First patient to receive gene therapy treatment developed at Children's Hospital of Philadelphia living pain-free
A new gene therapy developed at the Children's Hospital of Philadelphia has been approved by the FDA and is being used to transform lives. The first patient to receive this gene therapy after it was FDA cleared is now living pain-free for the first time.
Bike riding is fun now for 12-year-old Rahemeen Nabeel, who lives in Cherry Hill, New Jersey.
"I do lots of sports, and now that I have like more energy and stuff, I actually joined clubs and stuff," Nabeel said.
Energy is something that was in short supply for most of her life. Nabeel was born with an inherited blood disorder called beta thalassemia.
"It was just, like, so painful," she said. "Like I was in constant pain."
And there were constant blood transfusions — a struggle for the family, who lived in Dubai.
"So we took her to Paris, we took her to Italy, Rome, Switzerland, and then we took her to Turkey, Korea," said Zainab Nabeel, the 12-year-old's mother.
They finally ended up at the Children's Hospital of Philadelphia, which had done extensive research on a gene therapy where the patients' own stem cells are reprogrammed to fix the blood disorder.
"It was like a nightmare, but also like a really big blessing," Rahemeen Nabeel said.
The treatment includes chemotherapy that allows the gene therapy to work.
"It was a long, stressful journey, but we are really, really happy," Zainab Nabeel said.
It's been two years since Rahemeen Nabeel became the first patient to receive the FDA-approved treatment. Her mother said she's considered cured now.
No longer needing transfusions, Rahemeen Nabeel is back to enjoying her pet bird, Mango, and is doing well in 6th grade.
"Now I'm a bit too energized," she said.
The new gene therapy treatment, called Zynteglo, is being called a game changer.
"To see a therapy like this move into being widely available for patients, this is why we do what we do," said CHOP's Dr. Timothy Olson.
CHOP has about 17 patients in various stages of treatment with the new gene therapy.
Rahemeen Nabeel, who led the way, said she eventually wants to do something that combines science with her love for animals.
