Researchers from the Perelman School of Medicine at the University of Pennsylvania and Penn Medicine’s Abramson Cancer Center, have developed a new treatment for glioblastoma (GBM), the most aggressive type of brain cancer, which shows potential to reduce tumors in the short term.
The therapy targets two brain tumor-associated proteins instead of one, which has shown promise in reducing solid tumor growth in patients with recurrent GBM.
Two early clinical trials were conducted, which examined the safety and efficacy of CAR T-cell therapy.
In one trial, described in a paper published in the New England Journal of Medicine (NEJM), three patients with recurrent GBM participated, who had previously undergone standard radiation and chemotherapy.
In the second trial, published in the journal Nature Medicine, six patients with recurrent glioblastoma were treated with CAR T cells. Both studies confirmed that CART-cell therapy was safe and resulted in a reduction in tumor size for all nine patients.
The patient’s tumor size reductions occurred within one to two days in the Nature Medicine research, while in the NEJM study, the reductions occurred one to five days after treatment. One patient’s tumor almost completely regressed after five days, while another patient’s tumor decreased by 60.7% after 69 days following a single treatment.
Dr. Bryan Choi, the lead author of the NEJM study, expressed that the research “provides support for the possible impact of CAR-T cells in solid tumors, particularly in the brain.” He added that it enhances the hope that significant progress can be made.
Dr. Marcela Maus, co-senior author of the NEJM study and director of the Cellular Immunology Program at the Mass General Cancer Center in Massachusetts said, “These results are exciting, but they are also just the beginning — they tell us that we are on the right track in pursuing a therapy that has the potential to change the outlook for this intractable disease.”
However, the researchers stated that further data is necessary to assess the long-term effects of these treatments. Trials in larger and more diverse patient groups are needed to determine their broader applicability.
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