Recently, revakinagene taroretcel-lwey (Encelto, Neurotech) was FDA-approved as the first-ever treatment for slowing the progression of macular telangiectasia type 2 (Mac Tel). The device contains genetically modified cells that release ciliary neurotrophic factor. With any new therapy comes a myriad of questions and concerns. Some have likened Encelto to the complement inhibitors for geographic atrophy, as both slow down progression but don’t reverse it. The rate of ellipsoid zone loss with Encelto was more favorable, ranging from 31 to 55 percent over 24 months in two parallel Phase III trials, compared to around 20 percent with the complement inhibitors.1 However, neither therapy has demonstrated definitive functional vision preservation. Encelto showed significant reductions in retinal sensitivity loss in one of the two Phase III trials. Otherwise, visual acuity, reading speed and visual function questionnaires were similar between the treated and sham groups.

The other concern that has been voiced is that Mac Tel is slowly progressive and few patients complain of any deficits. I agree many patients fall into this category, but in speaking to some in more detail, I realize that many are just doing their best to adapt to their slowly changing reality. The majority of my patients have been referred in because they were noticing difficulties with vision in one eye. While we bank on the other eye carrying them through, what if it, too, goes down the tubes? We’ve then lost our opportunity to save vision.

Finally, there’s the surgical aspect that we must consider, including the risks involved and, ultimately, cost. The data seems to be showing better efficacy with earlier treatment, but is it risky to put this into someone’s better seeing eye, especially if the patients who really want it are the ones who have already lost significant central vision in the first eye? While the device seemed to have low rates of adverse events, there are still the usual risks of surgery. It’s also important to consider the issues with miosis and dark adaptation which are related to the release of CNTF. On the flip side, this can be a one-and-done procedure, unlike complement inhibitors that require repeated intravitreal injections. Compared to gene therapy where there is no kill switch, the device can be removed if there are adverse reactions.

We’re in an exciting time with new treatment options becoming available for both established and new indications. It’s great to be able to offer something now for our Mac Tel patients. While it won’t be for everyone, my sense is that some patients could see a lot of potential upside in being able to slow down the disease. RS

REFERENCE

1. Chew EY, Gillies M, Jaffe GJ, et al. Cell-based ciliary neurotrophic factor therapy for macular telangiectasia type 2. NEJM Evid 2025;4:8:EVIDoa2400481. doi:10.1056/EVIDoa2400481.