Experimental Cancer Drug Target Mistakenly Identified
Researchers found an oncology drug currently in clinical trials acts on an enzyme different from its intended target.
Updated on Oct. 13, 2026 in Cancer
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Scientists at The University of Sydney discovered that the experimental cancer drug zavondemstat, along with the related compound QC6352, targets the enzyme DHODH rather than KDM4 proteins. These findings impact the current clinical trials testing the medication for colorectal, pancreatic, and prostate cancers.
Why it matters
The study clarifies that these drugs function as DHODH inhibitors, an enzyme cancer cells use to produce DNA components, rather than the intended KDM4 inhibitors. Understanding this mechanism is critical for the appropriate use of these agents in future cancer treatment research.
Using patient-derived glioblastoma stem cells and tumor models, researchers demonstrated that zavondemstat and QC6352 primarily block the enzyme DHODH. While previous research used QC6352 to study KDM4 protein biology, these findings suggest that prior data relied on an incorrect mechanism.
The players
The University of Sydney
An Australian research institution that led the study investigating the mechanistic targets of cancer drug candidates.
The details
The researchers utilized genetic, mechanistic, and molecular experiments to evaluate how these drugs interact with cellular proteins. They found that instead of acting on KDM4, these agents inhibit DHODH, a vital enzyme used by cancer cells to synthesize essential DNA building blocks. The team also developed new compounds specifically designed to target KDM4 proteins without interfering with DHODH, which may help clarify KDM4's role in cancer biology moving forward.
Timeline
October 2026: Study findings were published in Nature Chemical Biology.
Health Landscape
This discovery updates our understanding of existing drug candidates currently undergoing clinical testing for various malignancies. It highlights the importance of identifying exact molecular targets to ensure that future oncological interventions are both effective and safe for patients.
Patients currently involved in clinical trials for zavondemstat should consult their oncology team regarding how these findings might relate to their individual treatment plan. It is important to discuss any questions about ongoing research with a doctor who can provide context based on the specific clinical trial protocol.
The takeaway
Understanding the precise biological mechanism of a drug is essential for determining its future role in cancer care. Always consult your physician for the most current information regarding participation in cancer clinical trials.
Further reading
For more on the current state of tumor research, visit the Cancer section.
Source note: This article includes information reported by Labonline.
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