New Treatment Approaches for Acute Myeloid Leukemia with RAS Activation
Official title: RNA Polymerase II as a Therapeutic Target in Acute Myeloid Leukemia (AML) with RAS Signaling Activation
Funded at University of California, San Francisco · NIH-11122335 · Data updated 2026-11-12
This project explores new ways to overcome treatment resistance in acute myeloid leukemia (AML) patients whose cancer has a specific genetic change called RAS activation.
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 start | July 1, 2022 |
| Project end | June 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $547,180 |
| NIH project number | 5R01CA266422-04 |
| Project ID | NIH-11122335 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is most relevant for patients with acute myeloid leukemia (AML) who have FLT3 mutations and have developed resistance to FLT3 inhibitors, particularly those with RAS pathway activation.
✕Less likely to help
Patients whose AML does not involve FLT3 mutations or RAS pathway activation may not directly benefit from this specific treatment approach.
What this research studies
Many patients with acute myeloid leukemia (AML) have a specific gene change called FLT3, and while treatments exist, the cancer often becomes resistant, especially when another pathway called RAS is also active. This research aims to find new ways to make current AML treatments more effective by focusing on a pathway called RNA Polymerase II. We believe that by targeting this pathway, we can help overcome the resistance that develops in AML cells with RAS activation. Our goal is to identify the most important parts of this pathway and test potential new therapies that could make a difference for patients.
Why it matters
Potential benefit: If successful, this work could lead to new treatment options for AML patients who have developed resistance to current therapies due to RAS pathway activation.
How similar studies have gone: This approach builds on preliminary findings that suggest targeting the RNA Polymerase II pathway could help overcome treatment resistance, representing a novel strategy in this specific context.
Where this research is happening
-
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
Related research
Other NIH-funded projects on the same topics.
-
Acute Myeloid Leukemia: Improving Targeted Therapies through Genetically Defined PDX Models
Researchers are using patient-like leukemia models to develop therapies that target specific genetic features of AML to improve survival.
-
A broader genomic risk picture to guide AML treatment
AIM to refine how genetic information guides treatment decisions for adults with acute myeloid leukemia by looking at how inherited and recurrent mutations affect risk.
-
RNA Polymerase II as a Therapeutic Target in AML with RAS Activation
This project explores a new way to treat a common type of blood cancer (AML) by blocking a key machinery (RNA Polymerase II) that cancer cells use when the RAS signaling pathway is overactive.
-
SHP2 and BCL2 inhibitors together for AML growth and resistance
A patient-focused look at whether combining SHP2 inhibitors with a BCL2 inhibitor can slow or stop AML, especially in people whose disease has FLT3 or KIT mutations.
-
Targeting RNA polymerase II in AML with RAS pathway 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.
-
New treatment approach for myelodysplastic syndrome and acute myeloid leukemia
This project aims to develop a new antibody-based therapy to help people with MDS or AML, especially older or frail patients who cannot tolerate high-dose chemotherapy.
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.