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An experimental drug that could combat the onset of Alzheimer’s may open up a whole new approach to medicines, according to scientists.
The agents, known as gamma-secretase modulators (GSMs), work in a revolutionary new way by acting on the structure of proteins.
Other drugs have indirect effects by targeting enzymes, the “catalysts” that assist biological responses, or the receptor molecules on cells which proteins bind to.
GSMs reduce the production of long pieces of amyloid beta protein (Abeta) that stick together and form clumps in the brain.
The new drugs also promote levels of shorter amyloid beta elements, a key trigger of Alzheimer’s, that may protect against the disease.
Study leader Dr Thomas Kukar, from the Mayo Clinic in Jacksonville, Florida, US, said: “These compounds may do three things that may be beneficial with respect to Alzheimer’s disease: they inhibit production of long Abeta, block aggregation of Abeta, and increase production of shorter Abeta peptides that may in turn inhibit Abeta aggregation.”
Senior researcher Dr Todd Golde, wrote in the journal Nature: “This broadens the notion of what drugs can do, and therefore, has wide-reaching implications for future drug discovery for many different disorders.”
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