DETROIT – Imagine needing to get a blood transfusion every two to four weeks for the rest of your life, simply to survive.
That is the reality for patients living with an inherited blood disease called beta thalassemia.
But there’s good news -- Children’s Hospital of Michigan announced a major breakthrough that’s now available in Detroit that could be a potential cure.
“We are happy to report on the status of our first case on July 30, 2024, a teenager began his gene therapy journey for thalassemia at our hospital,” said Dr. Sureyya Savansan, Children’s Hospital Transplant and Cell Therapy Director.
The patient wants to remain anonymous, but they’ve been a long-time patient at Children’s Hospital.
Beta thalassemia leaves the body unable to make enough red blood cells. The patient was unable to receive a bone marrow transplant to cure his illness because he couldn’t find a match.
“You can imagine our team’s joy and excitement that a full nine years after that initial transplant consultation, we were finally able to begin discussing the possibility of alleviating his need for blood transfusions,” said Liz Smythe, Children’s Hospital Bone Marrow Transplant nurse Coordinator.
The gene therapy is a three-step process. First, the patient’s cells are collected by a special machine. Then those cells are sent to a company that will put working copies of the abnormal gene into the patient’s cells -- that takes about 90 days.
The patient then undergoes four days of chemotherapy and the cells are returned to the patient.
“For years, they’ve been waiting for this to come in,” said Dr. Eman Al-Antary. “We are very, you know, like, glad that this is something that we are able to offer now for our patients and get them to have a life-changing treatment.”
The first patient’s cells have been collected and sent out. He’s expected to receive them in November.
“That’s always our goal here is just to make everything that’s available in the world, available to our patients here in Detroit,” said Dr. Alexander Glaros.
Eight patients are currently in the insurance approval process for the gene therapy. Additionally, Children’s Hospital of Michigan has begun the steps to treat their first sickle cell patient using gene therapy. That transplant is expected to take place sometime in 2025.