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 - By Sandy Leap. July 26, 2012.

Sandy LeapMany NBIA families, like mine, are eagerly awaiting a trial of the iron chelation drug, deferiprone. We hope to learn from a European trial called TIRCON, Treat Iron-Related Childhood-Onset Neurodegeneration, whether the drug can slow symptoms and perhaps even reverse the effects of iron overload in the brain.

Toronto-based ApoPharma, the maker of deferiprone, wanted to hear from affected NBIA families about their hopes for the drug. I was pleased to be able to share that perspective at a recent meeting with company officials and scientists in June.

Patty Wood, president of the NBIA Disorders Association, had met the company’s vice president of medical affairs, Dr. Fernando Tricta, at a TIRCON meeting in Munich last year. He was very moved when Wood described what it was like to care for her NBIA-affected daughter. He invited her to Toronto to share her story with his team, but Patty wasn’t able to go. She asked if I would make a presentation instead.

There was one hurdle. The presentation was set for June 25, the same day as Brittany’s 17th birthday. I couldn’t leave her. Tricta kindly invited the whole family to make the trip to Toronto. Lisa Tran, the company’s project leader in clinical research, and Marjorie Pinto, executive administrator of medical affairs, made the arrangements. Rich, Brittany and I made the 9 ½ hour drive from Virginia.

We shared our story with about 45 people who work in various departments of ApoPharma. All were genuinely interested in learning more about NBIA from a family’s perspective.

We shared our story because that is the one we live, the one we know. But we also made clear that there are many other families living their own stories, all different variations on the same theme. Every family wants a cure. We talked about the many forms of NBIA, the devastation we felt when Brittany was diagnosed, the progression of the disorder, as well as the joys.

We also showed a video of Brittany that included scenes before NBIA robbed her of most of her abilities. It is always very difficult for us to watch that video without becoming emotional. We let them know how difficult it is to live with a disorder that has no treatment. We stressed the importance of the deferiprone trial and of ApoPharma’s contribution to that work.

ApoPharma will be coordinating the use of the drug with the study locations, including the U.S. site, Oakland Children’s Hospital and Research Center led by Dr. Elliott Vichinsky.

We came away feeling the visit was very worthwhile. Tricta and his team now have a family story that makes their work about more than science. We were very grateful for the kindness and caring they showed.

Because it was Brittany’s birthday, we had that milestone to celebrate as well. The company had balloons and a cake. We all sang Happy Birthday. And because Brittany shares her birthday with Patty Wood’s daughter, Kimbi, we wished her a Happy Birthday, too.

 

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