Blocking stress-response pathways reduced disease-related damage in laboratory models of C9orf72 ALS and FTD
Original source
Targeting the integrated stress response or Ataxin-2 alleviates neurodegeneration in PolyGR models of C9orf72 associated frontotemporal dementia and amyotrophic lateral sclerosis. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human, Animal
Related topics
Blocking the integrated stress response or reducing Ataxin-2 activity reduced motor problems and poly(GR)-related toxicity in laboratory models of C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The work identified poly(GR) as an activator of this stress response in fruit-fly models and found effects in mouse primary neurons.
Why this matters
The findings point to the integrated stress response and Ataxin-2 as possible targets for future treatment research in C9orf72-associated disease. They were demonstrated in cells and animals, so they do not currently change treatment for people with ALS or FTD.
Limitations and context
This was preclinical research using Drosophila models and mouse primary neurons, not a clinical trial in people. The findings still need to be tested in additional disease models and human studies to determine whether targeting these pathways is safe and effective. The source is a primary research article, not evidence of a treatment benefit in patients.