In a major milestone for the use of CRISPR in cancer therapy, the first clinical trial testing CRISPR-edited T cells in patients with resistant leukemia has reported success, with three out of four patients responding to treatment.
The trial, which was led by University of Pennsylvania researchers, used CRISPR to edit the T cells of patients with acute lymphoblastic leukemia (ALL) who had relapsed after standard treatment. The goal was to create T cells that could better recognize and target the cancer cells.
After the edited T cells were infusion into the patients, three out of four patients achieved complete remission of their leukemia, and the fourth patient had a partial response.
The results are preliminary, but they offer hope that CRISPR-edited T cells could be a powerful new tool for treating leukemia, and potentially other types of cancer.
The trial is still ongoing, and the researchers are planning to enroll more patients. But the initial results are encouraging, and suggest that CRISPR-edited T cells could become a new standard of care for patients with resistant leukemia.
A new cancer treatment that uses CRISPR-edited T cells has shown promise in early clinical trials, offering hope to children with resistant leukemia.
In a Phase 1 clinical study, published in the journal Nature Medicine, 29 children with relapsed or refractory acute leukemia received a single infusion of CRISPR-edited T cells. Twenty-seven of the patients showed signs of disease remission, with no serious adverse effects reported.
The study used a CRISPR-based approach to edit the T cells, which are a type of white blood cell that helps the body fight infection. The edited T cells were then infused into the patients.
“This is a landmark study that demonstrates the potential of CRISPR-based therapeutics,” said co-lead author Dr. Ewelina Mamot, from the University of California, San Francisco. “The results suggest that this approach is safe and can achieve durable remissions in children with leukemia.”
The next step is to conduct larger clinical trials to confirm the efficacy of this new treatment approach. If proven effective, CRISPR-edited T cells could offer a new treatment option for children with leukemia who have few other options.