"This is Surreal": New Breakthrough Therapy for Cystic Fibrosis Gives Family Hope

When Stacy and Mike Riggs became first-time parents in 2003, they were ecstatic to bring home a healthy baby boy. Just two months later, they would feel their world flip upside down when they learned he had a serious chronic illness—one that could dramatically shorten his life.

At Lurie Children’s, Joe was diagnosed with cystic fibrosis (CF), a progressive, genetic disease that causes thick sticky mucus in the lungs and digestive tract, limiting the ability to breathe over time and impairing normal growth. Though the couple was shaken, “everyone on Joe’s medical team calmed our anxieties not just as first-time parents, but as parents of a baby with CF,” Mike says. 

Right away, Stacy and Mike learned how to administer treatments to their infant son—a process of caregiving he would with time take over for himself. Today, Joe completes daily vest treatments to loosen mucus in his lungs while doing nebulizer treatments to thin the secretions, stall bacteria growth and help him breathe easier. Every day, he takes around 30 pills, including digestive enzymes to help his body digest food. These treatments alone require about an hour and a half of Joe’s time each day just to maintain his health. The treatment time increases when he has a cold or an exacerbation, and sometimes hospitalization is also required.

As Joe got older and began taking notice of his treatments, he asked his parents about CF. What was it—and how long could he live with it? With advances in medical research, the life expectancy for someone with CF continues to climb. In the 1980s, life expectancy for someone with CF hovered in the late 20s, while a person born with CF today may have a life expectancy of about 46 years.

Philanthropy plays a critical role in these advances. Support from the Children’s Research Fund enables the Cystic Fibrosis Center at Lurie Children's to participate in clinical research efforts that broaden the understanding of cystic fibrosis and help develop new therapies.

Joe's family learned early about the importance of research, and he has participated in a number of studies. Now 16 years old, with a greater awareness of the potential limitations of his disease, Joe is all the more motivated to participate in research at Lurie Children’s that could eventually cure CF.

At their frequent visits to Lurie Children’s, the Riggs would ask their team of caregivers about research opportunities that Joe might be eligible for. A year ago, the family received the news they had been waiting for: Dr. Susanna McColley, Associate Chief Research Officer for Clinical Trials and Director of Clinical and Translational Research at the Stanley Manne Children’s Research Institute, told the Riggs family Joe was eligible for a clinical trial that they had been researching on their own. The drug, Trikafta, was being tested for children 12 years and older, and Dr. McColley was the Prinicipal Investigator at Lurie Children’s.

With this news, the family felt hope that Joe’s life could possibly be prolonged, and revolve less around time-consuming treatments.

Joe’s lung function had been consistently decreasing and hovered in the low 70s before he started the drug. Within the first month of taking the drug, the family noticed promising results.

“When he blew a 93 percent on his pulmonary function test, we thought, ‘This is surreal,’” Stacy says.

For the first time, Joe’s lung function appeared to be the same as someone without CF.

Nearly a year later, Joe can participate in football practice more often and more comfortably. It’s easier to play basketball, baseball and football without being short of breath.

Joe is not the only patient to benefit from this advancement. This October, the Federal Drug Administration approved Trikafta, which treats the most common gene mutation, representing about 90 percent of the CF population. Previous treatments treated only patients who had two copies of this gene mutation, while Trikafta is also effective in patients who have only one copy. It is also much more effective in patients with two copies of the mutation.

“It feels good to know that I might be helping people all around the world with CF,” Joe says.

“Philanthropy is key because you have to have the infrastructure in place to do these studies,” Dr. McColley says. “By having our state-of-the-art clinical research unit and by having the trained staff and services at the ready, we can attract industry partners and grants. We can get the studies up and going quickly so we can enroll kids to get new medicines approved faster. Philanthropy is critical to being a lead study site for many of the studies for CF and other serious childhood diseases.”

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