Assistant professor, University of Pennsylvania School of Medicine
- Department: Pediatrics
- Division: Oncology
- Primary Address:
The Children's Hospital of Philadelphia
Colket Translational Research Building, Office 3056
3501 Civic Center Blvd.
Philadelphia, PA 19104
- Assistant Professor of Pediatrics, University of Pennsylvania School of Medicine (2006 – present)
- M.D., Sackler School of Medicine, Tel Aviv, Israel (1997)
- B.A., Smith College (1993)
- B.A., Hebrew University, Jerusalem, Israel (1992)
My clinical and research specialty is neuroblastoma, a cancer that starts in the nerve tissues of infants and young children. At Children's Hospital, we have developed the nation's foremost clinical and research team devoted to children with neuroblastoma, and offer the possibility of clinical trials not available elsewhere.
In our lab, we have made great strides in our knowledge of the hereditary predisposition and progression of neuroblastoma. We 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 us translate our knowledge from the lab to new and -- one day -- curative ideas for patients. Along with the possibility of new therapeutics, we also can offer noninvasive screening for patients with a history that suggests a genetic predisposition to developing neuroblastoma.
Our neuroblastoma developmental therapeutics team involves oncologists, nurse practitioners, a clinical research assistant and a social worker, along with the child's primary oncologist. Our team approach means we all work together to help provide options for the families and patients who are referred to us or who seek us out on their own. Our team is always available to communicate with the family, an important consideration since many of our families come from outside our region. After the initial consult, I always let families know they are free to continue to communicate with us and ask questions. We believe that tough decision-making is best done in partnership with the family, which requires a deep understanding of the child and his/her history, and a sincere evaluation of all options both at the time of initial consultation and down the road. Our 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 our lab research involves the genetic mutations responsible for neuroblastoma. We recently discovered that a region of chromosome 2 was associated with the disease, and we 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 we are now working on translating our discovery to the therapeutic use of ALK inhibiting drugs. We currently have 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 I started as a fellow with Dr. John Maris, who is the premier expert in neuroblastoma, I found it intriguing to learn there were families with a history of the cancer. I 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 we do in the lab is focused on thinking about individual patients and how to develop new therapies. With a lot of determination, we have been able to translate our studies into clinical trials and move from patient to lab – and back to patient. I don't think I could do this kind of work anywhere else. We have a tremendous team and have created a model that is branching out from neuroblastoma to all childhood cancers.
Each child is unique and has something to teach us that will help us do better for the next child battling cancer. It is our privilege to learn from each individual child and to bring this wisdom back to the lab, and then back again to the clinic.
- Mossé Yaël P, Deyell Rebecca J, Berthold Frank, Nagakawara Akira, Ambros Peter F, Monclair Tom, Cohn Susan L, Pearson Andrew D, London Wendy B, Matthay Katherine K. Neuroblastoma in older children, adolescents and young adults: A report from the International Neuroblastoma Risk Group project.. Pediatric blood & cancer. 2013 Sep.
- Mosse Yael P, Lim Megan S, Voss Stephan D, Wilner Keith, Ruffner Katherine, Laliberte Julie, Rolland Delphine, Balis Frank M, Maris John M, Weigel Brenda J, Ingle Ashish M, Ahern Charlotte, Adamson Peter C, Blaney Susan M. 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. Vol 14(6) . 2013 May:472-80.
- Pugh Trevor J, Morozova Olena, Attiyeh Edward F, Asgharzadeh Shahab, Wei Jun S, Auclair Daniel, Carter Scott L, Cibulskis Kristian, Hanna Megan, Kiezun Adam, Kim Jaegil, Lawrence Michael S, Lichenstein Lee, McKenna Aaron, Pedamallu Chandra Sekhar, Ramos Alex H, Shefler Erica, Sivachenko Andrey, Sougnez Carrie, Stewart Chip, Ally Adrian, Birol Inanc, Chiu Readman, Corbett Richard D, Hirst Martin, Jackman Shaun D, Kamoh Baljit, Khodabakshi Alireza Hadj, Krzywinski Martin, Lo Allan, Moore Richard A, Mungall Karen L, Qian Jenny, Tam Angela, Thiessen Nina, Zhao Yongjun, Cole Kristina A, Diamond Maura, Diskin Sharon J, Mosse Yael P, Wood Andrew C, Ji Lingyun, Sposto Richard, Badgett Thomas, London Wendy B, Moyer Yvonne, Gastier-Foster Julie M, Smith Malcolm A, Auvil Jaime M Guidry, Gerhard Daniela S, Hogarty Michael D, Jones Steven J M, Lander Eric S, Gabriel Stacey B, Getz Gad, Seeger Robert C, Khan Javed, Marra Marco A, Meyerson Matthew, Maris John M. The genetic landscape of high-risk neuroblastoma. Nature genetics. Vol 45(3) . 2013 Mar:279-84.
