Dana's Angels Research Trust

Find a cure Niemann-Pick type C disease.

NPC disease is a rare, fatal genetic disorder that results in progressive decline in motor and intellectual function of affected individuals, primarily children. The NPC community has benefited from incredible research by world-class scientists funded by charities including Dana's Angels Research Trust, the Ara Parseghian Medical Research Foundation and the National Niemann-Pick Disease Foundation. The effort continues supporting pivotal NPC research; research that may also help millions of people suffering from HIV/Aids, Ebola, heart disease, stroke, Alzheimer's disease and other disorders that appear to be related to cholesterol metabolism.

DART and its SOAR collaborators are currently working with several different compounds at various stages of drug development. The drug pipeline was designed to generate maximum efficiency in moving promising drug candidates forward and to accomplish this, SOAR focuses on the following aspects:
-An open forum of discussion among SOAR scientists and affiliated labs to allow rapid identification of potential compounds to ameliorate NPC disease
-Discovery of biomarkers to provide critical tools for clinical drug evaluation
-Maintenance of several unique NPC animal models utilized for in vivo testing of compounds
-Complementary expertise among individual laboratories to provide a foundation for thorough analysis and evaluation of drugs of interest
-Actively seek out highly meritorious projects and additional collaborations aligned with the priorities of SOAR and aid in funding these pilot projects.

Central to DART's SOAR collaborative is a core of scientists from four internationally recognized NPC laboratories:
-Dr. Forbes Porter, NICHD/NIH
-Dr. Cristin Davidson, NHGRI/NIH
-Dr. Andrew Lieberman, University of Michigan
-Dr. Mark Schultz, University of Michigan
-Dr. Stephanie Cologna, University of Illinois, Chicago
-Dr. Charles Vite, University of Pennsylvania

These scientists maintain a close working relationship and function as a tightly integrated unit to allow for free scientific exchange. They work closely with an extensive network of scientific collaborators, including other intramural investigators at the National Institutes of Health [Therapies for Rare and Neglected Diseases (TRND), National Center for Advancing Translational Sciences (NCATS), and The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)], other academic institutions, and pharmaceutical companies.

While great progress has been made, the NPC community has yet to see its first FDA approved therapy. We are hopeful that Adrabetadex/cyclodextrin or Arimoclomol, each of which concluded FDA approved Phase 2/3 clinical trials in 2019 will be the first step in the creation of a drug cocktail that will work as a cure for NPC. Numerous additional therapies including gene therapy, are in our pipeline awaiting an opportunity to be tested clinically.