Jean Bennett and Albert Maguire: Gene Therapy to Reverse Near-Blindness
America's Obesity Problem
(by Mark Weber)
Bennett bred a virus to carry the gene. Maguire injected it into a patient's eye. And there was light
For Jean Bennett and Albert Maguire, sharing a human brain in a neuroanatomy class stirred up a personal chemistry that produced a marriage and three kids.
It led to a 25-year professional partnership as well, a collaboration that has yielded one of the first successful demonstrations of gene therapy in humans.
The partners have restored much of the vision in patients who have a rare genetic form of severely impaired eyesight called Leber's congenital amaurosis, in which a mutated gene prevents the retina from manufacturing a nutrient vital to eye health. The technique eventually could be tried to treat macular degeneration.
The trickiest part for Bennett, a gene therapy researcher at the University of Pennsylvania School of Medicine, involved finding a virus that would deliver normal copies of the defective RPE65 gene to malfunctioning retinal cells--harmlessly. "We needed a virus that wouldn't replicate or cause a dangerous immune reaction," she says.
Manufacturing the gene-carrying virus and injecting that first patient was done next door at Children's Hospital of Philadelphia , where Maguire is a pediatric eye surgeon. It was, Maguire says, a bit terrifying. Foremost on his mind was Jesse Gelsinger, the 18-year-old who died a decade ago at the University of Pennsylvania hospital from a severe immune response during a gene therapy study of his metabolic disorder. But the surgeon ultimately forged ahead. "If my own son had had this disease, I would have let him be the first to have the treatment," says Maguire, "which to me is the highest level of stringency."
The treatment surpassed all expectations. The first three young adults treated--all legally blind, with vision worse than 20/200 and a small visual field, like looking down a gun barrel--saw so much more clearly that they could even read additional lines off an eye chart, according to the results published in last year's New England Journal of Medicine.
The patients, all of whom had only their worse eye injected, are thrilled, says Bennett.
"Their main question is, 'Can I have my other eye injected?' " Most striking are the latest results, not yet published, for five children who have received the therapy over the past year. A 9-year-old Italian boy who once used a white cane, she says, now plays soccer for the first time in his life. And a video presented at a recent gene therapy meeting shows an 8-year-old American boy easily navigating an obstacle course of steps and hanging signs. A presurgery video shows him on a similar course completely bewildered, unable to take a step forward.
How long the inserted genes will last is uncertain, but the dog who got the first treatment (and whose silver-framed photo sits in Bennett's office), still has his vision nine years later.
Bennett, who hears every day from those hoping for cures for their blindness, says the next step is to seek approval from the Food and Drug Administration so that more of the 3,000 Americans with Leber's can regain at least some of their sight.
While gene therapy may still prove to be too tricky for metabolic disorders, its potential for other eye diseases appears promising.
"I think we redeemed gene therapy, in a way," says Maguire.
Anthony Atala: Grinding Out New Organs One at a Time
Anthony Atala was the first to build a functioning organ from scratch -- a bladder made cell by cell -- and put it into a patient, a child whose own bladder was congenitally deformed. Since that breakthrough a decade ago, the 50-year-old pediatric urologist, director of Wake Forest University's Institute for Regenerative Medicine, has moved on to cobbling up bones, heart valves, muscles, and some 20 other body parts.
Fear of Crowds. When You Need Help for Anxiety
Certain people find the feeling of a crowd pressing in on them to be extremely stressful. When such a feeling causes a panic attack, that's a sign of the anxiety condition claustrophobia. Feeling panicked by the mere presence of strangers is a condition known as agoraphobia
Repeated Exposure to Stomach Acid May Result in Barrett's Esophagus
Yvonne Romero, M.D., Gastroenterology and Hepatology, Mayo Clinic
Barrett's esophagus does increase your risk of developing esophageal cancer. But esophageal cancer is rare. About 7,000 cases of adenocarcinoma, the type of cancer associated with Barrett's esophagus, are diagnosed each year in the U.S. Less than 10 percent of people who have Barrett's esophagus ever develop esophageal cancer. And, when caught early, this type of cancer can often be effectively treated. The key is ...
If you have heartburn two or more times a week, it could be serious. The problem may be much more frequent than the occasional bout of indigestion. Frequent heartburn can also be a symptom of a more serious problem called Gastroesophageal Reflux Disease or GERD.
Guillain-Barre Syndrome a Rare But Serious Disorder
P. James Dyck, M.D., Neurology, Mayo Clinic
Guillain-Barre syndrome is an inflammatory disorder that can cause dramatic weakness and even complete paralysis. Most people recover but may require months, or even years, to regain their strength
Lifestyle Changes May Help Patients Cope With Peripheral Neuropathy
P. James Dyck, M.D., Neurology, Mayo Clinic
Peripheral neuropathy occurs as a result of nerve damage and often leads to pain, tingling, numbness and a lack of sensation in the limbs, particularly the hands and feet. Treatment may ease symptoms, and some lifestyle changes may also help
Avoid Tick-Borne Illness
Harvard Health Letters
The deer tick (also called the black-legged tick), which can carry and transmit the bacterium responsible for Lyme disease. Considered somewhat rare in the mid-1980s, Lyme disease is now the most common vector-borne illness in the United States. About 20,000 cases are reported annually to the U.S. Centers for Disease Control and Prevention
Some blood pressure drugs, particularly Cardura and Inderal, can cause weight gain, even up to 10 pounds a month in some susceptible individuals. But there are ways to control it ...
Garlic is prized as a culinary ingredient and herbal remedy by virtually every culture. The written and unwritten record of its virtues reaches back thousands of years.
(c) 2009 U.S. News and World Report