Understanding how axon transport is disrupted in a specific type of ALS
Mechanisms of disruption of axon transport of autophagic vesicles and lysosomes in C9orf72 ALS
['FUNDING_CAREER'] · BAYLOR COLLEGE OF MEDICINE · NIH-10872228
This study is looking into how problems with transporting important cellular materials in the body might lead to the nerve damage seen in people with a specific type of ALS, and it hopes to find new ways to help improve treatment for patients.
Quick facts
| Phase | ['FUNDING_CAREER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BAYLOR COLLEGE OF MEDICINE (nih funded) |
| Locations | 1 site (HOUSTON, UNITED STATES) |
| Trial ID | NIH-10872228 on ClinicalTrials.gov |
What this research studies
This research investigates the mechanisms behind the disruption of axon transport in patients with C9orf72-related Amyotrophic Lateral Sclerosis (ALS). The study focuses on how impaired transport of autophagic vesicles and lysosomes contributes to the degeneration of motor neurons. By using advanced models, including Drosophila and induced pluripotent stem cells from patients, the researchers aim to uncover the early pathogenic events that lead to ALS. This could help identify potential therapeutic targets to improve patient outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with C9orf72-related ALS or those with a family history of the disease.
Not a fit: Patients with other forms of ALS not related to C9orf72 may not benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that slow down or halt the progression of ALS in affected patients.
How similar studies have performed: Other research has shown promising results in understanding ALS mechanisms, but this specific approach focusing on C9orf72 is relatively novel.
Where this research is happening
HOUSTON, UNITED STATES
- BAYLOR COLLEGE OF MEDICINE — HOUSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BERTH, SARAH HATCH — BAYLOR COLLEGE OF MEDICINE
- Study coordinator: BERTH, SARAH HATCH
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