Gladstone, Stanford research offers new game plan for drugs targeting ALS

Faking out a key player linked to amyotrophic lateral sclerosis could provide a new game plan in drug researchers’ battle against the deadly disorder also known as Lou Gehrig’s disease, according to scientists at the Gladstone Institutes and Stanford University.

The work is no small feat. The protein implicated in ALS, which slowly destroys the nerve cells that control muscle movement even while patients’ minds remain intact, also is critical for the survival of cells, so researchers essentially found a way of luring excess toxic amounts of the protein, called TDP-43.
The findings from the Gladstone and Stanford researchers, published Sunday in the online version of the science journalNature Genetics, mean little for the survival of ALS patients today. Researchers worked with yeast cells and neurons from rats -- one of the earliest stages in scientific discovery -- and even if a drug were found tomorrow it would likely take a decade or more to run through safety and efficacy trials in humans.
Still, the discovery could provide drug researchers with a new path for blocking the toxic accumulation of TDP-43 at a time when there is only one Food and Drug Administration-approved drug to treat ALS.