Macular Degeneration Breakthroughs

Emerging Treatments & New Research for AMD

In This Article


Age-related macular degeneration (AMD) is the most common cause of blindness in the United States. The condition may be present in two forms, including wet AMD and dry AMD. There is currently no cure for AMD, and no treatment available for the dry form of the disease (other than preventative measures).

Scientists are working hard to find new solutions in the form of macular degeneration breakthroughs, emerging treatments, and new research that can help people with AMD keep their vision for as long as possible.

Types of AMD

Dry AMD and wet AMD have different characteristics.

Dry AMD

Dry AMD is the most common form of the disease; it involves the presence of very small yellow deposits—called drusen—which doctors can detect by performing an eye exam.

Drusen are present as a normal part of aging; but in AMD, these deposits begin to grow (in size and/or number). This increase in drusen can start the process of deterioration of the macula (an oval yellowish area near the center of the retina).

How Dry AMD Becomes Wet AMD
Verywell / Brianna Gilmartin

The macula is responsible for clear, straightforward vision). The retina is a layer of light sensitive cells that prompt nerve impulses, which are sent to the optic nerve, then travel to the brain, where images are formed. 

As dry AMD progresses, drusen begin to grow and/or increase in number and central vision may slowly become diminished due to deterioration of the macula.

Wet AMD

Dry AMD can progress to the wet form of the disease. Wet AMD involves abnormal, blood vessels that begin to develop under the retina. Wet AMD often progresses very quickly and can cause a person to experience vision loss due to edema or hemorrhage of these immature blood vessels, resulting in rapid damage to the macula.

Clinical Trial Phases

To understand just where a potential new treatment or drug stands, regarding the likelihood of becoming available to the consumer, it’s important to understand a little about medical research.

A new drug or treatment must successfully pass several phases of clinical trials before the product can be marketed or sold to the public. There are several phases of medical studies, these include:

  • Phase I: An experimental treatment or medication is tested on a limited number of people (usually between 20 to 80 study participants). This initial phase is aimed at testing the drug’s safety and identifying potential side effects.
  • Phase II: Once a drug or treatment is deemed potentially safe, it enters phase II testing aimed at continuing to observe its level of safety. This phase involves a larger group (usually between 100 to 300 study participants).
  • Phase III: After a drug or treatment is found to be relatively safe and effective, it is tested again (in a phase III trial) where scientists evaluate the effectiveness and safety, compared with standard treatment. This phase involves a much larger group (around 1,000 to 3,000) of study participants. Once a drug or treatment passes this phase, it qualifies to be evaluated for U.S. Food and Drug Administration (FDA) approval.
  • Phase IV: Once the new treatment or drug attains FDA approval, it is tested once more in a phase IV trial—aimed at evaluating its long-term safety and effectiveness— in those who are taking the new drug or being administered the new treatment.

Emerging Treatment for Wet AMD

If you have age-related macular degeneration, you may be excited to learn that there are some very promising new drugs and therapies on the horizon.

According to the American Academy of Ophthalmology, just 20 years ago, if a person developed wet AMD, vision loss was imminent. But in 2005, a groundbreaking new treatment, called anti-VEGF (including drugs such as Lucentis, Eylea and Avastin), became available.

These anti-VEGF drugs work to stop the blood vessels from growing, subsequently, controlling the leakage and slowing down the damage to the macula. According to the experts, the treatment is highly effective in preserving the central vision for people with wet AMD.

What Are Anti-VEGF Drugs?

The acronym VEGF— which stands for vascular endothelial growth factor— is a protein that is important in the growth and development of new blood vessels. When injected into the eye, anti-VEGF drugs help to stop the growth of these new, abnormal blood vessels.

Perhaps the primary drawback to the current treatment of wet AMD is the fact that the injections (giving directly into the back of the eye) of anti-VEGF drugs must be given every four to six weeks.

Today, there’s hope for new types of anti-VEGF treatments that will not need to be administered as often as the current, four- to six-week regimen. Some experts say that a few treatments being developed today, may possibly even cure the disease.

