Blocking a leukemia protein (PRC1) to make cancer cells mature
Targeting PRC1 in leukemia
New small-molecule drugs are being made to block a protein complex called PRC1 so chemotherapy-resistant leukemia stem cells in acute leukemia stop self-renewing and start to mature.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project ID | NIH-11161448 on NIH RePORTER |
What this research studies
Researchers are designing first-in-class small molecules that bind and inhibit the RING1B-BMI1 enzyme within the PRC1 complex, a key regulator of leukemia stem cell self-renewal. They used fragment-based screening and medicinal chemistry to create compounds that reduce the repressive H2Aub mark and push leukemic blasts toward differentiation. The project tests these compounds in lab-grown human leukemia cells and animal models to check whether they stop growth and lower relapse risk. If the molecules work in preclinical tests, the team would aim to move toward safety studies and early clinical trials.
Who could benefit from this research
Good fit: People with acute myeloid leukemia (AML) or related acute leukemias, especially cancers driven by HOXA9 or with high BMI1/PRC1 activity, would be the most relevant candidates for future trials.
Not a fit: Patients with non-acute blood disorders or leukemias not driven by PRC1/BMI1 biology, and anyone needing immediate standard treatment, are unlikely to benefit now because the work is preclinical.
Why it matters
Potential benefit: Could produce a new type of drug that eliminates chemo-resistant leukemia stem cells and lowers relapse rates in acute leukemia.
How similar studies have performed: Genetic studies show that removing PRC1 components can force leukemia cells to differentiate, but direct small-molecule inhibition of the RING1B-BMI1 ligase is largely novel and unproven in patients.
Where this research is happening
Ann Arbor, United States
- University of Michigan at Ann Arbor — Ann Arbor, United States (Active)
Researchers
- Principal investigator: Cierpicki, Tomasz — University of Michigan at Ann Arbor
- Study coordinator: Cierpicki, Tomasz
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.