Ben Schuster, photo

Once a week, 12-year-old Ben Schussler boards a 9 a.m. plane near his hometown of Eagan, Minnesota, and flies – usually with either a parent or a “Ben’s Team” member – to the Eastern Iowa Airport in Cedar Rapids, Iowa. From there, he and his escort drive 25 minutes to the clinical research unit located within University of Iowa Hospitals & Clinics.

Clinic staff all know Ben, and he enters the clinic and heads to his infusion bay, where for an hour he’ll receive an experimental treatment for Duchenne muscular dystrophy (DMD). After the infusion, staff will monitor Ben for side effects, and on some days he has to have a blood draw to test progress. He’s usually back home by 7 or8 p.m. that same day.

Ben is part of a clinical trial testing to see whether a new therapy will change the progression of Duchenne muscular dystrophy, says Katherine Mathews, MD, pediatric neurologist at University of Iowa Stead Family Children’s Hospital and the UI’s principal investigator for the study in which Ben is participating.  DMD is a genetic disorder marked by progressive degeneration of the muscles and increasing weakness. The disease mostly affects boys, although in rare cases it can also affect girls.UI Stead Family Children’s Hospital is also a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) and demanding optimal care for all people with Duchenne.

As of January 2019, Ben is about halfway through the second of two 96-week rounds of the clinical trial, both of which include weekly infusions. In the first part of the trial, participants didn’t know if they were being given a placebo or the actual treatment. In the second round, all participants are given the treatment.

“We think he was given the actual treatment in the first round, but we’ll never know for sure,” says Missy Schussler, Ben’s mom. “Ben broke his leg during that part of the trial and it healed well and he’s able to walk. That’s not usually the case for kids with DMD.”

When Ben was diagnosed with DMD at age 4, his parents, Missy and Frank Schussler, started researching the disease and searching for clinical trials. After years of research, a clinical trial compatible to Ben’s particular form of DMD opened up at the University of California-Los Angeles (UCLA).  Ben was screened and accepted into the trial. The same clinical trial was later started at UI Stead Family Children’s Hospital. He continued quarterly trips to Los Angeles for testing, but was able to move his infusions to Iowa.  Often times the trips to both sites meant missing two days of school for Ben.

The Schusslers are just one of dozens of out-of-state families who travel to Iowa City for a variety of pediatric and adult clinical trials. Many come from neighboring states, but others travel even farther.

“In many cases, this is the closest center active in the specific trial the family or patient wants to be involved in,” Mathews says. “In other cases, we are able to get a subject enrolled sooner than another site – similar to the reason Ben went to UCLA first. In rare instances, we may be the only site offering the study.”

Almost half of the studies done in the clinical research unit are pediatric studies. Mathews says children are different from adults, and there are important reasons for investigating new treatments in children.

“Many clinical trials are for diseases that start in childhood, so the best time to start treatment is childhood, before the disease is advanced,” Mathews says. “Other diseases are rapidly progressive and children won’t live to adulthood without effective treatment  so the treatment has to be in childhood. In other cases, children’s metabolism is different than adults and it is important to test a medication in children rather than simply assuming they will respond the same way an adult does.”

“We feel that the fact that we can offer the opportunity to participate in clinical trials for many pediatric diseases allows us to offer an important option for our patients and their families,” Mathews adds. “Participation in a trial is a major commitment on the part of the child and the family, but we learn something important from every trial, even those that don’t result in a change in therapy. We all appreciate the commitment of the patients and their families to clinical research and are happy to participate in the effort to improve outcomes for all of our patients.”

Missy and Frank decided early on in Ben’s diagnosis that they would do what they could to help their son but acknowledge it hasn’t been easy.

“It’s beyond stressful,” Frank says. “It would be impossible without the help we have from our family and friends taking turns as Ben’s escort.”

“It’s not for the weak, that’s for sure,” Missy adds.

Like Ben’s study, many other clinical trials are carried out in the clinical research unit (CRU), located inside UI Hospitals & Clinics. The unit looks like a typical medical clinic, with waiting areas, infusion suites, and patient rooms.

Though Ben brings his own tablet when he travels, he wanted to provide more things for patients – kids and adults alike – to do when they were in the unit for several hours as often required for the study. With the help of a friend at home, Ben donated gaming equipment to the clinical research unit as well as a cash donation to be used to help fund an initiative, spear-headed by Holly Clubb, RN, and Jackline Wangui-Verry, MSN, RN, to make the CRU more appealing and comfortable for children participating in studies.

“We just wanted to make the area more patient-friendly, give the patients something to do while they’re here,” Missy says.

For more information about Duchenne muscular dystrophy clinical trials carried out at the University of Iowa, please contact Carrie Stephan, nurse research coordinator, at  carrie-stephan@uiowa.edu. For more information about the UI CRU, please contact Jackline Wangui-Verry at jackline-wangui-verry@uiowa.edu or 1-319-353-6788.

Related Medical Services