Dallas hospital among those approved to provide new gene therapy for sickle cell disease
DALLAS - A Dallas hospital is one of a handful in the country approved to provide new gene therapy for sickle cell disease.
Children's Health is set up to provide one of two of the new gene therapies approved by the FDA last month.
The new gene therapy could be a game changer for the thousands of people suffering from sickle cell disease.
The Tucker family knows all too well the importance of blood drives like the one put on by the American Red Cross and the William McDonald YMCA in Fort Worth Wednesday.
Three of the five Tucker kids have sickle cell disease, a debilitating blood disorder that affects roughly 100,000 people in the U.S., most of them Black.
"Every time I have a pain crisis, I have to go to the hospital and get needles in my arm and I had surgery and blood transfusions," Nicholas Tucker said.
"It's been tough because whenever you're in a pain crisis, you have to have somebody take care of you," Kaden Tucker said.
Their mom, Crystal Dawn, said her three boys, ages 15,12, and 10, have spent countless hours at the hospital dealing with complications from the disease caused by a mutation of hemoglobin found in red blood cells that often leads to a rigid, sickle-like shape.
"Every day is a struggle. Every day is a battle," Dawn said.
Dr. Andrew Koh is a pediatric hematologist at Children's Health and the director of the cellular and immunotherapeutics program.
"This disease is, you know, a major impact in the U.S.," Dr. Koh said.
He's excited about a major breakthrough for the treatment of sickle cell disease.
In December, the FDA approved the first-ever gene therapy for people ages 13 and older that could cure the painful, inherited blood disorder.
Breakthrough sickle cell gene therapy treatments approved by FDA
The breakthrough sickle cell treatment is the first approved therapy based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.
"Essentially, you can take bone marrow derived, or blood derived stem cells, from the patient itself, genetically modify them to correct the sickle cell defect, and then re-infuse them back into the patient," Dr. Koh explained.
Children's Health is one of a handful of medical centers in the country currently qualified to dispense the gene therapy.
Dr. Koh is urging patients with sickle cell disease to talk to their providers to see if they might qualify for the therapy.
Dawn plans to do just that for her three kids.
"I’m so excited about the latest advancement in gene therapy. I can't wait to hear more about it," she said.
In the meantime, she's got plenty of people praying for her family.
Dr. Koh said patients who undergo the gene therapy will know within a few months whether they're making the right hemoglobin or not.
Then, because it's a newer therapy, there is a surveillance period with check ins, to make sure that there's no severe side.