Children's Hospital Oakland part of long-awaited drug trial

March 14, 2013 7:54:00 PM PDT
Children's Hospital Oakland is launching a long-awaited drug trial that could finally offer hope to children with a devastating and deadly disease. And researchers now believe the same drug could have an impact on other serious diseases as well.

For 5-year-old Tanner Young, the walls of Children's Hospital Oakland are full of adventure. But his parents are hoping his stay will be life changing as well. Tanner suffers from a rare degenerative disease.

"We took him to his neurologist and they did an MRI," said Tanner's mom Nicole Young. "And that's when they discovered the 'eye of the tiger.'"

The 'eye of the tiger' refers to a signature image in his brain scan. The dark spots that form the eye shapes are actually accumulations of iron. They're caused by a condition called Pantothenate kinase-associated neurodegeneration, which is known by the acronym PKAN.

Four years ago we met several patients; including Travis Brown, whose limbs were so twisted by the disease his parents transported him in a wagon.

"These kids really scared me in a way," Dr. Elliott Vichinsky said. "Because they were so handicapped they couldn't move, they couldn't talk, they were flat."

But now Dr. Vichinsky is beginning an international trial of a drug called Deferiprone which can cross into the brain and help filter out the iron for children like Tanner.

Having used it on a case-by-case basis, with an FDA waver, Dr. Vichinsky was able to stabilize the deterioration in a small number of early patients, including Travis Brown.

"Suddenly over a short time he started looking and was able to move and then he started communicating," Dr. Vichinsky said.

But Dr. Vichinsky says interest in Deferiprone also began to grow because PKAN, while extremely rare, shares some characteristics with other far more well-known diseases.

"We noticed that Parkinson's disease in adults, a large percentage of them, also just have isolated iron in the brain," Dr. Vichinsky said. "And data going back to the 1970's has demonstrated that Alzheimer's disease and dementia are also associated with excess brain iron."

The PKAN trial will include 130 patients on two continents. Children's Hospital Oakland is the only site in the U.S. The goal is to not only to stop the deterioration, but to reverse the effects of this devastating and deadly disease; possibly providing the first hope to families like Tanner's.

"Well, I hope it works and it helps all children with this," Nicole said. "We know it's not a cure, but it will definitely be a step in the right direction."

The trial is being funded by both the FDA and the European Union. Of the 130 patients participating, 40 will be treated at Children's Hospital in Oakland.

written and produced by Tim Didion


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