Years of Research, New Hope

For most of his life, Andrew Sabatino understood what it meant to live with a serious chronic disease. Diagnosed with cystic fibrosis (CF) at age 3, he grew up during a time when the outlook for many patients was far different than it is today. Treatments focused on managing symptoms, rather than addressing the underlying cause of the disease.

Andrew learned to adapt. He played sports, went to school and stayed active, but his daily routine revolved around treatments. “My goal was to try to think about it as little as possible,” Andrew said. “I didn’t want other kids to know I had CF.”

He spent hours every day using inhalers, airway clearance vests and medications to loosen the thick mucus that clogged his lungs and digestive system.

Now, advances in medicine, fueled by decades of research and clinical trials, are transforming care for patients with complex lung diseases.

For cystic fibrosis patients, the breakthrough came with Trikafta, a medication developed to treat the root cause of the disease. The drug reached patients after years of clinical research and studies, including clinical trials conducted at The University of Kansas Health System.

Andrew started taking Trikafta in January 2020. “Game changer is an understatement,” he said. “It was life changing.”

For physicians who have spent their careers treating CF, the results were unlike anything they had seen before.

“It is a miracle,” said Steve Stites, MD, chief medical officer and pulmonologist at The University of Kansas Health System. “It helps change the dynamic of how your lung works. You can breathe much more effectively and don’t trap infection in your lung.”

Doctors say the medication represents a fundamental shift in how the disease is treated. “Trikafta is the first therapy we have approved that actually treats the underlying cause of CF,” said pulmonologist Joel Mermis, MD.

Earlier treatments targeted symptoms such as mucus buildup and infections. Trikafta works by helping a defective protein function more normally, addressing the disease at its source.

For Andrew, that scientific advancement translated into a life that feels far more ordinary.

“Once Trikafta came out and I started using that, I wouldn’t even notice if I’d missed a treatment because I just felt so normal,” he said. “It was pretty amazing.”

The impact extends far beyond daily symptoms. When Andrew was growing up, many patients with CF expected significantly shortened lives. Today, doctors are having very different conversations.

“For a child born today with cystic fibrosis who can go on Trikafta, you probably are looking at a pretty normal life expectancy,” Dr. Stites said.

That progress has allowed Andrew to focus on milestones that earlier generations of CF patients often worried they might never reach. He is now a husband and father to his daughter, Juliet. “There’s definitely a lot to be hopeful for,” he said.

Researchers are already working on the next generation of treatments, including gene-editing and messenger RNA therapies aimed at helping the approximately 10% of CF patients who do not benefit from Trikafta. The goal, Dr. Stites said, is to continue pushing science forward “until it’s done.”

The same commitment to research is changing outcomes for patients with pulmonary fibrosis. Through earlier detection, clinical trials and new medications, patients such as Craig Ackerson and Madonna Mergemeyer are remaining active years after diagnosis. Craig, who was once told he might live only 3 to 5 years, continues to golf, kayak and hike. Madonna, diagnosed before her symptoms developed, has participated in multiple clinical trials and plays pickleball 3 times a week.

Together, their experiences show how research and clinical trials do more than advance science. They give patients something equally important: time, quality of life and hope for a future that once seemed out of reach.

Watch Andrew’s full story on Choices, Hope and Science.