On the first day of school, Hannah Sames had a bit more gear to lug onto the bus than most third-graders. The 8-year-old, who four years ago was diagnosed with giant axonal neuropathy (GAN), wears leg braces and gets around with the help of a walker.
“It’s getting harder and harder for her to get around, but she doesn’t complain,” said Hannah’s father, Matt.
GAN is a rare genetic disorder that slowly diminishes the ability to use the arms and legs, and to speak and swallow. It can also affect sight and hearing.
After Hannah’s diagnosis in March of 2008, her parents started the Hannah’s Hope Fund, a charity dedicated to funding research to find a treatment for the disease.
Financing the project is a perpetual challenge, but last week, the Sameses, who live in Clifton Park, received some exciting news. Doris Buffett, founder of the Sunshine Lady Foundation and sister of famed billionaire investor Warren Buffett, has agreed to give the fund a $450,000 challenge grant to help pay for a human clinical trial for children, which will involve gene replacement therapy.
“The challenge is we have until April 30 to raise $450,000 and if we fall short, she won’t give us any,” Matt Sames explained.
The family hopes to meet the challenge with a fundraising drive called Heroes for Hope. Donors are being asked to give at least $50 to the fund, send a solicitation to at least 100 potential donors and get three other people to commit to do the same.
The burden of funding the clinical trial has fallen on the organization the Sameses started because GAN is so rare, Hannah’s mom, Lori Sames, said.
“Pharmaceutical companies aren’t interested in incurring the expense to go through the clinical trial process to prove safety and efficacy. They’d rather focus their funding on treatments that impact more people for greater profit,” she explained.
If the gene replacement therapy planned for the clinical trial is successful it could help many people, her husband noted.
“This therapy could apply to other neurodegenerative diseases like spinal muscular atrophy [Charcot-Marie-Tooth disease] and up to perhaps 5 percent of those who suffer from ALS — Lou Gehrig’s Disease,” he said. “So our therapeutic approach could really have huge ripple effects in other disease communities.”
The gene replacement therapy is expected to stop the progression of the disease Hannah is fighting but won’t reverse nerve death that has already occurred.
“It can restore function to dysfunctional nerves but it’s not regenerative medicine, so the faster these kids receive treatment, the better,” Lori Sames said.
In 2009 Buffett supported the efforts of Hannah’s Hope Fund with a $500,000 matching challenge grant. The Sameses were able to drum up matching funds then and are hoping for a repeat performance with the $450,000 offered this time.
“If we fail it won’t be for lack of trying,” Matt Sames assured. “I think we can do it. We’ve got a strong network. The community has rallied behind us before and when people know that every dollar they give will be matched, that kind of inspires people to help us out.”
The family is grateful for the hope Buffett has once again offered.
“Thankfully Doris knows what’s important,” Lori Sames said. “She invests in things that are going to make a difference in the lives of children and adults.”
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