When their youngest daughter, Hannah, was diagnosed with Giant Axonal Neuropathy (GAN) – an extremely rare neurodegenerative disorder for which there is no known cure – Matt ’90 and Lori Sames resolved to do everything in their power to find one.
In 2008, the couple established Hannah’s Hope Fund. Their aim was to raise money to support and develop a treatment and a cure for GAN. It was no small feat, as there was no organization, scientific team or funding focused on the disease. At the time, the Sames family was fortunate to find one scientist, Dr. Steven Gray, to dedicate his work on this very rare disease. The stakes were high because GAN causes nerve death that eventually leads to paralysis, inhibits breathing and eventually takes the patient’s life in his or her teens or early twenties. Matt and Lori were racing against the clock.
Since then, Hannah’s Hope Fund has brought together a team of scientists from around the globe to research GAN and to work on therapeutic approaches. The organization has raised more than $7 million to drive these projects. It has been a long road, Matt Sames acknowledged, but their efforts have been well worth it; researchers at the University of North Carolina recently developed an innovative experimental gene transfer-based treatment for children afflicted with GAN.
The research that led to the treatment was headed by Dr. Gray, of UNC’s Gene Therapy Center, but was a collaboration of researchers from Columbia University, Northwestern University, as well as scientists from France and Quebec and others. The procedure that Gray and his colleagues developed involves using a genetically modified virus to deliver a missing gene into the cerebrospinal fluid of patients. Its aim is to help them regain some function they have lost in their central nervous system, which governs breathing, swallowing, movement and sensation throughout the body.
This clinical trial is now underway at the National Institutes of Neurological Disorders and Stroke of the National Institutes of Health in Bethesda, Md. The first two patients underwent treatment in May and November of 2015; a third child is slated to receive the treatment next month, Hannah is scheduled to receive it in April. While it will take time to determine the efficacy of the regimen, Sames said anecdotally the patients’ progress has been remarkably good. There have been no adverse effects and some gains in the children’s functionality.
Beyond aiding children with GAN, the treatment could also aid people living with other neurodegenerative diseases, such as ALS, Spinal Muscular Atrophy, Friedreich's Ataxia and more.
“My wife and I are thrilled that our team has developed a therapy in a little over seven years, and that it seems to have had some benefits,” Sames said. “We continue to work on a more efficacious approach for the central nervous system and we also need to save the peripheral nervous system, so our mission continues. The Le Moyne community has been a source of strength for us and we appreciate the continued support. Biomedical research is very expensive and we need all the help we can get. No donation is too small.”
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