Update on our progress from Lori Sames, Executive Director of HHF:
I participated in a clinical trials training course last week in DC. Immediately following the course was the American Society of Gene and Cell Therapy’s annual conference. It was amazing to see the efficacy and safety data for gene therapy for other diseases. Gene therapy has finally come of age and is successfully treating animal models of many diseases! Also, there are several human gene therapy trials underway.
While at this conference, I was fortunate to meet Corey Haas, the 8 yr old boy cured from blindness. Coincidentally, the Haas family lives about 35 minutes north of us. His disorder is also recessive. It’s called Leber’s Syndrome.
Our scientists were also at this conf. in DC. We had a meeting to plan the additional biodistribution study we need to conduct to fine-tune the surgical protocol for our “Investigational New Drug” (IND) that we have to file with the FDA. I was able to speak one-on-one with several of the people within the FDA who will be responsible for reviewing and approving our IND so we can begin a human trial. This Summer, we will have our first meeting with the FDA to present our pre-clinical studies we have to conduct over the next year, in order to gain approval to begin a clinical trial in the fall of 2011.
Also, a few weeks ago, I met the Executive Director of the SMA Foundation in NYC. Spinal Muscular Atrophy (SMA) is also a horrific recessive disorder whose treatment needs are identical to Giant Axonal Neuropathy (GAN). Both SMA and GAN patients need a healthy gene delivered to the spinal cord. We held a meeting in DC with SMA scientists, along with our lead scientist, Dr. Steve Gray. Two different SMA scientists have performed proof-of-concept studies in SMA mice and revealed gene therapy can rescue the mice! However, the route of administration of the gene therapy virus cannot be translated to a human. The SMA scientists were excited to learn from Steve how good our data looks for intrathecal injection to treat the spinal cord. Both Genzyme and the SMA Foundation would like Steve to present our findings to their scientists. We hope the SMA community will help fund the additional biodistribution study we have to conduct to fine-tune the route of administration (surgical protocol). SMA impacts 1 in 6,000 births and 1 out of every 35 people carry the SMA mutation! While SMA is considered an orphan disorder because it impacts less than 200,000 Americans, it is the leading fatal disorder in children. Hannah’s Hope Fund is blazing the trail for gene deliver to the spinal cord! Thank you all for helping to make this miracle happen!
Love, Lori and Matt