A drug candidate that blocks BAX protected motor neurons in laboratory studies
Original source
An enantioselective covalent inhibitor of BAX confers cytoprotection in vivo. (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.
Researchers developed a covalent inhibitor of BAX, a protein involved in programmed cell death. The candidate reduced cell death and protected motor neurons made from stem cells donated by people with amyotrophic lateral sclerosis (ALS) and healthy donors. In mice with severe liver injury, it also reduced liver-cell death and improved survival. [S1]
Why this matters
The findings suggest that blocking BAX could be a way to investigate cell death in ALS and other conditions involving cell loss. However, the motor-neuron results were from laboratory-grown cells, and the survival results came from a mouse liver-injury model, so this does not yet change ALS treatment. [S1]
Limitations and context
This was preclinical research involving laboratory-grown human motor neurons and mice, not people with ALS. The study does not establish whether the candidate reaches or protects motor neurons safely in humans, or whether it improves ALS outcomes.