New Drugs for Protein “Misfolding” Disorders
Proteins are the body’s workhorses—they are involved in almost every process that goes on in our cells. Protein folding is the process by which proteins become the shapes they need to be to get their work done.
Mistakes in this folding process happen naturally, and normally our bodies can dispose of misfolded proteins. However, in some people with genetic predispositions—and in others as they age—the body is unable to eliminate misfolded proteins, and becomes overwhelmed.
A variety of diseases are considered protein “misfolding” disorders, including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and type 2 diabetes. Traditionally, treatments for these conditions have focused on managing symptoms to increase quality of life for patients. What is not possible yet is to actually halt the disease’s progress.
As the Canada Research Chair in Drug Design for Protein Misfolding Disorders, Dr. Donald Weaver may be poised to change that. He is an international authority on clinical research on dementia and epilepsy, as well as computer-aided drug design and medicinal chemistry. He has designed and co-developed two drugs that have reached phase 3 human clinical trials, including one of the first Alzheimer’s drugs that actually attacks the disease.
Weaver will continue this work, using computational drug discovery and molecular modelling to design new drugs to target and prevent protein misfolding, with a particular focus on Alzheimer’s disease. Since that disease alone affects 47 million people worldwide, and is expected to double every 20 years, the potential impact of new drugs to treat it is enormous.