ERC grant for world-leading glaucoma research at KI

Glaucoma is the most common cause of irreversible blindness and affects 80 million people around the world. Pete Williams, researcher at Karolinska Institutet and St Erik Eye Hospital, has now been awarded a prestigious ERC Advanced Grant for his work on new treatments for the disease. The ERC Advanced Grant is one of the most eminent awards a researcher can receive.
“It’s testament not only to the confidence they have in the focus of our research programme but also to the quality of our work,” says Pete Williams, senior lecturer and research group leader at the Department of Clinical Neuroscience at KI and St Erik Eye Hospital. “Medical research in the field of eye care is often under-funded, which makes this grant as significant as it is strategically important.”
The grant of EUR 2.26 million (c. SEK 25 million) for five years will give the research team the impetus they need to carry their work forward.
The hunt for a treatment to protect vision
Glaucoma causes gradual damage to the optic nerve, leading to impaired vision and, at worst, blindness. Despite new surgical methods, laser technology, and improved pressure-reducing eyedrops, in Sweden over 40 percent of treated patients progress to blindness in at least one eye.
Dr Williams’s research has brought about a paradigm shift. His group has shown that retinal levels of a molecule called nicotinamide adenine dinucleotide (NAD) – which the cells need to produce energy and stay healthy – decline with age.
This leaves the retinal ganglion cells, the neurons that transmit visual signals to the brain, more vulnerable. Doses of nicotinamide, a form of vitamin B3, can, however, offset the deficiency and protect the neurons.
Since nicotinamide has been used safely for over 75 years, it has been possible to put it to clinical use much more rapidly than if it had been a new drug, Dr Williams explains.
“The most direct clinical effect of my research is the speed at which it’s been possible to have our results transferred to patients – it took only three years to show that nicotinamide could improve the vision of glaucoma patients.”
Several clinical projects are underway involving nicotinamide, including an international study of over 1,800 patients, one of the largest in the field of glaucoma to date (with sites in Australia, Singapore, Sweden, and the UK). The researchers will know by the next two to four years if it can become a new treatment.
Breakthrough for drug development
Dr Williams has also identified the enzyme NMNAT2, which is crucial to NAD synthesis in the retina and optic nerve, as an important target for drug development. His group has now teamed up with pharmaceutical chemists to develop new molecules that have proved to be even more effective than nicotine amide.
Their work has led to the formation of a biotech company that will eventually take the results of their research to clinical application.
“We’re currently working with chemists, materials scientists, and cell biologists to develop the next stage – a gene therapy able to both protect and regenerate the optic nerve in one single intervention, depending on the stage of the disease.”
The grant will fund new projects
The ERC grant gives the research group greater freedom to take more risks and invest in long-term projects with high innovation potential. It can attract more financing and boost the group’s self-confidence.
And as Dr Williams says, it also increases each member’s career opportunities and sense of security.
“This is real teamwork based on international collaborations, all of which is made possible by the research groups around the world bringing their knowledge and expertise to the table.”
Dr Williams hopes that glaucoma awareness will improve over the coming decade and that even more resources will have been dedicated to the field:
“I also hope that there’ll be more treatments combining neuroprotection with pressure-reduction, and that we’ll be seeing the first gene therapy studies and stem cell treatments.”