In the most recent issue of Cell Stem Cell, a new study out of the laboratory of Dr. Lee Rubin at the Harvard Stem Cell Institute, reports the identification of a new small molecule that might be effective in treating Amyotrophic Lateral Sclerosis (ALS). Otherwise known as Lou Gehrig’s
Reference: Cell.com
syndrome, ALS is a neurodegenerative disorder that results in motor neuron death, severe debilitation, and ultimately death. To date, there are few therapeutic options, with a median survival time from onset to death of around 39 months.
In this study, Yang et al. derived motor neurons from mouse embryonic stem cells and performed a drug screen in the culture dish to identify novel molecules that might preserve or inhibit the death of motor neurons. The group found many different hits, but one molecule that showed a robust and reproducible effect on motor neuron survival in many of their assays was kenpaullone. This molecule is a GSK3-beta inhibitor, and the authors go on to show that in addition to this, it also inhibits HGK kinase in motor neurons.
In addition to promoting the survival of mouse motor neurons, they also used human iPSC lines derived from ALS patients and showed a positive survival effect on human motor neurons. This new study demonstrated the power of pluripotent technology to search for novel drug candidates to use as therapeutics for debilitating diseases with no current cures.
Congratulations to Dr. Rubin and his group for this fantastic study, and good luck moving forward! For more information, please see the link below:
http://www.cell.com/cell-stem-cell/abstract/S1934-5909(13)00139-2#Summary
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