By: Trish Adkins
What if there's a better way to detect childhood cancer? A new way to outsmart a tumor? A treatment approach scientists haven't explored before?
Alex’s Lemonade Stand Foundation (ALSF) has funded 12 Innovation Grants to help researchers pursue bold ideas that could change the future of childhood cancer. These two-year-long projects will explore big questions in childhood cancer, challenge conventional thinking, and pursue discoveries that could lead to the next big breakthroughs for children with cancer. ALSF has funded nearly 290 Innovation grants since 2006.
For Robbie Majzner, MD, the answers could begin with a fundamentally different approach to treating neuroblastoma, a cancer that develops in young children.
“ALSF is allowing us to take a high-risk idea to develop a new platform technology and actually test that in the lab directly for pediatric cancer,” said Dr. Majzner, who was just awarded the World Gold Council Innovation Grant.
Neuroblastoma is the most common extracranial solid tumor in children. Although many children can be cured, those with high-risk disease may relapse despite intensive treatment. Dr. Majzner's team is exploring a new strategy: rather than simply blocking the proteins that drive tumor development, researchers will use specially designed antibodies to change how the protein is organized on the surface of cancer cells, potentially shutting down the signals that drive tumor growth. If successful, this approach could offer a new way to target neuroblastoma and potentially other cancers.
Dr. Majzner previously received an ALSF ‘A’ Grant to study combining immunotherapy and antibody therapy to treat neuroblastoma and osteosarcoma, a type of bone cancer. That work is helping propel his new project forward, illustrating how research funding can build momentum from one scientific question to the next.
Nathan Singh, PhD, another new Innovation Grant recipient, is also building on an earlier ALSF award. As a student, Dr. Singh received an ALSF POST grant that helped launch his research career studying CAR-T immunotherapy.
“Support from the POST allowed me to spend two years in Steve Grupp’s lab at CHOP and taught me the ins and outs of developing new cell therapies. I learned how to design CARs and engineer T cells, and also got to see this new therapy in action,” said Dr. Singh, who worked in the Grupp Lab when the first pediatric patient, Emily Whitehead, was treated in a groundbreaking CAR T-cell therapy trial.
CAR T-cell therapy has transformed treatment for some children with leukemia that has resisted other treatments. One CAR T-cell therapy, Kymriah, was approved by the U.S. Food and Drug Administration in 2017 for certain pediatric and young adult patients with B-cell acute lymphoblastic leukemia. Yet a major challenge remains: for many patients, the cancer eventually returns because the engineered immune cells stop working effectively over time.
Dr. Singh's Innovation Grant supports a bold new approach: redesigning the molecular components that tell CAR T cells how to respond. These specially engineered cells have the potential to be more durable and help stop leukemia relapse in children.
For Innovation grantee James Rutka, innovation means tackling a devastating complication of childhood brain tumors called leptomeningeal metastasis. This occurs when cancer cells leave the original tumor and spread through the cerebrospinal fluid in the brain and spinal cord. Once this spread occurs, treatment options narrow and survival is measured in months. This complication is common in aggressive brain tumors like medulloblastoma, where it can occur in up to 40% of children.
With support from an ALSF Innovation Grant, Dr. Rutka is aiming to understand how these tumor cells adapt to survive in the cerebral spinal fluid and then examine these adaptions across multiple brain tumor types. The hope is to find common weakness and target those with new or existing drugs.
“This funding is particularly valuable because it enables us to pursue an innovative approach to a devastating complication of childhood cancer,” said Dr. Rutka. “Our hope is that these discoveries will eventually lead to more effective, less toxic therapies and give more children the chance to live long, healthy lives after a cancer diagnosis.”
Across these 12 projects, researchers are exploring new ways to understand childhood cancer, overcome treatment resistance, detect disease earlier, and uncover vulnerabilities that could lead to new therapies. Each project begins with a question. With ALSF's support, researchers can investigate where those questions lead and what new possibilities they might open for children with cancer. Below, meet the newest group of Innovation Grantees and explore their breakthrough projects:
