UC San Francisco-based scientists have developed a new immunotherapy that was successful in wiping out all tumor cells inside the brains of the mice that were treated with this intervention. The success of this preclinical study has encouraged the researchers to expedite their plans to test the treatment in human subjects.
The study, which was led by Senthilnath Lakshmanachetty, focused on a deadly pediatric cancer called DMG, or diffuse midline glioma. This cancer is so aggressive that most patients die within a year after being diagnosed, and current treatments have had limited success in controlling this malignancy.
The UCSF scientists wanted to find a way to make immunotherapy helpful to patients diagnosed with this disease. The team noted that the CAR-T cells used in existing immunotherapies only worked for a short time and then the cancer would resume its progression.
The scientists wanted to find out whether engineering CAR-T cells so that they only get activated once they reach the brain could make the therapy more effective against DMG. They identified a protein that is only present inside the brain and they engineered CAR-T cells that can only trigger an anticancer response once they detect the presence of the brain-specific protein.
This meant that the immunotherapy could only become active once it entered the brain. When they infused these B-SYNC cells into mice having human DMG, the scientists were pleasantly surprised to find that the therapy eliminated all the tumors in the brains of the mice. They reintroduced DMG cells into the treated mice nearly two months after the initial treatment and the administered B-SYNC cells were still able to fight off the new tumor cells.
The data shows that the treatment remains active inside the brain long after initial administration. Actually, tests conducted 70 days after the initial treatment indicated that the B-SYNC cells were still actively on guard against tumor cells.
The researchers suggest that the success of their engineered immune cells could be because the cells remain inactive until they get inside the brain, giving them an opportunity to wage a spirited fight against tumors for a lot longer. They point to T-cell exhaustion as a reason why existing immunotherapies have a limited window of activity after which cancer progression resumes once the CAR-T cells are exhausted.
Their approach allows the engineered immune cells to only become active once inside the brain, thereby allowing the cells to pack a heavy punch that ends up eliminating the tumors completely and provides protective benefits long after the treatment was administered.
The team was so encouraged by their findings that they are working to start human trials soon, with April 2027 as a possible start date for clinical trials. The fight against cancer is an all-hands-on-deck undertaking, so other companies like CNS Pharmaceuticals Inc. (NASDAQ: CNSP) engaged in R&D programs geared at developing new treatments for brain cancer will be wishing the UCSF team well as they move B-SYNC to the next levels of testing.
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