Pan-RAS inhibitor approach for JMML in children and young adults
Official title: Investigating the Determinants of Response to Pan-RAS Inhibition in Juvenile Myelomonocytic Leukemia
Funded at University of California, San Francisco · NIH-11433355 · Data updated 2026-11-12
A study testing a Pan-RAS inhibitor to treat JMML, a pediatric leukemia, with potential implications for young patients and those with related RAS-driven diseases.
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 | August 1, 2026 |
| Project end | July 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $587,777 |
| NIH project number | 1R01CA316529-01 |
| Project ID | NIH-11433355 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with JMML or related RAS-driven leukemias, and potentially young adults with similar disease biology.
✕Less likely to help
Patients without RAS pathway–driven JMML or those who cannot tolerate targeted inhibitors may not benefit.
What this research studies
This project focuses on juvenile myelomonocytic leukemia (JMML), a rare pediatric cancer driven by RAS pathway mutations. Researchers will explore how a Pan-RAS inhibitor works with JMML cells and mouse models, building on prior MEK inhibitor findings. The work aims to understand why some patients respond less and how additional pathways like PI3K/AKT contribute to disease, with plans to test the inhibitor in systems that mimic human disease. The team will integrate molecular readouts, preclinical models, and translational insights to guide future clinical opportunities for children with JMML and potentially similar RAS-driven conditions.
Why it matters
Potential benefit: If successful, the approach could lead to more effective targeted treatments for JMML and related RAS-driven leukemias.
How similar studies have gone: Previous MEK inhibitor work showed clinical activity but lacked molecular responses, indicating room for improvement with broader RAS pathway targeting.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Stieglitz, Elliot — University of California, San Francisco
- Study coordinator: Stieglitz, Elliot
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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.