Gilead's CAR-T cell therapy shows promise in deadly brain cancer

A Gilead-backed CAR-T cell therapy targeting two proteins (EGFR and interleukin-13 receptor alpha 2) showed promising results in a study of 18 patients with recurrent glioblastoma, with tumors shrinking in 62% of those with measurable disease; however, the benefit was largely temporary, with many patients relapsing within a few months. Researchers at the University of Pennsylvania and Gilead's Kite cell therapy unit are working on a triple-target version of the therapy to improve persistence and plan to test it in newly diagnosed patients, reflecting significant unmet need given limited treatment options for this aggressive brain cancer.

Analysis

Gilead Sciences' (GILD.O) Kite cell therapy unit presented promising, albeit early, clinical data for its dual-target CAR-T therapy in recurrent glioblastoma, a highly aggressive brain cancer with limited treatment avenues. The study, involving 18 patients and presented at the American Society of Clinical Oncology meeting, demonstrated tumor shrinkage in 62% of the 13 patients with measurable disease, a significant outcome given the historical difficulty in achieving such responses in this cancer type. While these results, also published in Nature Medicine, are encouraging, the therapeutic benefit has been largely temporary, with many patients relapsing within two to three months, a key challenge highlighted by Kite's EVP Cindy Perettie who noted a lack of desired persistence. Manageable side effects, including fever and neurotoxicity, were observed. To address the durability issue, Kite is advancing a triple-target CAR-T construct, aiming for clinical trials next year, and also plans to evaluate the current therapy in newly diagnosed patients, where outcomes might be improved. The considerable patient demand for trial enrollment, with reported interest far exceeding available slots, underscores the substantial unmet medical need and the potential market for an effective glioblastoma treatment, for which only one approved therapy outside of radiation currently exists.

Key Decisions for Investors

• Investors should acknowledge the promising early efficacy of Gilead's CAR-T therapy in glioblastoma, reflected in the 62% tumor shrinkage, but remain cognizant of the significant hurdle of limited treatment persistence.
• Closely monitor Gilead's progress with the development of its triple-target CAR-T construct, expected to enter clinical trials next year, and upcoming data from trials in newly diagnosed glioblastoma patients, as these will be critical for assessing long-term viability.
• Given the high unmet need in glioblastoma, positive developments could be impactful for Gilead's Kite oncology franchise; however, the inherent risks of early-stage oncology drug development, particularly in solid tumors, necessitate careful risk assessment.