SHP2 and BCL2 Inhibition in Acute Myeloid Leukemia (AML)
Official title: Investigating the mechanism of SHP2 and BCL2 Inhibition in Acute Myeloid Leukemia (AML)
Funded at University of California, San Francisco · NIH-11122207 · Data updated 2026-11-12
This research explores new ways to treat acute myeloid leukemia (AML) by targeting specific proteins that help cancer cells survive.
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 | 2025 |
| Award (this year) | $456,661 |
| NIH project number | 5R01CA277031-03 |
| Project ID | NIH-11122207 on NIH RePORTER |
Who could benefit from this research
✓Could help
This research is relevant for patients with acute myeloid leukemia (AML), particularly those with FLT3 or KIT mutations, or those who have experienced relapse or resistance to current treatments.
✕Less likely to help
Patients without acute myeloid leukemia or those whose disease does not involve the specific genetic mutations or resistance mechanisms targeted by this research may not directly benefit from this particular approach.
What this research studies
Many patients with acute myeloid leukemia (AML) have mutations that lead to high relapse rates, and current treatments often face resistance. This project looks at how two proteins, SHP2 and BCL2, contribute to the survival of AML cells, especially those with specific mutations like FLT3 and KIT. Researchers are testing new SHP2 inhibitors in combination with an existing BCL2 inhibitor (venetoclax) to see if this combination can effectively stop cancer growth. They will also look for markers that predict who might respond best to this treatment and why some cells might become resistant. The aim is to find better treatment options for patients whose AML has relapsed or is resistant to current therapies.
Why it matters
Potential benefit: If successful, this work could lead to new, more effective combination therapies for patients with acute myeloid leukemia, especially those who have relapsed or have specific genetic mutations.
How similar studies have gone: While FLT3 inhibitors and BCL2 inhibitors like venetoclax are clinically active, resistance remains a challenge, making the combination of SHP2 and BCL2 inhibition a novel approach to overcome this resistance.
Where this research is happening
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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.