IMAGE:This image shows the cerebellum of a brain affected by Niemann-Pick disease type C at the end stage of the disease. The blue staining shows the dense pockets of... view more
Credit: National Institutes of Health, NIH/Eunice Kennedy Shriver National Institute of Child Health and Human Development
Researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disorders.
Lysosomal storage diseases, also known as lipid storage diseases, comprise about 50 rare inherited disorders that usually affect children. Fatty materials accumulate in the cells and tissues of the body. These diseases can result in damage to the brain, peripheral nervous system, liver, and other organs and tissues; they are often fatal.
Researchers at the National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), both parts of NIH, will conduct studies on NPC and other lysosomal storage disorders with funding provided by Vtesse.
"This is an excellent example of how launching a project to study the underlying biology of one disease can lead to advances that hold promise for an entire group of diseases -- the NCATS goal of finding what is common among diseases and the translational science process," said NCATS Director Christopher P. Austin, M.D. "I am grateful to all of the NPC patients, their families and patient support groups who have been equal partners in our efforts to find therapeutic solutions to these devastating disorders."
Vtesse will support the ongoing phase I clinical trial for NPC at the NIH Clinical Center, led by NICHD researchers who have been evaluating the safety of the drug cyclodextrin. Vtesse also plans to collaborate with NICHD to launch a second clinical study of cyclodextrin for the treatment of NPC in the United States and Europe, anticipated to begin in 2015. The use of cyclodextrin for NPC has been granted orphan drug designation in the United States and Europe, providing development incentives for products that demonstrate promise for the diagnosis and treatment of rare diseases or conditions.
Patients with NPC typically develop impaired coordination and movement (ataxia) and other neurological problems as a result of cholesterol buildup in their brain cells. "Patients and their families are desperate for an effective treatment for NPC," said NICHD Clinical Director Forbes D. Porter, M.D., Ph.D., who leads the phase I trial evaluating cyclodextrin at NIH. "Our role is to test promising new drugs and therapies to ensure that they are safe and effective."
Researchers in NCATS' Therapeutics for Rare and Neglected Diseases (TRND) program developed cyclodextrin as part of a project focused on finding treatments for NPC. The goal of TRND projects is to develop therapeutic candidates through a strategy known as de-risking, which involves the pre-clinical development of therapeutics to the stage where they attract biotechnology and pharmaceutical companies such as Vtesse to invest in their further clinical development.
The NPC project is a collaboration among government, academic and industry researchers, who worked with patient groups and the NPC community to advance knowledge about the disease. Before the TRND project, researchers had found that cyclodextrin showed promise as a treatment for NPC in studies of animal models for the disease. Additional NCATS efforts provided further evidence that cyclodextrin demonstrated potential as a treatment for NPC.
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NIH teams with industry to develop treatments for Niemann-Pick disease type C