An FDA-approved gene therapy for the rare blood disorder was effective but came at an incredible expense.
When she got the news that her young son had been diagnosed with the rare blood disorder known as transfusion-dependent beta thalassemia , Yusara Ahmed knew the drill. Her sister had also experienced the inherited condition and needed to undergo regular blood transfusions simply to survive.
Finally, the FDA approved the one-time treatment — betibeglogene autotemcel in 2022. By January 2024, the hospital was ready to treat Yusuf. At age 8, he became the first patient in the state of New York to undergo gene therapy for beta thalassemia. Millions of people have the thalassemia trait, especially in southern Europe, the Middle East, southeast Asia, and Africa, Dr Al-Samkari said.
Yusara Ahmed remembers her sister's endless visits to the hospital after she was diagnosed at age 4."We were all very traumatized by the hospital environment," she said. But good news came in 2008, a few years later, when her sister was able to get a stem cell transplant from their brother. In the bigger picture, she said gene therapy still has room for improvement. The need for chemotherapy is one target. According to her, it causes most of the complications related to gene therapy.
Genomic Medicine Biologic Therapy Biologics Beta Thalassemia Gene Therapy Thalassemia Thalassaemia Transplantation Of Stem Cells Transplantation Of Hematopoietic Stem Cells Hematopoietic Stem Cell Transplant Stem Cell Transplantation Stem Cell Therapy Stem Cell Haematopoietic Stem Cell Transplant
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