The U.S. Food and Drug Administration (FDA) has granted approval for a new oral medication, ziftomenib, aimed at treating patients with the most lethal form of blood cancer known as acute myeloid leukemia (AML). This breakthrough offers hope for those whose cancer has returned or is resistant to existing therapies, particularly patients with a mutation in the NPM1 gene.
Research originating from the University of Virginia School of Medicine has played a pivotal role in the development of this drug. The innovative work conducted by Jolanta Grembecka, Ph.D., and Tomasz Cierpicki, Ph.D., began in 2007 and has culminated in a treatment that could significantly improve survival rates for patients facing limited options. Grembecka and Cierpicki, who are now professors at the University of Michigan, expressed their satisfaction with the FDA’s decision, highlighting the importance of ziftomenib for patients without hope.
Significant Impact on AML Patients
According to the American Cancer Society, more than 22,000 individuals in the United States are diagnosed with AML each year, resulting in over 11,000 deaths. The disease is particularly aggressive and primarily affects those over the age of 68. Grembecka noted the dire need for effective treatments, stating, “Seeing our pioneering work on menin inhibitors evolve into an FDA-approved treatment for leukemia patients is extremely rewarding.”
The development journey of ziftomenib involved foundational discoveries made by Grembecka and Cierpicki, which were licensed to Kura Oncology in 2014. Following extensive research and collaboration, ziftomenib was brought to market under the brand name Komzifti, thanks to the joint efforts of Kura Oncology and pharmaceutical group Kyowa Kirin.
How Ziftomenib Works
Ziftomenib targets the interactions of a cellular protein known as menin, which is essential for the growth and survival of leukemia cells. This drug allows the immature blood cells to develop into healthy white blood cells, rather than turning cancerous. Cierpicki explained the complexity of developing menin inhibitors, saying, “We had to pioneer an entirely new area of research—producing the human protein, creating robust biochemical assays, conducting high-throughput screening, and solving the crystal structure of menin.”
The clinical trials for ziftomenib began in 2019, and the FDA expedited its review process due to the urgent need for new treatments in this area. The approval was celebrated by experts in the field, including Mark Esser, Ph.D., head of the Paul and Diane Manning Institute of Biotechnology at UVA, who stated, “It is immensely gratifying to see the work of Drs. Grembecka and Cierpicki have this amazing impact for patients.”
Ongoing research is now investigating the potential of ziftomenib in combination therapies, targeting not only leukemia but also solid tumors. Esser emphasized the importance of accelerating drug development, noting that many patients do not have the luxury of time.
With the approval of ziftomenib, there is renewed optimism in the fight against acute myeloid leukemia, illustrating the direct impact of innovative research on patient care and treatment options. The Manning Institute remains dedicated to advancing such critical research, aiming to bring new therapies to patients as swiftly as possible.
