Dr Monje on the Role of a Novel GD2-Directed CAR T-Cell Therapy in Diffuse Midline Glioma

“We saw dramatic improvements in neurological function [such as] return of hearing, movement, taste sensation, and touch sensation. Patient 10 on the trial had a dramatic and ultimately complete response and he is still cancer-free.”

Michelle Monje, MD, PhD, the Milan Gambhir Professor of Pediatric Neuro-Oncology and a professor by courtesy of neurosurgery, pediatrics, pathology, and psychiatry and behavioral sciences at Stanford University, as well as a member of the Institute for Stem Cell Biology and Regenerative Medicine, the Maternal & Child Health Research Institute, the Wu Tsai Neurosciences Institute, and the Stanford Cancer Institute, discussed the role of a novel GD2-directed CAR T-cell therapy for the treatment of pediatric patients with H3K27M-mutated diffuse midline glioma, which includes diffuse intrinsic pontine glioma and spinal diffuse midline glioma.

Among the patients on the trial, 1 had ataxia and required a wheelchair for long distances at the hospital, Monje began. However, within 2 weeks following treatment with the CAR T-cell agent, the patient regained his coordination and was able to walk 2-mile hikes with his family, she noted. Dramatic improvements were observed with the CAR T-cell therapy, particularly with the neurological function, return of hearing, movement, taste sensation, and touch sensation, she explained.

Furthermore, another patient on the trial demonstrated a dramatic and complete response to the therapy; the patient is still cancer-free 4 years after starting treatment, Monje continued. Nevertheless, patients experienced a wide range of responses to the CAR T-cell therapy, she emphasized. Although several patients demonstrated antitumor activity, some children on the study did not respond to treatment, according to Monje. Specifically, some patients had achieved early and transient benefit, then lost it or had not demonstrated benefit at all, she asserted. Of note, there were no reported outliers with treatment, as the investigators evaluated the tumor volume as a measure of tumor response, she added.