Gene therapy for sickle cell disease and thalassemia

Offering life-changing gene therapy for kids with severe sickle cell disease and transfusion-dependent thalassemia

The MACC Fund Center for Cancer and Blood Disorders at Children’s Wisconsin is now offering the newly FDA-approved Casgevy therapy to children 12 years and older who have severe sickle cell disease or transfusion-dependent thalassemia. Casgevy works by adding a gene that makes fetal hemoglobin, which reduces the clinical effects of sickle cell disease or thalassemia.

Children's Wisconsin will be offering gene therapy in children with severe symptoms of sickle cell disease and children receiving blood transfusions to manage their thalassemia. Symptoms of sickle cell disease are broad and can range from severe acute intermittent pain, chronic daily pain, lung problems, stroke, enlarged spleens, kidney problems and bone necrosis in hips and shoulders.

Casgevy resulted in more than 93 percent of patients having at least 12 consecutive months without a severe vaso-occlusive crisis in the two-year study time after administration. Additionally, 92 percent of patients with thalassemia were transfusion-free for more than 12 months after receiving Casgevy.

How Casgevy works and what to expect

Casgevy is made from the patient’s own blood stem cells, which are modified using CRISPR technology, and returned to the patient in a single-dose infusion as part of a stem cell transplant. Prior to the infusion, the patient’s stem cells are collected for the modification, and then the patient goes through a round of high-dose chemotherapy intended to destroy the existing cells from the patient’s bone marrow that make the mal-formed blood cells. The gene-modified cells that are infused produce fetal hemoglobin that can overcome the need for blood transfusions in most patients and improve severe pain crises in patients with sickle cell disease. 

MACC Fund Center physicians estimate the total treatment will involve an approximate two-month hospital stay.

Contact our team to refer a patient to the MACC Fund Center for Cancer and Blood Disorders.