Targeting harmful EZH2 activity in aggressive leukemia

Dissecting and targeting canonical and non-canonical oncogenic functions of EZH2 in caner

['FUNDING_R01'] · DUKE UNIVERSITY · NIH-11258526

This project develops a new drug-like molecule that removes the EZH2 protein to try to stop aggressive MLL1-rearranged leukemia.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorDUKE UNIVERSITY (nih funded)
Locations1 site (DURHAM, UNITED STATES)
Trial IDNIH-11258526 on ClinicalTrials.gov

What this research studies

MLL1-rearranged leukemias are aggressive blood cancers with poor outcomes, and researchers are looking for better treatments. The team found that EZH2 not only represses genes but also helps activate cancer-driving genes by binding partners like c-MYC. They are using PROTAC technology to create degraders (including a lead compound called MS177) that pull EZH2 and its cancer-promoting partners apart and remove them from cells. In lab studies MS177 breaks down both the usual EZH2 complexes and the non-standard interactions and works better than enzyme-blocking drugs, with the goal of moving toward treatments for patients.

Who could benefit from this research

Good fit: Patients whose leukemia carries an MLL1 (MLL1-rearranged/MLL fusion-positive) abnormality would be the most likely candidates for related clinical trials.

Not a fit: Patients without MLL1 rearrangements or whose disease is driven by other pathways are less likely to benefit from this EZH2-targeting approach.

Why it matters

Potential benefit: If successful, this approach could lead to more effective therapies for people with MLL1-rearranged leukemia.

How similar studies have performed: Traditional drugs that block EZH2's enzyme activity have shown limited benefit, while PROTAC degraders like MS177 are a newer strategy with promising preclinical results but no proven patient benefit yet.

Where this research is happening

DURHAM, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.