UF Health Cancer Institute researchers have created a novel drug that shows promise for the treatment of aggressive, treatment-resistant forms of acute myeloid leukemia (AML).
AML is a common, aggressive blood cancer with among the worst survival rates of all leukemias. Current therapies have low cure rates, highlighting the need to develop more effective drugs.
In the new study, a research team led by Weizhou Zhang, Ph.D., identified two proteins that are essential for the progression of AML. In AML patients with poor outcomes, the two proteins — IKKβ and NR4A1 — work together to sustain AML cells.
Next, working with UF medicinal chemists in the lab of Guangrong Zheng, Ph.D., the team designed, developed and tested a drug called a proteolysis-targeting chimera (PROTAC) capable of degrading both proteins.
PROTACs are an emerging type of small-molecule drug that have shown promise in cancer. They work by delivering a target protein to the cell’s own disposal machinery so it can be destroyed.
“We found the PROTAC killed AML cells efficiently both in human AML cells and mouse models,” said Zhang, co-leader of the UF Health Cancer Institute’s Mechanisms of Oncogenesis research program and a professor and vice chair of research in the Department of Department of Pathology, Immunology & Laboratory Medicine. “Ultimately, the drug could be used to treat patients with difficult to treat, aggressive types of AML.”
The study was presented at the American Association for Cancer Research (AACR) Annual Meeting 2026 in San Diego by Chandra Maharjan, Ph.D., a research assistant scientist in Zhang’s lab and the study’s first author.
Now, the researchers are focusing on developing better, more specific degraders of one of the proteins, IKKβ. The team has patented the original PROTAC, and they’re working on more specific degraders for long-term patenting and eventual clinical use.
UF Health Cancer Institute member Jatinder Lamba, Ph.D., is a co-author on the study, which highlights how the institute is at the forefront of innovative drug discovery.
