Long-Term Benefits of Neuroprotective Implant for Macular Telangiectasia Type 2 Confirmed by New Study

A recent groundbreaking study has provided strong evidence that a surgically implanted device can slow vision loss in individuals suffering from macular telangiectasia type 2 (MacTel), a rare retinal disorder that progressively destroys central vision. The research, sponsored by Neurotech Pharmaceuticals and led by experts from Scripps Research and the National Institutes of Health (NIH), underscores the potential of neuroprotective therapies to change how we treat degenerative eye diseases.

MacTel remains without approved treatment options, leaving patients with limited options as their condition worsens. The newly developed implant, known as ENCELTO, delivers a continuous release of ciliary neurotrophic factor (CNTF), a naturally occurring protein that safeguards retinal neurons. This innovative treatment involves encapsulating genetically modified retinal epithelial cells within a collagen-based capsule, which is implanted in the back of the eye. The capsule design ensures long-term, localized delivery of CNTF while protecting the cells from immune rejection.

The two Phase III clinical trials, conducted across 47 international sites with 228 participants over 24 months, demonstrated that ENCELTO effectively slowed the degeneration of light-sensing cells in the retina. Results showed a significant reduction in ellipsoid zone loss, a key indicator of photoreceptor degeneration, with up to 54.8% in one trial and 30.6% in the other. Additionally, measures of visual function, including microperimetry and reading speed, indicated that the implant helped preserve practical vision, although outcomes varied between trials.

"This marks a pivotal shift in vision loss treatment," said Professor Martin Friedlander of the Lowy Medical Research Institute. "For the first time, we are seeing a therapy that can meaningfully alter the disease progression in MacTel, highlighting the promise of neuroprotection." The positive results contributed to the FDA approving ENCELTO in March 2025, making it the first authorized neuroprotective treatment for MacTel and for any neurodegenerative retinal disease.

Participants tolerated the implant well, with minimal side effects reported. The efficacy was consistent across different stages of disease and baseline vision levels, suggesting earlier intervention could maximize visual preservation. Future studies aim to evaluate long-term outcomes beyond 24 months and identify patient subgroups who may benefit most.

Furthermore, the success of ENCELTO paves the way for exploring its application to other neurodegenerative conditions affecting the eye and central nervous system. By delivering targeted, sustained doses of neuroprotective proteins, this platform technology holds promise for broader therapeutic use.

This study underscores that protecting retinal cells through innovative neuroprotective strategies could revolutionize treatment approaches for degenerative eye diseases, offering hope to those with currently untreatable conditions.

Source: Medical Xpress