Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease. The six new projects are part of the National Institutes of Health's Therapeutics for Rare and Neglected Diseases (TRND) program.
"These projects reinforce NIH's commitment to translational research and the need to accelerate potential new treatments that benefit patients with rare and neglected diseases," said NIH Director Francis S. Collins, M.D., Ph.D. "It is wonderful that TRND scientists and their collaborators can advance such promising projects that may have otherwise remained stalled due to a lack of scientific or fiscal resources."
Congress created the TRND program to facilitate the development of new drugs for rare and neglected diseases. TRND bridges the wide gap in expertise and resources that often exists between basic research discoveries and the development and testing of new drugs in human subjects. As it develops new treatments, TRND also conducts research aimed at improving the drug development process. In contrast to conventional drug development efforts, TRND will publish successes and failures so that the broader drug development community can benefit from the work done.
To develop new medicines, TRND establishes partnerships with academic, government, biopharmaceutical and patient advocacy groups. TRND and its collaborators focus on the optimization and pre-clinical development of new drugs, as needed, advancing them from Food and Drug Administration (FDA) investigational, new-drug approval to first, in-human clinical trials.
Typically, the success rate for such projects is low, with 80 to 90 percent of projects failing in the pre-clinical phase and never making it to clinical trials.
|Contact: Geoffrey Spencer|
NIH/National Human Genome Research Institute