(HealthDay News) — There’s more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.
“It’s encouraging that this gene-editing treatment continues to show promising efficacy for sickle cell patients,” said study lead investigator Dr. Rabi Hanna. He’s chair of the division of pediatric hematology oncology and blood and marrow transplantation at Cleveland Clinic Children’s.
Sickle cell anemia is a painful, inherited genetic disorder which creates misshapen sickle-shaped red blood cells. Over 100,000 Americans are thought to have sickle cell disease, which can shorten life spans and is much more common among Black Americans.
Recent advances in what’s known as CRISPR gene-editing technology helps correct the disorder by tweaking the underlying gene abnormality behind it.
The Cleveland Clinic CRISPR approach is called renizgamglogene autogedtemcel (shortened to “reni-cel”). It’s am experimental one-time treatment that uses the patient’s own stem cells to correct the genetic abnormality.
Two of the 18 patients in the new trial were treated at the Cleveland Clinic.
In the procedure, patients’ stem cells are harvested and then sent for gene editing in the lab. Patients also underwent chemotherapy to make room for the new stem cells to be infused into their bone marrow.
“Following treatment, all patients successfully regained their white blood cells and platelets,” according to a clinic news release. “Importantly, all patients have remained free of painful events since treatment, and those followed for five months or greater have seen their anemia resolve.”
The treatment also appeared to have no serious side effects, Hanna’s group reported.
“These latest results offer hope that this new experimental treatment will continue to show progress and get us closer to a functional cure for this devastating disease,” he said.
The findings were presented Thursday at the European Hematology Association 2024 Hybrid Congress (EHA) in Madrid. Such findings should be considered preliminary until published in a peer-reviewed journal.
The trial was funded by gene-editing company Editas Medicine.
Sickle cell disease (SCD) is a painful inherited blood disorder affecting red blood cells. Normally, these cells are round and flexible, carrying oxygen throughout the body. In SCD, a genetic mutation makes the cells sickle-shaped, stiff, and prone to clumping. These blockages hinder oxygen flow, causing severe pain, organ damage, and fatigue.
SCD disproportionately affects Black Americans. Roughly 1 in 365 Black babies are born with the disease, compared to 1 in 3,330 white babies. This disparity has historical roots. The sickle cell trait, carrying one copy of the mutated gene, actually offers some protection against malaria, a once-rampant disease in Africa, where many Black ancestors originated. While the trait offered a survival advantage, inheriting two copies (causing SCD) did not.
The consequences of SCD for Black Americans are significant. Patients experience frequent, excruciating pain episodes called crises, requiring frequent hospital visits and impacting work and school. Studies show Black patients with SCD are more likely to experience complications like blindness and require blood transfusions compared to other races.
Beyond the physical toll, SCD can lead to social and economic hardship. The unpredictable nature of crises can strain employment and education. Furthermore, historical distrust in the healthcare system due to past racism can lead to delays in seeking treatment.
More information
Find out more about sickle cell disease at the Sickle Cell Disease Association of America.
SOURCE: Cleveland Clinic, news release, June 14, 2024
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