Request for Applications: Pilot Grant for Research on Niemann Pick Type C

Request for Applications: Pilot Grant for Research on Niemann Pick Type C Eligibility All individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. Research Focus: The following areas of investigation will be considered in the context of a $50,000 pilot grant for Niemann Pick Type C. 1. EVALUATING INVOLVEMENT OF HEAT SHOCK PROTEINS IN THERAPY (but not by groups presently publishing on potential therapies already ex: HSP70) 2. Studies on methods and drugs to increase NPC1 protein expression to delay onset. 3. Studies intended to SPECIFICALLY treat NPC1-ASSOCIATED LUNG PATHOLOGY (surfactants, infused OR inhaled drugs SUCH AS CYCLODEXTRIN). Studies designed to show efficacy of FDA-approved drugs for symptom management or prophylaxis are not sought in this RFA. (i.e., antibiotics for pneumonia treatment or Mepron) 4. Studies willing to test the potential of combination therapy in fibroblasts/NPC animal models or work towards a Phase 1 trial of multiple FDA-approved and non-FDA-approved therapies in the population (i.e., Cyclo/tegretol/delta tocopherols for certain mutations) With the goal of finding concrete data that until this is cured we need mutation-specific combination therapies to best treat patients. 5. Stem cell/pluriponent stem cell studies for targeted correction of mutations. The timeline for the RFA/gr...

Source: ScanGrants feed - September 3, 2014 Category: Research Authors: Center for Orphan Disease Research and Therapy University of Pennsylvania Source Type: funding