TRND began five pilot projects to establish the proof-of-principle and operating protocols for the program soon after it was established in 2009. Those projects include potential treatments for the neurodegenerative disease Niemann-Pick type C, the neuromuscular disorder hereditary inclusion body myopathy, the blood disorder sickle cell disease, a rare blood cancer known as chronic lymphocytic leukemia, and the parasitic worm diseases schistosomiasis and hookworm. The chronic lymphocytic leukemia and the sickle cell disease projects have recently received investigational new drug approval from the FDA and are in clinical trials.
Earlier this year, TRND approved its first four drug development projects from its initial solicitation. They focus on potential new treatments for Duchenne muscular dystrophy, a degenerative muscle disorder; fragile X syndrome, the most common inherited form of cognitive and developmental disabilities; cryptococcal meningitis, an infectious fungal disease; and core binding factor leukemia, a rare blood and bone marrow cancer.
"TRND selects projects based on their potential to move forward into human trials and transform patient care in diseases for which there is little or no therapy," said Christopher P. Austin, M.D., scientific director of the NIH Center for Translational Therapeutics, which oversees TRND and is administered by the National Human Genome Research Institute (NHGRI). "While such projects are high-risk, the scientific opportunities and medical needs are compelling. After rigorous scientific review, the new projects were selected to maximize the chance of success and to teach us important generalizable lessons about rare and neglected disease drug development."
The latest TRND projects, approved by the advisory council of the National Institute of Neurological Disorders and Stroke (NINDS) on behalf of NIH, and the collaboratin
|Contact: Geoffrey Spencer|
NIH/National Human Genome Research Institute