A chimeric antigen receptor (CAR) T-cell therapy approved for patients with large B-cell lymphoma has produced positive results in a pilot study involving patients with relapsed, treatment-resistant central nervous system (CNS) lymphoma. These findings from a small cohort were presented by Nayak et al at the 2024 ASCO Annual Meeting (Abstract 2006).
CNS lymphoma is a rare non-Hodgkin lymphoma in which malignant cells form in the lymph tissue of the brain or spinal cord. Although initial treatment is often effective, better treatments are needed when the disease recurs.
Axicabtagene ciloleucel was found to be safe and well tolerated in the 18 study participants and produced an overall response rate of 94%. At a median follow-up of 24.2 months, median duration of response was 13.4 months and 9 of 18 patients experienced disease progression. The median progression-free survival was 14.3 months (95% confidence interval [CI] = 6.3 months to not reached), and the median overall survival was 26.4 months (95% CI = 11.2 months to not reached). The median time to best response was 3 months. Seven patients have died, all from disease progression.
The most commonly reported side effects—cytokine-release syndrome and immune effector cell–associated neurologic syndrome (ICANS)—were manageable.
The study authors concluded, “Axicabtagene ciloleucel was safe and well-tolerated in patients with [CNS lymphoma], with encouraging efficacy and median progression-free survival and durability of response of more than 1 year. There was no apparent additional risk of adverse neurologic events, including ICANS, from axicabtagene ciloleucel.”
Disclosure: For full disclosures of the study authors, visit coi.asco.org.