Retinal Gene Therapy

One promising new treatment, for wet AMD, involves retinal gene therapy, as an alternative to monthly eye injections. The goal of gene therapy is to employ the body to make its own anti-VEGF by inserting a harmless virus (called an adeno-associated virus/AAV) carrying the anti-VEGF gene into a person’s DNA.

More specifically, RGX-314 gene therapy only requires one injection, but it must be performed via a surgical procedure. This treatment is currently getting ready to enter phase II of clinical research trials.

Studies on RGX-314

Now that retinal gene therapy has been approved by the FDA for other retinal eye conditions (other than AMD), this type of treatment looks very promising for people with AMD. RGX-314 could potentially work to block VEGF for years after it is administered; this, in turn, would help to inhibit the development of the symptoms of wet AMD, namely, the immature blood vessels that leak blood into the retina.

In a phase I/II clinical trial involving 42 people, 9 out of 12 study participants did not require any further anti-VEGF injections for six months after one RGX-314 injection. In addition, there were no side effects observed during the study.

ADVM-022

Another potentially effective type of gene therapy can be administered in an outpatient setting (such as the doctor’s office). This therapy is called ADVM-022 and it is also moving into phase II clinical trials. It’s estimated that both of these therapies (ADVM-022 as well as RGX-314) could be available to people with wet AMD in as little as three years (approximately 2023).

The Port Delivery System

The Port Delivery System (PDS), is a very small (smaller than a grain of rice) apparatus that can store anti-VEGF medication. The PDS is implanted into the eye during a surgical procedure; it functions to provide a continuous release of anti-VEFG medication into the eye.

The Port Delivery System could allow people with wet AMD to avoid eye injections altogether. The procedure enables people with wet AMD to go up to two years without needing a treatment.

Refilling the medication can be done via a doctor’s office visit. But, the procedure to refill the medication is a bit more complex than the anti-VEGF injections that are currently the standard treatment for wet AMD.

This innovative treatment is now in phase three clinical trials and could possibly be available for consumer use within the next three years (around the year 2023).

Study of Lucentis (Ranibizumab) Port Delivery System

A 2019 phase II randomized controlled clinical trial (considered the gold label of medical studies), published by the American Academy of Ophthalmology evaluated the safety and effectiveness of the Port Delivery System with the anti-VEGF drug Lucentis (ranibizumab) for wet AMD treatment.

The study found that the PDS was well tolerated and that in people with age-related AMD, the PDS resulted in a response comparable with monthly intravitreal (inside the back of the eye) injections of anti-VEGF (ranibizumab) treatments.

"The PDS was found to be well tolerated with the potential to reduce the treatment burden [the workload of healthcare caused by a chronic condition] in nAMD [age-related AMD] while maintaining vision,” wrote the study authors. Note, the ranibizumab PDS treatment study has (as of 2020) progressed to a stage III trial.

Eyedrops

Anti-VEGF eye drops for wet AMD is another new treatment modality for AMD that is in the initial stages of clinical trial—but hasn't yet been used on humans. The treatment has been tested on animals.

Once the medicated eye drops are considered safe enough for human use, clinical trials will begin. It may take more than 10 years (around the year 2030) for anti-VEGF eye drops for wet AMD to be available for consumer use.

Oral Tablets

An anti-VEGF pill, to be taken orally (by mouth) may be available to the public in the next five years (approximately 2025). The pill form of the medication will enable people with wet AMD to eliminate or reduce the frequency of anti-VEGF injections.

Now in the phase II stage of clinical research trials, developers of oral medication for wet AMD are trying to work out the bugs. The medication has many side effects right now, such as nausea, leg cramps and liver changes.

Once the medication can be considered safe, and dangerous side effects can be eliminated, it can be considered for consumer consumption.

Longer-Lasting Anti-VEGF Injections

Several new anti-VEGF medications—aimed at reducing the frequency of injections—are being developed by the drug industry. These include drugs such as Abicipar, and Sunitinab, which are estimated at around three to five years (in the year 2023 to 2025) before approval for consumer use.

