CHAPEL HILL, N.C. Even if the patients hadnt been as young as 4 months old, the surgery would have been harrowing: six holes bored into the skull, six tiny tubes inserted directly into targeted parts of the brain, then a solution containing hundreds of millions of viruses pumped in.
But the rare degenerative illness it fights is even scarier. Canavan disease strikes infants, essentially making the brain attack itself with a toxic chemical, stopping and reversing development. It then kills, usually before age 10.
The procedure used in the study though, slows Canavans progress and improves and may even help extend their lives, according to a study that appeared last month in the online journal Science Translational Medicine. It does that by using viruses as microscopic trucks to deliver missing genetic material precisely where its needed in the kids brains.
This form of gene therapy was created at UNC-Chapel Hill, and the viral vehicle and genetic cargo used in the study were developed there at the medical schools Gene Therapy Center. Center director R. Jude Samulski was a senior author of the study, which began in 2001 and tracked 13 children who received the treatment.
The youngest was 4 months old, the oldest 83 months when they got the operation. After the procedure, the researchers, led by Paola Leone, an associate professor of cell biology at the University of Medicine and Dentistry of New Jersey, followed them to see how the therapy affected their illness.
To an outsider, the results might not even be noticeable. To the families, though, the changes began quickly and were nothing short of dramatic.
Right away, we saw a significant change in his eyes, said Jordana Holovach of Rye, N.Y., whose son Jacob participated in the study. He then was able to regain some of the strength he had lost in grasping, improved his head control, his immune system clearly got better, and he was even with some assistance able to take steps, something we never thought wed be able to see.
Jacob, who had the operation in 2001, will be 17 years old in February. He has grown so much that he doesnt have the strength to take steps now, but attends a mainstream high school, albeit with substantial help.
Ilyce Randell of Buffalo Grove, Ill., whose son Max was diagnosed when he was little more than 4 months old, said that he hadnt seemed to use his eyes at all before the operation, which he had when he was 3 years old. Not long after it, though, he clearly was focusing on things, and began using his arms.
To this day, his sight is improving, and he barely needs glasses. Thats a huge thing, when youre trapped in your body, she said.
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UNC scientists use virus to deliver genetic material to slow kids' illness