Alzheimer's disease could be treated by the use of a molecular inhibitor molecule. The molecule may be able to halt the development of the disease. This disorder is the result of amyloid fibrils, a form of protein, improperly becoming attached to other proteins, creating clusters of the structures, eventually leading to the loss of cognitive function.
Brichos, a molecule now shown to arrest progression of the disease, develops naturally in the human body, researchers report. This molecule acts as a molecular chaperone, attaching to dysfunctional proteins, preventing them from forming the dangerous clusters.
Prevention of toxic protein clusters is considered a goal of researchers looking to develop treatments for Alzheimer's. Recognition of the role Brichos plays in this process could lead to new drugs designed to treat the disease.
"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," Samuel Cohen, a Research Fellow at St John's College in Cambridge, England, said.
Proteins can preform important tasks through the process of folding, but when this function goes wrong, diseases and disorders such as Alzheimer's can result. The oligomers which result from clustering of proteins produce amyloid fibrils, which are highly toxic to nerve cells. These structures then cause other proteins to also fold incorrectly, building the fibril. When Brichos encounters an amyloid fibril, it binds to the formation, effectively creating a coating that prevents the structure from growing.
Modeling of how Alzheimer's disease progressed on the molecular level allowed researchers to predict the effects of halting the disease at various points.
Live brain tissues from mice were exposed to amyloid-beta, the protein that is responsible for the formation of amyloid fibrils. Samples exposed to the protein exhibited formation of the dangerous structures, while progression was limited in tissues supplied with Brichos.
Carefully examining the progression of a disease, and predicting which molecules may stop the progression of an illness could be utilized by investigators in other fields of medical research.
As many as five million Americans might suffer from Alzheimer's disease, according to the National Institute on Aging. The disease is usually diagnosed in patients older than 65. Some researchers believe the condition could begin with damage to the brain, starting a decade or more before symptoms appear.
Discovery of the role of Brichos in preventing the progression of Alzheimer's disease was detailed in the journal Nature Structural & Molecular Biology.