New gene therapy extends life for sickle cell patients
A new era of hope — and the opportunity to live longer and without excruciating pain — has arrived for patients battling sickle cell disease. WashU Medicine and St. Louis Children’s Hospital have become one of only a few academic medical centers in the U.S. to offer a newly FDA-approved, life-changing gene therapy.
Each year, an estimated 2,000 children in the U.S. — most of them Black — are born with sickle cell disease, a life-threatening condition that makes red blood cells sticky and prone to clumping, blocking blood flow and oxygen from reaching muscles and organs. This oxygen deprivation leads to severe pain episodes known as crises, as well as strokes and organ damage. Monthly blood transfusions only temporarily ease symptoms by introducing healthy red blood cells and can lead to complications including infections.
This cutting-edge treatment, called Lyfgenia, utilizes a patient’s own genetically modified stem cells to produce healthy red blood cells. Unlike traditional donor transplants, this approach eliminates the grueling search for a matching donor and significantly reduces immune complications.
The results speak for themselves. Martin Mwita Jr., a 21-year-old from Nebraska who has spent his entire life suffering from debilitating pain crises, strokes and frequent hospitalizations, became the region’s first patient to undergo the procedure. Just six weeks after receiving the infusion, a remarkable 85% of his red blood cells were healthy. Now, despite being told as an infant that he likely wouldn’t live past age 35, Martin is finally free to live an active life, ride bikes and pursue his dreams of studying business without the shadow of his disease.
Shalini Shenoy, MD, a professor of pediatrics at WashU Medicine and a renowned pediatric hematologist and oncologist of Siteman Kids at St. Louis Children’s Hospital, led the orchestration of the intensive treatment, which is reserved for patients with severe forms of sickle cell disease and requires specialized clinical expertise unavailable outside of an academic setting. Because of the demands of gene therapy, which requires patients to first be treated with high-dose chemotherapy, patients undergo extensive evaluation to ensure they are physically and mentally able to endure the procedure and potentially severe side effects.
“We could not have imagined curing sickle cell disease with gene therapy even 10 years ago,” said Shenoy. “It’s a huge step forward.”
