Why NCATS’ New Program Can Make a Difference (UPDATED)

Earlier this month, we wrote about the exciting new program that was announced at the National Institutes of Health’s National Center for Advancing Translational Sciences. The program takes abandoned compounds developed by the pharmaceutical industry and enlists academia to see if there are new uses for those compounds.

The hope, NIH Director Francis Collins, MD, PhD, said at the time, was to shorten the time from discovery to application. And that’s just one of the benefits; our polling has consistently shown that the public believes collaboration across sectors is a good thing. Moreover, there is knowledge to be gained by putting these compounds to the test; maybe one is a dud across the tests, but something discovered sparks another discovery down the line.

But this isn’t just pie-in-the-sky thinking.

Last week, the Los Angeles Times reported on a nearly parallel process that could pay big dividends in global health. According to science writer Thomas H. Maugh II, PhD, a team of researchers – led by James McKerrow, PhD, MD, and Anjan Debnath, PhD, both of the University of California, San Francisco, developed a process of screening drugs in the lab for amoebic dysentery. During their research, they were able to get their hands on 900 compounds developed by Iconix Biosciences, which was going out of business.

Debnath and McKerrow found that one compound was particularly effective against not only amoebic dysentery, but also giardiasis; its previous function had been to treat arthritis. Its potential for impact is significant: The compound, auranofin, was found to be 10 times more effective than the current drug of choice, and could cost as little as $2.50 per dose. (And if you think that matters only for the developing world, remember this: The most recent Centers for Disease Control and Prevention report focusing on waterborne disease outbreaks and other health events associated with recreational water, published in September 2011, found that in 2007-2008, 38 cases of giardiasis occurred in four states. That’s not a high number – unless you’re on of the 38.)

Maugh writes that the compound has received orphan drug status from the Food and Drug Administration, which will accelerate testing. Clinical trials are being organized to test the drug in humans.

It happened with auranofin. There’s no reason not to believe it can’t work with the NCATS program.

UPDATE: If you’re looking for another example, Reuters has one in a story published Thursday on new therapies for autism. One new therapy, developed by Cambridge, MA-based Seaside Therapeutics, includes an active ingredient from baclofen, a drug that has been used since the 1960s to treat cerebral palsy. That ingredient has been found to work particularly well against Fragile X syndrome, the most common known genetic cause of autism.

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