'Landmark' discovery brings a pill for Alzheimer's closer to reality, researchers say

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The discovery of a compound that stops diseases like Alzheimer's and Parkinson's in their tracks is a “landmark” step toward a pill to treat these conditions, according to researchers in the United Kingdom.

In Alzheimer's and similar diseases, the brain responds to the buildup of “misfolded” proteins by shutting off production of new proteins altogether, which leads to cell death. An orally administered compound has been found to intervene in this process and restore protein production in mice with neurodegenerative disease, according to researchers at the Medical Research Council Toxicology Unit at the University of Leicester.

The discovery means that a pill to treat Alzheimer's disease is now “a real possibility,” stated team leader Giovanna Mallucci, M.D.

However, the compound had serious side effects that degraded pancreas health and led to serious weight loss and mild diabetes, the researchers wrote. A pill that would be viable for humans is still “a long way away,” according to Mallucci.

The compound originally was developed by pharmaceutical company GlaxoSmithKline for a different purpose, the team noted.

The findings were published Wednesday in Science Translational Medicine.