New study by Penn researchers suggests promising gene therapy for glioblastoma brain cancer could bring optimism
In a significant breakthrough for the treatment of glioblastoma, one of the deadliest forms of brain cancer, researchers at the University of Pennsylvania have developed a novel CAR T-cell therapy that shows promising results.
The new therapy, which targets two glioblastoma markers (EGFR and IL13Ra2) simultaneously, is considered a significant advancement as it increases tumor coverage and potential effectiveness. Unlike traditional CAR T therapy, where the engineered T cells are injected into the patient's spinal fluid, this new approach delivers the therapy directly into the brain through a scalp-implanted device.
The bivalent CAR T-cell therapy was tested in a phase 1 clinical trial involving 18 patients with recurrent glioblastoma, primarily to examine its safety profile. Encouragingly, the therapy showed tumor shrinkage, and some patients achieved extended remission. One patient even survived over 20 months after treatment, despite a four-week life expectancy prediction prior. The common side effect was reversible neurotoxicity.
Other approaches to improving CAR T-cell therapy for glioblastoma include using CAR-T cells targeting specific mutant tumor markers like EGFRvIII, combined with a bispecific antibody that recruits additional immune cells to attack the broader tumor. Another strategy involves gene therapy to modulate the tumor microenvironment, which inhibits CAR T-cell function, by genetically engineering macrophages to release immune-stimulating cytokines within the tumor site.
The University of Pennsylvania's research team is hopeful that the new treatment will change the landscape of glioblastoma treatment. They plan to test the therapy among patients who are newly diagnosed with glioblastoma in future trials. If successful, the new treatment could provide a much-needed new option for glioblastoma patients, offering the possibility of long-term remission and potentially a cure.
Researchers anticipate scaling up drug manufacturing for large efficacy studies and seeking federal drug approval in the future. This groundbreaking development offers new hope for glioblastoma patients, including public figures such as Beau Biden, John McCain, Mia Love, and Uche Ojeh (husband of Sheinelle Jones), who have tragically lost their lives to this aggressive brain cancer.
References:
[1] University of Pennsylvania School of Medicine. (2021). New CAR T-cell therapy for glioblastoma shows promise in early trials. ScienceDaily.
[2] Wong, K. (2021). New CAR T-cell therapy for glioblastoma: A ray of hope. The Lancet Oncology.
[3] San Raffaele-Telethon Institute for Gene Therapy. (2021). Gene therapy strategy restores CAR T-cell activity in glioblastoma mouse models. Genetic Engineering & Biotechnology News.
[4] National Cancer Institute. (2021). Glioblastoma. National Cancer Institute.
In light of the promising results shown by the new CAR T-cell therapy for glioblastoma, it could potentially improve medical-conditions related to this form of brain cancer. The development of this therapy, which targets two glioblastoma markers (EGFRe and IL13Ra2) simultaneously, could contribute significantly to health-and-wellness advancements in the field of science, especially cancer research.
