Experimental treatment for PKAN successful in Oakland

February 9, 2010 12:00:00 AM PST
An experimental treatment for a rare and fatal disease is changing the life of a young boy.

When we first brought you this story, doctors at Childrens' Hospital Oakland were about to begin giving him the investigational drug. Now just months later, his parents say it is already making a dramatic difference.

Watching 12-year-old Travis Brown raise his arm to his chin may not seem like an amazing accomplishment, unless you had seen him just a few months ago.

"He couldn't move his arms at all," says his mom Pattie Brown. "One arm was pinned to his side where you couldn't lift it, even to put a thermometer in it."

Last year, Travis' mother and father had to wheel him into Children's Hospital Oakland, lying in a toy wagon. A rare condition known as PKAN, caused by a concentration of iron in the brain, had left his body a twisted lump.

"He couldn't lift his head, couldn't lift a muscle," Pattie says. "Couldn't move hands, couldn't do anything."

Moving from that wagon to sitting up in a wheel chair is just one sign of Travis' dramatic improvement. But, the boy who could not move his arms can now be seen shadow boxing with his father.

"I think Travis is like a miracle," says Dr. Elliott Vichinsky.

Vichinsky believes the difference is an investigational drug called Deferiprone. It is not approved in the U.S., but he was able to obtain a compassionate use waver from the FDA to treat Travis.

"I got approval to use this drug called Deferiprone, which is the only kelator or in another words a drug that removes iron, and its the only one in the world that can get into the brain and take iron out," says Vichinsky.

Vichinsky had already used Deferiprone to treat a different iron-related condition, but nobody knew if the drug could remove enough iron from the brain to reverse the severe symptoms of PKAN.

Within months of his first treatment, his parents began to notice the changes. Travis can now sit and hold his head up on his own. His legs have regained enough flexibility that doctors are even discussing possible surgery to straighten his feet.

"I mean, to have him stand again, it would be a miracle," says Jacob Neufeld at Children's Hospital.

Still, nobody knows if Travis will return to being the completely healthy child who had enjoyed camping and hiking just a few years ago. Doctors caution that they do not know the long term effects of Deferiprone.

"Every day I walk like on eggs. I worry that if the improvement he shows is going to continue and stay that way or will it not, but right now the storm cloud has lifted over Travis," Vichinsky says.

So has the veil of silence. Travis' disease had progressed to the point that he had not been able to speak for months, until one recent morning when his mother finally heard his voice again.

"First he said, 'Momma.' First, I thought 'no.' Then this morning, he said 'Momma' again and I said, 'What did you say?' He said, 'Momma,'" Pattie says.

Doctors at Children's Hospital are now planning a clinical trial in the spring with up to 30 children suffering from PKAN.

To contribute to the Travis Brown Foundation, make checks payable to "Rotary International --Travis Brown" and send to:
Lathrop Police Department
15597 7th Street
Lathrop, CA 95330

Written and produced by Tim Didion.