Six months after being treated with base editing — a technique that modifies the genetic code to reduce side-effect risk — and a second bone marrow transplant, the 13-year-old patient, Alyssa, is still in remission and is doing well, according to a GOSH news release.
It’s tricky to treat T-cell leukemia with traditional CAR T-cell therapy because T-cells will kill each other before they can be used in treatments, but by altering a donor’s CAR T-cells, base editing ensures the cells only attack cancerous ones and not themselves.
Alyssa was diagnosed with leukemia in May 2021, and despite receiving chemotherapy and a bone marrow transplant, full remission couldn’t be achieved.
“Only after she received her CD7 CAR-T cell therapy and a second bone marrow transplant in GOSH she has become leukemia free,” Robert Chiesa, MD, a bone marrow transplant consultant at GOSH, said in a statement. “This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months.”
The researchers plan to recruit 10 patients with T-cell leukemia “who have exhausted all other treatment options” to further test the base editing technique.
At the Becker's 11th Annual IT + Revenue Cycle Conference: The Future of AI & Digital Health, taking place September 14–17 in Chicago, healthcare executives and digital leaders from across the country will come together to explore how AI, interoperability, cybersecurity, and revenue cycle innovation are transforming care delivery, strengthening financial performance, and driving the next era of digital health. Apply for complimentary registration now.
