New models and insights for desmoplastic small round cell tumor
Desmoplastic small round cell tumor: harnessing new insights and new models
This project is creating the first genetically engineered mouse models and analyzing tumor samples to help find better treatments for adolescents and young adults with desmoplastic small round cell tumor.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Sloan-Kettering Inst Can Research NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11180183 on NIH RePORTER |
What this research studies
From my perspective as someone affected by DSRCT, the team at Memorial Sloan Kettering will build the first mouse models that carry the EWSR1-WT1 fusion found in patients and will also test how loss of genes like p53 and ARID1A changes tumor behavior. They will use genomic editing tools (Cas9 and guide RNAs) to recreate the chromosome change that drives DSRCT and grow tumors in mice to study how they form and progress. The investigators will also profile abnormal 'neotranscripts' and other molecular changes in tumors to find vulnerabilities. The goal is to pinpoint biological targets that could guide new drug development or future clinical trials.
Who could benefit from this research
Good fit: Ideal participants for any patient-facing parts of this work would be people diagnosed with DSRCT—especially adolescents and young adults—or patients willing to provide tumor samples for research.
Not a fit: People without DSRCT or patients needing immediate clinical treatment are unlikely to get direct benefit from this preclinical and model-development project.
Why it matters
Potential benefit: If successful, this work could reveal the biological drivers of DSRCT and point to new targeted treatments and clinical trials for adolescents and young adults.
How similar studies have performed: Creating engineered mouse models and using CRISPR-driven chromosomal engineering has worked for other sarcomas, but this is a novel and first-in-field effort specifically for DSRCT.
Where this research is happening
New York, United States
- Sloan-Kettering Inst Can Research — New York, United States (Active)
Researchers
- Principal investigator: Ladanyi, Marc — Sloan-Kettering Inst Can Research
- Study coordinator: Ladanyi, Marc
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.