![New Niemann-Pick Treatment Shows Promise]()
In the genetic disorder Niemann-Pick disease type C1 (NPC1), abnormally high levels of lipids accumulate in cells and damage organs including the liver, spleen and brain. NPC1 is usually diagnosed when children develop neurological symptoms such as clumsiness or learning difficulties. Symptoms progressively worsen and patients usually die 10 to 15 years following diagnosis. No FDA-approved treatment exists. Steven Walkley, D.V.M., Ph.D., has co-authored a study in the August 10 issue of The Lancet showing that the drug 2-hydroxypropyl-β-cyclodextrin (HPβCD) can safely slow NPC1’s progression. Patients received monthly or bi-weekly spinal injections of the drug for 18 months. Following the treatment period, biochemical and neurological tests showed that, compared with historical data for patients the same age, patients treated with the drug experienced significantly less cognitive dysfunction, with minimal side effects. Dr. Walkley is director of the Rose F. Kennedy Intellectual and Developmental Disabilities Research Center and professor in the Dominick P. Purpura Department of Neuroscience, in the department of pathology, and in the Saul R. Korey Department of Neurology at Einstein.
Posted on: Monday, October 02, 2017