A groundbreaking study led by Scripps Research and the National Institutes of Health (NIH) has provided strong evidence that a neuroprotective surgical implant can slow the progression of vision loss in individuals with macular telangiectasia type 2 (MacTel), a rare retinal disease.
The treatment, ENCELTO, is the first cell-based therapy for MacTel and was approved by the US Food and Drug Administration (FDA) in March 2025. The implant delivers a natural protein called ciliary neurotrophic factor (CNTF), which helps protect retinal neurons.
Two phase 3 clinical trials, conducted over 24 months at 47 sites worldwide, enrolled 228 people with MacTel. The trials demonstrated that ENCELTO slowed the decline of light-sensing retinal cells and preserved vision in individuals with MacTel. One trial showed a stronger effect, likely reflecting differences in disease severity.
The treatment was effective regardless of the participant’s baseline vision or disease stage, suggesting that earlier intervention may help preserve more functional vision as MacTel progresses.
While ENCELTO has shown promise, its estimated cost of $266,000 is significantly high. However, researchers believe that it represents a versatile platform technology that could potentially be adapted to treat other neurovascular degenerative diseases beyond MacTel.
For individuals like the writer’s 30-year-old daughter who lost her vision at age 7 due to an arachnoid cyst, this breakthrough treatment offers new hope. The estimated cost of ENCELTO is around $260,000, which may be out of reach for many patients.
Source: https://scitechdaily.com/first-ever-treatment-for-rare-eye-disease-wins-fda-approval-after-landmark-trials