SOAR is accelerated research project designed to find a cure for Niemann Pick Type C (NPC), a rare genetic disease for which there is no current effective treatment of cure. NPC ravages the bodies and minds of its victims, who are all likely to experience seizures, psychosis, depression and hallucinations. Their speech becomes slurred; they have difficulty swallowing, often requiring a feeding tube. Their median lifespan is 16 years old.

Because it is a progressive, neurological disorder, one of NPC's primary symptoms is dementia. As a result, research into NPC also offers a profound opportunity to understand – and someday to treat and cure – more common forms of dementia such as Alzheimer's, which our parents, grandparents, and we ourselves may face.

SOAR was created by parents and scientists as a grassroots response to the absence of treatments for this terminal disease. The SOAR collaborative is comprised of scientists from leading institutions where NPC research is conducted, including the Albert Einstein College of Medicine, Washington University of St. Louis, Mt. Sinai School of Medicine and Oxford University. Advisors from the Mayo Clinic and the National Institutes of Health complete this collaboration.

A race against time

Experts estimate that it takes 15 years and a billion dollars to develop a new drug. By focusing on compounds that do not need new approval by the Food and Drug Administration, SOAR has accomplished in just three years, what few similar projects have achieved. A trial to test a treatment that could slow the progression of NPC was conducted last year at the National Institutes of Health. For the first time, the trial also validated a biomarker – a biological indicator of the disease's progression.

Researchers have found at a number of additional compounds that may lessen the severity of NPC. One of these, cyclodextrin, is already being used in "compassionate use" trials with some children. SOAR researchers are working with other scientists to better understand how cyclodextrin works, what dosage is optimal and safe, and the best way to deliver it, with the goal of launching a full-fledged clinical trial at the NIH in 2012