Exciting clinical trials underway and the next frontier of treatment for retinal conditions

Nimesh Patel, MD, a retina specialist focusing in pediatric retina at the Mass Eye and Ear Infirmary in Boston Children’s Hospital and Assistant Professor of Ophthalmology at Harvard Medical School and the Director of Pediatric Retina, discussed exciting advancements for retinal diseases and his “dream” for the next frontier of research.

Question:

Are there any promising clinical trials currently underway for retinal diseases that you are excited about?

Nimesh Patel, MD:

Yeah, there are a lot. I’m probably going to offend someone by leaving them off because there are so many really good clinical trials that are out there and some moving into a later phase. I’d say, one area that I’m interested in is gene therapy for inherited retinal diseases that we’re part of clinical trials for. Things like X-linked retinitis pigmentosa that, like you said prior, were incurable diseases. There’s some new treatments available for subretinal injection of gene therapy for inherited retinal conditions that are moving through clinical trials. We do have an approved product, Luxturna, that has shown us that it’s possible, and now it’s just a matter of applying the same technology and delivery to other forms of inherited retinal diseases. That’s probably more in the niche.

I think in the broader scope is probably looking at longer-term delivery devices or delivery methods for the current treatments we have in macular degeneration; there’s been implants approved by the FDA. There were some problems with those initially, and it’s yet to see if those will be a sustainable option. But when they do work, they are really extending the treatments out much longer than they were before. Then, I think one of the ones that I’m really excited about is the gene therapy option for macular degeneration and diabetic retinopathy. Again, seeing if we can put the gene therapy under the eye, have the eye produce the anti-VEGF medication instead of injecting it in the eye, that could be a really good option in the long run if it’s sustainable. I think those are probably different delivery methods of some of the current technologies probably what I’m most excited about at this point going forwards. Then, of course, some of these new compounds that we’re looking at, they’re probably still a little bit early in the pipeline.

Question:

What do you see as the biggest challenges facing ophthalmologists in the treatment of retinal diseases?

Nimesh Patel, MD:

Fortunately, we have pretty good treatments available. I think really our limitation often is more outside factors, clinic space, our patient volumes are exceeding our demand. A lot of the patients have trouble coming back to clinic for the treatments and they need rides and things like that. I think the biggest limitation often for the retinal specialist is how can we get the volume of patients that need us into the clinics on time without missing follow-ups. That’s probably the biggest challenge that we face as doctors, as patients missing appointments or having other comorbid conditions that put them in the hospital. Part of our area that we need to improve. There is then durability and hoping that these medications can last longer and they don’t have to keep coming back in.

Question:

What are some areas of continued need or research that will be important to advance the field of retinal diseases?

Nimesh Patel, MD:

Yeah, I think that part of this is what we’ve discussed is going over how can we increase the duration of the current treatments we have and how can we prevent cellular death. That’s probably the biggest one in not just ophthalmology and retina. Also, glaucoma in Alzheimer’s disease is once you have a progressive retinal condition, how can you preserve the cells that are there? That’s probably the next pipeline. The next pipe dream is how can you preserve the retinal cells? Then, on top of that, I would say some retinal implant is probably the next frontier is can you implant retinal cells and have them survive in a way that they can be functional? I think those are the next 2 areas that we’re probably going to be looking at in the long term.