Combination therapies for malignant peripheral nerve sheath tumors (MPNST)
Preclinical-Clinical Trials Collaboration to effectively advance new combination therapies for malignant peripheral nerve sheath tumors
Trying new drug combinations to treat MPNST in people with neurofibromatosis type 1 and others who have these tumors.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11123275 on NIH RePORTER |
What this research studies
If you or your child has MPNST, this project aims to move new combination drug treatments from the laboratory into patients more quickly. Researchers will use what they learn about tumor-driving mechanisms and immune responses to design combinations, test them first in a validated MPNST mouse model, and then open clinical trials. The clinical plan emphasizes testing more than one combination within the same trial so promising regimens can be identified faster. Work will be coordinated across Brigham and Women's Hospital, the NIH Clinical Center, and Children's National to enroll patients and bring results back to the lab.
Who could benefit from this research
Good fit: Ideal candidates are people (including children with NF1) who have confirmed MPNST and meet trial safety and measurable-disease criteria for the combination therapy trials.
Not a fit: People without MPNST, those whose tumors are fully removable by surgery, or individuals who do not meet trial safety or eligibility criteria (for example due to poor organ function or pregnancy) are unlikely to benefit from these trials.
Why it matters
Potential benefit: If successful, this work could produce new combination therapies that slow tumor growth, shrink inoperable tumors, and improve survival for people with MPNST.
How similar studies have performed: Preclinical studies support combination approaches for MPNST but clinical successes to date have been limited, so this approach is promising but still experimental.
Where this research is happening
Boston, United States
- Brigham and Women's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Cichowski, Karen M — Brigham and Women's Hospital
- Study coordinator: Cichowski, Karen 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 below.