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New drug approach could lead to cures for multiple diseases

Category: Hormones and Metabolism

Following research conducted by the Oregon Health and Science University in Portland, a new approach to treating misfolded proteins could lead to cures for a wide range of diseases. Lab tests carried out on mice may bring about revolutionary changes in treatment for ailments such as cystic fibrosis and cataracts.

The study, published in the Proceedings of the National Academy of Sciences, was led by Michael Conn, Ph.D., former professor of physiology and pharmacology at OHSU. His team looked to discover a new method to fix misfolded proteins and restore them to make cells function correctly. Using male mice incapable of fathering offspring, the researchers were able to cure the mice and perfect the process through lab testing. They believe the same treatment could be used on humans.

"The opportunity here is going to be enormous because so many human diseases are caused by misfolded proteins. The ability of these drugs - called 'pharmacoperones' - to rescue misfolded proteins and return them to normalcy could someday be an underlying cure to a number of diseases," affirmed Conn.

Previously, scientists believed inactive proteins were naturally non functional. But Conn and his team of researchers showed that when the proteins were misfolded, their purpose was rerouted and caused a malfunction. The pharmacoperones are used to correct the routing problem and correctly fold proteins for functioning.

"We expect that these studies will change the way drug companies look for drugs, since current screening procedures would have missed many useful pharmacoperone drugs."

According to Conn, the next step in the process will be clinical trials on humans.

What are misfolded proteins?
Lab tests show that misfolded proteins are caused by genetic mutations and are capable of retaining their function but are incorrectly routed in the cell and lead to disease. A wide range of ailments are caused by these proteins, including Alzheimer's disease, Parkinson's disease, cystic fibrosis and different types of diabetes.

According to Nature Education, misfolded proteins develop a toxic configuration but are still able to communicate with native proteins around them. When this occurs, they repeat their function in a loop, intensifying their toxicity until they eventually impair function or kill the cell.

At the moment, there is no definitive treatment for prevention of these diseases. However, with the conclusions drawn by Conn and his research team, as well as improvements in blood testing and the increased availability of lab tests online, doctors and patients can start to hope for a cure in the near future.