Myocilin is a protein produced in the eye, but its role is not clear. People with mutations in the MYOC gene produce a different version of the protein, which can aggregate to form clumps in the cells which regulate pressure in the eye.

 

Dr Raquel Lieberman, an associate professor at the Georgia Institute of Technology in the US, led the research which scanned a large number of potential compounds to target the myocilin clumps. 

 

The mutant variation of the protein was mixed with a fluorescent marker which emitted more light when the protein was free. When the group tested the chemical candidates they identified two compounds which bound tightly to the protein, resulting in a drop in fluorescence. Further tests in cell cultures showed that this binding to the myocilin blocked the formation of the protein clumps.

 

“When we saw that these compounds inhibited aggregation, then we knew we were onto something good,” said Dr Lieberman, adding: “Because aggregation underlies the pathogenesis of this form of glaucoma.”

 

“The underlying problem with myocilin is that it has been studied for 14 years and still nobody really knows what its biological role is inside the eye,” said Dr Lieberman. The group are now working to resolve this by conducting further research to determine the structure and function of the protein.

 

The findings were announced by Dr Lieberman at a conference in California last week (January 20) and the research was published in the journal ACS Chemical Biology.

 

Commenting on the findings, Cecilia Fenerty, a consultant ophthalmic surgeon at Manchester Royal Eye Hospital, said that the research was an “exciting and very promising development”.

 

“It is a development of a drug treatment which targets the underlying disease process, rather than treating the consequence of the disease, and that’s a novel approach,” said Ms Fenerty. “Treatment success may depend on early detection or targeting those particularly at risk of early-onset, inherited glaucoma. Although this novel approach to treating the disease may in future be translated to other types of glaucoma.”

 

Learn more about how to detect glaucoma here.