United States – European HematologyAnother trial has revealed more positive outcomes of using the CRISPR genetic technique in treating the patients with sickle cell disease, as reported by HealthDay.
Expert Insight
“We are pleased with the results of this gene-editing treatment because of its promising efficacy in sickle cell patients, according to the lead investigator of the study Dr. Rabi Hanna. He is also the division head of pediatric hematology oncology and blood marow transplantation at Cleveland Clinic Children’s.
Sickle cell anemia is an inherited condition that causes painful complications and leads to the formation of abnormally shaped red blood cells known as sickle cells. Sickle cell disease is estimated to affect more than 100,000 Americans and can decrease life expectancy while being more prevalent in Black population.
New developments in gene therapy on what is called CRISPR technology assist in rectifying the disorder by making changes to the defective gene that causes it.
The Cleveland Clinic CRISPR method is known as renizgamglogene autogedtemcel or, for short, reni-cel. It is an experimental procedure that can only be performed once, and it involves using the stem cells of a patient to fix the problem gene.
Details of the Trial
Two of the 18 patients recruiting in a new trial were from the Cleveland Clinic.
In it, patients’ stem cells are collected, followed by their transportation to the laboratory for genetic alteration. Patients also received chemotherapy for the purpose of demolishing the existing stem cells in the bone marrow before the infusion.
“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 seemed to have no significant adverse effects, according to the reports given by Hanna’s group.
“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.
Presentation and Sponsorship
The results were unveiled Thursday at the European Hematology Association 2024 Hybrid Congress (EHA) in Madrid. Findings like these should be taken with a pinch of salt until the details have been published in a research journal, as reported by HealthDay.
The trial was sponsored by gene-editing company Editas Medicine.