KATY – Right now, the only normal way to cure sickle cell disease is with a bone marrow transplant. Doctors say it is incredibly rare to find a matching donor for that kind of transplant.
Now, a new health development may be creating another option for those in need.
Doctors at the Texas Children’s Hospital said they have found a way to eliminate the need for a third-party donor where the patient can be their own.
Helen Nduku, 17, of Katy was the first patient in the gene cell therapy trial at Texas Children’s Hospital.
Nduku’s trial was successful, and now she’s sharing her story with others.
“I can live a day-to-day life without needing narcotics or feeling like very tired or sleepy or shut down,” Nduku explained.
Nduku did not have a matching donor, as most people don’t, but Dr. Tami John, a pediatric bone marrow transplant specialist, was able to collect Nduku’s cells, modify them through gene therapy and transplant them back. Nduku is the first patient in Houston to receive gene therapy for sickle cell.
“Gene therapy for sickle cell disease is still a therapy that is under experimental evaluation. There are more and more types of gene therapy that are becoming available,” said John. “It is not yet though available for everybody because we are still trying to understand the acute and long-term implications of the therapy.”
Nduku will be part of this study for the next decade.
For Nduku, it was curative, John said. He added that it’s likely going to give her a better quality and longer life.
“At certain points, you become comfortable with the idea that you’re going to die sooner than you probably should. So, for this to have worked for me and to give me a life, it’s kind of [like] I want to do everything!” Nduku said. “So, I think it’s... I just think it’s beautiful.”
John added that they will be able to expand gene therapy to other diseases, however, the main thing slowing them down is the efficiency and being able to replicate the process quickly.
She expects more patients could have access to this in the next few years.
