Niemann-Pick Research Recognized — Dr. Francis Collins, director of the National Institutes of Health, honored Einstein and several other institutions last June when he presented the NIH Director’s Award to the Niemann-Pick Disease type C (NPC) Therapeutic Development Team. The award recognized the team’s outstanding accomplishment in identifying and developing a treatment for NPC. This rare, inherited disease affects young children and involves progressive mental and physical deterioration. In 2009, Einstein researcher Dr. Steven Walkley and graduate student Cristin Davidson published a study in PLoS One showing that the drug cyclodextrin was effective in a mouse model of NPC. This research was crucial in persuading the FDA to approve cyclodextrin as an investigational new drug now being tested in a phase I clinical trial at the NIH. Dr. Walkley is professor of neuroscience, of pathology, and of neurology and director of the Rose F. Kennedy Intellectual and Developmental Disabilities Research Center.
Posted on: Friday, December 06, 2013