South SFV Today

An immune-cell therapy trial at Stanford Medicine has shown promising results in treating a lethal childhood brain cancer. The trial, which uses engineered CAR-T cells, demonstrated significant tumor reduction and improved neurological function in participants. Notably, one patient experienced a complete disappearance of the tumor.

The study focused on children with diffuse intrinsic pontine glioma (DIPG) and other related tumors. Of the 11 participants treated with CAR-T cells, nine showed benefits, including reduced tumor size and improved physical abilities. One participant's tumor vanished entirely from brain scans.

Michelle Monje, MD, PhD, the lead author of the study and professor at Stanford Medicine, stated, "This is a universally lethal disease for which we’ve found a therapy that can cause meaningful tumor regressions and clinical improvements." The findings were published in Nature on November 13.

The trial received support from various institutions, including the U.S. Food and Drug Administration's regenerative medicine advanced therapy designation. This designation aims to expedite the approval process for treatments showing potential against life-threatening conditions.

CAR-T cell therapy involves modifying a patient's T cells to target specific cancer cells. Although previously successful against blood cancers, this trial marks one of the first successes against solid tumors using CAR-T cells.

Crystal Mackall, MD, senior author of the study and professor at Stanford Medicine, remarked on the unexpected clinical benefits observed: "We could see clear evidence of reversibility."

The trial continues to enroll participants as researchers work to refine treatment protocols and reduce side effects like cytokine release syndrome. Future research will focus on enhancing treatment efficacy by targeting biological aspects of tumors.

Drew, a participant who experienced complete tumor remission after treatment, shared his hopes for future patients: “I’m hoping they’ll learn from all my successes to help other kids.” Diagnosed with DIPG in 2020 during high school, Drew now pursues college studies while receiving periodic CAR-T cell infusions.

The research team acknowledges contributions from families who donated tissue samples after losing loved ones to similar diseases. These donations have been instrumental in advancing research efforts.

Stanford Medicine continues its collaboration with other institutions to improve outcomes for pediatric cancer patients through immunotherapy advancements.