Pioneering gene therapy as a treatment for sickle cell disease
Gene therapy is a new and exciting treatment option available for patients with sickle cell disease (SCD). Children’s National Hospital is one of the few pediatric hospitals in the country that offers both FDA-approved sickle cell disease gene therapies: CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel).
What this means
Gene therapy involves an autologous transplant, taking the patient’s own stem cells, genetically changing those stem cells and then, after chemotherapy, infusing those stem cells back into the patient to make healthy blood.
“I’m excited about gene therapy for sickle cell disease. I think it has the potential to be a curative option for every single child with sickle cell disease,” said Robert Nickel, M.D., hematologist at Children’s National.
Currently both treatments are only approved for patients 12 years and older with severe disease. Children’s National was the first hospital in the world to collect stem cells for the LYFGENIA™ treatment.
Moving the field forward
Clinical trials hold incredible promise to advance the care of SCD. Children’s National continues to pioneer transplant therapies to cure SCD and is one of the leading centers participating in clinical trials of new treatments for this condition.
Experts at Children’s National are leading a multi-site clinical trial of a chemotherapy-free transplant approach for SCD using a matched sibling donor. This chemotherapy-free approach has less toxicity and side effects for children undergoing transplant.
In addition, Children’s National has been leading the way with innovative approaches to support sickle cell patients. “We’re providing alternative approaches to pain such as healing touch, acupuncture, massage VR technology, physical therapy and exploring other ways of treating pain in an integrated manner,” said Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease Program at Children’s National.
The team is also exploring non-opioid treatments, such as intravenous citrulline, a naturally occurring amino acid that has been proven to enhance blood flow and potentially alleviate pain in treated patients in preliminary studies under the direction of Suvankar Majumdar, M.D., chief of Hematology at Children’s National.
