Trametinib for juvenile myelomonocytic leukemia (JMML)
PROJECT 3: A High Content Clinical Trial of the MEK inhibitor Trametinib in JMML
This gives the drug trametinib to children with JMML to try to block overactive MEK signaling and control their leukemia.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11181022 on NIH RePORTER |
What this research studies
Children with neurofibromatosis type 1 (NF1) and other Ras-pathway mutations can develop JMML, an aggressive childhood leukemia with high relapse after stem cell transplant. This project treats pediatric JMML patients with the MEK inhibitor trametinib to target the overactive Ras/Raf/MEK/ERK pathway that drives the disease. The team used genetically engineered mouse models that responded well to MEK inhibitors and opened a national phase to offer trametinib to affected children. Participants will be seen at specialized pediatric cancer centers for treatment, monitoring, and genetic testing as part of the protocol.
Who could benefit from this research
Good fit: Children (typically infants and young children up to about 11 years) diagnosed with JMML, especially those with NF1 or other Ras-pathway mutations and those with residual or relapsed disease, are the intended candidates.
Not a fit: Children whose leukemia is driven by unrelated genetic pathways or who cannot tolerate MEK inhibitors are less likely to benefit from this approach.
Why it matters
Potential benefit: If successful, trametinib could reduce disease activity, lower post-transplant relapse, and offer a targeted therapy option for children with JMML.
How similar studies have performed: Preclinical mouse models showed strong activity of MEK inhibitors, and MEK inhibitors have shown benefit in other RAS-driven conditions, but targeted therapy in JMML remains relatively new.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Shannon, Kevin M. — Indiana University Indianapolis
- Study coordinator: Shannon, Kevin M.
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 below.