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Support Of Accelerated Research for NPC Disease

Experts estimate that it takes 15 years and a billion dollars to develop a new drug – much too long and expensive for those afflicted with NPC.

By focusing on compounds that do not require approval by the Food and Drug Administration (FDA) as new chemical entities, SOAR has accomplished what only a small number of similar projects have achieved.

Over the past five years, SOAR has made significant progress in developing a drug pipeline for NPC therapeutics and in the discovery of disease biomarkers to facilitate clinical testing of these drugs. There are eight potential therapeutic compounds either in trial or currently being tested in the SOAR-NPC drug pipeline.

In 2013, a Phase 1 trial to test cyclodextrin, a treatment that could significantly slow the progression of NPC, was initiated at the NIH. SOAR researchers continue to work with other scientists in order to better understand how cyclodextrin works, what dosage is optimal and safe, and the best way to deliver it. Click here to learn more about the NIH Cyclodextrin Trial.

Although cyclodextrin and several other compounds are promising, no single potential therapeutic intervention is expected to impact all aspects of NPC disease and it is quite likely that a combination of two or more agents will lead to the most successful amelioration of Niemann-Pick type C disease. Hence, there is a continuing need to address new therapies for this devastating disorder.