Children’s Hospital of Michigan in Detroit is the first and only medical center in the state to offer a new gene therapy for the rare condition beta thalassemia, helping patients avoid chronic anemia and a lifetime of blood transfusions.
Zynteglo adds a gene to the patient’s own bone marrow stem cells, which are then infused back into the patient in a single, potentially curative treatment. The process takes several steps over several months, including preparing the patient for the infusion much like a patient who receives a bone marrow stem cell transplant. It is available only at qualified treatment centers, like Children’s Hospital of Michigan.
“This is a potentially life-long solution for our beta thalassemia patients, who otherwise would experience continuous medical treatment throughout their life, as well as possible significant side effects,” says Dr. Süreyya Savaşan, medical director of the transplant and cell therapy Program at the Children’s Hospital of Michigan. “This is a game-changer.”
The severe anemia caused by beta-thalassemia often causes patients to rely on blood transfusions every three to four weeks. Children’s Hospital of Michigan manages one of the largest groups of thalassemia patients in the country who are dependent on blood transfusions – and who could potentially be cured by this new treatment.
Children’s Hospital of Michigan became a zynteglo qualified treatment center in May 2023 and is currently in the process of screening patients to begin the months-long treatment process.
Sites are chosen based on their expertise in stem cell transplantation, cell and gene therapy, and beta thalassemia. The transplant and cell therapy team at Children’s Hospital of Michigan has performed hundreds of stem cell transplants, including traditional stem cell transplants from matched donors for children with beta thalassemia. Patients must meet a specific clinical and laboratory profile for zynteglo treatment eligibility.
The availability of this new treatment at Children’s Hospital of Michigan represents an important milestone in a long legacy of thalassemia care. It comes almost exactly 100 years after beta thalassemia was first described in 1925 by Dr. Thomas Benton Cooley, the head of Children’s Hospital of Michigan’s Pediatric Division in the 1920s and ‘30s.
Beta thalassemia (β-thalassemia) or beta-thal, is a rare genetic disease that researchers believe affects about 1500 people in the United States, most commonly among individuals of Mediterranean, South Asian, and Middle Eastern descent. It’s named for mutations in the gene for β-globin, a primary component of hemoglobin, the protein within red blood cells that carries oxygen throughout the body.
Even with transfusions, patients can experience symptoms ranging from chronic fatigue, weakness, and pain to potentially deadly complications like liver disease, heart failure, and diabetes. Previously the only curative treatment for beta-thal was a stem cell transplant. This remains an option for some patients, but the patient needs to have a matched donor for a good outcome. Even with a good match, transplant recipients can experience transplant rejection or graft-versus-host disease, a severe complication that can lead to organ damage.
