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