A new medication developed from years of research at the University of Virginia (UVA) is giving fresh hope to patients with one of the deadliest types of blood cancer—acute myeloid leukemia, or AML.
The U.S. Food and Drug Administration (FDA) has approved the drug ziftomenib for patients whose cancer has come back or no longer responds to treatment and who have a mutation in the NPM1 gene.
This is a major breakthrough for people with very few options left. Ziftomenib is taken as a pill once a day and offers a potential lifeline to patients who previously had no effective treatments available.
The journey of ziftomenib began in 2007 when scientists Jolanta Grembecka, Ph.D., and Tomasz Cierpicki, Ph.D., were working at UVA in the Department of Molecular Physiology and Biological Physics.
They collaborated with their former mentor, John Bushweller, Ph.D., on early discoveries that laid the groundwork for the new drug. In 2009, Grembecka and Cierpicki moved to the University of Michigan, where they continued their research as professors in the Department of Pathology.
Their work focused on blocking a protein called menin, which helps leukemia cells grow and survive. Normally, this protein interacts with other proteins in the cell to keep cancer going. By stopping menin from doing its job, ziftomenib allows blood cells to grow normally instead of turning into cancer cells.
“It’s a wonderful achievement,” said Dr. Mark Esser, chief scientific officer at UVA’s Paul and Diane Manning Institute of Biotechnology. “This is exactly the kind of important research the Manning Institute was created to support. We want to speed up the development of treatments for the most difficult diseases.”
AML is an aggressive blood cancer that mostly affects older adults—especially those over 68. About 22,000 people in the U.S. are diagnosed each year, and around half of them die from the disease. AML accounts for roughly one-third of all blood cancer cases.
Dr. Grembecka described the impact of their discovery: “Acute myeloid leukemia is a very aggressive blood cancer with poor clinical outcomes. Seeing our work lead to an FDA-approved treatment is extremely rewarding. It’s something we once only dreamed of.”
The research from UVA and Michigan was licensed to Kura Oncology in 2014. Kura, along with pharmaceutical company Kyowa Kirin, developed the drug and released it under the brand name Komzifti. Clinical trials for ziftomenib started in 2019, and the FDA gave the drug a priority review because of the urgent need for new treatment options.
Dr. Cierpicki explained that their work involved building a whole new field of study. “We had to produce the human protein, design tests, run massive screenings, and even solve the crystal structure of menin. At that time, many in the pharmaceutical world didn’t believe we could successfully target protein-protein interactions. But we did.”
Dr. Bushweller, now at UVA Comprehensive Cancer Center, praised their success. “It’s incredibly rewarding to see their research lead to a drug that helps patients. This opens the door for more targeted treatments in the future that can transform cancer care.”
Ongoing clinical trials are also studying how ziftomenib might work when combined with other treatments, possibly expanding its use to solid tumors as well.
As Dr. Esser pointed out, “Drug development is often slow, but patients don’t have time to wait. That’s why UVA’s Manning Institute is focused on finding and delivering life-saving treatments faster.”
The approval of ziftomenib shows what can happen when scientists, universities, and pharmaceutical companies work together. For people with AML who previously had no good options, this new treatment could mean more time, better health, and renewed hope.
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