Innovative Dual-Drug Approach Shows Potential in Treating Aggressive Leukemia

Researchers at Peter Mac have identified a promising new combination therapy for certain subtypes of acute myeloid leukemia (AML), offering hope for improved treatment outcomes. The therapy involves using a well-established Menin inhibitor, revumenib, alongside a novel drug called PF-9363, which is currently in clinical development. PF-9363 functions by blocking enzymes KAT6 and KAT7 that are crucial for cancer cell growth and survival, thereby enhancing the efficacy of Menin inhibitors.

The combination has demonstrated in preclinical studies that it significantly halts the proliferation of leukemia cells and induces their death, potentially overcoming resistance to existing treatments. Dr. Shellaina Gordon from Monash University explained that when used together, these drugs rapidly suppress leukemia cell multiplication, offering a more targeted approach to therapy.

Professor Mark Dawson, leading the research, highlighted that Menin inhibitors have already revolutionized AML treatment by turning off cancer-causing genes. However, resistance often develops over time. The addition of PF-9363 could address this by enhancing anti-leukemic effects and extending treatment durability.

The study, published in Cancer Discovery, shows that combining these drugs in animal models increases survival rates and counters resistance mechanisms. This advancement paves the way for future clinical trials involving AML patients, aiming to provide more effective and lasting treatment options. Collaboration with researchers from Dana-Farber Cancer Institute and Hannover Medical School underscores the significance of this breakthrough.

Overall, this dual-drug approach offers a promising new avenue for tackling aggressive leukemia, bringing us closer to more effective precision therapies for AML patients.