Backgroundkids at NIH

In August of 2008, HHF brought 20 research scientists together in Boston for the first ever symposium on GAN. The goal of this meeting was to discuss everything known about GAN, and prioritize therapeutic approaches, as well as prioritize basic research studies needed to learn more about underlying disease mechanisms. Gene therapy was prioritized as the #1 therapeutic approach coming out of this meeting.

Dr. Jude Samulski, Director of the University of North Carolina (UNC) at Chapel Hill Gene Therapy Center, sent research associate, Dr. Steven Gray to the symposium to assess whether or not gene therapy was a viable approach for GAN. HHF began funding Dr. Gray’s work on GAN Oct. 1, 2008. Due to the efforts of Dr. Gray, utilizing more than 25 years of experience obtained at the UNC Gene Therapy Center, there is now a gene therapy approach for GAN! None of this would be possible without the multitude of HHF supporters who donated and helped raise funding.

On Jan. 26th, 2012, HHF and the UNC clinical team had a Pre Investigational New Drug (Pre IND) meeting with the FDA, and with their guidance, we now have a clear path forward to a GAN gene therapy clinical trial. The GAN disease community will likely be the first disease community to receive a therapeutic gene to the spinal cord. The goal is to stop disease progression. Gene therapy will not reverse cell death, so it’s critical these kids receive treatment ASAP. Every clinical trial is a human experiment. It will not be known if gene therapy will be an effective treatment for GAN until our clinical trial(s) end.

We recognize that once we rescue the central nervous system, we need a drug therapy approach to rescue the peripheral nerves, the nerves outside of the central nervous system. Hannah’s Hope Fund is committed to the GAN disease community long-term.

We are deeply appreciative of our loyal supporters who have made all of this possible.