Another new drug, Beovu has already been approved for use in the U.S. Beovu injections can last as long as three months and the innovative medication is said to be more effective at drying fluid that has accumulated in the retina due to wet AMD.

Combination Drug Treatments

New combination medications for AMD include a combination of drugs that are already on the market, for treatment of AMD. The goal is a multi-faceted treatment approach aimed at increasing the benefit of the drugs and making the injections last longer.

One such combination is an eye drop to treat glaucoma, called Cosopt (dorzolamide-timolol), being tested in combination with anti-VEGF injections. Studies indicate that these two drugs, when given together, may help to lower retinal fluid better than just anti-VEGF injections can accomplish alone.

Radiation Therapy

Radiation therapy, similar to the type of treatment commonly used to treat cancer, is thought to help slow the growth of abnormal blood vessels caused by wet AMD. Radiation therapy is said to work the same way it does in cancer treatment. This is accomplished by destroying fast-growing cells.

But long-term safety must still be evaluated before radiation therapy can be considered as a mainstream option for AMD treatment. There are two types of radiation therapy available in the UK and Switzerland and they will soon be tested in the United States. Clinical trials are anticipated to begin within a year (around 2021).

Emerging Treatment for Dry AMD

The majority of the cases of AMD involve the slow-developing type of AMD, called dry AMD. Currently, as of 2020, there are no treatment options available for dry AMD, but some promising new therapies are in the pipeline.

Stem Cell Therapies

Stem cell therapy is gaining momentum for all types of treatment today, including many forms of cancer, as well as for dry AMD. The goal of stem cell therapy for AMD is that the new stem cells will be able to replace retinal cells that have been damaged or destroyed by symptoms of AMD. 

Stem cells are often introduced into the body’s blood circulation, via IV infusion. But, researchers are working on how to transplant the stem cells directly into the eyes. One strategy involves placing the stem cells into a fluid suspension that can be injected under the retina.

Although stem cell therapy for AMD has only been studied in small clinical trials, the experts say this treatment regime shows great promise. The drawback is that it may take another 10 to 15 years (around the year 2030 or 2035) for stem cell therapy to be proven effective and safe for consumers.

Study of Stem Cell Therapy for AMD

A small study, involving people with wet AMD, published by the New England Journal of Medicine, found that using a person’s own stem cells to replace damaged retinal cells, resulted in maintaining visual acuity for one year after the procedure.

The study authors wrote, “This seems to indicate the surgery helped to halt the progression of the disease.” Although this study does not indicate that stem cell therapy is effective for dry AMD, many scientists are confident that upcoming studies on stem cell therapy for dry AMD will be promising.

Injections for Dry AMD

Apl-2 is a drug that can be injected intravitreally (directly into the back of the eye) to help slow the progress of dry AMD, protecting the retinal cells from being destroyed. This treatment is in phase III trial stage and is expected to be available in approximately three to five years (around year 2023 to 2025).

Other Potential New Dry AMD Treatments

There are several other potentially effective new treatment modalities on the horizon for dry AMD, these include:

  • Oracea: An oral antibiotic with anti-inflammatory properties, it may be available for people in the late-stage of dry AMD. Oracea is currently in phase III trials and may be available as early as 2021.
  • Metformin: A drug commonly given to people who have diabetes, it was found to lower the risk of development of AMD. This may be due to metformin’s anti-inflammatory properties. Metformin is in a phase II trial as of 2020.

A Word From Verywell

Your ophthalmologist (or other healthcare provider) is the expert when it comes to which type of new AMD treatment might be right for you. There are many factors to consider, such as the type of AMD, your symptoms, as well as the level of progression of the disease, and more.

In addition, there is no single treatment that exists that does not have drawbacks. Some emerging treatments may be found to have very few side-effects, but the patient selection criteria (the criteria used to qualify as a study participant) may be very strict (such as for surgically implantable telescope lenses). Other treatments/medications can have side effects.

In the end, it’s important to stay open to new possibilities while working with your healthcare team to discover the best new AMD treatment for you.

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Article Sources
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