- Associated Press - Saturday, December 23, 2017

SIOUX FALLS, S.D. (AP) - As they stood outside Sanford Children’s Hospital waiting for a valet to bring a car, Raena Brendtro’s mother asked her if she wanted to sit down.

Raena responded no. She wanted to stand while she still could.

Two hours earlier, they had arrived for an appointment that changed their lives. After weeks of waiting for lab tests to return, they had learned that Raena was suffering from a rare disorder that was attacking her nervous system, known as Friedreich’s Ataxia. Those who suffer from the disorder are often confined to wheel chairs by the time they graduate from high school. Many die in their 20s after the disease attacks the heart and other organs.

Her parents had been bracing for the diagnosis.

“You kind of hold out hope that somehow it’s going to be something else,” said her father, Dan Brendtro. “The problem is, that the other things that are on the list are brain cancer and things that were just as bad.”

Today, four and a half years later, Raena, 16, is a junior at Lincoln High School. The disease is robbing her of her mobility.

But in a world where freakish coincidences happen, there’s a ray of hope from Raena’s own home town. One year before her diagnosis, Pete Vitiello, a scientist at Sanford Research, had been part of a study on a protein that is central to the disorder. The study found that the protein was linked to an antioxidant enzyme, but from there, the study ended, mothballed.

Now Dan Brendtro is raising money to resume the study, the Argus Leader reported . With Vitiello on board, Brendtro launched a fundraiser this week to raise $75,000 that would enable Vitiello to begin research by the New Year. Typically, research studies rely on foundation or government grants that can take years before the funding comes through. As his daughter slips further into the grips of Friedreich’s Ataxia, Brendtro hopes to bypass the slow, traditional route of funding studies by raising money directly.

If they can get started, the goal, Vitiello said, is to determine whether the antioxidant enzyme helps cause the disease, or whether it’s simply a bystander. They could start to get answers within months of resuming the study.

“This is one of those instances where I feel something can be done,” Vitiello said. “Dan and his daughter are pretty special. That’s been a motivating force for me.”

Three months ago, Brendtro had no idea that Sanford and Vitiello had already made a foray into Friedreich’s Ataxia. But the drive to find a cure put Bendtro on a path to find Vitiello.

It started with the Sioux Falls Half Marathon. Brendtro, a casual runner, decided a few days before the event that he always wanted to run a half marathon, so why not. Following that, he decided he wanted to run a marathon. He contacted his marathoner cousin, Angie Roth, for advice. The two eventually agreed to run the Twin Cities Marathon. They joined with Tom Batta - a fellow Friedreich’s Ataxia parent from Watertown - in running to raise money for Friedreich’s Ataxia Research Alliance. Batta’s team raised nearly $45,000 while Brendtro raised nearly $30,000.

As he pounded out more than 26 miles for a marathon he hadn’t done much training for, Brendtro thought about what he could do next for his daughter and for 4,000 other kids and young adults in the U.S. who also suffer from the disorder. He came up with an idea: Why not raise $100,000 for a study at Sanford Research.

It was naïve, he says now, thinking he could just show up and ask Sanford to take money for a study. But nonetheless, that’s what he did.

What Brendtro didn’t know about was Vitiello and the study that had ended in 2012. Vitiello had worked with the Mayo Clinic’s Dr. Gracia Isaya, a noted researcher into Friedreich’s Ataxia. And although their study had shown promise, it came to an end when its funding also came to an end.

Brendtro’s offer got kicked over to a group that runs Sanford’s registry for patients suffering from rare diseases. A former lab technician of Vitiello’s was working with the group and knew about the mothballed study. That’s how Brendtro and Vitiello got connected.

Vitiello came up with a budget to see the study through. Brendtro started raising the money.

Because it’s a rare disease - only about 100 people a year are diagnosed with Friedreich’s Ataxia in the United States - it doesn’t get the research money that common diseases get, said Jennifer Farmer, the executive director of the Friedreich’s Ataxia Research Alliance. Typically, there’s about $4 million to $5 million a year available for research.

“One of the key things is finding the researchers with the expertise and interest in the disease,” said Farmer. That is only going to get worse as Mayo’s Isaya retires.

The original study that Vitiello and Isaya started opened a new path in conquering the disease, Farmer said. Vitiello’s decision to reopen the study is the next step on that path.

“What’s cool about this project is it’s really opening a new direction,” Farmer said.

Nobody is laboring under the illusion that the study is a sure thing. But even if it ends up demonstrating that the enzyme in question has no role in the disease, researchers can check if off their list and move onto something else.

“I don’t know if it’s going to be important or not, but I’m willing to give it a shot,” Vitiello said.

Brendtro, who is raising money at TheFinishLine.org, has a goal of hitting $75,000 in three weeks.

“What that would mean is we could give a Christmas gift of a research study to the 4,000 kids, young adults, in the United States who have this disease, and to their 8,000 parents,” Brendtro said.

___

Information from: Argus Leader, http://www.argusleader.com

Copyright © 2018 The Washington Times, LLC.

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