Scientists at the University of Southern California have come up with a new approach to study disease mechanisms of amyotrophic lateral sclerosis (ALS, motor neurone disease, or Lou Gehrig's disease). The multinational team of scientists established an in vitro (in a dish) model system that involves directly changing a blood cell into a motor neuron, the main cell type that is killed in ALS. So, the team can take a blood cell from an ALS patient and turn it into a motor neuron to study the disease. The patient derived induced motor neurons actually die more rapidly in vitro than motor neurons derived from the blood of healthy control counterparts, thus recapitulating the disease and creating a model to study ALS in the lab.
Perhaps of most notable scientific significance, this paper establishes a new model system for studying neurodegenerative diseases in the cell type effected by the disease. The group exhaustively started this model system, and screened thousands of different drug-like molecules on the cells to identify molecules that could help the diseased cells survive longer in the dish. These findings will hopefully translate to help patients who have the disease.
Interestingly, many different mutations can cause ALS, but this paper proposes a mechanistic convergence in one cellular pathway that can be modulated by drugs identified in their drug screen. Very exciting research indeed!
References: Shi, et al. Nature Medicine doi:10.1038/nm.4490