Benjamin Huang, MD

The research in our laboratory is focused on cancer modeling, molecular therapeutics, functional genomics, and biomarker discovery in hematologic cancers. Our research focuses primarily on pediatric acute myeloid leukemia (AML), which is characterized by frequent co-occurrence of fusion oncogenes and signal transduction mutations. While targeted inhibitors directed against either class of alteration have shown activity in AML, patients invariably relapse with treatment-refractory leukemia, underscoring the need for both a deeper understanding of how transcriptional and signaling mutations cooperate to drive leukemia growth and how these genetic alterations create novel dependencies. Thus, we are harnessing promising small molecule inhibitors and synthetic lethality approaches to uncover therapeutic vulnerabilities. We are also collaborating with the Children's Oncology Group (COG) to study the largest pediatric AML genome and transcriptome sequencing project to date (NCI TARGET). We are uncovering distinct transcriptional and epigenetic landscapes in pediatric AML, informing novel candidate biomarker signatures and immunotherapy targets. These data have also allowed us to achieve unprecedented residual disease detection using ultrasensitive molecular platforms in pediatric AML. Our overall goal is to develop new therapeutic and genomic strategies in AML to inform biology-driven, genetically stratified trials of innovative drug combinations.