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.
Education and Training
MD - Sackler School of Medicine, Tel Aviv, Israel
Pediatrics - Children's Hospital of Philadelphia, Philadelphia, PA
Pediatrics - Children's Hospital of Philadelphia, Philadelphia, PA
Pediatric Hematology/Oncology - Children's Hospital of Philadelphia, Philadelphia, PA
Pediatric Hematology-Oncology – American Board of Pediatrics
Smith College Northampton, MA
Hebrew University, Jerusalem, Israel
Titles and Academic Titles
Patricia Brophy Endowed Chair in Neuroblastoma Research
Associate Professor, Perelman School of Medicine at the University of Pennsylvania
Departments and Services
Areas of Focus
Laboratories & Research Programs
Hereditary predisposition and progression of neuroblastoma
Wood AC, Krytska K, Ryles HT, Infarinato NR, Sano R, Hansel TD, Hart LS, King FJ, Smith TR, Ainscow E, Grandinetti KB, Tuntland T, Kim S, Caponigro G, He YQ, Krupa S, Li N, Harris JL, Mossé YP. Dual ALK and CDK4/6 inhibition demonstrates synergy against neuroblastoma. Clin Cancer Res. 2017;23(11):2856-2868.
Infarinato NR, Park JH, Krytska K, Ryles HT, Sano R, Szigety KM, Li Y, Zou HY, Lee NV, Smeal T, Lemmon MA, Mossé YP. The ALK/ROS1 Inhibitor PF-06463922 Overcomes Primary Resistance to Crizotinib in ALK-Driven Neuroblastoma. Cancer Discov. 2015 Nov 10. [Epub ahead of print]
Devoto M, Specchia C, Laudenslager M, Longo L, Hakonarson H, Maris J, et al. Genome-wide linkage analysis to identify genetic modifiers of ALK mutation penetrance in familial neuroblastoma. Hum Hered. 2011;71(2):135-9. Epub 2011 Jul 6. Cited in PubMed: PMID 21734404.
Johnson K, McGlynn B, Saggio J, Baniewicz D, Zhuang H, Maris JM, et al. Safety and efficacy of tandem (131) I-metaiodobenzylguanidine infusions in relapsed/refractory neuroblastoma. Pediatr Blood Cancer. Epub 2011 Apr 14. doi: 10.1002/pbc.23062. Cited in PubMed: PMID 21495159.
Nguyen le B, Diskin SJ, Capasso M, Wang K, Diamond MA, Glessner J, et al. Phenotype restricted genome-wide association study using a gene-centric approach identifies three low-risk neuroblastoma susceptibility loci. PLoS Genet. 2011 Mar;7(3)e1002026. Epub 2011 Mar 17. Cited in PubMed: PMID 21436895.
Cole KA, Huggins J, Laquaglia M, Hulderman CE, Russell MR, Bosse K, et al. RNAi screen of the protein kinome identifies checkpoint kinase 1 (CHK1) as a therapeutic target in neuroblastoma. Proc Natl Acad Sci U S A. 2011 Feb 22;108(8):3336-41. Epub 2011 Feb 2. Cited in PubMed: PMID 21289283.
Wang K, Diskin SJ, Zhang H, Attiyeh EF, Winter C, Hou C, et al. Integrative genomics identifies LMO1 as a neuroblastoma oncogene. Nature. 2011 Jan 13;469(7329):216-20. Epub 2010 Dec 1.
Lipsitz E, Moorthy G, Mosse Y, Fox E, Adamson PC. A sensitive and selective liquid chromatography/tandem mass spectrometry method for determination of MLN8237 in human plasma. J Chromatogr B Analyt Technol Biomed Life Sci. 2010 Sep 1;878(25):2369-73. Epub 2010 Jul 3. Cited in PubMed: PMID 20685183.
Yu D, dos Santos CO, Zhao G, Jiang J, Amigo JD, Khandros E, et al. miR-451 protects against erythroid oxidant stress by repressing 14-3-3zeta. Genes Dev. 2010 Aug 1;24(15):1620-33. Cited in PubMed: PMID 20679398.
Diskin SJ, Hou C, Glessner JT, Attiyeh EF, Laudenslager M, Bosse K, et al. Copy number variation at 1q21.1 associated with neuroblastoma. Nature. 2009 Jun 18;459(7249):987-91. Cited in PubMed: PMID 19536264.
Capasso M, Devoto M, Hou C, Asgharzadeh S, Glessner JT, Attiyeh EF, et al. Common variations in BARD1 influence susceptibility to high-risk neuroblastoma. Nat Genet. 2009 Jun;41(6):718-23. Epub 2009 May 3. Cited in PubMed: PMID 19412175.
Attiyeh EF, Diskin SJ, Attiyeh MA, Mossé YP, Hou C, Jackson EM, et al. Genomic copy number determination in cancer cells from single nucleotide polymorphism microarrays based on quantitative genotyping corrected for aneuploidy. Genome Res. 2009 Feb;19(2):276-83. Epub 2009 Jan 13. Erratum in: Genome Res. 2009 Mar;19(3):520. Cited in PubMed: PMID 19141597.
Wood A, Laudenslager M, Haglund EA, Attiyeh EF, Cole KA, Diskin S, et al. Potent Inhibition of ALK mutated neuroblastomas by the selective inhibitor PF02341066 [abstract]. 2009 ASCO Annual Meeting; Orlando, FL, 2009 May 30-Jun 3.
Editorial and Academic Positions
Reviewer for journals
Cancer Genetics Cytogenetics
Journal of Clinical Oncology
Medical and Pediatric Oncology
Pediatric Blood and Cancer
University of Pennsylvania
2008, Full member, Abramson Cancer Center of the University of Pennsylvania
The Children's Hospital of Philadelphia
2008, Member, Clinical Research Quality Assurance Committee
2008, Member, Institutional Review Board
2006, Member, Genes Genomes and Pediatric Diseases Research Affinity Group
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