New treatment strategies for children with juvenile myelomonocytic leukemia.
Implementation of an interventional, risk-adapted clinical trial for children with newly diagnosed juvenile myelomonocytic leukemia.
This study is looking for ways to help children with juvenile myelomonocytic leukemia (JMML) get better treatment by using gentler options based on their unique genetic makeup, so they can avoid tough side effects from stronger therapies, while testing a mix of existing medicines to find the best approach for each child.
Quick facts
| Grant type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-10919211 on NIH RePORTER |
What this research studies
This research focuses on improving treatment outcomes for children diagnosed with juvenile myelomonocytic leukemia (JMML), a serious blood disorder. The study aims to identify patients who may benefit from less intensive treatments based on specific genetic markers, potentially avoiding the harsh side effects of standard therapies like stem cell transplantation. By using a combination of existing medications, azacitidine and trametinib, the research seeks to enhance treatment effectiveness. Patients will be monitored closely to determine the best risk-adapted therapy for their individual conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are children aged 0-11 years who have recently been diagnosed with juvenile myelomonocytic leukemia.
Not a fit: Patients with JMML who are older than 11 years or those who have already undergone extensive treatment may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and less harmful treatment options for children with JMML.
How similar studies have performed: Previous research has shown promise with similar approaches, particularly in identifying biomarkers that predict treatment outcomes in JMML.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Stieglitz, Elliot — University of California, San Francisco
- Study coordinator: Stieglitz, Elliot
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.