New treatments for lymphatic malformations in children
Official title: Next Generation Treatments for Lymphatic Malformations
Funded at Arkansas Children's Hospital Res Inst · NIH-11295428 · Data updated 2026-11-12
Targeted, personalized drug approaches and lab testing aim to find safer, more effective treatments for children with cystic lymphatic malformations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Arkansas Children's Hospital Res Inst NIH-funded |
| Lab location | 1 site (Little Rock, United States) |
| Project start | March 15, 2024 |
| Project end | February 28, 2029 |
| Funding year | 2026 |
| Award (this year) | $627,552 |
| NIH project number | 5R01HL173107-03 |
| Project ID | NIH-11295428 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children from newborns through 11 years old with cystic lymphatic malformations—especially those with neck masses, recurrent infections, or known PIK3CA pathway changes—are the most likely candidates.
✕Less likely to help
Adults or patients whose malformations do not involve PI3K/PIK3CA pathway abnormalities may not receive direct benefit from this project.
What this research studies
Researchers will study cells taken from children's cystic lymphatic malformations using gene, microRNA, and protein analyses to understand what drives growth and spread. They will use those lab findings to test targeted drug combinations and other therapies in cell and laboratory models to find options that work better than current treatments. The team is focusing on mutations like PIK3CA and pathways that make some patients resistant to existing PI3K/mTOR drugs. The goal is to match treatments to each child’s lesion to reduce infections, inflammation, and the number of surgeries needed.
Why it matters
Potential benefit: Could lead to personalized medicines that shrink malformations, reduce infections and inflammation, and lessen the need for repeated surgeries and harsh drugs.
How similar studies have gone: Some patients have experienced size reductions with PI3K/mTOR inhibitors, but many do not respond and none are cured, so this work builds on partial successes to find better, individualized options.
Where this research is happening
-
Arkansas Children's Hospital Res Inst
Little Rock, United States Active
Researchers
- Principal investigator: Strub, Graham M — Arkansas Children's Hospital Res Inst
- Study coordinator: Strub, Graham 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.