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Finding Treatment Targets for Age-related Macular Degeneration

In This Video

  • Clinician scientists at Mass Eye and Ear/Massachusetts General Hospital have been behind some of the biggest breakthroughs in treating age-related macular degeneration (AMD), but they believe more work needs to be done
  • Their ultimate goal is to develop treatments for early AMD and prevent vision loss from occurring
  • AMD is challenging because it is a complex disease, and a team of researchers is taking a multidisciplinary approach to treatment and detection, including metabolomics and artificial intelligence

Clinician scientists at Mass Eye and Ear/Massachusetts General Hospital Department of Ophthalmology have been behind some of the biggest breakthroughs in treating age-related macular degeneration (AMD), but they believe more work needs to be done. In this video, members of the AMD research team, Joan W. Miller, MD, chief of Ophthalmology at Mass Eye and Ear and Mass General; Demetrios Vavvas, MD, PhD, director of the Retina Service at Mass Eye and Ear; and Deeba Husain, MD, site director of Mass Eye and Ear Retina Consultants, explain their current research to develop new treatment targets for AMD and their efforts to detect the blinding disease at an earlier stage to prevent vision loss.


One of the world's leading ophthalmologists, Dr. Miller was a key member of the team that developed the first treatments for patients suffering from age related macular degeneration (AMD). The idea that new treatments developed at Mass Eye and Ear could limit and lessen vision loss was a game changer.

"These treatments were revolutionary and the anti-VEGF treatments are now given to more than a million patients around the world every year for wet AMD and for other conditions," said Dr. Miller. "We were incredibly pleased and excited to be able to now have a treatment that could really improve people's vision and hold it in good shape for a number of years."

But that doesn't mean Dr. Miller and her AMD research group are satisfied.

"The ultimate goal is to have treatments for early AMD and be able to prevent any vision loss from AMD," explained Dr. Miller.

Dr. Miller's colleague, Dr. Vavvas, is studying light sensing cells called photo receptors. The death of these cells leads to AMD, and he is working to understand what cause photo receptor death and how to prevent it.

"We're trying to look at the shared pathways of cell death in this different set of diseases," shared Dr. Vavvas. "The main root of our research is to develop therapies that are applicable to as many people as possible in as soon a time as possible."

But Dr. Vavvas is also investigating ways to provide more personalized care to AMD patients along with his colleague, Dr. Husain.

"AMD is challenging because it's a complex disease," said Dr. Husain. "It occurs in our body because of a combination of factors, which includes our genetic makeup as well as external factors such as our diet, our lifestyle, and other environmental factors."

Because of this, the Mass Eye and Ear retina team is attacking the problem in multiple ways.

"Under the leadership of Dr. Joan Miller, we have this large repository of images. With the help of artificial intelligence experts and computer engineers, we are analyzing these images to better understand disease and do subtyping of diseases, including age-related macular degeneration. We actually can assess when vision loss happens," explains Dr. Husain.

Another tool the team is using is metabolomics, the study of small molecules called metabolites, key in identifying AMD at its earliest stages.

"Metabolites have been found in many diseases to help as early biomarkers for disease," shared Dr. Husain.

The goal is that in the not-so-distant future, a scenario like this could be a reality.

"We envision treatments for AMD being such that you come into the doctor's office, and something will be picked up on one of these tests that we're identifying, and someone will say, 'listen, you need to take drug A by mouth like a vitamin once a day, and that'll prevent you from really developing any serious form of AMD and certainly prevent any vision loss,'" said Dr. Miller.

Dr. Miller's patient Laura Brennan suffers from wet AMD, but her vision has improved after starting treatment.

"This disease is affecting the baby boomers right now. It's affecting my friends. It's affecting people I know and love, and we need some help," said Brennan.

"Patients are so happy when we're able to offer a treatment for wet AMD. And when vision actually gets better after that treatment, it's really like a miracle," shared Dr. Miller.

"It's made my life so much better. I can't imagine not being able to see," continued Brennan. "Dr. Miller is brilliant. She's a genius. I can't imagine going through this without her."

"I'm extremely excited," said Dr. Vavvas. "We're following up a tradition at Mass Eye and Ear of great success every decade helping patients. And the findings so far of our research has shown much promise to have therapies for our patients in the future."

"We're not going to be satisfied until we can prevent any vision loss from AMD," said Dr. Miller.

Learn more about the Department of Ophthalmology at Mass Eye and Ear/Mass General

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By assessing genomic–metabolomic associations in age-related macular degeneration, Deeba Husain, MD, of the Department of Ophthalmology, and colleagues have obtained additional evidence of the pathogenic role of the LIPC gene and glycerophospholipid metabolism, which may represent novel treatment targets.


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