A Black Texas teenage girl with sickle cell disease is the first patient to receive gene cell therapy at the Texas Medical Center in Houston.
Helen Nduku, 17, of Katy, Texas, is the first patient in the Texas Medical Center to receive gene therapy for sickle cell disease.
Currently, there is only one cure for sickle cell disease– a bone marrow transplant from a matched sibling donor.
Unfortunately, Nduku does not have a sibling whose marrow matches hers, so Texas Medical Center doctors tried something different. The approach involves extracting stem cells from a patient’s own blood.
“You’re essentially your donor,” said assistant professor, Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine Dr. Tami John.
Once the stem cells are collected, they are modified in a lab while patients undergo chemotherapy. Afterward, the patient receives a transfusion of their own modified stem cells, which plants the seeds for healthy red blood cells to form.
The pain Nduku experienced from sickle cell disease was debilitating and pushed her to enroll in the trial at 15.
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“Imagine someone squeezing and stabbing you at the same time,” the 17-year-old Katy resident said, according to The Houston Chronicle. “It’s very much like that. It’s intense, down in your bones.”
It was so bad that Nduku had to take narcotic painkillers for relief and regularly went to the hospital for weeks.
“Helen has been a patient at Texas Children’s for most of her life,” John said. “She’s had significant complications from her sickle cell disease. Gene therapy just became available. We decided together that this route would be best for her.”
At age 17 and after joining the trial, Nduku’s life is genuinely starting.
She has been accepted into the University of Pittsburgh and plans to take a gap year before studying chemistry or business.
There are 100,000 cases of sickle cell disease in the U.S., according to data from the Centers for Disease Control and Prevention.
Sickle cell disease is an inherited blood disorder that affects red blood cells. People with sickle cell disease have red blood cells that contain mostly hemoglobin S, an abnormal type of hemoglobin. Sometimes these red blood cells become sickle-shaped (crescent-shaped) and have difficulty passing through small blood vessels.
When sickle-shaped cells block small blood vessels, less blood can reach that part of the body. Tissue that does not receive a normal blood flow eventually becomes damaged. This is what causes the complications of sickle cell disease.
Nduku recommends gene therapy to others with sickle cell disease and wants other patients to know that this life-changing option exists.
“I’m very glad that I did it,” she said. “Don’t expect to be all fixed in two days. It’s going to take time. But the time it takes is so worth it.” “I hope to give other people hope,” she added.
