Department of Pediatrics

By: Ada Chong
April 2023

Jason Yustein, MD, PhD, joined the Emory Department of Pediatrics in July 2022 as a Professor of Pediatrics in Hematology/Oncology and Pediatric Hematologist/Oncologist at Children’s. Dr. Yustein has been involved in research as a faculty member for the past 15 years.

Dr. Yustein’s primary research focuses on pediatric sarcomas, which are bone and soft tissue cancers. His work centers on modeling these diseases better and understanding the biology behind these malignancies. Dr. Yustein and his team study high-risk conditions, including metastatic and therapeutic resistance disease. Patients who have diseases that spread to other locations in their body or who do not respond to frontline therapy typically equates to that patient doing very poorly. Dr. Yustein tests therapies for these advanced stages and looks to identify therapeutic avenues to target these diseases. His top goals for his research are to understand the fundamental aspects of why these tumors resist therapy and how tumors are successful in metastasizing in a patient’s body. Currently, there is no standard of care for metastatic or therapeutic resistant diseases.

During the research process, Dr. Yustein and his team develop a pipeline that integrates basic biology, pre-clinical testing, and getting treatment to patients. The first step is to learn how to better understand the biology of sarcomas. Later, Dr. Yustein integrates what he learned from patient samples and publicly available data. He also looks at identifying pathways of key enzymes that are happening inside and outside the tumor cell to understand their roles that contribute to the disease stages. He hopes to learn questions such as, “Are there alterations in certain proteins, enzymes and pathways that are targetable by itself or in combination with other therapies?” “How do we test therapies and combinations of therapies?” “Could these lead to the development of a clinical trial to improve outcomes for these high-risk conditions?”

The next steps are testing the therapies and taking the insights and translating them to therapeutic interventions.

Another research project Dr. Yustein is working on involves partnering with pharmaceutical companies and other faculty members to see if they can work together toward testing better therapies to target high-risk sarcomas. For example, in Dr. Yustein’s previous faculty position at Baylor College of Medicine he studied osteosarcoma, which is the most common bone tumor in the pediatric population. Dr. Yustein and his team identified a pathway that was dysregulated in osteosarcoma. A pharmaceutical company he worked with had a small molecule targeting a key protein in the osteosarcoma cell. They utilized his lab’s experience in modeling metastasis to see if the drug was effective in targeting metastatic osteosarcoma. From these critical pre-clinical studies, they developed a clinical trial in 2021 for the treatment of children with relapsed solid tumors.

Dr. Yustein began his research during his fellowship at The Johns Hopkins University/National Cancer Institute when he saw sarcoma patients. He says he saw what patients went through. “One day kids are playing soccer or in the marching band and the next day they are diagnosed with a potential deadly disease,” says Dr. Yustein. Sarcomas compared to other pediatric cancers hasn’t made much progress in overall survival rates. “We’re 65-75% in overall survival rates. We’ve made improvements, but there’s still a 1/3rd of patients succumbing to the disease, which is inferior to most other pediatric cancers. Furthermore, those patients with metastatic disease, their survival rate is less than 30%. These patients think about the future and have goals and I want to help them get there,” says Dr. Yustein.