Macular Degeneration Research

Reviewed by: HU Medical Review Board | Last reviewed: May 2023 | Last updated: May 2024

Scientists believe that many factors can influence the development, severity, type, and rate of progression of macular degeneration. Genetics, diet, age, and environment may all play a role. Unfortunately, it is not well understood how these factors interact to lead to someone developing macular degeneration.1

Although the exact causes of macular degeneration are not well known, research is promising in treating the disease.

Gene therapy

Gene therapy may be an alternative to current therapies. A single injection of a harmless virus carrying the therapeutic gene is inserted into the eye. The goal of this therapy is to help the person with macular degeneration make their own protection against further damage from the disease. New gene therapy options are currently in clinical trial, and expected to be available to the public within a few years.1-4

Stem cell therapy

Scientists are studying the possibility of using stem cells to regenerate certain macular cells in people with advanced age-related AMD. Stem cells are special cells capable of growing and changing into many different cell types, including retinal pigment epithelial cells.2

Stem cell therapy has generated great excitement and shows some early promise as a possible treatment to restore sight. This therapy may take up to 15 years to be proven safe and effective for human use.2

Medicines for macular degeneration

Years of research have yielded major gains in developing drugs used to treat wet AMD. Additionally, research gains are showing promising results for the treatment of dry AMD.

Anti-VEGF drugs

In the past, drugs that target a specific protein have been the only injectable medicine available. These medicines, known as anti-vascular endothelial growth factor (anti-VEGF), need to be injected into the eye every 4 to 12 weeks, depending on the person. Anti-VEGF drugs decrease the amount of fluid from wet AMD.5

A pill form of anti-VEGF is currently being developed. However, this drug has many unwanted side effects. This type of drug will likely take at least 5 years to prove its safety and efficacy.2

Longer-lasting anti-VEGF drugs provide treatment with fewer injections. Beovu® (brolucizumab-dbll) is approved by the US Food and Drug Administration (FDA) for the treatment of wet AMD. About 1 in every 3 people taking Beovu can wait up to 3 months before their next injection. Beovu is new, but it may provide a better option for those with wet AMD.2,6

Two additional longer-lasting anti-VEGF drugs, Abicipar-pegol and Sutent® (sunitinib), are in clinical trials for treating wet AMD and may take years to receive FDA approval.2

Complement inhibitors

Exciting new treatments for dry AMD are being developed. As of late 2023, 2 complement inhibitors have been FDA-approved to treat the advanced form of dry AMD known as geographic atrophy (GA):7,8

APL-2 and Zimura® (avacincaptad pegol) are injectable drugs that work to stop the complement activation system that is responsible for inflammation, tissue damage, and blood vessel growth. Both drugs have shown promising results in slowing the long-term degeneration in dry AMD and are currently in clinical trials.2,9

Researchers are also looking into developing injectable drugs that will block runt-related transcription factor 1 (RUNX1), which is linked to abnormal blood vessel growth in the retina.10

Other medicines

The oral antibiotic Oracea® (doxycycline) is being tested for the treatment of dry AMD and the geographic atrophy that may result. Geographic atrophy may occur in advanced dry AMD and refers to the areas of the retina where cells waste away and die. Oracea is not yet approved for use. Clinical trials suggest that some side effects include stomach upset, rash, and sensitivity to sunlight.2,11

Metformin is a popular drug that treats diabetes. Metformin is currently in clinical trial to treat dry AMD inflammation. Results of previous trials have been promising.2

Eye drops

An eyedrop for the treatment of wet AMD could potentially be the first at-home treatment for wet AMD. This drug has had animal testing and requires human clinical trials to be proven safe and effective.2

Radiation therapy

Radiation therapy is being tested on the safety and efficacy of slowing the growth of blood vessels in wet AMD. Radiation therapy may decrease the need for injection therapy. However, radiation therapy has its own side effects. This type of therapy is still in clinical trials and is not yet approved by the FDA.2

Port delivery system

The size of a single grain of rice, the port delivery system (PDS) is an implantable device that stores anti-VEGF drugs directly into the eye. PDS drugs slowly release medicine into your eye, allowing for continuous treatment. The PDS provides the same results as monthly eye injections, but you only need treatment twice a year. Refills of the device may occur in a doctor's office.

The FDA has approved PDS with Susvimo™ (ranibizumab) for those with wet AMD. Susvimo has a boxed warning, the strictest warning from the FDA. It has this warning because Susvimo increases the risk of endophthalmitis, inflammation of the eye that can lead to blindness.2,12,13

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