DIPG in Children: Promising B7-H3 CAR-T Therapy Results from Seattle and the First Known Cure with Everolimus

Diffuse intrinsic pontine glioma (DIPG) is one of the most aggressive and heartbreaking pediatric brain tumors. It forms in the brainstem (pons), making surgery impossible, and responds poorly to traditional chemotherapy. Each year in the United States, approximately 200–300 children are diagnosed with DIPG or the related diffuse midline glioma (DMG). The median survival is only 8–11 months, and fewer than 1% of children survive five years.

For decades, radiation has been the only standard treatment, offering temporary symptom relief but no cure. Families and researchers have long sought better options. Two recent developments — one involving advanced immunotherapy and another involving a repurposed drug — are generating cautious hope.

Promising Results from Seattle Children’s B7-H3 CAR-T Trial (BrainChild-03)

In January 2025, researchers at Seattle Children’s Research Institute published first-in-human results from the BrainChild-03 Phase 1 trial in the journal Nature Medicine. The trial tested a B7-H3-targeted CAR-T cell therapy delivered directly into the brain’s ventricles (intracerebroventricular or ICV delivery).

Key findings from the trial (21 children and young adults with DIPG):

• The treatment was generally well tolerated.
• Median overall survival from diagnosis was approximately 19.8 months — nearly double the historical average.
• Median survival after the first CAR-T infusion was 10.7 months.
• Three patients who started treatment before disease progression remained alive at 44, 45, and 52 months post-diagnosis.
• One notable participant, diagnosed at age two, has far outlived initial expectations and continues to thrive years later.

The therapy uses a patient’s own T cells, genetically modified to recognize B7-H3 (a protein highly expressed on DIPG tumor cells). By delivering the cells directly into the cerebrospinal fluid, researchers bypassed many systemic side effects while getting the immune cells closer to the tumor.

A follow-up preclinical study published in January 2026 further enhanced the CAR-T cells by adding a CXCR3 receptor to improve their ability to migrate into the tumor. BrainChild Bio (the company advancing this therapy) has now received FDA Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations and is preparing for a pivotal Phase 2 trial.

Important context: This remains an early-phase experimental treatment. While the survival signals are encouraging, larger controlled trials are needed to confirm benefit and long-term outcomes.

The Remarkable Case of Lucas Jemeljanova – The First Known DIPG Cure with Everolimus

In a completely different approach, a Belgian boy named Lucas Jemeljanova has become the first child in the world known to be cured of DIPG.

Diagnosed at age six, Lucas enrolled in the BIOMEDE clinical trial at Gustave Roussy Cancer Centre in France. He was randomly assigned to receive everolimus, an mTOR inhibitor already approved for other cancers (such as kidney, breast, and pancreatic) but never before successfully used against DIPG.

• Over a series of MRI scans, his tumor gradually shrank and eventually disappeared completely.
• Lucas stopped taking the medication more than a year before doctors confirmed it was safe to do so.
• He is now 13 years old with no evidence of disease — a true cure in a cancer long considered incurable.

Seven other children in the same trial were classified as “long responders” (no progression for more than three years), but Lucas is the only one whose tumor vanished entirely. Researchers believe a rare genetic mutation in his tumor made it exceptionally sensitive to everolimus. They are now studying his tumor samples and creating lab-grown organoids to understand how this could help other patients.

What These Developments Mean for Families

These stories highlight two very different paths forward: advanced cell therapy that trains the immune system and deeper understanding of why certain tumors respond to existing drugs.

If your child or a loved one has been diagnosed with DIPG or a diffuse midline glioma, independent research into clinical trials, expanded access programs, and the latest evidence can help identify realistic options quickly. Our team offers a free case assessment to review medical records and outline tailored pathways — no obligation.