Fall 2025
By Hannah White Hannah
Cookies for Kids’ Cancer has awarded two grants totaling $600,000 to Dana-Farber Cancer Institute to support groundbreaking pediatric cancer research. The funding supports two projects: one led by A. Thomas Look, MD, focused on high-risk neuroblastoma, and another led by Scott Armstrong, MD, PhD, targeting a rare and aggressive form of childhood leukemia. This is part of their ongoing commitment, having previously funded more than $2.6 million in pediatric cancer research grants to Dana-Farber through a rigorous peer-reviewed process.
Look received a $300,000 grant in 2025 to advance treatments for children with high-risk neuroblastoma. These patients often endure a year of intensive treatment followed by post-consolidation therapy, which includes isotretinoin and an anti-GD2 antibody. While this regimen improves survival, many children relapse due to treatment limitations. Look’s research arose from a screen that identified a drug, PF-9363, which works synergistically with isotretinoin to extend suppression of cancer cell growth and increase the expression of GD2, making neuroblastoma cells more vulnerable to immunotherapy that specifically targets this cell surface glycolipid. This combination therapy significantly improved the killing of human neuroblastoma cells growing in immunosuppressed mice and was well tolerated. His team is now testing a three-drug regimen—including PF-9363, isotretinoin, and the anti-GD2 antibody—in this model system to validate a treatment schedule suitable for testing to improve outcomes for children with neuroblastoma. If successful, this could lead to clinical trials and offer new hope for children with treatment-resistant neuroblastoma.
“There’s a critical need to develop smarter, more targeted treatments for these children,” Armstrong said. “Our goal is to bring therapies to the clinic that directly address the biology of their disease and improve their chances of survival.”
Scott Armstrong, MD, PhD, Dana-Farber Cancer Institute
Cookies for Kids’ Cancer awarded Armstrong a $300,000 grant in 2024 to study childhood leukemia with NUP98 gene rearrangements, which are associated with poor outcomes and limited treatment options. His team is targeting the Menin-KMT2A complex, a key driver of leukemia gene expression, and exploring a second target, KAT6A, for combination therapy.
“There’s a critical need to develop smarter, more targeted treatments for these children,” Armstrong said. “Our goal is to bring therapies to the clinic that directly address the biology of their disease and improve their chances of survival.”
Thanks to funding from Cookies for Kids’ Cancer and the revolutionary work of two Dana-Farber researchers, these studies represent a significant step toward better outcomes for children with some of the most challenging pediatric cancers.