In July 2011, Danielle Kerkovich and Amy Drew wrote a Cerebrum essay for us on how to design a plan for discovering drugs that might work in cases of rare disease. Pharmaceutical firms can be unwilling to bet on a drug that might help only a few thousand people because it costs so much to develop a single drug and there's no guarantee a promising one will actually pan out. Meanwhile, bench scientists who do the basic research aren't equipped to translate it into a drug.
Kerkovich and Drew argued that researchers in academia and in the drug industry, with support from government and from nonprofits, should work together to promote translational research. The example they used was potential drug development for Batten disease, a rare and fatal disorder that affects young children. They write:
These nontraditional and direct efforts to foster collaborative research environments spanning from academia to industry may guide future approaches to crossing the dreaded valley of death. With the institutional framework and necessary tools in place thanks to heavy lifting by the NIH, FDA, and other organizations, nonprofit foundations now have the opportunity to construct a clear, navigable pathway that will empower researchers to turn an ordinary molecule into vital medicine.
Part of the challenge is to explain the idea and get buy-in from all sides. So besides the Cerebrum story, they recently created an animated video outlining their plan. It's called "Beyond the Valley of Death: A strategic plan toward a cure for Batten disease," and at less than five minutes long might be more appealing to non-readers: