Swiss gene therapy for blindness makes further encouraging progress

ddd Gaetan Bally / Keystone

Eye researchers at the Swiss city of Basel are hopeful of a breakthrough in the treatment for blind people after initial positive indications that a new form of therapy works.

Blind people learning to see. What sounds like a fairy tale is becoming reality – at least in a certain sense. A new therapy for the retinal disease retinitis pigmentosa, which has so far only been tested on a small number of people, is showing astonishing results.

With its help, blind people were able to recognize at least the outlines of certain objects. This is reported in the renowned scientific journal New England Journal of Medicine.

Stefan Futterknecht from the Institute of Molecular and Clinical Ophthalmology Basel (IOB) is a co-author of this recently published study. He is convinced that these findings will be very beneficial.

“This has laid the foundation for the development of more precise and effective therapies in the future, said Futterknecht, who described the study as a “milestone for restoring vision.”

Outlines of objects are seen

Five years ago, the first study of this gene therapy was conducted, at that time in Paris and with a single person. That man gained his first visual impressions through the therapy.

After initial treatments proved effective on the anonymous test subject, the IOB have moved to the next stage.

Of the ten participants in the expanded, new study, most experienced an improvement, said Futterknecht. “Seven out of ten people had an increase in light sensitivity.” Furthermore, four out of eight people were able to see the outlines of even small objects, such as a matchbox.

Being able to recognize a door could greatly simplify everyday life, according to the study. Furthermore, those who practiced more with the glasses saw better.

However, this milestone is far from being the end of the road. “We are still a long way from patients being able to read or having the kind of vision we are used to,” said Futterknecht. Now, work is underway to improve various elements of the therapy, including the proteins that make cells light-sensitive.

Potential benefits

Ideally, patients treated should be able to see again without glasses. The long-term hope is “to restore vision as much as possible,” says Futterknecht.

Specifically, all participants in the study suffer from retinitis pigmentosa, but its origins differ. Futterknecht says this shows that the new gene therapy has the potential to benefit many blind people, not just a specific, small group.

Optogenetic therapy does not address every single cause of the various forms of this disease, in which, in advanced stages, the light-sensitive retinal cells barely function. Instead, it enables other cells in the retina to respond to light.

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Translated from German, reviewed by an English Department journalist.