At Children's Hospital, I work with a team of world-class clinicians and scientists dedicated to rapidly translating research about childhood cancers from the laboratory to the patient. We are one of the nation’s leaders in early phase clinical research that intently focuses on improving pediatric cancer cure rates and minimizing long-term side effects. We design studies to help benefit children who have refractory or relapsed diseases for which treatment options may be limited. As we make new discoveries, our goal is to bring them quickly to young patients. I also serve as chairman of the national Children’s Oncology Group Neuroblastoma Committee, a research consortium of more than 200 member institutions.
My own specialty is neuroblastoma, an often intractable cancer that starts in young nerve cells and can be extremely aggressive. I've been interested in this disease since before medical school, when I had the opportunity to work with noted scientists Drs. Audrey Evans and Britton Chance. I later became convinced that neuroblastoma is a genetic disease, and that we could make critical insights by discovering the genes that cause the cancer.
To learn more and make a difference as quickly as possible, I developed a laboratory and translational research program at Children's Hospital to help us focus on the genetic abnormalities in hereditary and sporadic neuroblastoma. We strive to be the world’s leading neuroblastoma research and treatment center as we work to exponentially increase our understanding of this cancer and its underlying biology. Our challenge is to impact clinical practice – an effort we approach with urgency and a large measure of recent success. In the past few years, our team at Children's Hospital has been able to identify the main genes associated with this cancer. As a result, we moved some of these discoveries toward new therapies, a number of which are now in clinical trials.
To help support our investigations, my colleagues and I have gathered the largest collection of pediatric neuroblastoma cases ever assembled. This allowed us to perform studies that led to the discovery of neuroblastoma genes and identify who is likely to get the disease. Dr. Yael Mosse and I, along with our colleagues at Children's Hospital, found that mutations of the ALK (anaplastic lymphoma kinase) oncogene explain most hereditary neuroblastomas. The gene is also mutated in cancer cells -- making it a therapeutic target for this malignancy. Trials of an ALK-attacking compound for neuroblastoma patients are currently underway.
Our neuroblastoma team has made enormous progress as we translate our basic and clinical research into improved therapies. Patients, their families and referring physicians may be confident that our outstanding clinicians and researchers provide the most comprehensive and cutting edge care possible for children with this difficult cancer.
- Education and Training
MD - University of Pennsylvania School of Medicine, Philadelphia, PA
Pediatrics - The Children's Hospital of Philadelphia, Philadelphia, PA
Hematology/Oncology - The Children's Hospital of Philadelphia, Philadelphia, PA
- Titles and Academic Titles
Giulio D'Angio Chair in Neuroblastoma Research
Professor of Pediatrics, University of Pennsylvania School of Medicine
- Departments and Services
- Research Interests