Breakthrough CAR T Cell Therapy Offers Options for Children with Deadly Brain Tumor, DMG

A groundbreaking clinical trial at Seattle Children’s started by Dr. Nicholas Vitanza, who received early funding from Cookies for Kids’ Cancer, has shown highly promising results in treating diffuse midline glioma (DMG), one of the deadliest pediatric brain tumors. By using an innovative form of targeted immunotherapy called CAR -T cell therapy, Dr. Vitanza and his team are giving new hope to families facing this devastating diagnosis. Some children with DMG are seeing signs of real progress — including tumor shrinkage and extended survival — in a disease long considered untreatable. This research marks a major step forward in pediatric cancer treatment and the future of precision medicine. 

What Is DMG and Why It's So Hard to Treat 

Diffuse midline glioma (DMG), which, when located in a critical part of the brain called the pons, is named DIPG, is one of the most aggressive and lethal brain tumors in children. It typically grows diffusely through critical brain and spinal cord structures, making it impossible to completely remove with surgery. Traditional treatments like radiation can temporarily slow tumor growth, but there is no known cure — and the average survival is only 12 months. 

Meet Dr. Nicholas Vitanza and His Team at Seattle Children’s 

Dr. Nicholas Vitanza, a pediatric neuro-oncologist at Seattle Children’s, is one of the leading researchers working to change that. His team is pioneering the use of CAR T cell therapy — a cutting-edge form of immunotherapy that has already shown success in treating certain blood cancers — to target and attack DMG tumors directly within the brain and spine. Cookies for Kids’ Cancer initially funded Dr. Vitanza in 2020 and 2021.  As a “young” investigator, our peer-reviewed funding opened other doors for Dr. Vitanza, which allowed him to expand his research and grow his lab from a team of 3 to more than 10, all focused on curing pediatric brain tumors.  

“Government support for pediatric cancer research has always been low, making up less than 4% of all cancer funding, but available money continues to shrink. When I completed my training, I was determined to dedicate my year to exclusively searching for cures for childhood brain and spinal cord tumors, but it would have been impossible to get started without funding through Cookies for Kids’ Cancer. Without that early support from Cookies and similar groups, I almost definitely would not have my lab or been able to open our clinical trials so quickly.” 

Inside the CAR T Cell Therapy Trial: What the Team Found 

In a first-in-human early-phase clinical trial, children with DMG received CAR T cells. This means a small amount of a patient’s own white blood cells are removed from the blood, taken to the lab where they are engineered to fight markers on the surface of cancer, grown into the millions, and then given back to patients directly into a fluid compartment in their brain called the lateral ventricle. The results were among the most encouraging ever seen for this disease: 

• Repeated intracranial CAR T cell doses were manageable, mostly as an outpatient even in patients as young as 2 years 

• Tumor reduction was observed in multiple patients 

• Some children are still alive multiple years from diagnosis 

• This is the first time CAR-T therapy has shown such an impact on DMG, offering real evidence that the immune system can be harnessed to fight this otherwise unstoppable tumor. 

Why This Matters: A Turning Point in Pediatric Oncology 

DMG has long been considered incurable — an impossible diagnosis for a family to hear. Dr. Vitanza’s development of the first ever intracranial CAR T cell trials for children has proven that targeted local immunotherapy is possible for these patients and can trigger a local immune response. This opens the door for more refined, personalized treatments for childhood brain cancers and beyond. 

What’s Next for CAR T Therapy and DMG Research 

Seattle Children’s continues to refine their approach, exploring: 

• New CAR T engineering to increase potency and reduce side effects 

• Combination therapies with radiation, drugs, and other immunotherapy 

• Expanded trials to hospitals across the U.S. in order to reach more children and young adults 

Seattle Children’s is also collaborating with other pediatric research centers to accelerate discoveries and bring promising treatments to families sooner. 

Frequently Asked Questions (FAQ) 

What is CAR T cell therapy? 

• CAR T (chimeric antigen receptor T -cell) therapy involves modifying a patient’s T cells to recognize and attack a specific target, in this case a protein on cancer cells. 

What is DMG? 

• DMG (diffuse midline glioma). is a rare and deadly pediatric brain tumor that primarily affects children under 10. 

Can families join the trial? 

• Seattle Children’s has opened 4 CAR T cell trials for children with brain and spinal cord tumors, with the first two studies closed but potentially the ability to enroll on BrainChild-03 or BrainChild-04 (https://www.seattlechildrens.org/clinics/cancer/research-and-clinical-trials/t-cell-immunotherapy-for-brain-and-central-nervous-system-tumors/). 

When will this treatment be widely available? 

• Multiple leading institutions including Baylor, City of Hope, St. Jude, and Stanford currently have trials open in the U.S. with more phase 1 and phase 2 clinical trials planned.