07 March 2022

Gene therapy has enabled more than 90% of patients with transfusion-dependent thalassemia to end monthly blood transfusions, according to an international phase 3 clinical trial.

The trial included 22 patients, aged from four years to 34. The treatment, called beti-cel, used the patient’s own stem cells that were treated in the lab with a modified virus to add functional copies of the HBB gene that is defective in thalassemia.

Before the new cells were infused, patients received chemotherapy, which typically involves a hospital stay of at least four to five weeks for close monitoring.  

Trial participants were monitored from 13 months to four years after receiving their new cells. The participants typically reached transfusion-free status about one month after the autologous stem cell transplantation.

Writing in the New England Journal of Medicine, the authors say treatment-related adverse events were typical of autologous hematopoietic stem cell transplantation and the required chemotherapy.

Four patients had at least one adverse event that was considered to be related or possibly related to gene therapy. These were all non-serious, except for one case of thrombocytopenia.

There were no cases of secondary cancer due to the chemotherapy or gene therapy.

Study co-author Dr Jennifer Schneiderman, from the Center for Cancer and Blood Disorders at Lurie Children’s Hospital and associate professor of paediatrics at Northwestern University Feinberg School of Medicine, said: “It is impressive that so many patients in the study, including the youngest patients, acquired transfusion independence that was durable.

“The current study represents the next step in moving this intervention into clinical practice, which will increase access to a potential cure for patients with transfusion-dependent thalassemia.”

Source: Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. (2022) “Betibeglogene Autotemcel Gene Therapy for Non–β0/ β0 Genotype Beta-Thalassemia.” New England Journal of Medicine, doi: 10.1056/NEJMoa2113206

Link: https://www.nejm.org/doi/full/10.1056/NEJMoa2113206

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