Recent clinical trial results indicate CAR-T cell therapy’s first successes against solid tumors, offering hope for children with deadly brain and spinal cord tumors like diffuse intrinsic pontine glioma (DIPG). Published in Nature, the study involved 11 participants, nine of whom showed benefits, including tumor shrinkage and improved neurological function. One participant experienced a complete response, with all detectable traces of cancer disappearing. The trial, conducted at Stanford Medicine, targeted the GD2 surface marker prevalent in DIPG tumors. CAR-T cells were administered both intravenously and intracerebroventricularly, the latter method resulting in fewer side effects. Most participants showed improved symptoms and reduced tumor size, with some regaining lost abilities. The trial’s promising results, including a median survival of 20.6 months and one participant still healthy four years post-diagnosis, have prompted further research to optimize the therapy. This breakthrough has earned a regenerative medicine advanced therapy designation from the FDA, expediting future approval processes.

To read more, click here.

[Source: GEN, November 13, 2024.]