Kite, a Gilead Company, announced data from a pilot study in collaboration with Dana-Farber Cancer Institute that demonstrate Yescarta® (axicabtagene ciloleucel) is well-tolerated in patients living with relapsed or refractory (R/R) primary or secondary central nervous system lymphoma (PCNSL and SCNSL). The findings, which also suggest a trend in efficacy, were presented in an oral session (Abstract #2006) at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Central nervous system lymphoma (CNSL) is an aggressive and rare form of non-Hodgkin lymphoma that has either originated in (primary) or spread to (secondary) the brain, eye, spinal cord or cerebrospinal fluid.

The prognosis of PCNSL has historically been poor with a five-year survival rate of only 30%. More than half of the patients experience a relapse after front-line treatment, with a median survival of approximately two months. R/R CNSL is considered an area of unmet clinical need with no standard of care treatment options.

In this pilot study of 18 patients with CNSL, at a median follow-up of 24.2 months, no treatment-limiting toxicities and no apparent additional risk of adverse events were reported. Immune effector cell-associated neurologic syndrome (ICANS) was observed among 44% of patients (27.8% Grade >3). The objective response rate was 94.4% and the complete response rate was 66.7%.

The median time to best response was three months. The median duration of response was 13.4 months and 9 patients had progressed. At a median follow-up of 24 months, the median progression-free survival was 14.3 months (95% CI: 6.3-NR) and median overall survival was 26.4 months (95% CI: 11.2-NR).

90% of patients developed grade 1 or 2 cytokine release syndrome; two patients developed Ommaya-related meningitis requiring explant with subsequent recovery. One patient developed grade 3 electrographic focal status epilepticus that resolved with anti-epileptic agents. Seven patients have died, all from disease progression.

The Yescarta U.S. Prescribing Information has a BOXED WARNING for the risks of CRS and neurologic toxicities, and Yescarta is approved with a Risk Evaluation and Mitigation Strategy (REMS) due to these risks; see below for Important Safety Information.