Yael P. Mossé
雅尔·莫斯
MD
Associate Professor of Pediatrics; Attending Physician, Division of Oncology儿科学副教授，肿瘤科主治医师
👥Biography 个人简介
Yael P. Mossé, MD is a physician-scientist at Children's Hospital of Philadelphia whose work has been pivotal in translating the discovery of ALK mutations in neuroblastoma into clinical practice. Co-discoverer of ALK as the hereditary neuroblastoma predisposition gene in 2008, she subsequently led the first pediatric trials of crizotinib (COG ADVL0912) and lorlatinib in ALK-driven pediatric cancers, including neuroblastoma, anaplastic large cell lymphoma, and inflammatory myofibroblastic tumors. Her research spans mechanistic studies of ALK oncogenic signaling, preclinical modeling of resistance mechanisms, and the design of early-phase pediatric oncology trials through the Children's Oncology Group. She is widely credited with establishing ALK as the first tractable precision oncology target in neuroblastoma.
🧪Research Fields 研究领域
🎓Key Contributions 主要贡献
Co-Discovery of ALK Mutations in Neuroblastoma
As co-first author of the 2008 Nature Genetics paper, identified germline and somatic ALK mutations in neuroblastoma families and sporadic cases, providing the molecular rationale for targeting ALK with small-molecule inhibitors.
First Pediatric Clinical Trials of Crizotinib
Led COG study ADVL0912, the first Phase I/II trial of crizotinib (a dual ALK/MET/ROS1 inhibitor) in children with relapsed or refractory solid tumors and anaplastic large cell lymphoma, demonstrating safety and early efficacy signals that led to FDA pediatric approval.
Lorlatinib in Pediatric ALK-Driven Tumors
Designed and leads the COG ADVL1622 trial of lorlatinib (third-generation ALK/ROS1 inhibitor) in pediatric patients with ALK-driven neuroblastoma and other malignancies, targeting both primary and crizotinib-resistant ALK mutations.
Resistance Mechanisms to ALK Inhibition
Characterized compound ALK mutations and bypass resistance mechanisms that emerge under selective pressure of ALK inhibitors in neuroblastoma, informing rational combination strategies and next-generation inhibitor development.
Representative Works 代表性著作
Identification of ALK as a major familial neuroblastoma predisposition gene
Nature (2008)
Co-first author study identifying ALK germline mutations (F1174L, R1275Q) as the primary cause of hereditary neuroblastoma and somatic driver in sporadic disease.
Crizotinib in pediatric patients with advanced ALK-driven tumors: phase I study
Journal of Clinical Oncology (2017)
COG ADVL0912 phase I/II results demonstrating tolerability and activity of crizotinib across ALK-positive pediatric cancers including ALCL and neuroblastoma.
Phase 2 study of crizotinib in children and adolescents with ALK-positive neuroblastoma
Journal of Clinical Oncology (2021)
Pivotal phase II results from the neuroblastoma cohort of COG ADVL0912 showing objective responses to crizotinib in heavily pretreated patients with ALK-mutated neuroblastoma.
🏆Awards & Recognition 奖项与荣誉
📄Data Sources 数据来源
Last updated: 2026-04-06 | All information from publicly available academic sources
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