HONG KONG — A research team from the University of Hong Kong developed a combination therapy, QUIZOM, for chemotherapy-refractory FLT3-mutated acute myeloid leukemia. The therapy, combining Quizartinib and Omacetaxine Mepesuccinate, was evaluated in a Phase 2 clinical trial conducted from November 2017 to September 2020.

The clinical trial enrolled 40 patients, ranging in age from 23 to 81, who had chemotherapy-refractory FLT3-mutated acute myeloid leukemia. The trial documented a composite complete remission rate of approximately 83%. Participants had a median leukemia-free survival of 10 months and a median overall survival of 12.9 months.

Thirteen patients who received the QUIZOM therapy moved on to allogeneic hematopoietic stem cell transplantation. The study results were published in the journal Nature Communications in June 2026. Single-cell gene expression data indicated that the therapy disrupts protein metabolism within cancer cells and activated patient T-cell immune responses.

Multi-omics analysis identified a population of drug-resistant leukemic stem cells in patients who relapsed. These resistant cells use PLD1-mediated phospholipid metabolism to facilitate protein folding and promote cell survival. Laboratory testing confirmed that adding a PLD1 inhibitor suppresses the regenerative capacity of these resistant leukemic cells.

Anskar Leung Yu-hung, Chair Professor of Medicine at the university, said: "This study demonstrates the clinical potential of QUIZOM, enabling more high-risk patients to become eligible for HSCT, and provides a comprehensive understanding of its mechanisms. It provides a clear and promising therapeutic direction for overcoming drug resistance in leukemia. The research team has filed a patent application regarding the discovery of PLD1 inhibition in leukemia, aiming to improve treatment regimens for acute leukemia and ultimately benefitting more patients with haematological malignancies."

No independent assessment was available for this report.