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.
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.
