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GAINESVILLE, Fla. (Ivanhoe Newswire) -- Around the world, there are more than 1,500 gene therapy trials going on to treat everything from Parkinson's to blindness to clogged arteries. Could the key to healing be in the body's building blocks?
Dale Turner was barely old enough to read when doctors told him his world would soon go dark.
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"The doctors said I would be completely blind by age 10," Turner told Ivanhoe.
He has an inherited form of blindness called Leber's Congenital Amaurosis. A defective gene prevents his retina from producing the nutrients his eyes need. His vision was like looking down a gun barrel.
Turner got used to the fact he'd be legally blind for the rest of his life, until his parents heard about an experimental therapy -- injecting new, healthy genes into the eye.
"You always have the hope that there will be something for your incurable condition," Turner said. "Growing up, that there will be something for my eyes someday that will allow me to see."
In a trial, doctors injected hundreds of billions of copies of the working gene beneath the retina in the back of the eye. Four out of six patients regained vision.
"The improvement in vision corresponded exactly where the genes were replacing the defective gene," William Hauswirth, Ph.D., Professor of Ophthalmic Molecular Genetics at the University of Florida College of Medicine in Gainesville, Fla., told Ivanhoe.
To test the success of the surgery, patients in a British study walked through a maze. Before treatment, one patient bumped into walls six times and took almost a minute and a half to get through the course. After surgery, no bumps, and he finished in 14 seconds.
"It's likely the treatment it will last for a long time, if not for the life of these patients," Dr. Hauswirth said.
Turner will never forget the first time he went outside after surgery.
"I could see colors like never before, and it was just like a blue sky I had never seen before, and I had this feeling, like what have I been missing out on this whole time?" he said.
Eye diseases are the focus of just 17 of the 1,537 gene therapy trials going on around the world. Nearly 1,000 trials are searching for a gene treatment for cancer 140 trials are underway for heart conditions.
Brenda Hill's heart is counting on one of those trials.
"It was like when I got up in the morning, I had to pull myself out of bed," Hill told Ivanhoe. "I just couldn't stand up."
She has peripheral artery disease. The arteries that supply blood to her legs are blocked.
"It was like a toothache that never stopped, just ached and ached, just wouldn't stop," Hill explained.
Medication wasn't working, so she enrolled in a gene therapy trial. Doctors inject genes into the leg muscles. The hope -- trigger the growth of new blood vessels.
"Some of the results have been encouraging, and some of the results have been disappointing," Jeffrey Snell, M.D., director of Interventional Cardiology at Rush University Medical Center in Chicago, Ill., told Ivanhoe.
In this trial, those who improved only saw their endurance increase about 5 percent. Researchers are still trying to pinpoint the right genes to help more people.
Hill says for her, it worked. She went from hobbling to hopping across the dance floor a few months after her injection, and cut her meds by half.
"It made me feel good, made me feel like I'm progressing," Hill said. "I'm not ready to sit down yet."
From standing to seeing, gene therapy is still a work in progress, but it could change our world in the future.
HIV is one of the latest targets for gene therapy. In a recent clinical trial, scientists found an injection of an anti-HIV gene could make the body resist the AIDS virus.
More Information
Click here for additional research on Genes to the Rescue
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If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Melissa Medalie at mmedalie@ivanhoe.com
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John Pastor
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This article was reported by Ivanhoe.com, who offers Medical Alerts by e-mail every day of the week. To subscribe, go to: http://www.ivanhoe.com/newsalert/.
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