Emerging Treatments | Ophthalmology

Session Focus

Ahmad framed the session as covering both cornea and retina across a period of unusual activity. The conversation moved from the field’s long history of firsts through the shift toward regenerative approaches, the durability problem in retina, and where the next five to ten years lead.

A Field Defined by Firsts

Reza Dana MD went back 120 years rather than twenty to make a point about ophthalmology’s role in medicine generally.

Many medical firsts occurred in ophthalmology, and many of those in cornea specifically. The first successful allotransplants came in 1905, roughly half a century before the first successful kidney transplant at the Peter Bent Brigham in the 1950s. Ophthalmology was first to advance laser technology for shaping tissue in the 1980s and 1990s. Successful stem cell transplantation followed in the late 1980s and 1990s, developed in his own department, after bone marrow transplantation. Topical biologics likewise found their leading indication in ophthalmology.

The principal change of the last twenty years, in his account, is a shift toward pro-regenerative therapies that remove the indication for surgery.

Keratoconus is his clearest example. Until roughly ten years ago it was a surgical condition: mild cases were fitted with a contact lens, and corneal thinning past a certain point meant a corneal transplant. Patients now receive cross-linking, and that technology has itself advanced considerably.

His second example is recombinant nerve growth factor therapy for corneal healing, now roughly a $1.7 billion annual market, addressing what was previously either untreated or surgical.

Twenty-Five Years in Retina

Rishi Singh MD traced the retina arc from nothing to something.

Anti-VEGF therapy arrived in 2004 as a first-in-class improvement across blinding disorders: neovascular AMD, diabetic retinopathy, diabetic macular edema and retinal vein occlusion, all leading causes of blindness addressed by a single drug.

His assessment was more complicated than that sounds. It was not so great because of the influx of patients, the number of injections being given, and durability problems.

The field has moved on to Ang-2 biology, with some Ang-2 inhibitors now state of the art and more in the pipeline he described as genuinely exciting.

Beyond that: geographic atrophy, previously untreated, now has a first-in-class therapeutic option. Inherited retinal degeneration has Luxturna, which he called an amazing feat. And the first encapsulated cell technology, again developed in Boston, has reached patients with macular telangiectasia.

He noted a personal stake, as the son of a parent receiving these injections, in seeing their value directly.

On aging, he agreed these are largely age-related conditions while noting a genetic underpinning in retina alongside environmental and lifestyle contributions. He mentioned studies on GLP-1s improving lipid profiles in patients, with a possible anti-inflammatory profile that might carry outcome benefit, while adding that the field is still not achieving what it needs to.

Durability, Not Acuity, Is the Unmet Need

Ahmad asked what the remaining unmet need is for anti-VEGF: fewer injections, better vision, or longer intervals before rescue.

Singh first corrected the premise. These drugs have biosimilars rather than generics, though average selling price has dropped significantly, making them more affordable.

His answer on unmet need was unambiguous. It is not improvement in visual acuity. It is durability.

He quantified the patient burden. Patients come in five to six times a year, spend four hours in the office, then go home and cannot do anything for twenty four hours. Many of these are working-age adults, which makes the quality of life impact substantial.

That framing matters for development priorities: the field is not primarily seeking better vision outcomes from this class, but relief from the treatment schedule required to maintain them.

Key Takeaways

1. Ophthalmology performed the first successful allotransplant in 1905, half a century before the first kidney transplant.

2. Regenerative approaches are removing surgical indications, with keratoconus moving from transplant to cross-linking.

3. A single drug class addressed four leading causes of blindness from 2004, at the cost of an unsustainable injection burden.

4. Ang-2 biology is now state of the art in retina, with further pipeline agents in development.

5. Geographic atrophy, inherited retinal degeneration and macular telangiectasia all now have first-in-class options.

6. Durability rather than visual acuity is the unmet need. Five to six visits a year and 24 hours of recovery each time.

7. Anti-VEGF agents have biosimilars, not generics, though average selling price has fallen substantially.