The Daily Tar Heel

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Wednesday January 19th

Hannah' Hope Fund raises money for researching an incurable disease

Family seeks to win Pepsi grant

In March 2008, Lori and Matt Sames received the worst news a parent can imagine: their three-year-old daughter had been diagnosed with an incurable disease.

Now, their hope lies with Steven Gray, a researcher at UNC’s Gene Therapy Center, who believes that with appropriate resources a cure is within reach.

Giant Axonal Neuropathy
  • Very rare neurodegenerative disease, similar to Lou Gehrig’s disease (ALS)
  • Caused by a mutation in a single gene, called gigaxonin.
  • Children’s nerves slowly deteriorate and die.
  • Confined to wheelchair by early teens.
  • In later teens and early 20s require artificial life support
  • Usually die in third decade of life.
  • Mental function of patient is fine.
  • Referred to as death sentence.
  • No FDA-approved treatments.
You can vote at least 3 times a day as follows:
  1. Go to http://www.refresheverything.com/hannahshopefund
    (You must click the “Vote For This Idea” button AFTER you log in for your vote to count.)
  2. Text the message 104051 (for Hannah’s Hope) to 73774 (for Pepsi).
  3. Use the Facebook Pepsi Refresh Voting app.
  4. Use the iPhone voting app which can be downloaded from iTunes.

Hannah, the Sames’ youngest daughter, was diagnosed with Giant Axonal Neuropathy, or GAN, after their pediatric neurologist stumbled upon the rare disease in a medical textbook.

Lori Sames said that receiving the news about her daughter left her family in a state of shock, anger and disbelief.

Poised to overcome what some call a “death sentence,” the family turned to action.

After days of grieving, the family decided someone had to be the first to be cured of GAN. In August 2008, Lori Sames brought 20 researchers together in Boston to create a symposium on GAN in an effort to compile research and ideas for a cure.

Until then, the information was scattered.

Soon, Sames realized that little had been discovered about the disease, as pharmaceutical companies dismissed GAN as a relatively rare ailment. She then started a grassroots fundraising campaign to cure GAN — also known as an “orphan disorder” — which affects less than 200,000 people.

“Pharmaceutical companies don’t usually look to develop therapies for orphan disorders — they look to cure diseases that affect mass amounts of people,” Sames said.

“The whole effort is really driven by the families of the children with the disorder.”

To lead that effort, the Sames family founded a 501©(3) non-profit organization called Hannah’s Hope Fund dedicated to finding a cure for the disorder.

“There is no energy on this planet stronger than a parent’s love for their children,” said Heather Tutwiler, Lori Sames’ cousin and an advocate for Hannah’s Hope Fund.

Since June of 2008, Sames said the foundation has raised $2.1 million toward GAN research.

Last August, Hannah’s Hope Fund also won a $250,000 grant through the Pepsi Refresh Project and is competing again for the same grant in November.

As of Monday, Hannah’s Hope Fund is in third place. The top two projects each month receive grants.

Voting for the November prize ends tonight at midnight.

Gray is leading the research for Hannah’s Hope Fund.

“What we’ve been doing is trying to develop viruses as vehicles to put the gene back in the body,” he said.

“We’re trying to target these viruses to the spinal cord to repair nerves that are being damaged.”

Researchers around the globe are now working on developing a therapeutic approach to curing GAN, but must abide by the FDA’s standards and guidance.

“It’s realistic within the next two years to actually move forward into testing therapies on humans with the disease,” Gray said. “But it all depends on how the FDA proceeds.”

Contact the University Editor udesk@unc.edu.

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