New Drug Method to Cure Disease--from Cystic Fibrosis to Alzheimer's

There may be a new technique to cure disease. Scientists have demonstrated a revolutionary new method in mice that could cure a wide range of human diseases--from cystic fibrosis to cataracts to Alzheimer's disease--that are caused by "misfolded" protein molecules.

Misfolded protein molecules are caused by gene mutation. While they're capable of maintaining their function, they're misrouted within the cell and can't work normally. This causes several different types of diseases. Yet in this latest study, the researchers discovered a way to use small molecules that enter cells, fix the misfolded proteins and allow the proteins to move to the correct place and function normally again.

"The opportunity here is going to be enormous because so many human diseases are caused by misfolded proteins," said P. Michael Conn, one of the researchers, in a news release. "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. Drugs that act by regulating the trafficking of molecules within cells are a whole new way of thinking about treating disease."

That's not to say this method has been used in humans--yet. However, researchers have managed to cure mice of a form of disease that causes males to be unable to father offspring. The identical disease occurs in humans and the same concept could potentially be used to cure them, as well.

Proteins need to fold into three-dimensional shapes in precise ways to do their work within human cells. Before recent discoveries, though, scientists believed that proteins that were inactive were intrinsically non-functional. However, it seems that misfolded proteins actually are misrouted within the cell and cease to function only because of this misrouting. Pharmacoperones can fix these misfolded proteins and make them functional again.

"These findings show how valuable laboratory animals are in identifying new treatments for human disease," said Conn in a news release. "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."

The findings reveal a potential new method for treating disease. That said, it will be quite some time before this technique is used in humans. However, the findings have paved the way for future research and treatments that could help patients.

The findings are published in the journal Proceedings of the National Academy of Sciences.