American Society of Hematology

NCATS Launches Collaborative Program with Industry and Researchers to Spur Therapeutic Development, Seeks Feedback

Published on: May 08, 2012

The National Institutes of Health (NIH) unveiled a collaborative program that will match researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients. The initiative, Discovering New Therapeutic Uses for Existing Molecules, will direct researchers' attention to a part of the therapeutic pipeline that traditionally has been difficult for them to access: compounds that already have cleared several key steps in the development process, including safety testing in humans.

NIH's new National Center for Advancing Translational Sciences (NCATS) has partnered initially with Pfizer, AstraZeneca, and Eli Lilly and Company which have agreed to make about two dozen of their compounds available for this initiative's pilot phase. The compounds passed through preclinical testing and through phase I safety trials in humans, but were abandoned later for lack of efficacy or because the company moved out of a given therapeutic area. The companies will provide the researchers with access to the compounds and related data. The compounds, whose particular molecular targets are already mostly known, are not intended to be screened against a myriad of random targets as is often done in the first steps of the drug discovery process, but rather be studied as an intentionally designed connection between a drug and a disease.

Dr. Kathy Hudson, the acting deputy director of NCATS, who is the center's point person on the collaboration with $20 million budget, said that the NCATS expects to award eight to ten grants starting in 2013, each worth up to $2 million in the first year. During that year, a successful awardee will be expected to reach certain milestones in completing preclinical work, providing evidence of the biological rationale for the drug's new use. If these are met, the investigator will receive money for up to two additional years to undertake early phase II clinical trials establishing efficacy against the disease. The expectation is that success achieved in these early stages will draw a commercial sponsor interested in carrying the molecule through larger, more expensive, late-stage trials.

Early details for prospective applicants are available through the Notice of Intent announcement; more will appear in a request for applications that the NIH intends to publish in early June. The first application due date will be as soon as mid-July. Dr. Hudson expects that the award competition will involve two peer-reviewed stages: peer review of a project summary, and then peer review of a full proposal, which must include a signed agreement between the researcher and the company.

Feedback Requested 

In the meantime, NCATS is soliciting input from the biomedical research community, potential biotechnology and pharmaceutical company partners, and other members of the public on this proposed therapeutics discovery program and more broadly on how the government can partner with the private sector in this area. NCATS is particularly interested in receiving input on the following issues:

  1. Innovative strategies and practices that have proven successful to develop novel uses for discontinued agents that have no known development limitations and are safe for use in humans, including: most significant challenges for public-private partnerships; options NCATS can consider to nurture academic efforts to foster greater translation of drug discovery efforts; and identification of other partners that would like to provide drugs and biologics that are no longer being pursued internally to the NIH research community for investigation for new therapeutic uses.
  2. Views of potential academic and industry partners on the transaction cost of developing individual agreements as well as the desirability of using template agreements and incorporating them into this program. NCATS interested in comments on how the use of template CDAs and CRAs in general and the current CDA and CRA might affect one's institution's participation in the therapeutics discovery program. Applicants will be able to access these agreements prior to submitting an NIH X02 pre-application.
  3. Comments on how working with a Clinical and Translational Science Award site (CTSAs) could advance drug rescue research projects, particularly for rare and neglected diseases.
  4. Comments on how working with NIH Intramural Research Program investigators and the NIH Clinical Center resources could advance drug rescue research projects.
  5. Comments on whether the goals and incentives of the NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules are sufficient for biotechnology and pharmaceutical companies to participate in the program.
  6. Comments on the resources that a biotechnology or pharmaceutical company partner might realistically contribute to an NCATS program on therapeutics discovery in addition to the agents and the associated data (and the type of information about the molecules that a partner would be willing to disclose publicly).
  7. Comments on the pharmacologic activity or biological target of the drug candidate that the investigator needs access to in order to test his/her biological hypothesis of disease intervention.

ASH members are encouraged to submit responses directly to NCATS by June 1, 2012. If you have specific comments on these issues that you think should be submitted on behalf of the Society, please contact ASH Senior Manager for Scientific Affairs, Ulyana V. Desiderio, PhD, at or (202) 776-0544.

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