2023 Department of Pediatrics Academic Annual Report

the Sunshine Project consortium of the National Pediatric Cancer Foundation (NPCF). Finally, Dr. Metrock is director of the Pediatric Neuro-oncology Fellowship Program, one of 23 such programs in North America to train the next generation of leaders in pediatric neuro-oncology as well as the Global Pediatric Neuro-oncology Fellowship Program in collaboration with Children’s Cancer Hospital Egypt 57357 and St. Louis Children’s Hospital at Washington University in St. Louis Girish Dhall, M.D., is an international leader in the field of pediatric neuro-oncology. He chairs the Head Start 4 clinical trial—an investigator-initiated, international clinical trial of high-intensity chemotherapy to avoid cranial irradiation for infants with malignant central nervous system (CNS) tumors. Dr. Dhall recently presented the results of the low-risk arm of this protocol at the International Symposium on Pediatric Neuro-oncology held in Hamburg, Germany, where he showed that this treatment strategy provided a cure to 95% of infants and young children with a subtype of medulloblastoma without the use of cranial irradiation. He will be co-chairing an international trial to treat low-risk medulloblastoma patients with irradiation-avoiding strategies along with Stefan Rutkowski, M.D., a senior neuro-oncology researcher in Hamburg, Germany. Drs. Rutkowski and Dhall have been successful in obtaining $2.5 million from the German Ministry of Education for this trial, which is anticipated to open in early 2024 at multiple sites across North America, Australia and Europe. Dr. Dhall is the Deputy Director as well as institutional site PI for the Neurofibromatosis Clinical Trials Consortium, which includes 21 sites across the United States and Australia engaged in clinical research in neurofibromatosis. Dr. Dhall is also the institutional PI for PNOC, which is an international consortium focused on understanding how brain tumors develop in children and identifying personalized therapy strategies with sites in the US, Europe, Asia and Australia. He co-chairs the CNS Germ Cell Tumor Working Group within PNOC. He is also a member of the Steering and Protocol Review Committees of the Sunshine Project of NPCF as well as their CNS Tumor Working Group. Dr. Dhall serves on the Steering Committees of the CNS Disease Group, Adolescent and Young Adult (AYA) Discipline Group, and Young Investigators (YI) Committee within COG. Dr. Dhall serves as the director of Pediatric Developmental Therapeutics Program at Children’s of Alabama/UAB, one of only 21 members on the NCI PEP CTN and COG Developmental Therapeutics Committee. As site PI, he has been personally responsible for conducting/supervising 15 to 20 active Phase I/II studies, including multiple industry trials. He contributes to the scientific mission of the committee by reviewing scientific concepts and protocols to help advance innovative therapies. An example of discoveries taken from bench to bedside includes the research led by Rintaro Hashizume, M.D., Ph.D., in the field of neuro-oncology. Dr. Hashizume conducts bench-to-bedside translational research to treat diffuse intrinsic pontine glioma/diffuse midline glioma, which is one of the hardest to cure pediatric brain tumors. His research includes developing new targeted therapeutic agents to cure this difficult disease as well as developing innovative methods of drug delivery to the brain, which includes convection enhanced delivery (CED) and intranasal drug delivery. LEUKEMIA, LYMPHOMA, AND HISTIOCYTOSIS PROGRAM Matthew Kutny, M.D., conducts research focused on improving treatments for leukemia. He serves as the institutional PI for COG, the institutional PI for the North American Consortium for Histiocytosis, and site PI for several trials of targeted therapies and immunotherapies in leukemia. He is the vice chair of the Scientific Committee of the International Society for Pediatric Oncology, North American Branch. He is the oncology lead for UAB/Children’s of Alabama Pediatric Cellular Therapy Program, including Kymriah for acute lymphoblastic leukemia (B-ALL). He led the recently completed international COG trial, testing arsenic trioxide and all-trans retinoic acid in treatment of newly diagnosed children with acute promyelocytic leukemia. He is investigating the causes of bleeding/thrombosis and early death in children with acute promyelocytic leukemia. Dr. Kutny is leading an effort to decrease burden of care by developing a trial to evaluate oral (instead of IV) form of arsenic trioxide. He is leading a trial that tests a novel antibody drug combination in patients with relapsed leukemia. Dr. Kutny is leading efforts to optimize CNS treatment in AML and develop best strategies for risk stratification to tailor therapy based on genetic and disease response. Dr. Kutny also serves on the COG Cancer Control and acute lymphoblastic leukemia steering committees. He is studying the role of PRMT1 inhibition in acute megakaryoblastic leukemia in collaboration with Dr. Xinyang Zhou. Ana Xavier, M.D., conducts clinical trials in pediatric lymphoma. She currently is the principal investigator in two investigator initiated trials, NCT05253495, “Reducing the burden of oncologic chemoradiotherapy and radiation exposure from diagnostic imaging by utilizing targeted immunotherapy in children, adolescents, and young adults with lymphoma,” and NCT03719105, “Chemoimmunotherapy and allogeneic stem cell transplant for NK/T-cell leukemia/lymphoma” for children, adolescents, and young adults with Hodgkin or non-Hodgkin lymphoma. In addition, Dr. Xavier has developed prominence as a leader in rare pediatric non Hodgkin lymphomas, serving on guidelines and research committees for the National Comprehensive Cancer Network, Children’s Oncology group, and International BFM/EICNHL/COG. In additional, she has been able to participate in review submitted abstracts, and moderate/present several oral sessions in national and international meetings, including for the American Society of Hematology Annual Meeting. SOLID TUMOR PROGRAM The Pediatric Solid Tumor Program is a multidisciplinary team that is committed to excellence in clinical care and research in pediatric solid tumors. The team, led by Elizabeth Alva, M.D., consists of four pediatric oncologists, one surgical oncologist, one orthopedic oncologist and one radiation oncologist who work collaboratively to provide ongoing improvements to care for our patient population. Dr. Alva is an active member of the NCI-COG pediatric Molecular Analysis for Therapy Choice (MATCH) committee. She is currently the study chair on a sub-protocol of the Pediatric MATCH trial that she developed titled, “AG-120 (Ivosidenib) in Patients with Tumors Harboring IDH1 Mutations.” Dr. Alva is also active on the Target Agent Prioritization committee for the Pediatric MATCH through COG. She is the site PI of the Beat Childhood Cancer Consortium, which offers clinical trials for patients with medulloblastoma and neuroblastoma. In addition, she is the site PI for the Sunshine Project/NPCF consortium, which also provides early phase trials for patients with solid tumor and brain tumors. She is a member of the protocol committee for the Sunshine Project as well. She serves as the site PI for multiple COG studies focusing on solid tumors, including neuroblastoma, hepatoblastoma and germ cell tumors. Her expertise also includes retinoblastoma, and she works closely with the ocular oncologist, Dr. John Mason, to provide multidisciplinary

2023 Academic Annual Report

78