The United States Food and Drug Administration (FDA) has approved a new oral medication, ziftomenib, designed for patients suffering from the most lethal form of blood cancer, acute myeloid leukemia (AML)NPM1 gene mutations who experience either recurrent or treatment-resistant forms of the disease. The approval marks a significant advancement for individuals facing limited treatment options.
The development of ziftomenib originated from research conducted at the University of Virginia School of Medicine, where two researchers, Jolanta Grembecka, Ph.D., and Tomasz Cierpicki, Ph.D., began their work in 2007. Both scientists, who have since moved to the University of Michigan as professors in the Department of Pathology, collaborated closely with their former mentor, John Bushweller, Ph.D. This collaboration has led to a breakthrough that could transform the lives of many patients.
Significance of Ziftomenib for AML Patients
Each year, more than 22,000 Americans are diagnosed with AML, a particularly aggressive blood cancer primarily affecting individuals over the age of 68. According to the American Cancer Society, over 11,000 people succumb to the illness annually, making it one of the deadliest forms of blood cancer.
Dr. Grembecka expressed her satisfaction with the FDA’s endorsement, stating, “Seeing our pioneering work on menin inhibitors evolve into an FDA-approved treatment for leukemia patients is extremely rewarding. It is remarkable to witness the direct impact of our research.”
Ziftomenib works by disrupting the interactions of a cellular protein known as menin, which plays a crucial role in the growth and survival of leukemia cells. By inhibiting this protein, the drug allows cells to mature into healthy white blood cells rather than becoming cancerous.
Collaboration and Future Prospects
The journey of ziftomenib began with foundational discoveries made by Grembecka and Cierpicki, which were licensed to Kura Oncology in 2014. The drug has been brought to market under the brand name Komzifti through a partnership between Kura and the pharmaceutical group Kyowa Kirin. Clinical trials commenced in 2019, with the FDA granting ziftomenib a priority review due to the urgent need for new treatment options.
Mark Esser, Ph.D., head and chief scientific officer at UVA’s Paul and Diane Manning Institute of Biotechnology, praised the achievement, stating, “Ziftomenib is a long-awaited and desperately needed new option for patients for whom other treatments have failed.” He emphasized the institute’s commitment to accelerating research that leads to new treatments for complex diseases.
Further clinical trials are currently underway to explore the potential of ziftomenib in combination with other therapies to target both leukemia and solid tumors. This ongoing research underscores the dedication of the scientific community to finding effective solutions for patients in need.
Dr. Bushweller highlighted the significance of this achievement, saying, “Their success portends a future with more such targeted agents that dramatically improve cancer care.” As the medical community continues to innovate, ziftomenib represents a beacon of hope for many battling acute myeloid leukemia.
