Gene Therapy Shows Promise in Lowering Stroke Risks for Sickle Cell Patients

A recent study from St. Jude Children's Research Hospital indicates that gene therapy could be an effective approach to reducing stroke risk factors in individuals with sickle cell disease. Sickle cell disease disrupts normal blood flow due to abnormally shaped red blood cells that can clog small blood vessels, including those in the brain, increasing the likelihood of brain ischemia and stroke. One major contributor to this risk is the elevated speed of blood flow in the brain, which hampers oxygen delivery to neural tissues.

The research involved three patients undergoing gene therapy, with blood flow in their brains monitored via MRI before and after treatment. Results showed a significant decrease in blood flow velocity—ranging from 22% to 43%—bringing levels close to normal and remaining stable over time. Dr. Akshay Sharma, the study's lead author, stated that these findings provide promising physiological evidence that gene therapy can mitigate neurovascular complications associated with sickle cell disease.

The study explains that in sickle cell disease, the crescent-shaped red blood cells struggle to pass through small vessels, causing blockages and oxygen deprivation in the brain. To compensate, the body accelerates blood flow, which paradoxically increases stroke risk. By normalizing blood flow, gene therapy could potentially prevent these adverse events.

Compared to standard treatments like hydroxyurea or blood transfusions, gene therapy demonstrated more durable and effective blood flow improvement. Hydroxyurea only modestly affects cerebral blood flow, while transfusions have short-term benefits requiring continuous administration. Although bone marrow transplants also restore normal blood flow long-term, gene therapy may offer a similar benefit with less complexity.

This preliminary evidence supports expanding clinical trials of gene therapy to include patients at risk for stroke, who were previously excluded due to high-risk status. As Dr. Sharma noted, gene therapy emerges as a promising new option alongside bone marrow transplants to safeguard brain health in sickle cell disease patients.

These findings, published in the American Journal of Hematology, pave the way for further research to optimize gene therapy for neurovascular protection, potentially transforming treatment strategies for stroke prevention in sickle cell disease.

Source: https://medicalxpress.com/news/2025-06-gene-therapy-factors-sickle-cell.html