Alzheimer’s disease could soon be prevented with a simple pill after scientists have identified a molecule that can slow the progress of the disease or even have its symptoms reversed in the early stages. The discovery is a starting point for creating a drug that could be taken by millions of middle-aged people to prevent dementia. The future drug could be used when the first symptoms of Alzheimer’s disease appear, in the same way that pills prevent heart disease today, according to scientists from Cambridge University.
The molecule found by the scientists can serve as an inhibitor in the formative stages when the accumulation of brain plaques, a buildup of toxic protein, cause irreparable damage to the synapses. Called amyloid fibrils, the proteins eventually form clusters called oligomers together with other proteins with which they come into contact. This stage is called secondary nucleation and leads in the end to Alzheimer’s disease. But, as the team from Cambridge found out, natural proteins called Brichos can stick to the proteins and prevent the forming of damaging clusters that enable the condition to proliferate in the brain. The molecule essentially forms a coating that prevents the fibrils from assisting other proteins in misfolding and nucleating into toxic oligomers.
“A great deal of work in this field has gone into understanding which microscopic processes are important in the development of Alzheimer’s disease; now we are now starting to reap the rewards of this hard work. Our study shows, for the first time, one of these critical processes being specifically inhibited, and reveals that by doing so we can prevent the toxic effects of protein aggregation that are associated with this terrible condition”, says Dr Samuel Cohen, lead author of the report.
Scientists also believe the technique could help them identify other molecules that could have a similar effect simply because now they know what to look for. “It’s striking that nature – through molecular chaperones – has evolved a similar approach to our own by focusing on very specifically inhibiting the key steps leading to Alzheimer’s. A good tactic now is to search for other molecules that have this same highly targeted effect and to see if these can be used as the starting point for developing a future therapy”, Dr Samuel Cohen explained.
The research was carried out by an international team comprising academics from the Department of Chemistry at the University of Cambridge, the Karolinska Institute in Stockholm, Lund University, the Swedish University of Agricultural Sciences, and Tallinn University. Their findings were published in the journal Nature Structural & Molecular Biology.