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Yael P. Mossé, MD

Attending Physician

Assistant Professor, Perelman School of Medicine at the University of Pennsylvania

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.


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