A clinical trial at Stanford Medicine has yielded promising results for children with deadly brain and spinal cord tumors, including diffuse intrinsic pontine glioma (DIPG). The study demonstrated that CAR-T cell therapy can shrink tumors, restore neurologic function, and, in one case, eradicate all detectable traces of cancer.
The findings, published in Nature, have led to the FDA granting the therapy a regenerative medicine advanced therapy designation, which fast-tracks the approval process.
CAR-T Cell Success Against Solid Tumors
This trial represents one of the first successes of CAR-T cells against solid tumors. DIPG, a rare and aggressive brain cancer, has a dismal five-year survival rate of less than 1%.
The trial included 13 participants with DIPG or similar brain and spinal cord tumors. Of the 11 who received the treatment, nine experienced significant benefits, such as improved physical abilities and reduced tumor sizes. Notably, one patient achieved a complete response, with the tumor disappearing entirely. Four years after diagnosis, this individual remains healthy.
"This disease is usually fatal, but this therapy has shown meaningful tumor reductions and clinical improvements," said Dr. Michelle Monje, lead researcher of the trial. "While there's still work to be done, this one case gives us hope."
The CAR-T cell therapy was designed to target GD2, a marker present in DIPG cells. Initial side effects, including fever and inflammation, were observed, but patients showed notable progress. The patient who experienced a complete response, diagnosed at 16, regained abilities lost to the disease, including walking unassisted, and is now studying forestry in college.
CAR-T Cell Therapy and Solid Tumors
CAR-T cells are engineered from a patient's immune cells to target cancer cells. While CAR-T cell therapy has shown success in hematological cancers, its application to solid tumors has been challenging due to factors such as the hostile tumor microenvironment and tumor heterogeneity. Physical barriers within solid tumors can also impede CAR-T cell infiltration, reducing therapeutic efficacy. Recent clinical trials offer promise in treating specific solid tumors, but further research is needed to enhance the potential of CAR-T cell therapy.
