Researchers Corrected Mechanism of Cancer Drug
A new study revealed that the experimental drug zavondemstat targets DHODH enzymes rather than KDM4 proteins.
Updated on Oct. 13, 2026 in Cancer
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Researchers discovered that the experimental cancer drug zavondemstat and its relative QC6352 primarily block the enzyme DHODH. This finding corrects the previously held belief that these drugs primarily acted on KDM4 proteins.
Why it matters
The correction is critical as zavondemstat is already in clinical trials for various cancers based on an incorrect mechanism. Understanding its true target helps refine how these drugs are used and developed for cancer treatment.
The study analyzed drug interaction using patient-derived glioblastoma stem cells and tumor models. Researchers established that DHODH is the primary enzyme inhibited, which cancer cells utilize to produce essential DNA components.
The players
The University of Sydney
This Australian public research university led the study investigating the drug interactions of zavondemstat and QC6352.
The details
The research team conducted genetic, mechanistic, and molecular experiments to compare the effects of QC6352 against known KDM4 inhibitors. They have since developed new compounds capable of inhibiting KDM4 proteins without affecting the DHODH enzyme.
Timeline
The study findings were published in Nature Chemical Biology in October 2026.
The Big Picture
This discovery forces a reassessment of the ongoing clinical trials for zavondemstat by invalidating the mechanism that originally justified its use in human patients. It marks a significant shift in the development of cancer therapies by distinguishing between DHODH and KDM4 inhibition.
Patients currently enrolled in clinical trials for zavondemstat may see changes to study protocols as researchers adjust their understanding of the drug's activity. The findings suggest that future therapeutic efforts will focus on separating DHODH inhibitors from KDM4-specific treatments.
The takeaway
Scientific precision in identifying drug targets is essential to ensure that clinical trials are based on accurate biological data. Researchers are now prioritizing the creation of specialized inhibitors that target proteins without unintended interference.
Further reading
For more information on current therapeutic developments, visit the Cancer section.
Source note: This article includes information reported by Labonline.
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