Even if the patients hadn't 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, and stopping and reversing development. It then kills the child, usually before age 10.

There is a procedure that appears to slow Canavan's progress and improves -- and may even help extend -- sufferers' 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.

This form of gene therapy was created at University of North Carolina-Chapel Hill, and the viral vehicle and genetic cargo used in the study were developed there at the medical school's 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 nearly 7 years old 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 we'd 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 doesn't 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 hadn't 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.

there's nothing you can do," she said.

More here:
New gene therapy shows promise for kids with Canavan's disease

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