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Assessing the feasibility of MT-011, a first-in-class drug to treat glaucoma and other neurodegenerative diseases via a breakthrough mechanism-of-action

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New class of glaucoma drugs could sharpen brains as well as eyes

A drug that protects optic nerves offers hope to the one third of glaucoma patients for whom no effective treatment exists. Intriguingly, the drug may also slow Alzheimer’s.

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Glaucoma is a neurodegenerative disorder that causes deterioration of eyesight starting with the periphery, causing ‘tunnel vision’ to develop. It is a major cause of irreversible blindness, affecting 1 in 10 over-75s in Europe and 60 million people worldwide. Current treatments are focussed on reducing pressure in the eyeball, but for the 30 % of patients who have ‘normal tension’ glaucoma, no effective treatments are available. The EU-funded SA-VOIR project seeks to develop a new drug that can delay or even prevent blindness in these individuals.

Cell scaffolds

“Ophthalmologists prescribe the same treatment to patients presenting normal tension as they do for high-tension glaucoma, even though they know it will be less effective,” says Siem van der Laan, SA-VOIR project coordinator. “We want to find a solution that protects the nerve from degeneration, primarily for normal-tension glaucoma – although the high-tension kind may also benefit.” MT-act, host of the SA-VOIR project, has been investigating small molecule drugs which target the enzymatic machinery that regulates microtubules, the scaffolds on which a cell is built.

Tau dysfunction

As well as providing the cell’s molecular architecture, these microtubules are essential for the transport of molecules around the cell. Damage to these microtubules is a characteristic early symptom of glaucoma, and it’s hoped that the drug candidate known as MT-011 can help cells resist this degradation. As the condition affects optic nerves, glaucoma is considered a neurodegenerative disease like Alzheimer’s and Parkinson’s. The first-in-class treatment targets early events in tau dysregulation provoking microtubule disintegration, meaning that MT-011 may also offer some protection against other tauopathies, a group of pathologies linked to tau dysfunction. However, van der Laan is staying focussed on glaucoma for now: “It is much easier to stratify and monitor patients with glaucoma, and the measurements are much less subjective,” he adds.


The project was supported through the EU’s Horizon 2020 programme. “The funding helped us to identify our approach and our position in the medical landscape,” explains van der Laan. “It was critical to polishing and advancing the business plan of the company.” “Our feasibility study also showed a major problem that ophthalmologists have is compliance of treatment,” notes van der Laan. “So, we’ll work with ophthalmologists to produce the drug in a formulation that is tolerable to patients.” Van der Laan says the next steps are mostly related to overcoming these technological and regulatory hurdles. If successful, the company hopes to move into Phase I clinical trials, before licensing the drug to a pharmaceutical company. This deal is expected to be worth some EUR 40 million, and major pharmaceutical firms have already indicated an interest.


SA-VOIR, glaucoma, blindness, Alzheimer’s, neurodegenerative, disorder, tau, tauopathies, molecule, ophthalmologist

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