‘Extraordinary Measures’ film features treatment given at UNMC

Fred Haan didn’t need to see “Extraordinary Measures,” a movie about a fatal genetic disorder, on the big screen to know how it ends. He lives the story of Pompe disease every day.

Symptoms of this rare degenerative muscle condition became evident in his early 30s via upper body weakness. A muscle biopsy revealed the diagnosis. Over time, Haan has lost his ability to walk. While his strength has gradually decreased, the 55-year-old’s hopes are still high.

Extraordinary Measures is based on a true story about a family with two children who have Pompe disease, a fatal genetic disorder. UNMC was involved in the clinical trials for the treatment featured in the movie and currently treats three patients with the disease.

Every other week, the Sioux Center, Iowa, native makes the 2 ½ hour trek to UNMC for a four-hour infusion of enzyme replacement therapy.

“We didn’t have treatment for Pompe disease until a few years ago when the Food and Drug Administration approved the replacement enzyme, Myozyme, which helps prevent the progression of the disease,” said William Rizzo, M.D., a pediatric geneticist with the UNMC Munroe-Meyer Institute (MMI).

Some of the first clinical trial patients were treated right here at UNMC. Currently, three patients with Pompe disease receive the treatment described in the movie here at UNMC. Haan receives his treatment as part of a clinical trial. He passes the four hours with naps, television, reading material or chit-chats with his wife of 35 years, Cheryl, and their three grown children.

Haan did show some improvement in his symptoms early on; however, the enzyme replacement therapy is only expected to slow the progression of Pompe. Still, it’s better than no treatment at all.

“In the past we just threw up our hands, but now we have something to offer people,” Dr. Rizzo said. “So many doctors don’t think about the disease because they don’t know about it. Hopefully the movie will generate more awareness.”

Haan knows it already has.

“I was watching the movie and suddenly it hit me, all these people in the theater are going to know about (Pompe disease),” he said. “We need that awareness in order to keep the research going and help find a cure.”