KRISTEN VANHEYST, DO
University Hospitals Rainbow Babies & Children’s Hospital (Cleveland, OH)
CureSearch Young Investigator Award: 2023-2025
Focus: Pediatric Osteosarcoma; Tumor Microenvironment; Immunotherapy
Project title: Tumor Microenvironment Modulation as an Effective Therapy for Osteosarcoma
Osteosarcoma is the most prevalent aggressive primary malignancy of the bone affecting children and young adults. Although overall survival in patients with osteosarcoma has improved with advances in therapy, there have been no significant improvements in survival outcomes in patients with metastatic pulmonary osteosarcoma. Due to the complex genetic heterogeneity of osteosarcoma, molecular-targeted therapy approaches are not feasible. For this reason, novel therapeutic approaches are desperately needed.
Kristen VanHeyst, DO, from Rainbow Babies and Children's Hospital, is working to establish the efficacy of a novel TGF-beta I receptor inhibitor for the treatment of osteosarcoma. The presence of TGFß contributes to making the tumor microenvironment more hospitable to tumor cells and dampens the ability of the patient's own immune system to eradicate this tumor. This project is a novel possibility that may highly impact the survival of pediatric and adolescent/young adult patients with metastatic pulmonary osteosarcoma or refractory osteosarcoma by offering real clinical opportunities for treatment.
"This project will expand upon our current trial design. We aim to ultimately lower the age limit for enrollment onto the trial, which will allow more pediatric patients access to novel therapeutic options.”