Understanding how plexiform neurofibromas become malignant nerve sheath tumors and finding better treatments
PROJECT 1: From Neurofibroma to MPNST: Models, Biology and Translation to Clinic
['FUNDING_OTHER'] · INDIANA UNIVERSITY INDIANAPOLIS · NIH-11181016
Testing drug combinations and lab models to help adults with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas or are at risk for malignant peripheral nerve sheath tumors (MPNST).
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | INDIANA UNIVERSITY INDIANAPOLIS (nih funded) |
| Locations | 1 site (INDIANAPOLIS, UNITED STATES) |
| Trial ID | NIH-11181016 on ClinicalTrials.gov |
What this research studies
This project uses genetic studies from patient tumors together with genetically engineered mouse models to learn how benign plexiform neurofibromas (PNF) progress to atypical neurofibromas (ANF) and malignant peripheral nerve sheath tumors (MPNST). Researchers are building on earlier clinical successes with the drugs selumetinib and cabozantinib and are testing whether combining these therapies works better. Laboratory experiments and preclinical drug testing will guide which combinations move into clinical trials. The goal is to translate laboratory findings into treatments that reduce tumor burden and lower the chance of developing MPNST in adults with NF1.
Who could benefit from this research
Good fit: Adults (21+) with neurofibromatosis type 1 who have plexiform neurofibromas, atypical neurofibromas, or are at elevated risk for MPNST are the most likely candidates for related trials.
Not a fit: People without NF1, those who do not have plexiform or atypical neurofibromas, and likely children under age 21 would not be expected to benefit from this project.
Why it matters
Potential benefit: If successful, this work could lead to drug combinations that shrink plexiform neurofibromas, ease symptoms, and reduce the risk of progression to deadly MPNST.
How similar studies have performed: Earlier clinical trials showed that selumetinib and cabozantinib can produce meaningful shrinkage of many plexiform neurofibromas, so this project builds on those clinical signals.
Where this research is happening
INDIANAPOLIS, UNITED STATES
- INDIANA UNIVERSITY INDIANAPOLIS — INDIANAPOLIS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CLAPP, DAVID W — INDIANA UNIVERSITY INDIANAPOLIS
- Study coordinator: CLAPP, DAVID W
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.