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.