Francis S. Collins, M.D., director of the National Institutes of Health (NIH), 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 Steven U. Walkley, D.V.M., Ph.D., 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 U.S. Food and Drug Administration to approve cyclodextrin as an investigational new drug now being tested in a phase I clinical trial at the NIH. Dr. Walkley is a professor in the Dominick P. Purpura Department of Neuroscience and in the Saul R. Korey Department of Neurology; a professor of pathology; and director of the Rose F. Kennedy Intellectual and Developmental Disabilities Research Center.