Children’s Hospital of Michigan announced Monday that it now is treating the first person in Michigan — a teenage boy — with a newly approved gene-editing therapy for the rare inherited blood disorder known as beta thalassemia .
Called Zynteglo , the treatment will essentially rewrite the genetic code of a portion of the boy’s own stem cells so that his bone marrow can begin to make healthy hemoglobin, the component of red blood cells that carries oxygen.
The boy, who lives in Michigan but does not want to be identified, has needed blood transfusions every three to four weeks of his life because his body doesn’t produce healthy hemoglobin, said Dr. Sureyya Savasan, medical director of the Transplant and Cell Therapy Program.
Those transfusions over time can cause iron to build up within organs such as the liver, lungs and heart and can cause damage severe enough to affect his overall health and life expectancy.
“We are truly excited today (to be) offering this life-changing treatment for our thalassemia patients at the Children’s Hospital of Michigan,” Savasan said, adding that it’s just the first of two breakthrough gene-editing treatments for blood disorders available at the Detroit hospital.