January 2012

Forthcoming projects for NIH Therapeutics for Rare and Neglected Diseases Program

Researchers will embark on six new drug-development projects for rare and neglected diseases as part of the National Institutes of Health’s Therapeutics for Rare and Neglected Diseases(TRND) program. The treatments will target, among others, a musculoskeletal disorder, a tropical parasitic worm and a virus that affects the central nervous system of newborns.

The projects cover a broad range of illnesses. For example, Kenneth D. Bloch at the Harvard Medical School and Massachusetts General Hospital will lead a team that will analyze a compound’s use against fibrodysplasia ossificans progressive, a rare inherited disorder in which muscle and connective tissue get replaced by bone. The compound has been shown to be effective in a mouse model.

Julie F. Liu at Concert Pharmaceuticals Inc. will spearhead an initiative to produce compounds to thwart the parasitic Schistosoma worm, which infects more than 200 million people in tropical regions and causes severe anemia, diarrhea and internal bleeding.

David W. Kimberlin at the University of Alabama at Birmingham and colleagues will study candidate compounds to treat neonatal herpes simplex virus, an infection transmitted from mother to child during childbirth. Other projects focus on Duchenne muscular dystrophy, creatine transporter deficiency and a rare lung disease, pulmonary alveolar proteinosis.

TRND creates partnerships between academic, government, pharmaceutical and patient advocacy groups to develop new drugs for rare and orphan diseases. The collaborators don’t receive grants but get support, laboratory space and other resources from the NIH’s Office of Rare Diseases Research, which oversees the TRND program. The teams focus on the preclinical development of medicines, which involves getting U.S. Food and Drug Administration approval for investigating new drugs and culminates in clinical trials with the medicines.

Each project has to meet certain milestones. Projects that don’t hit their milestones are terminated to make way for others. Instances in which a project can be terminated include when the treatment doesn’t appear to be effective in animal models or when it shows toxicity in preclinical testing.

The next solicitation for TRND projects will open in the spring. Visit http://nctt.nih.gov/TRND for more information.

Raj_MukhopadhyayRajendrani Mukhopadhyay (rmukhopadhyay@asbmb.org) is the senior science writer for ASBMB Today and the technical editor for JBC.

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