Developing early phase clinical trials to identify therapeutic biomarkers and novel combination therapies for children with high-risk sarcomas and liver tumors.
The prognoses for children with high-grade sarcomas and high-risk liver tumors have historically been poor, and this has remained unchanged for decades. This, in part, is due to the rare nature of these cancers, the lack of useful preclinic models and the lack of molecular understanding of these cancers. To increase survival rates and treatment options for children with sarcomas and liver tumors, I develop early phase clinical trials to identify therapeutic biomarkers, partner in the creation of useful preclinical models and develop combination therapies.
The survival rate for children with localized sarcomas is approximately 60-70%, and this statistic has remained unchanged for over 30 years. For those with metastatic and recurrent disease, the survival rate is less than 30%. Children diagnosed with rare liver tumors have similar outcomes. These statistics highlight the importance of finding successful treatment strategies for children diagnosed with these rare cancers.
Unfortunately, our ability to understand these malignancies and find successful treatments has been stalled due to the lack of clinical trials and the lack of relevant and sustainable model systems that inform our basic science and translational studies. My work is focused on partnering with pre-clinical scientists to harness the power of early phase clinical trials to create useful model systems to study sarcomas and liver tumors as well as develop combination therapies for future early phase clinical trials.
Many sarcomas are grouped into a large heterogenous group and exhibit variable responses to chemotherapy. I aim to increase our understanding of these sarcomas by comparing tumor types across all risk levels. The genetic information gained from this work will guide the design of early phase clinical trials and inform treatment strategies for sarcomas such as desmoplastic small round cell tumors and malignant rhabdoid tumors, among others.
The survival rate for children with rare liver cancers, such as fibrolamellar carcinoma, can be below 20%. By developing clinical trials driven by biological questions and creating useful tumor models to be used in basic and translational laboratories, we stand to increase our understanding of pediatric hepatocellular carcinomas and fibrolamellar carcinomas and increase survival rates.
Dr. Gartrell is a pediatric oncologist who received her MD from the University of Nebraska Medical Center in Omaha, Nebraska. She then completed a pediatric residency at the University of Tennessee Health Science Center and fellowships in pediatric hematology/oncology and pediatric solid tumor at St. Jude Children’s Research Hospital. She also earned a graduate certificate in clinical and translational research from the University of Cincinnati. Dr. Gartrell is deeply passionate about developing early phase clinical trials for children with high-risk sarcomas and liver tumors. Through these clinical trials, she focuses on identifying therapeutic biomarkers and discovering novel combination therapies for these tumors. She also serves as the principal investigator of the Pediatric Early Phase Clinical Trials Network for St. Jude.