Response to US study of CAR-T in childhood brain tumors

One of the eleven children and young adults with the incurable brain tumor DIPG who received CAR-T cell therapy appears to have been cured. Four years after the treatment, this 20-year-old young man is still alive. Nine of the eleven children in the study saw an improvement in their symptoms. In four of them, the tumor shrank. The average survival time increased by seven months. The study was published in Nature today.

Read the article in De Volkskrant here (behind a paywall).

Dr. Jasper van der Lugt, pediatric oncologist specializing in brain tumors, says: ‘The results are promising, but I am cautious. You had to be well enough and your tumor had to be small enough to take part in this study. And many of the children and young people in the study had received radiation shortly before the CAR-T cell therapy. That makes it hard to untangle the effect of the radiotherapy and the CAR-T treatment.

‘All children and young people in the study experienced serious side effects. With an incurable brain tumor, you sometimes take more risks, but it remains a precarious balance. Children with DIPG usually do not have long to live. It is important to carefully weigh the pros and cons of participating in a study of a new treatment. At the Máxima, we hope to open our own clinical trial at the end of next year into another type of CAR-T for children with a brain tumor, including DIPG.’

Dr. Friso Calkoen, pediatric oncologist specialized in CAR-T, adds: ‘So far, CAR T-cells have mainly worked on forms of blood cancer, such as leukemia. It is more difficult to get them to work in tumors in organs or tissues, because they have a kind of protective wall around them. They are also more difficult to distinguish from healthy tissue around them. All over the world, including here at the Princess Máxima Center, scientists are studying whether this promising form of immunotherapy can also work for brain and solid tumors.’