A Slow and Steady… Boom?

In 1999, Time quoted geneticist W. French Anderson, MD, as saying, “Twenty years from now gene therapy will have revolutionized the practice of medicine. Virtually every disease will have gene therapy as one of its treatments.”¹

In that Time article, Dr. Anderson was touting recent advances in gene therapy for heart disease that were finally showing some promise after 8 years of work. At the time, researchers were encouraged by signs of efficacy but had yet to see a successful protocol, as a whole, for gene therapy.

Alas, we have surpassed the 20-year mark (21 to be exact) since Dr. Anderson’s remarks, and we are still a far cry from achieving his vision. Gene therapy remains in its infancy for the vast majority of medical specialties, and it’s an FDA-approved option for only two conditions: Leber congenital amaurosis (approved in 2017) and spinal muscular atrophy (approved in 2019).

For most retina specialists, the fact that the eye was the organ privileged with the first successful gene therapy came as no surprise, considering the eye’s unique features. It is self-contained and provides easy viewing. Because of that, we’ve had something of a boom in ocular gene therapy research. In fact, so many novel agents are under investigation that we couldn’t discuss all of them in this issue’s featured article, “The New Frontier: Gene Therapy for AMD.” Instead, we focused on a subset of therapeutics for this leading cause of adult blindness in the developed world.

Although the approved gene therapy for Leber congenital amaurosis is ground-breaking and certainly life-changing for appropriately treated patients, just imagine how many patients we could help if one of the several proposed gene therapies for AMD pans out.

But let’s not get ahead of ourselves. The promise of a one-and-done treatment for AMD is alluring, but trials are in early stages, or just starting pivotal programs, and much can change as the data roll out and long-term follow-up reveals the lasting effects. (By the way, stay tuned for part 2 of the discussion, where we will dig into the specifics of gene therapies for inherited retinal diseases in the July/August issue.)

While we watch the gene therapy story slowly unfold, we have plenty to keep us on our toes in the clinic every day. We have ever-evolving imaging tools, as well as a robust armamentarium of AMD therapies, thanks to advances in anti-VEGF agents and delivery methods.

This issue, focused on AMD, touches on all of it: a new OCT classification system for macular atrophy, at-home OCT monitoring, the anti-VEGF therapy journey, pipeline drugs for nonexudative AMD, and treatment pearls for tough AMD cases.

The days of sitting AMD patients down and telling them that there’s nothing we can do to preserve their vision are mostly in the rear-view mirror, but there is still a great deal of work to be done. We might have to reeducate patients, constantly, on the importance of compliance with their treatments, but it’s worth it. Because vision matters.