Understanding how nerve cell proteins change in ALS
Identifying mechanisms of neurofilament regulation and turnover in amyotrophic lateral sclerosis
['FUNDING_R01'] · WASHINGTON UNIVERSITY · NIH-11180495
This project aims to understand how certain proteins in nerve cells, called neurofilaments, are regulated and cleared in people with ALS.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | WASHINGTON UNIVERSITY (nih funded) |
| Locations | 1 site (SAINT LOUIS, UNITED STATES) |
| Trial ID | NIH-11180495 on ClinicalTrials.gov |
What this research studies
Amyotrophic lateral sclerosis (ALS) is a severe condition that causes motor neuron loss, leading to muscle weakness and breathing difficulties. Researchers are focusing on specific proteins, neurofilaments (NfL and pNfH), which are important markers of nerve damage in ALS. We know that a new treatment for a specific type of ALS can lower these neurofilament levels, but we need to better understand how these proteins are regulated and removed from the body. This work uses advanced techniques to look at these proteins in detail, including finding new forms of NfL in spinal fluid.
Who could benefit from this research
Good fit: This foundational research is for patients with Amyotrophic Lateral Sclerosis (ALS) who are interested in how biomarkers are used to understand and potentially treat their condition.
Not a fit: Patients not diagnosed with Amyotrophic Lateral Sclerosis (ALS) would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to better ways to track ALS progression and develop more effective treatments by targeting the specific ways these nerve cell proteins are managed in the body.
How similar studies have performed: While a recent therapy for a specific type of ALS has shown success in lowering neurofilament levels, this project aims to uncover the underlying mechanisms, which is a novel area of focus.
Where this research is happening
SAINT LOUIS, UNITED STATES
- WASHINGTON UNIVERSITY — SAINT LOUIS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LY, CINDY V — WASHINGTON UNIVERSITY
- Study coordinator: LY, CINDY V
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.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease