Rare Tumor Clinical Research Specialist - Rare tumors, while individually uncommon, account for 25% of cancer deaths. These cancers have been understudied, leading to less improvement in outcome than for common cancers. Obstacles to conducting clinical trials for patients with rare tumors are many, including limited biospecimens resulting in a lack of understanding of the biology and therapeutic targets, small patient numbers, and the need for novel trial designs. Additionally, most rare pediatric cancers occur in the adolescent and young adult population, and care for these patients is often fragmented between numerous pediatric and adult subspecialists. My role as a rare tumor clinical research specialist is to address each of these barriers at both the institutional and National Clinical Trials Network (NCTN) levels. Nationally, I serve as chair of the Children’s Oncology Group (COG) Rare Tumor Disease Committee, and study chair for three COG clinical trials. With R50 support, I will expand the availability of rare tumor clinical trials within COG, focusing on trials of immunotherapies and molecularly targeted and genomically informed therapies for rare cancers. This will include developing novel studies for patient populations never previously studied within COG, including thyroid cancer and melanoma. Importantly, I will continue to build partnerships with the adult NCTN groups to design trials that span the adolescent-young adult divide. Additionally, through leadership of rare tumor efforts for the NCI/COG Molecular Characterization Initiative and our institutional biobank, I will expand the collection and study of clinically and molecularly annotated biospecimens. At the Abramson Cancer Center, I will develop a pediatric-adult rare tumor program, built upon my leadership of the Pediatric Very Rare Malignant Tumor Program and role as institutional PI of the NCI-funded Pediatric Early Phase Clinical Trial Network (PEP-CTN). I will lead both NCTN and institutional trials that span the pediatric-adult age range. Through this program, I will increase enrollment to both NCTN and investigator-initiated precision medicine trials for both children and adults with rare tumors and develop a software tool enabling physicians to search for appropriate trials for their patients, regardless of whether they are open at the pediatric or adult hospital. Finally, I will mentor the next generation of rare tumor and developmental therapeutic clinical trialists to design and lead studies through COG/NCTN. Together, this award will substantially expand the pipeline of rare tumor clinical trials within the NCTN, increase participation in NCI-funded rare tumor clinical research across the age spectrum at ACC, augment both institutional and COG biobanks to enable the discoveries that will drive the next generation of national cooperative group trials, and develop the next generation of rare tumor leaders within the COG.
