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Researchers propose a new way to discover new glaucoma treatments

23 September 2021

In a study recently published in the Journal of Clinical Medicine, researchers at Karolinska Institutet and St. Erik Eye Hospital, present a new way of identifying potential therapeutics based on exploring common genetic changes in models of glaucoma. The study showed that by using this approach, multiple glaucoma-relevant therapies could be pre-clinically tested. Enhancing the ability to identify potential therapeutics is vital for improving patient outcomes in the future.

The Williams lab research group behind the study: Pete Williams, Tim Enz and James Tribble.

Glaucoma affects 80 million patients globally and approximately 100,000 – 200,000 in Sweden. In glaucoma, retinal ganglion cells, which connect the eye to the brain, are progressively damaged, leading to a loss of vision. The only treatment strategies currently available target the pressure in the eye, but despite the availability of the treatment, many patients continue to lose vision. New treatment options are greatly needed.

In the study, the scientists used data from RNA-sequencing, a technique that is becoming increasingly important in pre-clinical research of glaucoma, to map changes in retinal ganglion cells. RNA is needed to convert the information stored in the DNA into proteins. Changes to the amount of RNA which corresponds to specific genes and proteins can indicate potential key mechanisms of disease.

"We compared RNA-sequencing data from multiple datasets to find changes that are common to different forms of retinal ganglion cell injury. This is a different way to discovering glaucoma relevant mechanisms and a more efficient way to identify potential therapeutic targets. We then searched for drugs that are known to interact with these genes in other contexts and tested their potential to protect retinal ganglion cells. We identified a number of promising drugs that warrant further testing," says Tim Enz, ophthalmologist and researcher, and the first author on the paper.

The researchers believe that this data driven approach is a better way to identify new therapeutics and begin pre-clinical testing, especially for testing multiple drug candidates. Importantly, it uses publicly available resources so that many labs will be able to apply this approach. 

"This work exemplifies the lab’s commitment to encouraging clinicians to engage in fundamental and pre-clinical research," says the principal investigator and group leader Pete Williams. 

23 September 2021