Clinical trials of deferiprone for NBIA to start

March 20, 2012 7:47:24 PM PDT
Doctors at Children's Hospital Oakland are about to begin a clinical trial that could change the lives of children with a devastating disease. The condition was once thought incurable, but an experimental drug has already shown dramatic results.

Delivering the valedictorian speech is an accomplishment for any high school senior, but especially for one who was in danger of losing his ability to speak at all. For nearly a decade, Brent Bonfiglio has been fighting a rare neurodegenerative disease called NBIA -- caused by an accumulation of iron in the brain.

"His speech was becoming more and more slurred, so he and other people were frustrated asking him to repeat what he said," said Veronica Bonfiglio, Brent's mom.

We first met Brent and his mom three years ago, shortly after he began an experimental treatment for the disease. Although his symptoms had not progressed to a critical stage yet, another young patient we met that same day had. Travis Brown's parents wheeled him into Children's Hospital Oakland in a red wagon. Unable to speak, his body was too badly twisted to use a wheelchair.

"I look back and think he went through heck," said Pattie Brown, Travis' mom.

Both families ultimately turned to Elliott Vichinsky, M.D., at Children's Hospital Oakland, who was using a drug called deferiprone to remove iron from the bodies of children with a different condition. He agreed to request a compassionate use waver from the FDA.

"Which means if you have a fatal disease or a disease that's devastating and there's no effective therapy, they'll allow you on an individual basis to try some potential therapy that's unproven," said Vichinsky.

Brent and Travis became the first two patients to receive deferiprone for NBIA. The families say they began to notice a difference within months.

"I can sit without falling," said Brent.

Travis, now uses a wheelchair and can move his arms enough to embrace his mom.

Word spread quickly among families of children with the most common form of NBIA, known as Pantothenate kinase-associated neurodegeneration, or PKAN. Despite the drug's early successes, there simply was not enough data to get deferiprone approved for wider use.

So Vichinsky began writing grant requests, first to the FDA, then the European Medicines Agency, both ultimately agreed to fund clinical trials.

"So now we're integrating both grants to lead a Phase 3 trial that will hopefully result in the approval of the drug worldwide," said Vichinsky.

Vichinsky says the study will include 90 patients. He expects the trial to take about 18 months. If successful, he believes approval could come quickly after that. Both Travis and Brent's families are hoping others will ultimately benefit from the drug they helped pioneer.

"I guess I would say hang in there guys, and help will come to you," said Brent.

And perhaps deliver the same second chance at life, that Brent has taken advantage of.

One cautionary Note: Vichinsky is concerned about families potentially obtaining deferiprone from third party sources. The dosages used for treating NBIA are drastically different than other conditions deferiprone is used for.

Children's Hospital clinical trial contact - Nancy Sweeters: 510-428-3885 ext. 4151
For more info: NBIADisorders.org

Written and produced by Tim Didion


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