Collaborative launch of immunotherapy clinical trial for children with brain tumors

August 25, 2016

September, 2016 - NEW YORK, NY: A Kids' Brain Tumor Cure Foundation, Solving Kids' Cancer and the Ty Louis Campbell (TLC) Foundation announce their joint financial support of a Phase 1 clinical trial to test the safety and efficacy of combination checkpoint inhibitors in the treatment of children with brain tumors.

The collaboration of these three like-minded nonprofits will result in a total contribution of $185,000 to bring this clinical trial to fruition for children battling brain tumors, today. The trial will be led by Dr. Ira Dunkel, Memorial Sloan Kettering Cancer Center (MSKCC) and enrollment is expected to open some time in the first quarter of 2017. The International trial will be available to children in six esteemed cancer centers to start, including MSKCC, Dana Farber/Boston Children's Hospital, Johns Hopkins/Sidney Kimmel Comprehensive Cancer Center, Great Ormond Street Hospital (UK), Institut Curie Research Centre (France) and Children's Hospital Colorado. It is also likely that this innovative study will expand to additional centers in upcoming months.

"Pediatric brain tumors can be very difficult to treat because of the unique anatomy of the central nervous system, making brain cancer the number one cause of cancer-related deaths among children," said Principal Investigator Dr. Ira Dunkel, Pediatric Oncologist, Memorial Sloan Kettering Cancer Center. "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."

Checkpoint inhibitors are humanized antibodies that take the "brakes" off immune cancer cells to effectively and efficiently kill cancer cells. This combination treatment targets the immune system rather than the cancer itself.

In this case, children will be treated with a combination approach leveraging two agents, an anti-PD1 antibody (nivolumab), and another antibody targeting CTLA-4 (ipilimumab), a combination that has been highly effective in several high-risk adult cancer trials already. The hope is that children with malignant brain tumors will have similar results while reducing the toxicities that these kids usually receive from treatment.

"Checkpoint combination therapy is the most exciting breakthrough in cancer research this decade. Adults with previously untreatable cancers like metastatic melanoma and lung cancer have been cured in clinical trials, and we are passionate about partnering with A Kids' Brain Tumor Cure Foundation and the TLC Foundation to lead the charge in bringing this innovation to children battling brain tumors," said Scott Kennedy, Executive Director, Solving Kids' Cancer.

By working together across borders, nonprofits and medical institutions can move the needle faster, which has otherwise been quite stagnant for pediatric brain tumor patients. Progress has often been slow in recent decades, having little to no improvement in survival rates for many young brain tumor patients with relapse or metastatic disease.

"One of the most frustrating roadblocks we have faced in the childhood cancer community is the lack of collaboration and information sharing - not only across institutions, but also across borders and among nonprofits. This clinical trial is so exciting because the parameters for funding ensured that the study would be offered across several medical institutions, and it is co-funded by three nonprofits with a shared mission. It's a win-win that breaks down these barriers and puts children first," said Amy Weinstein, Executive Director, A Kids' Brain Tumor Cure Foundation.

This innovative trial marks the first time this encouraging immunotherapy combination of agents is being used in children with brain tumors. All three collaborating partners expect this study may provide a completely new avenue for treating deadly brain tumors in children.

Cancerous brain tumors are the #1 cancer related death in children. Current treatments are toxic, cause permanent cognitive and physical impairments, and severely diminish quality of life. Pediatric brain cancer research is severely under-funded, and as a result, there have been no new treatment breakthroughs in over 20 years. The mission of A Kids' Brain Tumor Cure (AKBTC/PLGA Foundation) is to raise funds and awareness for scientific research leading to new, promising treatments and a cure for children's brain cancer. For more information, visit; contact; or connect on social media at, or #AKBTC or @PLGA


Solving Kids' Cancer is not just our name, it's our mission. We focus on aggressive childhood cancers with low survival rates--because Every Kid Deserves to Grow Up. Solving Kids' Cancer helps accelerate new, next-generation treatments, including immunotherapy, cancer vaccines, and new drugs by applying an understanding of the entire childhood cancer research landscape to wisely invest in innovative projects. Visit, @SolveKidsCancer, or for more information.


The TLC Foundation is a nonprofit organization that funds innovative research and clinical trials specifically geared toward the treatment of pediatric cancers (including brain and spinal cord tumors). TLC seeks less toxic, more effective treatments designed for children fighting cancer. Their ultimate mission is to help fund the intelligence and technology that will uncover new ways to cure children with cancer. Visit, @SuperTyCampbell or for more information.

Solving Kids' Cancer

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