In 2008, Hannah Sames of Rexford, NY, was diagnosed with Giant Axonal Neuropathy (GAN), a very rare, neurodegenerative disorder. Her parents Lori and Matt were told that their daughter was going to die. She was going to weaken, lose the ability to move, speak and swallow, and would typically succumb to pneumonia in her late teens or early 20’s. They decided to fight! What happened next is nothing short of a miracle.
We immediately started Hannah’s Hope Fund, a 501(c)(3) public charity and the only organization in the world dedicated to funding a treatment and cure for GAN. Through grassroots efforts, HHF has raised $3.3 million, and the collaborative team of scientists working with us have developed what they feel is an effective therapy. GAN is considered an “orphan disorder” because it doesn’t impact enough people to attract the attention of Pharmaceutical and Biotechnology companies.
Miraculously, just three years and three months after we began funding the UNC at Chapel Hill Gene Therapy Center, we had our Pre Investigational New Drug meeting with the FDA for GAN gene therapy. GAN patients will likely be the first disease community in the world to receive a therapeutic gene to the spinal cord, and the process will be the first in-human one with the AAV9 viral vector. The viral vector is a “delivery truck” that transports healthy genes to cells. The delivery mechanism we are using may apply to many neurological genetic disorders. Following this meeting with the FDA, we have a clear path forward to a Phase 1 clinical trial and the FDA required safety studies are currently underway. However, after paying $649,000 for the safety studies, Hannah’s Hope Fund is out of money.
Recently, HHF was given an amazing opportunity. Doris Buffett’s Sunshine Lady Foundation gave Hannah’s Hope Fund an “all-or-nothing” matching grant challenge. We have until April 30th, 2013 to raise $450,000, and if we do, The Sunshine Lady Foundation will award our charity a $450,000 grant. It’s critical we achieve national attention, both to help more children get properly diagnosed and raise awareness of our mission and challenge grant.
While our mission began with Hannah, we quickly realized the broad implications of our efforts for other disorders, and we continue to be inspired by families with GAN who find us from all over the world. Please visit our website, and please view this video that will introduce you to Hannah and other children that are living with GAN.