Professor Yaël Mossé

Dr. Mossé is a tenured Professor at The Children's Hospital of Philadelphia and the Perelman School of Medicine of the University of Pennsylvania. She is a physician-scientist who has focused for two+ decades on the childhood cancer neuroblastoma with the dual goals of improving patient outcomes and using the disease as a model to understand cancer in general. She received her M.D. from the Sackler School of Medicine, Tel Aviv, Israel, in 1997, joining the Children's Hospital residency program that year, followed by fellowship training at the same hospital starting in 2000.One unique aspect of her work is the translational nature that seamlessly straddles the laboratory and the clinic. Her seminal discovery of gain-of-function mutations within the tyrosine kinase domain of the Anaplastic Lymphoma Kinase (ALK) gene as the major cause of hereditary neuroblastoma, and the demonstration that these same alterations are the most common somatic single nucleotide mutations in the sporadic forms of the disease have positioned ALK is the only mutated oncogene tractable for targeted therapy in NB. ALK inhibition therapy has now moved upfront in the currently enrolling COG Phase 3 clinical trial for patients with newly diagnosed high-risk neuroblastoma, initially with crizotinib, but more recently with lorlatinib as the ALK inhibitor of choice. 

In parallel, her lab is developing newer and more precise strategies for targeting ALK as she learns from patients about de novo and acquired resistance, including the development of immunotherapeutic strategies to target cell surface ALK, which is expressed on most neuroblastomas and select other pediatric and adult cancers.