Breaking Barriers in Pediatric Brain Tumor Research

CHRI: Working Together for a Better Tomorrow

In Nebraska, brain tumors are the most common form of childhood cancer and the leading cause of cancer-related death in kids. At Children’s Nebraska, our researchers are tackling this urgent need head-on.

Through the Child Health Research Institute (CHRI), a partnership between Children’s Nebraska and the University of Nebraska Medical Center (UNMC), more than 600 experts are working together to turn scientific discoveries into real treatments for children. From launching clinical trials to developing targeted therapies, CHRI ensures research translates directly into better care.

Nebraska’s Role in Advancing Pediatric Cancer Research

Pediatric cancer research is historically underfunded — just 4% of National Cancer Institute funding goes toward childhood cancers with even less dedicated to brain tumors.

Thanks to the leadership of Don Coulter, MD, and CHRI’s Pediatric Cancer Research Group, Nebraska is one of the few states in the nation to dedicate a line item in its state budget to support pediatric cancer research. This unique state funding ensures lifesaving discoveries continue to move forward for kids and families here at home.

Today, Children’s Nebraska and UNMC are conducting 10 clinical trials to evaluate new medications for pediatric brain tumors. Eight are funded by government sources while two are supported by pharmaceutical partners.

Childhood Brain Tumors in Nebraska

Nebraska ranks 7th in the nation for pediatric brain tumor incidence and has the highest rate in the Midwest. In Nebraska, on average:

• One child is diagnosed with cancer every four days

• One child dies from pediatric cancer nearly once a month

These numbers underscore why research here matters so much — and why Children’s Nebraska is leading the charge.

Physician Champions in Brain Tumor Research

CHRI researchers include multiple physician leaders working together to tackle pediatric brain tumors. Sidharth Mahapatra, MD, PhD, is a critical care physician at Children’s Nebraska and an associate professor at UNMC. He is a co-champion of the Pediatric Cancer Research Group and serves as chair of the Nebraska Brain Tumor Research Collaborative. Dr. Mahapatra’s research focuses on Group 3 medulloblastoma (G3MB), one of the most aggressive and hardest-to-treat childhood brain tumors. His lab, supported by eight different funding sources, collaborates with experts in Neurosurgery, Oncology, Pharmacy and other specialties to develop new and more effective treatments.

What is Medulloblastoma in Children?

Medulloblastomas are the most common malignant brain tumor in children, accounting for about 20% of all pediatric brain tumors. The disease usually affects children between ages 3 and 8. These tumors grow in the cerebellum, which controls movement and balance. Because of their aggressiveness, these tumors require medications that act quickly and effectively. CHRI researchers are focused on developing these treatments.

Developing New Treatments for Medulloblastoma

Dr. Mahapatra and CHRI researchers are investigating innovative strategies to keep medulloblastomas from developing and spreading, including:

• Repurposing FDA-approved drugs such as antidepressants and anti-malarials to work alongside chemotherapy, increasing the effectiveness of treatment and reducing side effects for children with medulloblastoma

• Identifying and targeting epigenetic drivers of MYC (a gene that promotes cancer cell growth)

• Identifying targets and developing contrast agents for image-guided surgical research to improve medulloblastoma therapies

• Investigating how epigenetic changes and abnormal cell signaling drive the development and progression of medulloblastoma tumors in children

• Identifying epigenetic changes during medulloblastoma therapy that contribute to therapy resistance in, and testing new ways to overcome treatment resistance and prevent tumor recurrence

• Determining how the stem cell gene regulator SOX2 (a protein that controls how certain genes work) changes the activity of medulloblastoma cells, and identifying drugs that can block SOX2 to stop tumor growth

About the Child Health Research Institute

CHRI unites more than 600 pediatric researchers across Nebraska and the Midwest. Its Pediatric Cancer Research Group is dedicated to improving the lives of children diagnosed with cancer through innovative research, multidisciplinary collaboration and state-supported funding.

Together, Children’s Nebraska and CHRI are shaping the future of pediatric brain tumor research — right here in Nebraska.

Learn more about how Children’s Nebraska and CHRI are leading the way in groundbreaking research.