TUESDAY, June 18, 2024 (HealthDay News) — There’s more encouraging news in the battle against sickle cell disease, as another trial finds the CRISPR gene-editing therapy offers impressive results for patients.
“It is encouraging that this gene-editing treatment continues to show promising efficacy for patients with sickle cell anemia,” said the study’s principal investigator, Dr. Rabi Hanna. He is chairman of the division of pediatric hematology, oncology, and blood and marrow transplantation at Cleveland Clinic Children’s Hospital and Children’s Hospital.
Sickle cell anemia is a painful inherited genetic disorder that creates deformed, sickle-shaped red blood cells. More than 100,000 Americans are believed to have sickle cell disease, which can shorten life expectancy and is much more common among African Americans.
Recent advances in what is known as CRISPR gene editing technology help correct the disorder by modifying the underlying genetic abnormality behind it.
The Cleveland Clinic’s CRISPR approach is called renizgamglogene autogedtemcel (abbreviated as “reni-cel”). It is a one-time experimental 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 collected and then sent to the laboratory for gene editing. Patients also underwent chemotherapy to make room for the new stem cells to infuse into their bone marrow.
“Following treatment, all patients successfully regained their white blood cells and platelets,” according to a news release from the clinic. “Importantly, all patients have remained free of painful events since treatment, and those followed for five months or longer have seen their anemia resolve.”
The treatment also did not appear to have serious side effects, Hanna’s group reported.
“These latest results offer hope that this new experimental treatment will continue to show progress and bring us closer to a functional cure for this devastating disease,” he said.
The findings were presented Thursday at the European Hematology Association’s Hybrid Congress (EHA) 2024 in Madrid. These findings should be considered preliminary until published in a peer-reviewed journal.
The trial was funded by the gene editing company Editas Medicine.
More information
Learn more about sickle cell disease from the Sickle Cell Disease Association of America.
SOURCE: Cleveland Clinic, press release, June 14, 2024
