CHICAGO — Early results from the first-in-human phase 1 clinical trial of gamitrinib, a first-in-class mitochondria-targeting cancer therapy, were presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. The study showed that the drug was safely administered to 18 patients with advanced cancer who had exhausted standard treatment options.
Gamitrinib is engineered to act inside cancer cell mitochondria, disrupting key survival functions tumors use to grow and resist treatment. The therapy was designed to concentrate within mitochondria, where cancer cells may be especially vulnerable. Pre-clinical work conducted in collaboration with the Wistar Institute in Philadelphia previously demonstrated that gamitrinib could slow or stop tumor growth in multiple cancer types.
The ongoing phase 1 trial includes adults with advanced solid tumors or lymphoma whose disease has stopped responding to standard therapies. Patients receive gamitrinib as a weekly intravenous infusion, with doses gradually increased to evaluate safety. To date, 18 patients have been treated across multiple dose levels, and no dose-limiting side effects have been observed. Blood samples confirm the drug behaves as expected in the body.
“These early findings suggest the drug can be administered safely at the dose levels tested to date.” said Anthony Olszanski, Vice Chair of Clinical Research, Department of Hematology/Oncology and Director of the Early Clinical Drug Development Phase 1 Program, Fox Chase Cancer Center. “That’s a critical first step in developing a completely new approach to cancer treatment.”
The trial remains open, and patient enrollment is continuing. Researchers aim to determine the optimal dose for future studies and to assess early signals of efficacy, including whether the drug can slow or shrink tumors. The study, titled “A phase 1, first-in-human, open-label, dose escalation and expansion study of Gamitrinib, a first-in-class, mitochondria-directed inhibitor of the molecular chaperone Heat Shock Protein-90 (Hsp90) in patients with advanced cancer,” represents a novel strategy in oncology by directly targeting mitochondrial function in cancer cells.