Funded Projects - UF

Scientists Tackle Lethal Childhood Brain Cancer

BRAVO Phase I trial for children with newly-diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

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Dear Andrea & Brian - I want to thank you for your terrific friendship and support in our shared mission of finding effective treatments and cures for kids dealing with brain cancer.  I am especially excited about our current joint project -- working with Dr. Duane Mitchell and his team at the University of Florida Brain Tumor Immunotherapy Program to bring to life their new BRAVO Phase I trial for children with newly-diagnosed diffuse intrinsic pontine glioma (DIPG).

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As you know, until now the outcome for children with DIPG has been dismal.  The standard therapy of external beam radiation given alone or in combination with chemotherapy has not worked and there is a desperate need for new therapeutic options.  We believe that immunotherapy, a strategy that uses the patient’s own immune system to attack their tumors, offers great promise, especially for treating difficult cancers such as DIPG.  

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Their new BRAVO Phase 1 trial employs a novel adoptive next-generation cellular therapy approach that has never been used before to treat DIPG patients.  Normally, a supply of tumor tissue is required to enable production of a tumor vaccine.  Because DIPG tumors are located within the brain stem, a highly critical and inoperable portion of the child’s brain, surgical resection of the tumor and harvesting of tissue has not been possible. 

The Mitchell team has developed an approach where they can amplify the RNA content from a few hundred isolated tumor cells to generate RNA libraries suitable for production of a workable vaccine.  This removes the need for surgery and allows the use of a small core biopsy to obtain a tumor sample suitable for vaccine production. 

 

Applying this technology now to newly-diagnosed inoperable DIPG tumors marks a major new step forward in treating this disease. The Mitchell team has recently completed the successful biopsy and amplification of tumor RNA from a single pediatric patient with DIPG.  This patient, treated under a special expanded access/compassionate use single patient protocol, went on to have adoptive cellular immunotherapy treatment without complications.  The work we are now jointly funding will enable the Mitchell team to expand on this single-patient experiment and move into a full clinical trial that will make the approach available for testing tested in a broader population of patients.  Our hope is that the lessons learned in this trial will one day lead to a whole new way to effectively treat this disease.