Targeting RNA polymerase II in AML with RAS pathway activation
RNA Polymerase II as a Therapeutic Target in Acute Myeloid Leukemia (AML) with RAS Signaling Activation
This project tests drugs that block RNA polymerase II to help people whose acute myeloid leukemia has RAS pathway activation and is resistant to FLT3 inhibitors.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11210635 on NIH RePORTER |
What this research studies
You would be part of work where researchers test existing RNA polymerase II pathway inhibitors in lab models and in leukemia samples from patients to see if these drugs can overcome resistance driven by RAS/MAPK signaling. The team will pinpoint which parts of the Mediator/RNA Pol II system are most important for resistance and then confirm the key genes that allow leukemia cells to survive. They will combine studies in cell and animal models with analyses of patient samples to guide which therapies look most promising. Successful findings would be used to design future clinical trials that patients with resistant AML might join.
Who could benefit from this research
Good fit: Ideal candidates are people with acute myeloid leukemia, especially those with FLT3 mutations and evidence of RAS/MAPK pathway activation or relapse after FLT3 inhibitor therapy.
Not a fit: Patients whose leukemia lacks RAS/MAPK activation or who cannot provide samples or participate in follow-up studies are less likely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this could lead to new treatments that overcome resistance to FLT3 inhibitors for patients with RAS-activated AML.
How similar studies have performed: Related approaches targeting transcriptional machinery have shown encouraging preclinical activity but remain early and are not yet standard clinical care.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Smith, Catherine Choy — University of California, San Francisco
- Study coordinator: Smith, Catherine Choy
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.