Pediatric Brain Tumor Foundation, partners announce investment in novel immunotherapy research at the Chad Carr Pediatric Brain Tumor Center at Michigan Medicine

July 31, 2019  //  FOUND IN: Michigan Medicine News

The Pediatric Brain Tumor Foundation (PBTF) has partnered with the Samson Research Fund and the Charles Woodson Clinical Research Fund, both at C.S. Mott Children’s Hospital, to invest more than $500,000 in a novel immunotherapy approach for treating pediatric high-grade gliomas (pHGG), a common form of highly aggressive pediatric brain cancer. This investment represents almost two-thirds of the $800,000 necessary to fully fund this three-year collaborative project between Michigan Medicine’s departments of pediatrics and neurosurgery. 

pHGG tumors account for the leading cause of death by disease among children in the U.S. While improvements have been made in treatment outcomes for leukemia and other pediatric cancers, outcomes for children with pHGG have shown only modest improvements in recent decades. 

Maria G. Castro, Ph.D., and Pedro R. Lowenstein, M.D., Ph.D.

“There is an unmet need for developing better treatments for pHGG patients, and immunotherapy could offer a novel, safe and highly efficacious approach,” said Maria G. Castro, Ph.D., R. C. Schneider Collegiate Professor of Neurosurgery, professor of cell & developmental biology at U-M and Pedro R. Lowenstein, M.D., Ph.D., Richard C. Schneider Collegiate Professor of Neurosurgery, professor of cell & developmental biology at U-M. “The treatment that our team has pioneered uses a gene therapy-mediated delivery of therapeutic genes into the tumor — eliciting the reprogramming of the patient’s own immune system so that it will recognize and kill brain tumor cells.”

pHGG tumors are particularly difficult to treat due to the inability to perform total surgical resections of the tumor and the challenges the blood brain barrier cause in reaching brain tumor tissue with chemotherapy. Although radiation is part of the standard of care for adult brain tumor patients, it is limited in use in children with high grade gliomas due to its significant toxicity to the developing brain. The treatment strategy being pioneered at Michigan Medicine has been approved by the FDA for adult patients with glioblastoma multiforme, the most aggressive form of brain cancer, and a Phase I clinical trial has recently completed patient enrollment.

“Because of the Pediatric Brain Tumor Foundation’s generous investment, we now will be able to perform the necessary experimental work in pre-clinical models in order to get FDA approval to implement this desperately needed therapy in children. We are grateful for their support and excited about the potential this new partnership holds for kids afflicted with these devastating tumors,” added Castro and Lowenstein. Castro and Lowenstein are joined on the project by co-investigator Karin Muraszko, M.D., chair and Julian T. Hoff, M.D., professor of neurological surgery.

The funds invested in this research project were raised through the PBTF’s annual Think Fit for Kids Family Fitness Festival, which has raised nearly $2 million over the last nine years with 100 percent of its proceeds supporting cutting-edge pediatric brain tumor research. 

“Kids diagnosed with brain tumors do not have the luxury of time,” said Kimberly Gilman, chairperson of the PBTF’s Think Fit for Kids and Michigan alumna. “As such, we are thrilled to have the opportunity to streamline the research process by leveraging a Michigan Medicine treatment strategy that has already been approved for adults in order to benefit the often-neglected community of pediatric patients. Collaboration between committed foundations and dedicated medical professionals is the only way to change the currently unacceptable outcome for the 13 children diagnosed each day in the U.S. with a brain tumor. “In partnership with research institutions like Michigan Medicine, the Pediatric Brain Tumor Foundation is answering the increasingly urgent call to put an end to this terrible disease,” said Bill Tiller, PBTF president and CEO. “As the leader in the childhood brain tumor community, we are committed to making cures possible for all children with brain tumors. We are grateful to our partners in mission for collaboratively funding research that offers hope to families who have run out of treatment options.”

Email kgilman@curethekids.org to get involved with Think Fit for Kids, and visit www.curethekids.org/research to learn more about this grant and other PBTF research funding initiatives.

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