Announcing This Year’s 2026 Grant Recipients!

Brian Crompton, MD – Dana-Farber Cancer Institute

Advancing Liquid Biopsies to the Clinic for Patients with Ewing Sarcoma

Treatment for aggressive pediatric sarcomas has remained largely unchanged for the past 15 years, with outcomes plateauing around 70%. A major challenge is the lack of reliable biological markers to identify which patients are likely to be cured and which are at risk of relapse. Dr. Crompton aims to validate a new clinical assay that detects circulating tumor DNA (ctDNA) from a simple blood draw as a surrogate for tumor burden in Ewing Sarcoma. Early identification of poor responders could enable timely, patient-specific treatment modifications, similar to approaches used successfully in pediatric leukemia. The study will also examine tumor heterogeneity and validate ctDNA as a clinical return-of-results tool.

Elliot Stieglitz, MD, PhD – University of California, San Francisco

Direct Targeting of the Ras Pathway in Juvenile Myelomonocytic Leukemia Using RMC-7977

Juvenile myelomonocytic leukemia (JMML) is a rare and aggressive blood cancer in young children, currently treated primarily with bone marrow transplantation, which cures only about half of patients. JMML is driven by mutations in Ras pathway genes. Dr. Stieglitz will test a promising new Ras inhibitor, RMC-7977, in mouse models of JMML, alone and in combination with other targeted therapies. Successful results could rapidly advance this approach into clinical trials and reduce reliance on toxic transplants.

Lev Gorfinkel, MD, PhD – Dana-Farber Cancer Institute

Next-Generation Hematopoietic Stem Cell Transplant Conditioning Through Antibody-Drug Conjugates

Stem cell transplantation can cure lethal blood cancers but requires toxic chemotherapy or radiation that causes severe long-term side effects, especially in children. Dr. Gorfinkel will test a targeted antibody-drug conjugate (CD45-ADC) designed to selectively eliminate blood-forming cells while sparing healthy tissues. Using a non-human primate model, the study will assess engraftment success, immune recovery, and reduced toxicity, with the potential for rapid clinical translation.

Rubia Isler Mancuso, PhD – Yale University

Genetic Dependencies Driving Leukemogenesis in an RBM15-MKL1 Mouse Model

Dr. Mancuso is studying acute megakaryoblastic leukemia (AMKL), a pediatric leukemia driven by the RBM15-MKL1 fusion protein. Using a novel inducible mouse model, Dr. Mancuso will examine how leukemia begins—potentially before birth—and identify additional genetic changes that cooperate with RBM15-MKL1 to drive disease. These insights may uncover new therapeutic targets.

Rebecca Ronsley, MD – Seattle Children’s Hospital

Biomarkers of Response to Delta-24-RGD Therapy in Pediatric Diffuse Midline Glioma

Diffuse midline glioma (DMG) is a devastating pediatric brain tumor with extremely poor prognosis. Dr. Ronsley will evaluate biomarkers of response to Delta-24-RGD, an oncolytic virus delivered directly into tumors. By analyzing cerebrospinal fluid, blood, and tumor tissue, the project aims to develop tools for monitoring response and guiding personalized therapy in collaboration with leading pediatric oncology consortia.

Anupriya Agarwal, MD – Oregon Health & Science University

Intervention and Prevention Strategies in RUNX1-Mutated Leukemia Evolution

RUNX1 mutations are associated with poor outcomes in acute myeloid leukemia and familial platelet disorder, with a high risk of progression to leukemia. Despite affecting thousands of individuals, no preventive strategies exist. Dr. Agarwal seeks to identify interventions to halt leukemic evolution in RUNX1-mutated disease, addressing a major unmet clinical need.

Carl Allen, MD, PhD – Baylor College of Medicine

Curing Pediatric Langerhans Cell Histiocytosis Through Combined Chemotherapy and MAPK Inhibition

Langerhans cell histiocytosis (LCH) affects multiple organs and is driven by mutations in the MAPK pathway. Current chemotherapy fails to cure over half of patients and often causes long-term harm. Leveraging new biological insights and the North American Consortium for Histiocytosis (NACHO), Dr. Allen will test a novel combination of MAPK inhibition and chemotherapy with the goal of curing patients.

Zachary Morris, MD, PhD – University of Wisconsin–Madison

Theranostic Radiopharmaceuticals Alone or Combined with CAR T Cells for Pediatric Medulloblastoma

Medulloblastoma is a leading cause of cancer-related death in children, and current radiation treatments cause severe long-term toxicities. Dr. Morris will test a targeted radiopharmaceutical, NM600, delivered intrathecally to precisely treat tumors while sparing healthy brain tissue. Combining NM600 with CAR T cells may further enhance efficacy and enable curative treatments with reduced toxicity.

Ibrahim Chamseddine, MD – Massachusetts General Hospital

Predictive Risk Score for Hearing Loss in Pediatric Brain Cancer Radiotherapy

Permanent hearing loss affects up to 60% of children treated with brain radiotherapy. Dr. Chamseddine will develop a predictive risk model using data from proton therapy clinical trials to identify children at highest risk before treatment begins. The tool aims to guide personalized treatment planning and early intervention to improve survivors’ quality of life.