Understanding the varied effects of neurofibromatosis type 1 to improve treatment and risk assessment
Official title: Modeling neurofibromatosis-1 disease heterogeneity to optimize risk assessment and treatment
Funded at Washington University · NIH-11134859 · Data updated 2026-11-12
This study is looking at neurofibromatosis type 1 (NF1) to understand how different genes and other factors affect the symptoms people experience, so we can create better, personalized treatments just for you.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | May 2, 2025 |
| Project end | April 30, 2033 |
| Funding year | 2025 |
| Award (this year) | $877,001 |
| NIH project number | 1R35NS142919-01 |
| Project ID | NIH-11134859 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with neurofibromatosis type 1, particularly children and young adults experiencing neurological problems or tumors.
✕Less likely to help
Patients with other unrelated genetic conditions or those who do not have neurofibromatosis type 1 may not benefit from this research.
What this research studies
This research focuses on neurofibromatosis type 1 (NF1), a genetic condition that leads to a wide range of neurological issues and tumors. The project aims to identify the genetic and molecular factors that contribute to the diverse symptoms experienced by patients with NF1. By analyzing how these factors interact with both internal and external influences, the research seeks to develop personalized treatment strategies that are more effective for each individual. Patients may benefit from improved risk assessments and tailored therapies based on their unique disease characteristics.
Why it matters
Potential benefit: If successful, this research could lead to more effective and personalized treatment options for patients with neurofibromatosis type 1.
How similar studies have gone: Previous research has shown promise in understanding genetic conditions through personalized approaches, suggesting potential for success in this area as well.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Gutmann, David H — Washington University
- Study coordinator: Gutmann, David H
Related research
Other NIH-funded projects on the same topics.
-
Understanding variations in Neurofibromatosis type 1 to improve risk and care
This project uses genetic, cellular, and computer models to learn why Neurofibromatosis type 1 causes different problems in different people and to guide better care for children and adults with NF1.
-
Online program providing personalized NF1 care letters plus videos or peer navigator support for adults with low health literacy
This study will test whether giving adults with NF1 and low health literacy personalized care letters plus either short, plain-language videos or a call with a trained peer navigator helps them better understand NF1 symptoms and care before their primary care visit.
-
Understanding genetic causes of neuroendocrine tumors in Mexican patients
This study is trying to find out the genetic changes that cause neuroendocrine tumors in Mexican patients to help develop better treatments.
-
Combination therapy to shrink plexiform neurofibromas in NF1 patients
This trial tests whether the drugs cabozantinib and selumetinib can be safely given together to see if they help shrink progressive or painful plexiform neurofibromas in adolescents and adults with NF1.
-
Study on the use of Koselugo for treating Neurofibromatosis 1
This study is testing how well Koselugo works and how safe it is for people with Neurofibromatosis 1 in everyday treatment settings in South Korea.
-
Monitoring for cancer risk in neurofibromatosis type 1 tumors
This study is trying to find out which harmless tumors in people with neurofibromatosis type 1 might turn into cancer so that doctors can better monitor patients at risk.
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.