ABC2 made this grant possible through a funding partnership with the Lyla Nsouli Foundation, 3000 Miles To A Cure and the BraveCort Foundation.
What Is DIPG?
Diffuse intrinsic pontine glioma (DIPG) is an aggressive brain tumor found in a part of the brain stem called the pons. The pons controls essential bodily functions such as heartbeat, breathing, swallowing, eye movement, eyesight, and balance. DIPG affects children almost exclusively. Approximately 200-400 children in the United States are diagnosed with DIPG each year. DIPG tumors are insensitive to traditional chemotherapy. And although radiation can alleviate symptoms, it’s not curative, and most children with DIPG die within 2 years of diagnosis.
A New Approach
This phase 1 trial evaluates the safety, immunogenicity, and preliminary clinical effects of a personalized dendritic cell vaccine made from tumor RNA obtained from a biopsy of the patient's own DIPG tumor.
This personalized vaccine is combined with adoptive T cell therapy of ex vivo expanded tumor-specific lymphocytes and autologous stem cell transfer. Researchers at UF have demonstrated that this combined cellular approach is highly effective against multiple brain tumors, including resistant DIPG models, and they are hopeful that this approach will be beneficial to children with DIPG.
BRAVO incorporates a newly developed process for stimulating and expanding tumor-specific lymphocytes that allows researchers to achieve 300-1000X more activated T cells than previously and also incorporates a new process for differentiating these lymphocytes into long-lived memory T cells. Also, importantly, this study will examine whether administering the vaccine during recovery from standard chemotherapy (temozolomide) is effective in enhancing the immune response to treatment or not.
**Families seeking clinical trials for children with newly-diagnosed DIPG can learn more about the study at ClinicalTrials.Gov. Importantly, children must enroll before they start radiation therapy in order to collect a pheresis to make the cellular therapy products. This means the study must identify and enroll patients in the 3-4 week window between diagnosis and starting radiation therapy. Families also need to be able to travel to UF in Gainesville, FL for biopsy and administration of the vaccines, T cells, and stem cell therapies on a monthly basis.