In DepthBiomedicine

Battle over rare disease drug ensnares NIH

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Science  07 Oct 2016:
Vol. 354, Issue 6308, pp. 18-19
DOI: 10.1126/science.354.6308.18

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A bitter fight involving the National Institutes of Health (NIH), two companies, and opposing camps of researchers and parents is marring the emergence of a promising therapy for children with a rare, inherited, and uniformly fatal disorder of cholesterol metabolism. The 5-year-old National Center for Advancing Translational Sciences (NCATS), part of NIH, shepherded the early development of a cyclodextrin—a doughnut-shaped sugar molecule—as a treatment for Niemann-Pick type C-1 (NPC). Not long ago, NCATS handed the drug off to a small Maryland company, Vtesse, for further human trials and, hopefully, commercialization. But in a recent letter to NIH Director Francis Collins, copied to several members of Congress, CTD Holdings Inc., a disgruntled, competing, Florida-based firm, complains that the agency is "choosing winners and losers in the development programs of private enterprises." Also at issue—and dividing parents and scientists—is whether the Florida firm's intravenous-only delivery of its competing cyclodextrin, now moving into clinical trials, is appropriate. Cyclodextrins do not cross the blood-brain barrier, and it is almost always brain damage that kills the young victims of NPC. But in studies of mouse models of the disease, cyclodextrins, even when administered peripherally, have significantly impacted the neurological damage; it's not known how.