Area Children’s Cancer Organizations Help Fund Breakthrough Study

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Three nonprofit organizations dedicated to fighting children’s cancer have partnered to help fund the first phase of an immunotherapy study in hopes of finding groundbreaking treatment for children with brain tumors.

The Chappaqua-based A Kids’ Brain Tumor Cure Foundation, the Ty Louis Foundation of Carmel and Solving Kids Cancer in Manhattan have collaborated to donate more than $180,000 toward this clinical trial under the direction of Dr. Ira Dunkel of Memorial Sloan Kettering Cancer Center. The trial is expected to begin with 36 children by early next year.

Amy Weinstein, executive director of A Kids’ Brain Tumor Cure Foundation, said the study will test the safety and efficacy of using checkpoint inhibitors on children. Checkpoint inhibitors are drugs containing certain antibodies that attack cancer cells. The approach has shown success in fighting other types of cancers, particularly melanoma and Hodgkin lymphoma.

This is the first time this is being used in children with brain tumors. Treatments such as chemotherapy and radiation continue to be used on the patients, but with little progress.

“The goal is to give these researchers the funding they need to try their ideas out,” Weinstein said. “The challenge facing the research community is funding and we want to help them and we want to help them with their ideas.”

The trial will be conducted in six of the world’s top cancer centers: Sloan Kettering, Dana Farber/Boston Children’s Hospital; Johns Hopkins/Sidney Kimmel Comprehensive Cancer Center, Great Ormond Street Hospital in the United Kingdom, Institut Curie Research Centre in France and Children’s Hospital Colorado, Weinstein said. It may be expanded to more hospitals later on.

Launching this innovative study with the help of some of the biggest cancer institutions is a breakthrough for not only the three organizations but for pediatric cancer research.

Cindy Campbell, founder of the Ty Louis Campbell Foundation and the mother of Ty who died in 2012 at four years old from an aggressive brain tumor, said seeing a new study provides encouraging news for parents and families of children who have grown accustomed to bracing for the worst.

The hope is that the study can lead to additional funding and research and eventually more effective treatments, she said.

In a statement, Dunkel said researchers are hopeful that this could yield encouraging results against brain tumors, the leading cause of cancer deaths among children. To this point, they have been difficult to treat because of the unique makeup of the central nervous system.

“Checkpoint inhibitors are not showing the same kind of side-effect profiles or toxicities seen in the standard treatments kids with brain tumors currently receive like high-dose chemotherapy and radiation, which can be critical for increasing quality long-term survival,” Dunkel said.

In this study, children will be treated with an approach leveraging two agents, an anti-PD1 antibody (nivolumab) and another antibody targeting CTLA-4 (ipilimumab), according to a joint release form the three foundations. This combination has been highly effective in several high-risk adult cancer trials.

The hope is that children with malignant brain tumors will have will have similar results while reducing the toxicities that the children typically receive from chemo or radiation treatment.

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