My research has focused on clinical trials for developing novel treatments for pediatric solid tumors and pediatric tumor predisposition syndromes, including neurofibromatosis and multiple endocrine neoplasia type 2 (MEN2)-associated tumors.

I am the chair for a phase 2 trial of cabozantinib that is conducted through the Children’s Oncology Group consortium,where we are studying the activity of this drug in bone and soft-tissue sarcomas, and other solid tumors in children and young adults.

Neurofibromastosis type 1 (NF1) is an autosomal dominant disorder caused by mutations in the NF1 tumor suppressor gene. Plexiform neurofibromas are benign nerve sheath tumors seen in NF1 that can cause severe morbidities and have the potential for transformation to malignant peripheral nerve sheath tumors (MPNST), an aggressive sarcoma with poor outcomes. My research has focused on the analysis of plexiform neurofibroma growth using volumetric MRI measurements, and the identification of precursors for malignant transformation in plexiform neurofibroma based on imaging and growth characteristics. The overall goal of my projects is to help guide the design of meaningful treatment trials for NF1-associated plexiform neurofibroma, and to identify precursors of malignant transformation to help in early diagnosis and potential prevention of MPNST.

MEN2 is a rare cancer predisposition syndrome caused by a germline activating mutation in the RET proto-oncogene.It is characterized by childhood medullary thyroid cancer (MTC), pheochromocytoma, and other non-tumor manifestations. There are no curative treatments for patients with advanced MTC that cannot be removed completely by surgery. I developed a natural history trial of MEN2-associated MTC with the goal of better understanding the biology, natural history, and mechanisms of resistance to targeted therapies in this rare disease.