CLIFTON PARK Hannah Sames got a special delivery at school Tuesday in honor of her ninth birthday.
Her dad brought in her favorite menu item from Moe’s Southwest Grill — a beef burrito with everything on it except rice.
She and her family are also celebrating another milestone: exceeding a $450,000 fundraising goal that resulted in a matching grant from Doris Buffett, founder of the Sunshine Lady Foundation and sister of famed billionaire investor Warren Buffett.
The money is needed to fund a human clinical trial for children, which will involve gene replacement therapy. The Sameses hope the therapy will help stop the progression of giant axonal neuropathy, a rare genetic disorder that Hannah was diagnosed with in March 2008.
GAN 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, her parents started the Hannah’s Hope Fund, a charity dedicated to funding research to find a treatment for the disease.
GAN afflicts only a small percentage of the population, but the Sameses hear from a new family that has a child with the disease about every four to six weeks, Hannah’s father, Matt Sames, said.
“It’s still very rare, but we’re the only organization in the world for it,” he explained. “Because we are getting some notoriety in the medical field and with scientists, families that were either undiagnosed or misdiagnosed, now the disease is being recognized, and when they go to the Internet to look for research or support, we’re it.”
The clinical trial the Hannah’s Hope Fund has been working to finance was scheduled to start this spring but has now been delayed, probably until July, said Sames. Close to 40 children battling GAN will participate.
“As with everything, it just seems like something else pops up and we have to do another study. When you’re going into children with an experimental therapy, they just really want to make sure that it’s safe; they want to be very thorough,” he said.
The gene replacement therapy planned for the clinical trial may stop the progression of the disease but won’t reverse nerve death that has occurred, so time is of the essence.
Hannah is holding her own health-wise, her father said.
“She’s still using a walker and we do a ton of [occupational therapy] and [physical therapy]. We try and keep her as flexible and as strong as we can but you can really notice her energy level has its ups and downs and that’s hard,” he said.
Although the Hannah’s Hope Fund has raised the money needed to fund phase one of the clinical trial, the research will continue and the bills will keep coming, so the fundraising will go on. The Sameses hope to generate $1 million by April 30. So far, $904,000 has been raised, including the matching funds from Doris Buffett.
Sames can tell all sorts of stories about generous donors.
“It’s just been incredible,” he said.
For more information or to make a donation, visit www.hannahshopefund.org.