Macular Degeneration Treatment Breakthrough

Macular Degeneration Research

He has a special interest in age-related macular degeneration research and in the genetics of AMD. He is the principle investigator for a groundbreaking study that is looking at the retinal changes that lead to dry macular degeneration called the COMPLETE Study.

1. Compared to wet macular degeneration, why are there so few trials for dry AMD?

Much of the research over the past 20 years has focused on wet AMD and the growth of abnormal blood vessels. Wet AMD also benefited from the breakthroughs in cancer therapy where the drugs to treat wet AMD were originally developed to treat the growth of blood vessels into tumors.

Our approach to dry AMD dry AMD is based on our clinical exams, human autopsy studies looking at eyes from patients with AMD, and the genetic breakthroughs that resulted in 2005 once the human chromosomes were sequence.

The first complex human genetic disease that was mapped following completion of the human genome project was AMD and it was mapped to genes that are found in the complement pathway, a part of the human immune system. So, in some ways, we can think of AMD as an autoimmune disease in which the disease results from abnormal activation of the immune system.

2. Why should a person consider participating in a clinical trial?

3. As the principle investigator for the COMPLETE Study, tell us a little about the importance or significance of this study.

From genetic studies we know that complement genes have been implicated as a cause for AMD. We also know from the autopsy studies, that complement is deposited in eyes with AMD and normal eyes don't have this complement accumulation.

It is the abnormal accumulation of complement that we believe cause the damage in the eye and the loss of vision.

In the COMPLETE Study, we are using the only FDA approved drug that has been proven to block the activation of complement.

The drug was approved for a rare disease that causes young people to die; the disease is called paroxysmal nocturnal hemoglobinuria. The drug, known as eculizumab, blocks complement and restores a normal quality of life to these young people.

Thousands of people have been treated with eculizumab and we know it's safe as long as we follow certain precautions. Just like we borrowed the wet AMD drugs from cancer therapy, we are borrowing this dry AMD drug from a different field of medicine and using it for the treatment of an eye disease.

4. In a previous post you mentioned, that,"genetic studies have unambiguously shown that complement activation is associated with all forms of AMD." Can you explain complement activation to us?

In AMD, it is believed that people with AMD carry mutations in their DNA that cause the complement system to become over-activated and the pathway accidentally destroys normal tissue.

In AMD, the normal tissue that is destroyed resides in the back of the eye. By blocking this abnormal activation, we hope to prevent the damage of the normal tissue and give the eye a chance to recover and stop degenerating.

5. The drug being used in the COMPLETE study is, eculizumab. Tell us in general terms how this drug works, how it will be given in the study and how often. What are the possible side effects of this drug?

The drug known as eculizumab (also known commercially as SOLIRIS) and this drug blocks a specific protein in the complement pathway known as C5.

By blocking C5, we can prevent the damage that results from complement activation. The drug is an antibody similar in structure to Avastin , which is one of the drugs we use for wet AMD.

When we give eculizumab, we also give it as a systemic infusion. What's good about a systemic infusion is that the drug gets to both eyes and AMD is always in both eyes.

The drug has to be infused every week for 5 weeks then every 2 weeks thereafter. The infusion takes about 30 minutes.

The greatest risk of this drug is that it puts you at risk for meningococcal meningitis, a rare bacterial infection of your brain surface. This is an infection that is most frequently seen on college campuses among young people.

That's why all college students should get the meningococcal vaccine. In our study, all patients get the vaccine before we start the therapy, and the vaccine successful prevents the infection. Remember, this drug is already FDA approved and has been used in thousands of patients.

6. Explain briefly a randomized, double blind, placebo controlled study. What are the chances of a patient receiving the active drug?

To perform a scientifically valid study, it is necessary to randomize patients so some patients get the drug and some patients get the placebo. We randomize patients 2:1 to get the drug. That means 2 out of 3 patients, or 67%, get the drug and 1 out of 3 get the placebo.

One way to view your odds are to realize that if a patients doesn't participate in the study, then they have a 0% chance of getting the drug, and if they participate in the study, then they have a 67% chance of getting the drug. I don't know and the patient will not know if they received the drug until the end of the study.

7. What is the criteria for a patient to be eligible for this study?

To be enrolled in the study, patients need at least one eye with dry AMD. The dry AMD can be characterized as just large soft drusen or a feature of AMD known as geographic atrophy. Vision has to be 20/60 or better. The best way to know if a person qualifies for the study is to have their doctor send us an image of their eyes.

The image should be either a photograph or an autofluorescence image. In addition, an OCT image would be helpful.

8. If a person is interested in participating in the COMPLETE study who should they contact?

They should contact my office at 305-326-6148 or send their question and/or images to prosenfeld@med.miami.edu. Or, they can contact my coordinator Maria Esquiabro at 305-326-6508 or at mesquiabro@med.miami.edu.

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