Associate Professor
The Children’s Hospital of Philadelphia, University of Pennsylvania
I’m a pediatric oncologist and lead both the Developmental Therapeutics Program (DVL) and the Very Rare Malignant Tumors Program (VRMTP) at the Children’s Hospital of Philadelphia. I have national leadership roles in each of these areas within the Children’s Oncology Group (COG).
In the DVL program, I conduct early phase clinical trials of new treatments for children with difficult to treat cancers. My particular area of focus includes molecularly targeted therapies that are designed to block specific genetic mutations identified in patients’ tumors. In particular, I am the academic leader of the development of larotrectinib for children with NTRK gene fusions, and now lead a COG nationwide clinical trial studying the ability to use larotrectinib instead of chemotherapy for children with one of these gene fusions.
While individually very uncommon, together very rare malignant tumors comprise over 10% of childhood cancers. Given the rarity of each individual diagnosis, these cancers have been poorly studied and for many patients there are not standard, defined treatments. As the chair of the Rare Tumors Committee for the Children’s Oncology Group, I am working to advance the study of these tumors and define new ways to treat patients. I am hopeful that we can advance the care of patients with these tumors by using cancer genomics to identify targetable mutations as well as studying immunotherapy, which has revolutionized the care of many of these cancers in adult patients.