ELVIN WAGENBLAST, PHD
The Tisch Cancer Institute at Mount Sinai
CureSearch Young Investigator Award: 2023-2025
Focus: Pediatric Leukemia; Acute Myeloid Leukemia
Project title: Targeting Therapy Resistance in NUP98-NSD1 Rearranged Childhood Leukemia
This project is supported in part by the Team Steve: Steven Crowe Legacy Fund
Leukemia is the most frequent cancer in children. Although acute myeloid leukemias (AMLs) only account for 20% of pediatric acute leukemias, it is the leading cause of childhood leukemia mortality. NUP98-NSD1 rearranged leukemia is observed in about 15% of cytogenetically normal pediatric AML cases and represents a poor prognostic group. There is a critical need to identify and characterize novel therapeutic interventions in cases where standard chemotherapy is ineffective in children with AML.
Elvin Wagenblast, PhD, from the Icahn School of Medicine at Mount Sinai, is approaching the study of pediatric leukemia in a different way by characterizing the evolution of leukemia from a developmental perspective. His team is researching the molecular mechanisms of NUP98-NSD1 rearranged pediatric AML using an innovative CRISPR/Cas9 mediated genomic engineering approach in human blood stem cells obtained from cord blood to model leukemia initiation and progression. The team is working to identify and characterize novel therapeutic targets that prevent leukemic maintenance and progression in NUP98-NSD1 rearranged leukemia.
"My vision is to understand the earliest steps of how and why childhood leukemia initiates and
to use this knowledge to innovate therapeutic intervention focused on targeting the
preleukemic and leukemic phase.”