Understanding and treating Langerhans cell histiocytosis
Defining Pathogenic Mechanisms and Therapeutic Opportunities for Langerhans Cell Histiocytosis
['FUNDING_R01'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11253935
This project aims to understand what drives Langerhans cell histiocytosis and develop new treatments for people with LCH.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11253935 on ClinicalTrials.gov |
What this research studies
You would be joining work that looks at the actual immune cells in LCH lesions, especially those with MAPK pathway changes like BRAF V600E, to see how the cell type, patient age, and the tissue around the cells shape the disease. The team will study whether a cellular aging program (senescence) helps drive lesions and affects outcomes. They will use patient biopsy samples and laboratory models to explore how these factors cause disease. From that understanding, they will try targeted therapies that block MAPK signaling or change the local tissue environment to prevent or shrink lesions.
Who could benefit from this research
Good fit: Adults diagnosed with Langerhans cell histiocytosis, particularly those with biopsy-confirmed MAPK pathway mutations (for example BRAFV600E), would be the most relevant candidates to engage with this work.
Not a fit: People without LCH or whose disease does not involve MAPK pathway mutations (or unrelated conditions) are unlikely to receive direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could lead to more precise, targeted treatments that reduce lesion growth, limit disease spread, and improve quality of life for people with LCH.
How similar studies have performed: Targeted MAPK/BRAF inhibitors have shown promise in LCH patients with BRAFV600E, but they are not universally curative and this project aims to build on and extend those findings.
Where this research is happening
NEW YORK, UNITED STATES
- ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MERAD, MIRIAM — ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
- Study coordinator: MERAD, MIRIAM
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.