SHP2 and BCL2 inhibitors together for AML growth and resistance
Official title: Investigating the mechanism of SHP2 and BCL2 Inhibition in Acute Myeloid Leukemia (AML)
Funded at University of California, San Francisco · NIH-11353878 · Data updated 2026-11-12
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.
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 | 2026 |
| Award (this year) | $528,493 |
| NIH project number | 5R01CA277031-04 |
| Project ID | NIH-11353878 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with AML, particularly those with FLT3 or KIT mutations.
✕Less likely to help
Patients without these molecular features or those who cannot tolerate the drug combination may not benefit.
What this research studies
This project tests a drug combination aimed at AML patients whose leukemia cells rely on RTK signaling. Researchers will use a new SHP2 inhibitor together with venetoclax to see if cancer cells shrink or die more effectively than with either treatment alone. They will study how the drugs affect signaling pathways in patient-derived leukemia cells, including single-cell analyses to spot markers of response and resistance. The work includes lab experiments and analyses intended to guide future trials in people with AML. If successful, this approach could extend remission and improve outcomes for patients with specific genetic AML.
Why it matters
Potential benefit: If successful, patients with AML could experience longer responses and better control of the disease through a new drug combination.
How similar studies have gone: Early studies suggest SHP2 inhibitors plus BCL2 inhibitors can be effective in some RTK-driven leukemias, but this combination in AML is still being explored.
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.
-
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.
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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.
-
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.
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Making genetic risk predictions more accurate for adults with acute myeloid leukemia
This project is developing better genetic-based risk categories to help guide treatment decisions for adults with acute myeloid leukemia.
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.