Yael P. Mossé, MD
Areas of expertise: Neuroblastoma
Locations: Buerger Center for Advanced Pediatric Care
About Yael P. Mossé, MD
Dr. Yael Mossé’s clinical and research specialty is neuroblastoma, a cancer that starts in the nerve tissues of infants and young children. The Children's Hospital of Philadelphia has developed the nation's foremost clinical and research team devoted to children with neuroblastoma, and offers the possibility of clinical trials that are not available elsewhere.
In Dr. Mossé’s lab, researchers have made great strides in the knowledge of the hereditary predisposition and progression of neuroblastoma. They discovered the gene mutations that are the primary cause of the inherited version of neuroblastoma and that also play a significant role in high-risk forms of the more common, non-inherited form of the disease. These findings are helping translate knowledge from the lab to new and — one day — curative ideas for patients. Along with the possibility of new therapeutics, physicians at CHOP can also offer noninvasive screening for patients with a history that suggests a genetic predisposition to developing neuroblastoma.
The neuroblastoma developmental therapeutics team involves oncologists, nurse practitioners, a clinical research assistant and a social worker, along with the child's primary oncologist. This team approach means CHOP’s experts all work together to help provide options for the families and patients. The team is always available to communicate with the family, an important consideration since many families come from outside our region.
After the initial consult, Dr. Mossé always let families know they are free to continue to communicate with her and ask questions. She believes that tough decision-making is best done in partnership with the family, which requires a deep understanding of the child and his history, and a sincere evaluation of all options both at the time of initial consultation and down the road. Dr. Mossé’s goal is to help patients and families go home after a serious appraisal of all options and strong consideration of quality of life.
The focus of Dr. Mossé’s lab research involves the genetic mutations responsible for neuroblastoma. The team recently discovered that a region of chromosome 2 was associated with the disease, and identified mutations in the anaplastic lymphoma kinase (ALK) gene. ALK is an oncogene (cancer causing gene) whose mutations are both genetic and acquired. Many pharmaceutical companies already make drugs that turn off the ALK gene, so Dr. Mossé’s team is now working on translating its discovery to the therapeutic use of ALK inhibiting drugs.
Dr. Mossé’s lab currently has a multi-institutional trial open through the Children’s Oncology Group for all children with relapsed neuroblastoma with the goal of bringing this therapy up-front for the treatment of newly diagnosed patients with neuroblastoma.
When Dr. Mossé started as a fellow with John M. Maris, MD, who is the premier expert in neuroblastoma, she found it intriguing to learn there were families with a history of the cancer. She decided to investigate this, and what had started as a side project – with luck and perseverance – turned out to result in a major scientific discovery.
Every experiment Dr. Mossé and her team does in the lab is focused on thinking about individual patients and how to develop new therapies. With a lot of determination, they have been able to translate the studies into clinical trials and move from patient to lab – and back to patient. Dr. Mossé believes she works with a tremendous team. Together, they have created a model that is branching out from neuroblastoma to all childhood cancers.
Titles
Attending Physician
Patricia Brophy Endowed Chair in Neuroblastoma Research
Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania
Certifications
Pediatric Hematology-Oncology – American Board of Pediatrics
Awards and Honors
2024, William Osler Patient Oriented Research Award, University of Pennsylvania Perelman School of Medicine
2024, Dr. Audrey E. Evans Award of Excellence to Drs. John Maris and Yael Mossé
2022, Frank A. Oski Memorial Award, American Society of Pediatric Hematology/Oncology
2021, Elected Member, American Pediatric Society
2018, Patricia Brophy Endowed Chair in Neuroblastoma Research, The Children's Hospital of Philadelphia/Alex's Lemonade Stand Foundation
2013, ASCO Leadership Development Program, American Society of Clinical Oncology
2013, Leadership Development Program, American Society of Clinical Oncology
2012, First Inaugural James B. Nachman ASCO Junior Faculty Award in Pediatric Oncology, American Society of Clinical Oncology
2012, Election to Membership, American Society of Clinical Investigation
2012, First Inaugural James B. Nachman Junior Faculty Award in Pediatric Oncology, American Society of Clinical Oncology
2006, Foerderer-Murray Award, Children's Hospital of Philadelphia
2004, Career Development Award, American Society of Clinical Oncology
2003, Young Investigator Award, American Society of Clinical Oncology
Leadership and Memberships
2021-present, American Pediatric Society
2021-present, American Association for Cancer Research
2013-present, American Society of Clinical Oncology
2012-present, American Society of Clinical Investigation
Research Interests
Hereditary predisposition and progression of neuroblastoma
Education & training
Undergraduate Degree
Smith College Northampton, MA
Graduate Degree
Hebrew University, Jerusalem, Israel
Medical Degree
MD - Sackler School of Medicine, Tel Aviv, Israel
Internship
Pediatrics - Children's Hospital of Philadelphia, Philadelphia, PA
Residency
Pediatrics - Children's Hospital of Philadelphia, Philadelphia, PA
Fellowship
Pediatric Hematology/Oncology - Children's Hospital of Philadelphia, Philadelphia, PA
Publications
Selected Publications
Mosse YP, Laudenslager M, Khazi D, Carlisle AJ, Winter CL, Rappaport E, et al. Germline PHOX2B Mutation in Hereditary Neuroblastoma. Am J Hum Genet. 2004;75(4):727-30.