- Mosse Yael P, Lipsitz Emily, Fox Elizabeth, Teachey David T, Maris John M, Weigel Brenda, Adamson Peter C, Ingle Mark A, Ahern Charlotte H, Blaney Susan M. Pediatric Phase I Trial and Pharmacokinetic Study of MLN8237, an Investigational Oral Selective Small-Molecule Inhibitor of Aurora Kinase A: A Children's Oncology Group Phase I Consortium Study. Clinical Cancer Research. Vol 18(21) . 2012 Nov:6058-64.
- Carpenter E L, Haglund E A, Mace E M, Deng D, Martinez D, Wood A C, Chow A K, Weiser D A, Belcastro L T, Winter C, Bresler S C, Asgharzadeh S, Seeger R C, Zhao H, Guo R, Christensen J G, Orange J S, Pawel B R, Lemmon M A, Mosse Y P. Antibody targeting of anaplastic lymphoma kinase induces cytotoxicity of human neuroblastoma. Oncogene. Vol 31(46) . 2012 Nov:4859-67.
- Carpenter Erica L, Mossé Yael P. Targeting ALK in neuroblastoma--preclinical and clinical advancements.. Nature Reviews Clinical Oncology. Vol 9(7) . 2012 Jul:391-9.
- DuBois Steven G, Chesler Louis, Groshen Susan, Hawkins Randall, Goodarzian Fariba, Shimada Hiroyuki, Yanik Greg, Tagen Michael, Stewart Clinton, Mosse Yael P, Maris John M, Tsao-Wei Denice, Marachelian Araz, Villablanca Judith G, Matthay Katherine K. Phase I study of vincristine, irinotecan, and ¹³¹I-metaiodobenzylguanidine for patients with relapsed or refractory neuroblastoma: a new approaches to neuroblastoma therapy trial.. Clinical cancer research : an official journal of the American Association for Cancer Research. Vol 18(9) . 2012 May:2679-86.
- Bosse Kristopher R, Diskin Sharon J, Cole Kristina A, Wood Andrew C, Schnepp Robert W, Norris Geoffrey, Nguyen Le B, Jagannathan Jayanti, Laquaglia Michael, Winter Cynthia, Diamond Maura, Hou Cuiping, Attiyeh Edward F, Mosse Yael P, Pineros Vanessa, Dizin Eva, Zhang Yongqiang, Asgharzadeh Shahab, Seeger Robert C, Capasso Mario, Pawel Bruce R, Devoto Marcella, Hakonarson Hakon, Rappaport Eric F, Irminger-Finger Irmgard, Maris John M. Common variation at BARD1 results in the expression of an oncogenic isoform that influences neuroblastoma susceptibility and oncogenicity. Cancer research. Vol 72(8) . 2012 Apr:2068-78.
- Y.P. Mosse, F.M. Balis, M.S. Lim, J Laliberte, S Voss, E Fox, R Bagatell, B.J. Weigel, P.A. Adamson, M.A. Ingle, C.H. Ahern, S.M. Blaney. Efficacy of crizotinib in children with relapsed/refractory ALK-driven tumors: A Children’s Oncology Group Phase 1 Study. ASCO Annual Meeting. 2012.
- A.C. Wood, M. Laudenslager, K. Krytska, M. Sarmady, J. Perin, E.F. Rappaport, J.M. Maris, M. Devoto, Y.P. Mosse. Identification of a new hereditary neuroblastoma predisposition locus at chromosome 2p25. Advances in Neuroblastoma Research (ANR) Meeting. 2012.