NIH Injects New Moxie Into Drug Development

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Like making a new pair of shoes out of supple old leather, The National Center for Advancing Translational Sciences (NCATS) spearheads the newly minted endeavor by the National Institutes of Health (NIH) to speed the process of delivering new and re-purposed therapeutics to the bedside. This $575 million dollar Translational Research Center will be lead by newly appointed Director, and NIH insider, Dr. Christopher Austin, who you can read more about in this recent Science Magazine post.

Established in December 2011, NCATS focuses on several existing programs to achieve its challenging goals. First is the Office of Rare Diseases Research coupled with the Therapeutics for Rare and Neglected Diseases (TRND).  These programs promote research and therapeutics development for diseases that afflict too few people to be a profitable and attractive research venture for pharmaceutical companies to take on. TRND is likely to benefit from one of the core initiatives of NCATS, which will attempt to re-purpose drugs that have already been investigated thoroughly but may not have been effective against the original target of interest. In collaboration with eight biopharmaceutical juggernautsAbbott, AstraZeneca, Bristol-Myers Squibb, Eli Lilly, GlaxoSmithKline, Janssen Pharmaceutical Research and Development, Pfizer, and Sanofi — NCATS has launched a pilot program, Discovering New Therapeutic Uses for Existing Molecules, to find alternate uses for 58 well-studied and safety-tested compounds. Investigators were encouraged to identify potential new uses for these compounds and to submit proposals to NCATS for funding. A smaller contingent of proposals will be selected from this original pool to submit full grant applications for funding in December and awards will be announced in the summer or fall of 2013.

NCATS also encompasses the NCATS Pharmaceutical Collection (NPC) a publicly available library of 3,800 small molecular compounds licensed for clinical use that is available for high throughput screening to identify new therapeutic targets and pathways. Collaborations with the NPC are already underway and are yielding promising results. One study recently published in the ACS Chemical Biology journal screened compounds from the NPC to identify potential drugs that may be used to treat Charcot-Marie-Tooth disease (CMT), a genetic neurological disease with no current treatment protocols, and found three promising candidates that may be used to combat this condition.

NCATS has far from cornered the market on NIH resources available to extramural investigators to promote new clinical therapies. In fact, the NIH Clinical Center (CC) is also launching a new grant program that offers extramural investigators access to the CC’s vast research resources. The Opportunities for Collaborative Research at the NIH CC program is designed to foster partnerships between extramural and intramural NIH scientists to increase opportunities for discovery of new treatments in human disease.

These new NIH initiatives represent the type of Government – Industry innovation and collaboration that will harmonize efforts and move medical science forward to new levels.  The programs have the potential to act as a catalyst for changing the way therapeutic research is conducted and hopefully will lead to a more collaborative and productive means of bringing new therapies to patients worldwide.

Please check back for future updates on these programs in future posts.  In the meantime, below are some additional resources for you to learn more.

For more information on NCATS, see their Web site:

For more information on funding opportunities and collaborating with NCATS see:

For more information on NIH CC collaborations and funding see:

Who to follow on Twitter from this post:

@nih_nhlbi                            @NIHClinicalCntr

@ScienceNow                         @ORDR

@SanofiUS                              @JanssenUS

@AbbottNews                         @AstraZenicaUS

@BMSNews                            @LillyPad

@GSKUS                                  @PfizerNews




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