Mosse YP, Laudenslager M, Longo L, Cole KA, Wood A, Attiyeh EF, et al. Identification of ALK as a major familial neuroblastoma predisposition gene. Nature. 2008;455(7215):930-5.
Bresler SC, Wood AC, Haglund EA, Courtright J, Belcastro LT, Plegaria JS, et al. Differential inhibitor sensitivity of anaplastic lymphoma kinase variants found in neuroblastoma. Sci Transl Med. 2011;3(108):108ra14.
Mosse YP, Lim MS, Voss SD, Wilner K, Ruffner K, Laliberte J, et al. Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children's Oncology Group phase 1 consortium study. The Lancet Oncology. 2013;14(6):472-80.
Bresler SC, Weiser DA, Huwe PJ, Park JH, Krytska K, Ryles H, et al. ALK mutations confer differential oncogenic activation and sensitivity to ALK inhibition therapy in neuroblastoma. Cancer Cell. 2014;26(5):682-94.
Krytska K, Ryles HT, Sano R, Raman P, Infarinato N, Hansel TD, et al. Crizotinib Synergizes with Chemotherapy in Preclinical Models of Neuroblastoma. Clin Cancer Res. 2015.
Infarinato NR, Park JH, Krytska K, Ryles HT, Sano R, Szigety KM, et al. The ALK/ROS1 Inhibitor PF-06463922 Overcomes Primary Resistance to Crizotinib in ALK-Driven Neuroblastoma. Cancer Discov. 2016;6(1):96-107.
Wood A, Krytska K, Ryles HT, Infarinato NR, Sano R, Hansel TD, et al. Dual ALK and CDK4/6 inhibition demonstrates on-target synergy against neuroblastoma. Clin Cancer Res. 2016.
Mosse YP, Voss SD, Lim MS, Rolland D, Minard CG, Fox E, et al. Targeting ALK With Crizotinib in Pediatric Anaplastic Large Cell Lymphoma and Inflammatory Myofibroblastic Tumor: A Children's Oncology Group Study. J Clin Oncol. 2017;35(28):3215-21.
Mosse YP, Fox E, Teachey DT, Reid JM, Sagfren SL, Carol H, et al. A Phase 2 Study of Alisertib in Children with Recurrent/Refractory Solid Tumors or Leukemia: Children's Oncology Group Phase 1 and Pilot Consortium ADVL0921). Clin Cancer Res. 2019.
Sano R, Krytska K, Larmour CE, Raman P, Martinez D, Ligon GF, et al. An antibody-drug conjugate directed to the ALK receptor demonstrates efficacy in preclinical models of neuroblastoma. Sci Transl Med. 2019;11(483).
Foster JH, Voss SD, Hall DC, Minard CG, Balis FM, Wilner K, et al. Activity of Crizotinib in Patients with ALK-Aberrant Relapsed/Refractory Neuroblastoma: A Children's Oncology Group Study (ADVL0912). Clin Cancer Res. 2021;27(13):3543-8.
Wolpaw AJ, Bayliss R, Buchel G, Dang CV, Eilers M, Gustafson WC, et al. Drugging the 'undruggable' MYCN oncogenic transcription factor: Overcoming previous obstacles to impact childhood cancers. Cancer Res. 2021.
Bosse KR, Giudice AM, Lane MV, McIntyre B, Schurch PM, Pascual-Pasto G, et al. Serial profiling of circulating tumor DNA identifies dynamic evolution of clinically actionable genomic alterations in high-risk neuroblastoma. Cancer Discov. 2022.
Berko ER, Witek GM, Matkar S, Petrova ZO, Wu MA, Smith CM, et al. Circulating tumor DNA reveals mechanisms of lorlatinib resistance in patients with relapsed/refractory ALK-driven neuroblastoma. Nat Commun. 2023;14(1):2601.
Goldsmith KC, Park JR, Kayser K, Malvar J, Chi YY, Groshen SG, et al. Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results. Nat Med. 2023.
Berko ER, Naranjo A, Daniels AA, McNulty SN, Krytska K, Druley T, et al. Frequency and Clinical Significance of Clonal and Subclonal Driver Mutations in High-Risk Neuroblastoma at Diagnosis: A Children's Oncology Group Study. J Clin Oncol. 2025:JCO2402407.
Matkar S, East MP, Stuhlmiller TJ, Witek G, Farrel A, Pastor S, et al. Kinome reprogramming of G2/M kinases and repression of MYCN contribute to superior efficacy of lorlatinib in ALK-driven neuroblastoma. Mol Cancer Ther. 2025.
Mosse YP, Polkosnik G, Pogoriler J, Mattei P, States LJ, Maris JM. Anaplastic Lymphoma Kinase Inhibition Therapy for Hereditary Neuroblastoma. JCO Precis Oncol. 2025;9:e2400886.