An antibody fragment reduced ALS-related damage in cells and mice
Original source
Neutralization of pathogenic PC-OxPL by AAV-delivered scFv as a therapeutic strategy for amyotrophic lateral sclerosis. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human, Mouse
Population inferred from the title and abstract by Compass.
Related topics
Researchers identified a pattern of oxidized phosphatidylcholines in cerebrospinal fluid from people with sporadic amyotrophic lateral sclerosis (ALS). In laboratory motor neurons, these molecules caused disease-related changes and TDP-43 pathology. An adeno-associated virus-delivered antibody fragment neutralised the molecules and prevented motor-neuron loss and behavioural problems in a mouse model; delivery also reached the central nervous system in minipigs.
Why this matters
The findings suggest oxidized phosphatidylcholines may contribute to ALS biology and identify a possible treatment strategy. However, the work was conducted in cells and animals, so it does not currently change treatment for people with ALS.
Limitations and context
This is preclinical primary research. The reported effects were observed in laboratory cells, a cerebrospinal-fluid-transfer mouse model and minipigs, not in people receiving treatment. The study would need to establish safety and effectiveness in human clinical trials.