January 12, 2010 - NPC parents help

get first drug recommendation!

A Food and Drug Administration advisory panel recommended Zavesca to treat NPC.  Phil Marella of DART, Cindy Parseghian of the Parseghian Foundation and Barb Vorpahl of the NNPDF spoke during the public portion of the hearing.

The panel of outside medical experts voted 10-to-3 in favor of a question that asked "in light of the safety and efficacy data presented in this application, does the risk-benefit profile of Zavesca support its approval for treatment of NPC?" The FDA usually follows the advice of its panels but is not required to.  The drugs sponsor, Actelion said the agency is expected to decide in March whether to approve Zavesca for treating NPC.

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DART is taking the lead in committing to a unique therapy development approach for additional NPC treatments called SOAR, Support Of Accelerated Research. Working with other NPC families and organizations, SOAR has created a multidimensional collaborative drug development program searching for an effective cocktail treatment for NPC.  The ambitious goal is to have at least two or three new NPC therapies ready for clinical trial in 2011.

Other efforts:

¨ DART has also funded initial research into possible gene therapy for NPC at Albert Einstein College of Medicine.

¨ Funding jointly with the Parseghian Foundation:

·   Two-year post-doctoral fellowships in neurology at two renowned research institutions; The Scripps Research Institute in La Jolla, California, and at the University of Texas Southwestern Medical School in Dallas, Texas.

·   Funding for the contracted nurse support for the NPC observational study at the National Institutes of Health in Dr. Forbes Porter’s lab, looking for potential disease markers and even therapies from the detailed, periodic evaluation of children with NPC.

·   Important studies looking for markers of the progression of NPC in cats afflicted by NPC and potential therapies to extend the lives of the afflicted cats at the University of Pennsylvania.

¨ DART’s study at Columbia University involved a natural compound, ubiquinone or Coenzyme Q10 in humans.  The goal of the study was to understand the benefit, if any, of CoQ10 as an NPC therapy.

¨ At the University of California, San Francisco, DART and NNPDF, funded research related to the potential use of the drug Prozac, as a therapy for NPC.  Unfortunately, they found that Prozac treatment had no effect on the lifespan of the NPC mice nor how the mice gained and lost weight over the course of their lifetime, or in their motor coordination and locomotion.