Understanding how nerve cell proteins change in ALS
Official title: Identifying mechanisms of neurofilament regulation and turnover in amyotrophic lateral sclerosis
Funded at Washington University · NIH-11180495 · Data updated 2026-11-12
This project aims to understand how certain proteins in nerve cells, called neurofilaments, are regulated and cleared in people with ALS.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | September 1, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $547,960 |
| NIH project number | 5R01NS138499-02 |
| Project ID | NIH-11180495 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
Patients not diagnosed with Amyotrophic Lateral Sclerosis (ALS) would not directly benefit from this specific research.
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.
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 gone: 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
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Ly, Cindy V — Washington University
- Study coordinator: Ly, Cindy V
Related research
Other NIH-funded projects on the same topics.
-
Investigating how DNA damage and repair mechanisms are altered in ALS and frontotemporal dementia.
This study is looking at how problems with DNA repair might contribute to conditions like ALS and frontotemporal dementia, and it’s testing ways to fix these issues in cells from patients to help develop new treatments.
-
Understanding how work exposures and genes influence ALS risk
A study is building a long-term group of people at higher risk for ALS to see how their jobs, exposures at work, and genes together affect if and when the disease might develop.
-
NUZ-001: a treatment option for ALS to protect nerve cells
A small molecule called NUZ-001 is being tested to help people with ALS by improving cell systems that keep proteins balanced and prevent harmful protein clumps.
-
Alpha5 integrin's role in ALS: how a cell switch worsens disease
Aims to understand how a specific protein called alpha5 integrin in immune cells affects ALS progression and whether blocking it can help extend life.
-
C9orf72 ALS/FTD Prevent: prepping for future treatment
This grant aims to get ready for a preventive treatment for people who carry the C9orf72 genetic change that can lead to ALS or frontotemporal dementia.
-
Air pollutants and ALS: mapping environmental links to motor neuron disease
This NIH grant explores whether different air pollutants are linked to the development of ALS and uses a large U.S. data set to look for environmental risk factors.
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.