The National Institutes of Health (NIH) has begun funding a new pilot partnership program between pharmaceutical companies and basic researchers. The program's goal is for basic researchers to find new uses for drug compounds that were discovered by pharmaceutical companies but have not lived up to their intended use.
This pilot project is called "Discovering New Therapeutic Uses for Existing Molecules" and was originally launched in May 2012. Eight pharmaceutical companies provided 58 potential drugs for the pilot program, and the NIH solicited ideas for new uses of these compounds. On June 18th, the NIH funded nine grant awards—totaling $12.7 million dollars—to academic research groups who will test compounds for their potential to treat various diseases including alcoholism, nicotine dependence, Alzheimer's disease, Duchenne muscular dystrophy, calcific aortic valve stenosis, peripheral artery disease, and schizophrenia. You can learn more about the specific projects on this website. These grants will provide up to two years of funding for proof-of-concept preliminary experiments.
There are several advantages to this program. The molecules that will be tested by the basic researchers involved in the pilot projects have already undergone Phase I clinical trials and thus—if preliminary testing for a new disease treatment is promising—can be rapidly transitioned into further human clinical testing. NIH has other funding mechanisms in place to move these compounds to Phase IIa trials. Of course, this is a partnership, so the industry partners will benefit as well. According to the program's frequently asked questions, the collaborating company will have the first shot at licensing any new intellectual property that arises from the new academic research. If there is no longer an active patent on the molecule or the company decides to pass on further commercial development, the academic researcher will be free to find another company to work with.
This program seems like an interesting idea, and I'll be curious to see if the preliminary grants bear fruit. It's certainly a good deal for the pharmaceutical company partners—according to the NIH, it takes an average of 13 years to take a new drug to market and 95 percent of potential drug compounds never make it to the clinic. So any potential new therapeutic use for 'failed' compounds is a win for them. It's more of a high-risk, high-potential reward scenario for the researchers who will spend a great deal of time and effort testing these molecules. But even if just one of these compounds shows promising results for one target disease, it could be a huge boon for patients desperately in need of new treatment options.