Understanding how SHP2 and BCL2 inhibitors work in treating Acute Myeloid Leukemia
Official title: Investigating the mechanism of SHP2 and BCL2 Inhibition in Acute Myeloid Leukemia (AML)
Funded at University of California, San Francisco · NIH-10879172 · Data updated 2026-11-12
This study is looking at how two new types of medicines might help people with Acute Myeloid Leukemia (AML) who have certain genetic changes and haven't responded well to other treatments, with the goal of finding better ways to tailor therapies just for them.
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, 2023 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $482,061 |
| NIH project number | 5R01CA277031-02 |
| Project ID | NIH-10879172 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with Acute Myeloid Leukemia, especially those with FLT3 or KIT mutations.
✕Less likely to help
Patients with other types of leukemia or those who do not have the specific mutations targeted by this research may not benefit.
What this research studies
This research investigates the role of SHP2 and BCL2 inhibitors in treating Acute Myeloid Leukemia (AML), particularly focusing on patients with specific genetic mutations. The study aims to understand how these inhibitors can work together to improve treatment outcomes for patients who have developed resistance to existing therapies. By using advanced techniques like single-cell multiomic sequencing, researchers will identify biomarkers that predict how well patients respond to these treatments. This approach could lead to more personalized and effective therapies for AML patients.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatment options for patients with Acute Myeloid Leukemia, particularly those who have not responded to current therapies.
How similar studies have gone: Previous research has shown promising results using similar approaches with targeted therapies in other cancers, indicating potential for success in this area as well.
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.
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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.
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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.
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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.
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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.
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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.
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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.