simply handing over compounds for study.

Crowdsourcing Ideas

The program’s goal in making the drug information publicly available, Colvis said, was to crowdsource the compounds in order to generate ideas about potential targets and indications for use. Within about 60 days NCATS received almost 160 pre-applications for potential new uses, many of which were for different indications using the same compound.

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