Axicabtagene Ciloleucel (axi-cel) in Relapsed/Refractory Central Nervous System Lymphoma (CNSL)

At ICML 2025, Prof Caron Jacobson from the Dana-Faber Cancer Institute presented a poster on a pilot study evaluating axicabtagene ciloleucel (axi-cel) in patients with primary and secondary CNS lymphoma, primarily in the second line and beyond. The study involved 18 patients, most of whom had refractory disease, with a mix of primary (two-thirds) and secondary (one-third) CNS lymphoma. Results showed that axi-cel was safe, with toxicity similar to systemic lymphoma patients, without increased risks of neurological toxicity or cerebral oedema. Preliminary efficacy data indicated a 67% complete response rate, with durable responses, a median PFS of 14 months, and a median OS not yet reached. Single-cell RNA sequencing (scRNAseq) analysis of CAR T-cells in the cerebrospinal fluid and peripheral blood revealed a distinct Type 1 IFNg gene expression signature in the CSF compared to blood.