The experimental treatment was used on 10 patients with leukemia that did not respond to chemotherapy and showed unprecedented remission results.
A pioneering gene therapy is offering new hope for patients with previously incurable leukemiaaccording to initial results from a clinical trial this week in the New England Journal of Medicine.
The experimental treatment, known as BE-CAR7, is the first of its kind in the world and has demonstrated impressive remission rates in children and adults with aggressive T-cell acute lymphoblastic leukemia (T-ALL).
The first patient to receive the therapy, Alyssa Tapley, now 16, was diagnosed in 2021 and did not respond to chemotherapy or a bone marrow transplant. With no conventional options left, he participated in the clinical trial in the hope that it could help others, even if it didn’t save his own life. Since then, the Tapley cancer remains undetectableand the young woman returned to school, resumed outdoor activities and now dreams of becoming a research scientist.
Developed by scientists at Great Ormond Street Hospital (GOSH) and University College London, BE-CAR7 is a multi-step gene editing therapy that transforms healthy white blood cells from donors into powerful cancer-fighting agents. Donor cells are altered to make them “universal” by removing receptors that trigger immunological rejection. Other immune markers, such as CD7 and CD52, are eliminated to protect the modified cells from both the patient’s immune system and the immunosuppressive medications used during treatment.
Finally, researchers insert a new DNA sequence that allows cells to express a chimeric antigen receptor (CAR) — the mechanism that allows them to identify and destroy cancerous T cells. Once infused into the patient, these modified cells quickly attack the leukemia, ideally eliminating the disease within four weeksat which point patients can undergo a bone marrow transplant to restore their immune system.
Recently released results from the phase 1 clinical trial, presented at the annual meeting of the American Society of Hematology, show that 82% of 10 participants (eight children and two adults) achieved deep remission and became eligible for transplant. 64% remain cancer freesome for three years already, says .
The researchers warn, however, that the therapy is intensive and not all participants survived. Recovery after transplantation can be prolonged and vulnerable to complications. Still, doctors describe the results as unprecedented for such an aggressive disease. “We saw impressive responses in eliminating leukemias that seemed incurable,” said hematologist Deborah Yallop.
With initial results so promising, philanthropic funding was secured for treat 10 more patients.
