Combination therapies for atypical neurofibromas in neurofibromatosis type 1
Preclinical-clinical trials collaboration to effectively advance new combination therapies for atypical neurofibroma in neurofibromatosis type 1
New drug combinations aim to shrink or stop atypical neurofibromas in people with neurofibromatosis type 1.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11294217 on NIH RePORTER |
What this research studies
This project brings together lab research and early human trials to find drug combinations that target atypical neurofibromas (ANF), the precursors to dangerous malignant peripheral nerve sheath tumors (MPNST). Researchers will study tumor samples and animal or cell models to identify promising combinations that overcome resistance to single drugs like MEK inhibitors. Promising regimens will move into early-phase clinical testing at participating centers, using scans, biopsies, and molecular tests to track tumor changes and biomarkers. The goal is to find safe combination treatments that reduce tumor growth or prevent progression to MPNST.
Who could benefit from this research
Good fit: People with neurofibromatosis type 1 who have atypical neurofibromas or high-risk plexiform neurofibromas with measurable lesions and who can undergo imaging and biopsies may be eligible.
Not a fit: People without NF1, those with unrelated cancers, or patients with fully developed, unresectable MPNST at presentation may not benefit from this preventive-focused research.
Why it matters
Potential benefit: If successful, the work could produce treatments that shrink ANF or block their progression to deadly MPNST, reducing the need for disfiguring surgery and improving survival.
How similar studies have performed: MEK inhibitors (like selumetinib) have worked well for many plexiform neurofibromas, but ANF have shown limited response so combination therapy for ANF is a newer and less-tested approach.
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.