Contributing to a Cure
Study participants receive quality care while researchers learn about diseases
By Allyson T. Collins, M.S.
NEI Science Writer/Editor
NEI clinical trials participant
In 2000, Rebecca Hatcher's 69-year-old sister lost vision in her left eye because of advanced age-related macular degeneration (AMD), in which abnormal blood vessels begin to grow and leak fluid within the retinal tissue in the back of the eye.
Soon after, Hatcher herself, who lives less than 25 miles from the Bethesda, Md., campus of the National Institutes of Health, searched for the National Eye Institute (NEI) online and e-mailed to ask if she could participate in a clinical study.
"Before my sister realized she had a problem, she couldn't see," Hatcher says. "I didn't want that to happen to me."
Clinical studies are a type of medical research in which people participate. According to www.clinicaltrials.gov, NEI has supported nearly 300 clinical studies, including 63 that are actively seeking participants.
"Everyone who comes to NEI is encouraged to take part in a clinical study," says Brian P. Brooks, M.D., Ph.D., chief of the NEI Unit on Pediatric, Developmental and Genetic Ophthalmology. Many of the studies Brooks conducts involve genetics.
Clinical trials and natural history studies are two types of clinical studies conducted at NEI. Clinical trials test a specific question, usually related to a treatment's safety, effectiveness or both. A clinical trial has at least one treatment group and may also have a control group that receives standard care or no treatment, Brooks explains. In clinical trials, researchers analyze a particular outcome, such as sharpness of a person's vision or the appearance of the retina in the back of the eye.
In contrast, natural history studies look at how a disease progresses over time. "Physicians need to see a variety of diseases and many patients with the same disease to maintain their skills, understand how a disease progresses and develop new treatments," Brooks says. "Natural history studies give us the opportunity for that."
People who want to take part in a study must fulfill the criteria required to participate, such as a certain age or diagnosis. When a person is enrolled in a clinical trial, a visit to NEI can take several hours and involve a variety of tests, including genetic testing. "Because we are a research hospital and we are trying to learn from patients, we tend to do more investigating and ask more questions than a private practice doctor might," he says.
The First Stop for AMD Treatment
NEI Staff Clinician Nida Sen, M.D., M.H.Sc., explains the importance of clinical studies to her patients.
Hatcher first participated in an AMD study involving lutein, a natural pigment in the eye that is also found in spinach, kale and broccoli, led by Emily Y. Chew, M.D., NEI deputy clinical director.
Five years later, she was seen by NEI ophthalmologist Wai Wong, M.D., Ph.D., who noticed she had progressed from signs of early AMD to intermediate AMD in both eyes.
"We found from the first Age-Related Eye Disease Study--known as AREDS--that if people had intermediate AMD in both eyes, they had a higher risk of progression to advanced AMD and losing vision as a result," Wong explains. "Ms. Hatcher had some of these changes, which allowed us to welcome her participation in a follow-up study called AREDS2. One primary goal of this second study is to find out if dietary supplements can decrease the risk of disease progression."
Previously, AREDS researchers, led by NEI scientists, found that certain antioxidant vitamins plus zinc could reduce the risk of advanced AMD by about 25 percent. AREDS2 researchers are trying to determine if lutein and a similar pigment zeaxanthin, as well as omega-3 fatty acids found in fish oil, can also be beneficial.
About 4,000 people from more than 80 clinical centers around the country have enrolled in the five-year study, which began in 2006. Hatcher was the first participant from NEI.
"Dedication and interest in participating in eye research are helpful as clinical studies can involve a long-term relationship with NEI physicians," Wong says. "My patients tell me they get the most satisfaction from feeling that they are giving back to the community and helping further medical knowledge and research."
Hatcher actually increased her commitment to clinical trials by enrolling in a second study when she developed wet AMD. Through this study, researchers will observe how one type of injected drug therapy, called Lucentis, alters new abnormal blood vessels.
"We know that the leaky blood vessels in wet AMD are like bad plumbing," Wong says. "We can often stop the leaking temporarily, but we don't know how to change the structure of the plumbing or remove leaky plumbing permanently yet."
Hatcher is pleased that she has maintained 20/20 vision in her right eye, and 20/40 vision in her left eye. "I've had the most wonderful experience," she says. "Sometimes there are three doctors looking at my records at the same time. I know they want the best for me, and you can't beat that."
In a Position to Study Rare Diseases
NEI clinical trial participant
Bobette Morgan has been coming to the National Institutes of Health since 1992. Her first clinical trial experience was through the National Institute of Dental and Craniofacial Research. She has Sjogren's syndrome, a disease in which the immune system mistakenly attacks healthy cells throughout the body. This condition often affects the moisture-secreting glands in the mouth and eyes first.
When Morgan began losing her vision in 2008, her doctors thought it was related to a medication she was taking. "It's like looking through a white curtain," Morgan says about her vision. "It's very foggy, but like Swiss cheese because I have a small part of my central vision left."
She consulted several specialists around the country before she came to NEI from her home outside Philadelphia in January 2010. NEI Staff Clinician Nida Sen, M.D., M.H.Sc., diagnosed her with autoimmune retinopathy, a disease where the immune system turns against the cells of the retina.
The disease is rare, difficult to diagnose and not completely understood, Sen says. "Once a person is diagnosed, they have likely already had the condition for one to two years," she explains. "For Bobette, tests showed that the function of her retina was already 70 to 80 percent below normal."
Morgan was the first person to enroll in an NEI clinical trial of the drug Rituximab. Researchers are studying its effectiveness in decreasing the body's harmful immune response toward the retina.
"I was enthusiastic about participating," Morgan says. "I always encourage people to take part in clinical trials. It's so much better when people take ownership of their condition, learn more about it and become useful to the medical community."
This 18-month pilot study of five patients is the first to look at Rituximab for autoimmune retinopathy. Sen and her colleagues designed the study after noticing that people with other autoimmune eye diseases and similar eye diseases benefited from using the therapy.
"These results encouraged us to go ahead with a clinical treatment trial for autoimmune retinopathy," she explains. "NEI is well-positioned to study rare diseases because we often provide a second opinion about these conditions. An ophthalmologist in the community may never see a patient with autoimmune retinopathy in his or her entire career, but we see one or two people every other month."
The most upsetting aspect of losing her vision, Morgan explains, has been giving up her independence. "I used to hop in my Jeep and off I went," she says. Now, she has difficulty finding a glass to take out of the cabinet, locating the grocery store exit and appreciating a beautiful day because the brightness of the sun hurts her sensitive eyes.
Morgan says that Sen and other NEI doctors have been frank about her diagnosis--she may never regain the vision she lost. However, she says, they are realistic about the possibilities.
"The best I can hope for is that my vision will stabilize," Morgan says. "But I really feel that it is important to participate in a clinical trial because I may help someone else."
For more information about how you can be part of eye research, visit The Eye Clinic.