A novel CAR-T cell therapy has demonstrated "promising results" in preclinical studies, potentially offering a new treatment avenue for glioblastoma, the deadliest form of brain cancer. Researchers from King's College London and McMaster University have developed a therapy that may eliminate aggressive tumors and lead to long-term disease survival.
Glioblastoma is notoriously difficult to treat due to its tendency to spread through the brain via thread-like extensions into healthy tissue. The cancer often resists conventional treatments such as surgery, chemotherapy, and radiotherapy, leading to a grim prognosis with a 95% mortality rate within five years of diagnosis. Approximately 3,200 Britons are affected by the disease annually.
The new study, published in the journal Nature, focused on a modified CAR-T cell therapy. CAR-T therapy, already in use for certain blood cancers, works by genetically modifying a patient's own immune cells to recognize and attack cancer cells. In this new approach, scientists engineered the CAR-T therapy to target a protein called GPNMB, which is found on glioblastoma cells and also on macrophages – immune cells often co-opted by the cancer to evade treatment.
In animal models designed to mimic human glioblastoma, the therapy was highly effective. In two key experiments, the treatment completely eradicated tumors in 12 out of 13 treated mice. These mice remained tumor-free for extended periods, exceeding four months in one group and five months in another. The study's lead author, Sheila Singh, a professor of neuro-oncology and neurosurgery, emphasized the need to view glioblastoma not just as a mass of cancer cells but as a complex "tumour-immune ecosystem." The therapy's success stems from its ability to target both the tumor and the supporting immune cells that help it thrive.
While this treatment has not yet been tested in humans, the strong preclinical efficacy suggests it could pave the way for future clinical trials. Current average survival for glioblastoma patients is typically between 12 to 18 months, with no significant treatment advances reported in two decades.
The study's co-author, Shan Grewal, highlighted that this method differs from previous approaches by simultaneously attacking both the tumor and the immune cells that facilitate its evasion of the body's defenses. "Our work suggests we may also need to dismantle the immune support system that helps glioblastoma survive," Grewal stated.
Glioblastoma has affected notable figures, including former Labour politician Dame Tessa Jowell, who died from the disease in 2018, and singer Tom Parker, who passed away in 2022 after an 18-month battle.
Symptoms of brain tumors can include persistent headaches, seizures, nausea, drowsiness, memory problems, weakness on one side of the body, or new difficulties with vision or speech. Individuals experiencing persistent or unusual symptoms are advised to consult a general practitioner. Professor Singh expressed her commitment to developing new treatments through global collaboration to improve outcomes for those affected by brain cancer.