Gene Therapy Paired with Special Goggles Improved Light Sensitivity in 7 of 10 Blind Retinitis Pigmentosa Patients

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The results, published Oct. 7 in the New England Journal of Medicine, come from a small early-stage trial led by researchers at the University of Pittsburgh and UPMC with international collaborators. The treatment did not restore normal vision or the ability to read. Some participants, however, became better at detecting, locating, and reaching for objects while wearing the goggles.

Retinitis pigmentosa has very few treatment options, and the approach is designed to work regardless of which gene caused a person’s disease, a key hurdle in this condition.

Retinitis pigmentosa destroys the light-sensing rod and cone cells in the retina, the thin tissue at the back of the eye. It affects more than 1.5 million people worldwide. People usually lose night vision first, then side vision, and many eventually become severely visually impaired or blind.

The disease can be caused by mutations in more than 100 genes, which makes gene-by-gene repair slow and expensive. Optogenetics takes a different route. Even after rods and cones die, other retinal cells called retinal ganglion cells often survive. In this trial, doctors delivered a gene for a light-sensing protein called ChrimsonR into those cells with a single injection into one eye.

ChrimsonR responds best to specific colors and intensities of light that ordinary daylight does not reliably provide. So participants wear goggles with a built-in camera that sends images to a portable processor. The processor turns the scene into patterns of light at the right wavelengths, and a projector in the goggles shines them into the treated eye.

The study is part of the PIONEER trial, a first-in-human, open-label study that tested three dose levels in 10 people with advanced disease who had little or no remaining vision. All participants knew they were receiving the treatment, and there was no comparison group.

After treatment, seven of the 10 showed improved light sensitivity, and six met the bar for a clinically meaningful change. Brain recordings using electroencephalography (EEG) in some participants showed evidence that visual signals reached and were processed by the visual cortex. Four of the eight participants who completed behavioral testing showed consistent improvements across several real-world visual tasks over months to years.

Dr. José-Alain Sahel, the study’s first author and chair of ophthalmology at the University of Pittsburgh, said the results show that even with profound vision loss, “the visual system retains a remarkable capacity to process new information.” Co-corresponding author Dr. Botond Roska of the Institute of Molecular and Clinical Ophthalmology Basel told Science News the work shows “optogenetics can bring back some visual activity [and] object sensitivity.”

Most eye-related side effects were mild or moderate, including temporary inflammation and short-lived rises in eye pressure. The study reported one severe event, a brief blockage of the central retinal artery right after the injection, which resolved within minutes after treatment with eye drops.

The trial was designed mainly to test safety, and its efficacy results are exploratory. With 10 participants, no control group and no blinding, the findings cannot show how well the therapy would work in a larger population. A joint release from GenSight Biologics quoted Sahel as saying the results need to be confirmed and further investigated.

The research was supported by GenSight Biologics, the French company developing the therapy, and one co-author is affiliated with the company. Sahel is also a co-founder of GenSight, STAT has reported. Those financial ties are common in early gene therapy research but are important context for readers weighing the results.

The study builds on a 2021 report in Nature Medicine that described partial recovery of visual function in a single blind patient using the same approach, the first clinical use of optogenetics in medicine.

Sahel was also senior author of a study last year on PRIMA, an implanted retinal device for geographic atrophy, an advanced form of dry age-related macular degeneration. PRIMA has received European approval but is not FDA-approved. The two approaches target different patients and are not interchangeable.

The therapy is not approved and is available only through clinical research. People with retinitis pigmentosa can ask a retina specialist about trials and about genetic testing, which can identify the gene involved and determine eligibility for other studies or treatments.

Patients should be cautious about clinics that charge for unapproved stem cell or gene treatments for blindness. Legitimate trials are listed on ClinicalTrials.gov and typically do not charge participants for the experimental treatment.

Sudden vision changes, such as a curtain over part of the vision, new flashes or floaters, or eye pain, need prompt evaluation by an eye doctor. Low-vision rehabilitation services can also help people with advanced disease make the most of remaining sight.

Larger, controlled studies will be needed to confirm the benefits and identify who is most likely to respond. MedicalDaily will follow any plans for the next phase of testing.

Seven of 10 people with advanced retinitis pigmentosa had improved light sensitivity after optogenetic gene therapy used with special goggles. Six had clinically meaningful gains.

No. Participants did not regain normal vision or the ability to read, but some could better detect, locate and reach for objects.

A gene for a light-sensing protein is delivered into surviving retinal ganglion cells. Goggles convert images into light patterns that activate those cells.

Most side effects were mild or moderate, such as temporary inflammation and rises in eye pressure. One brief artery blockage resolved within minutes.

The research was supported by GenSight Biologics, which is developing the therapy. One co-author is affiliated with the company, and the first author co-founded it.

No. It is experimental and available only through clinical trials. Patients can ask a retina specialist about research options.

It is an inherited disease that destroys light-sensing cells in the retina, usually causing night blindness, then side-vision loss and sometimes blindness.