CAR-T cell therapy shows promise against deadly childhood brain cancer

A clinical trial at Stanford Medicine has shown that CAR-T cell therapy, a cancer treatment, can effectively treat brain tumours in children.

In Short

• CAR-T cell therapy has shown promise in treating rare brain tumours in children
• It is a new cancer treatment that was earlier used in blood cancers
• A patient saw his brain tumour disappear entirely

A clinical trial has shown promising results in treating brain tumours in children using an immune-cell therapy known as CAR-T cells. The therapy not only shrank tumours but also restored neurological functions in several patients.

For one young participant, it eliminated all detectable traces of a typically incurable cancer called diffuse intrinsic pontine glioma (DIPG).

This is one of the first successes of CAR-T cells against solid tumours. DIPG, a rare and aggressive brain cancer, has a five-year survival rate of less than 1%, with most patients surviving only about a year after diagnosis.

The trial, conducted at Stanford Medicine, involved 13 participants with either DIPG or similar brain and spinal cord tumours. Out of 11 who received the treatment, nine showed significant benefits, including improved physical abilities and reduced tumour sizes.

One patient, named Drew experienced a complete response, meaning his tumour disappeared. Four years after his diagnosis, Drew remains healthy.

“This disease is usually fatal, but this therapy has shown meaningful tumour reductions and clinical improvements,” said Dr Michelle Monje, lead researcher of the trial. “While there's still work to be done, Drew’s case gives us hope.”

CAR-T cells are engineered from a patient's immune cells to target cancer. Previously successful in blood cancers, this trial tested their effectiveness on solid tumours. The treatment was tailored to target GD2, a marker found in DIPG cells.

Although the therapy caused some initial side effects like fever and inflammation, patients showed notable progress. Drew, who was diagnosed at 16, regained many abilities lost to the disease, including walking unassisted. He is now studying forestry in college.

The research team is refining the therapy to improve its safety and effectiveness, with hopes of wider availability in the future.