Seeking New Therapies in Childhood Neuroblastoma

Childhood cancer remains the leading cause of death from disease in children in high-income countries. For neuroblastoma, a common pediatric solid tumor usually found in children under the age of 5, survival rates are high when diagnosed while the tumor is localized or regional (low- and intermediate-risk). However, when the tumor metastasizes (high-risk neuroblastoma), survival rates drop to approximately 60%.

A recipient of a V Foundation All-Star Grant in 2023, Kimberly Stegmaier, M.D., aims to use cutting-edge technology to find new treatment options for high-risk patients, including neuroblastoma patients. She and her team previously discovered a new potential therapeutic target that has been used in other types of cancer, but not in neuroblastoma patients. In this grant, Dr. Stegmaier is testing drugs in high-risk neuroblastoma models in the lab, with the long-term hope of leading to a clinical trial for patients in the future.

Stegmaier, currently the Chair of the Department of Pediatric Oncology at Dana-Farber Cancer Institute, Associate Chief in the Division of Hematology/Oncology at Boston Children’s Hospital and the David G. Nathan Professor of Pediatrics at Harvard Medical School, is a veteran in the field of pediatric cancer research. She has seen the importance of consistent support, and the challenges of securing funding.

“It’s hard to fund childhood cancer research,” she said. “The diseases are rare, and typically, large companies are not going to do drug discovery for rare cancers. So, foundations like the V Foundation are essential for research in childhood cancer … Particularly, the focus on high-risk projects is super special, and we are so grateful for that support.”

Stegmaier first received funding from the V Foundation earlier in her career when she received a Translational Grant in 2012. She credits that grant for establishing her lab.

“I had a V grant a number of years ago that similarly focused on neuroblastoma and led to a clinical trial. It was at an early point in my career, and it informed really foundational work for my lab, led to a clinical trial, and really gave me a boost in my career. It was my early days of working on neuroblastoma. I became more introduced into the larger neuroblastoma community and part of larger collaborative grants and made new collaborations.”

“It really started with that V Foundation grant. The impact that the V is having is tremendous. And again, we’re so grateful that there’s a focus on pediatrics within the V Foundation.”