MINNEAPOLIS — A lot of people are traveling home for Thanksgiving this week, but one family from Alabama is happy to be spending the holiday hundreds of miles away, in Minnesota.
The Hamlett family is forgoing its traditional gathering because it's the only way they can gather with four-year-old Celia Grace Hamlett.
It has been nearly three months since Celia Grace left her home in Alabama in order to become the first person to receive a new gene therapy to treat metachromatic leukodystrophy (MLD).
Patients with MLD can't produce an enzyme that protects the brain and nervous system. If left untreated, it is often fatal just a few years after symptoms begin. Fortunately, Celia grace was still asymptomatic, which made her a good candidate to receive gene therapy.
"We've had a little friend that had the same disease and she was too far gone for the gene therapy and they were only able to do the bone marrow transplant. She passed away last week," said Celia Grace's dad, Gary Hamlett. "Just knowing that that could have been our baby, it's been rough. It's been a long journey."
That journey started this summer, when Celia Grace traveled to Minnesota so that her stem cells could be removed and sent to Italy to get re-engineered. For several years, a gene therapy trial in Milan has helped many patients stop the progression of MLD, but it took the University of Minnesota Medical School several years to get the FDA to approve the treatment in the US.
"We've got a protocol now with the FDA for the treatment stuff, for compassionate use," Gary Hamlett said. "We're so thankful about that and thankful that we're able to help other kids and other parents that are in the same situation."
Not that her situation has been easy. Celia Grace and her mom, Kassie Hamlett, have spent most of the past few weeks away from the rest of the family.
"I've never been away from my kids that long," Kassie Hamlett said. "The chemo was also really hard on her. I know we may face more obstacles along the way but as good as she's doing right now, we're thrilled."
Celia Grace's doctors are thrilled too, with a therapy that is proving to be as effective as they'd hoped.
"You know, the key thing is the enzyme levels that we're attempting to boost with the process, and they're above normal which is what we're trying to accomplish," said Dr. Paul Orchard, a Pediatric Blood and Marrow Transplant Physician at M Health Fairview. "We're really excited about how things have gone thus far."
And if they continue go well next week, Celia Grace will be home for Christmas.
"I'm thankful every day but I'm extremely thankful this year," Kassie Hamlett said. "I even bought her a shirt to wear on Thanksgiving, it says 'Extra thankful this year.' That's the best way that I can put it."
Dr. Orchard says Celia Grace is already having an impact on others. He says the FDA has already given clearance for two other children to receive gene therapy at M Health Fairview.
If they clear other hurdles, he says they might also receive the life-saving treatment in Minnesota in the months ahead.