A great leap forward in ophthalmic drug delivery?
“[Gene therapy may] dramatically reduce frequency of treatments.”
(Image credit: AdobeStock/Vink Fan)
It is not every day that a paper about treating eye disease appears in The Lancet.1 The article by Campochiaro et al1 on a clinical trial of gene therapy speaks to the potential impact of this technology to change not only how we care for patients with age-related macular degeneration (AMD) but also more generally how we treat patients whose diseases require long-term and repeated topical or intravitreal administration of therapeutic agents.
Depending on our patients to self-administer their medications or to reliably visit our clinics for their subsequent injections is a flawed model of drug delivery. Real-world evidence consistently shows that in clinical practice the outcomes with chronic medical therapy of eye disease are substantially and often dramatically inferior to those seen in the clinical trials that led to approval.2
In addition to the challenge of encouraging and convincing our patients to use their medications or return for injections reliably, the burden of administering these injections for neovascular AMD and, more recently, atrophic AMD, threatens to overwhelm the ability of retina clinicians to care for their large number of established patients while providing timely access for newly diagnosed patients. We are not training enough ophthalmologists today to manage the volume of aging patients with eye disease with today’s resource-intensive therapeutic regimens.
But that may change. The review by Veeral Sheth, MD, MBA, FASRS, FACS, in this issue describes gene therapy’s exciting potential to transition us from the current costly and burdensome model to either a single treatment or a dramatically reduced frequency of treatments. By greatly reducing the need to rely on patients to ensure the adequacy of therapy, this approach also offers the possibility that we will see results in the real world as good as those achieved in clinical trials. •
References
1. Campochiaro P, Avery R, Brown DM, et al. Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study. Lancet. 2024;403(10436):1563-1573.doi:10.1016/S0140-6736(24)00310-6
2. Mehta H, Kim LN, Mathis T, et al. Trends in real-world neovascular AMD treatment outcomes in the UK. Clin Ophthalmol. 2020;14:3331-3342. doi:10.2147/OPTH.S275977
Peter J. McDonnell, MD
is director of the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland, and chief medical editor of Ophthalmology Times®.
E: pmcdonn1@jhmi.edu
