Personalized Vaccines to Fight Childhood Brain Cancer

Title: Personalized anti-tumor vaccination for the treatment of brain cancer
Investigator: Duane A. Mitchell
Grantee: University of Florida

Harnessing the power of the immune system – the body's natural defense against invaders – is an ingenious way to fight brain cancer because the immune system already has specialists devoted to the fight.  Prominent among these specialists are "T cells", which act like soldiers that seek out and destroy invaders like cancer cells wherever those invaders are hiding.

However, using this strategy requires solving a difficult problem: how to teach T cells to attack only a patient’s tumor cells while leaving healthy cells of the brain and body unharmed.  This problem is especially tough in brain cancer because brain tumors contain a wide variety of cells that can look confusing to the immune system.

Dr. Duane Mitchell, Director of the University of Florida Brain Tumor Immunotherapy Program, is working to solve this problem by using vaccines.  His work exploits a new way to make personalized, brain tumor-targeting vaccines by extracting genetic material from a patient's brain tumor and using this material to train T cells to recognize the tumor.  Personalized vaccines made this way generate large numbers of T cells explicitly trained to attack a specific brain tumor – exactly what we want in a brain cancer-targeting vaccine.  Further, because making this type of vaccine requires only a few hundred tumor cells, any patient undergoing brain cancer surgery or biopsy can potentially get one.

ABC2 is now helping Dr. Mitchell put these new vaccines to practical use in a Phase 1/2 clinical trial for children with medulloblastoma.  This trial, jointly funded by ABC2, the Florida Brain Tumor Association, the Rendina Family Foundation, and Paul Hale, is the first pediatric brain cancer immunotherapy trial at the University of Florida.  It uses the new tumor-targeting vaccines to train patients’ immune systems to attack their own brain tumors.  The trial’s primary goals are to test the safety and effectiveness of the new vaccines and to identify factors that predict treatment response or failure.

If this clinical trial is successful, the new vaccine technology could move rapidly from the academic setting into a larger clinical setting taking its place as a much-needed pediatric medulloblastoma treatment.  In addition to driving the use of this technology to help children with brain cancer, the funding provided by ABC2 has magnetized additional outside financial support for the project and helped create a national network of treatment centers to speed and broaden future immunotherapy clinical trials.