Trinity College Dublin’s geneticists have made a groundbreaking discovery in the field of glaucoma treatment. With an estimated 80 million people around the world affected by glaucoma, and the number is predicted to surpass 110 million by 2040, this new development is a significant step towards providing a more effective therapeutic solution.

While topical eye drops are an essential element in preventing the progression of glaucoma, around 10% of patients develop resistance to treatment, putting them at risk of permanent vision loss. This novel gene therapy holds the potential to transform the way we approach glaucoma treatment, offering hope to those who are resistant to traditional methods.

Elevated pressure in the eye’s globe can cause severe damage to the optic nerve head, which transmits light signals to the brain and is responsible for our ability to see. The accumulation of unwanted proteins and blockages in drainage channels can cause fluid buildup and an increase in pressure over time.

The team’s findings suggest that a gene therapy-based approach can reduce intraocular pressure in pre-clinical models of glaucoma. This research provides hope for future glaucoma treatments and could potentially prevent serious optic nerve damage caused by elevated pressure in the eye.

The therapy involves using a viral vector, which is essentially a virus that has been repurposed to deliver specific instructions to cells in the body. By injecting the viral vector, the researchers were able to increase the flow of aqueous fluid from the front of the eye, thereby decreasing pressure in the eye. The key instructions delivered by the vector are for cells to produce an enzyme matrix called metalloproteinase-3 (MMP-3), which aids in initiating this process.

Professor Matthew Campbell, who is a professor in Genetics at Trinity and one of the researchers involved in the study, said: “This exciting project allowed us to bridge the gap between academia and industry and work very closely with a gene therapy company to develop a cutting edge therapy that we believe holds immense promise for patients in the future.”

The use of multiple models of disease and human eyes for testing adds to the credibility of the study, making the impressive results even more promising.

Recently, there has been a significant progress in gene therapies, resulting in the approval of several drugs by both the FDA and EMA.

However, it’s worth noting that all the authorized gene therapies to date are intended for the treatment of rare or ultra-rare conditions. Nevertheless, with the increasing sophistication of our knowledge about the fundamental mechanisms of common diseases, the prospect of utilizing gene therapy to treat common diseases is now feasible.

This new “approach to treating glaucoma using gene therapy,” according to first author Dr Jeffrey O’Callaghan, “is the culmination of over seven years of research.”

They “are now hopeful that this therapy will pave the way to the development of treatments for other forms of blinding eye diseases.”

Source: 10.1126/sciadv.adf6537

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