NIH Program Helps Developers Navigate Path to New Drugs for Neglected Diseases

Abstract

COMPETITION TO CROSS THE SOcalled Valley of Death has become rather stiff at the National Institutes of Health (NIH). Since 2009, the NIH’s Therapeutics forRareandNeglectedDiseases(TRND) programhassolicitedproposals fromscientists and pharmaceutical companies tryingtonavigatethatdeadzonebetween discovering promising compounds and getting them into human clinical trials. To date, TRND has received 125 applications for nonmonetary assistance; 10 have gotten the green light. “What this reflects is the really substantial need for novel therapeutics for rare and neglected diseases but also enormous opportunities because we’ve seen a lot of really good science,” said Christopher Austin, MD, scientific director of the NIH Center for Translational Therapeutics, which oversees TRND. Rather than award grant money directly, the congressionally mandated TRND program partners with chosen applicants to determine the steps and studies needed to submit compounds to the US Food and Drug Administration (FDA) for investigational new drug approval. “We combine our money with our know-how,” said Austin. “We pay [contractors] for the studies or we do them ourselves.” In the world of drug development, TRND has a molecularlevel budget: $24 million. The most recent round of project approvals, announcedlate lastyear,will aid in developing new treatments for fibrodysplasiaossificansprogressiva(FOP),a connective tissuedisorder; autoimmune pulmonary alveolar proteinosis, which causes breathing problems and can lead to respiratory failure; creatinine transporterdeficiency,acognitivefunctiondisorder; schistosomiasis, a parasitic infection; neonatal herpes simplex virus; and Duchenne muscular dystrophy. “Wereallytriedtochooseprojectswith the greatest medical need and the greatest scientific opportunity,” said Austin.