Translational Development of a tumor-selective pHLIP–exatecan conjugate for sarcoma
Outcomes remain poor for children with rhabdomyosarcoma (RMS) and Ewing sarcoma (EWS), two aggressive cancers that are very difficult to cure once they come back. Current treatments, like irinotecan, can cause significant side effects. A related drug, exatecan, is more powerful but has not been widely used because it can be too toxic.
CBX-12 is a new type of drug designed to deliver exatecan more directly to tumors. It uses a small peptide that homes in on the acidic environment commonly found in cancer cells, helping concentrate the drug in tumors while limiting damage to healthy tissue. Early studies in adults suggest that CBX-12 is safe and may be effective in hard-to-treat cancers.
Project Goals
This project will test how well CBX-12 works in laboratory models made from patient tumors, identify features that help predict which cancers will respond, and explore combinations with other treatments to improve its effectiveness.
The goal of this work is to support the development of a future clinical trial in children, with the hope of improving survival while reducing treatment-related side effects.
