In March 2024, an extraordinary discovery was made that could potentially transform cancer treatment.

A groundbreaking therapeutic experiment used to treat glioblastoma, a deadly form of brain cancer, showed “dramatic responses within days” for the participants in the trial.

Researchers and surgeons from the Mass General Brigham healthcare system employed a novel approach to CAR-T therapy, a cutting-edge, personalized treatment designed to activate the body’s immune system to target cancer cells.

CAR-T therapy typically treats leukemia by using the patient’s own immune system. The process involves extracting a patient’s blood, which is then sent to a lab where it is "trained" to fight cancer cells before being reintroduced to the patient’s body for treatment.

In this case, the team at Mass General Brigham combined an existing variation of CAR-T therapy with antibodies to treat three patients with recurrent glioblastoma. The results were nothing short of remarkable.

Within just two days, one patient saw an 18.5% reduction in tumor size, as reported in a study published in The New England Journal of Medicine. By day 69, the tumor had shrunk by an impressive 60.7%. According to Mass General Brigham, another patient’s tumor “regressed rapidly.”

An MRI taken five days after treatment for the third patient revealed that a single infusion had led to “near-complete tumor regression.”

“The CAR-T platform has completely changed how we approach cancer treatment,” said Bryan Choi, MD, PhD, a neurosurgeon and associate director of the Center for Brain Tumor Immunology and Immunotherapy, Mass General Cancer Center, and Department of Neurosurgery. “However, treating solid tumors like glioblastoma remains a challenge because cancer cells are not uniform, and the cells within the tumor vary.”

He added, “Our strategy combines two types of therapies, enabling us to approach glioblastoma treatment in a more expansive and potentially more effective manner.”

Mass General Brigham stated that studies like this offer “great promise for treating previously incurable conditions,” giving hope to those battling these diseases.

While CAR-T therapy is commonly used to treat blood cancers, this study demonstrates its potential to treat solid tumors as well.

This offers new hope for those affected by these conditions, but it’s crucial to note that this trial involved only three patients. Further research is needed before this treatment can be widely used for solid tumors.

Elizabeth Gerstner, MD, a neuro-oncologist at Massachusetts General Hospital and co-author of the study, remarked, “We have seen a dramatic and rapid response in these three patients. Our work shows progress, but we have much more to do.”

Marcela Maus, MD, PhD, director of the Cellular Immunotherapy Program, echoed this sentiment, stating, “These results are promising, but they are just the beginning. They confirm we are on the right path toward developing a therapy that could change the outlook for this challenging disease. We haven’t cured anyone yet, but that remains our ambitious goal.”