First-of-its-kind Study Explores if Stem Cells Might Lessen Inflammation in CF Patients

An Ohio cystic fibrosis (CF) patient was given an infusion of adult stem cells in a pioneering study that researchers hope will lead to therapies that reduce inflammation and infections linked to the disease.

“I am hoping the future generations of CF patients can get better treatments and that eventually a cure will be found for them,” Bob Held, 39, of Alliance, Ohio, said in a news release.

The Phase 1, open-label study (CEASE-CF, NCT02866721), supported by the Cystic Fibrosis Foundation, will enroll 15 adult CF patients at an Ohio center. All will be given a single infusion of human mesenchymal stem cells (hMSC), which are found in bone marrow. The cells will come from healthy adult volunteers selected after a rigorous process. [More information, including enrollment information, is available through this link.]

“It was a very exciting day for us with the very first participant in the first stem cell trial for cystic fibrosis,” said James Chmiel, MD, of University Hospitals Rainbow Babies & Children’s Hospital, and the study’s principal investigator.

Chmiel and his team will follow the participants for a year to ensure the safety of the treatment, which relies on the cells’ ability to detect environmental changes, such as inflammation.

“Once in the patient’s body, the stem cell tracks to the area where there’s a significant amount of inflammation, and they take up residence there,” Chmiel said. “The stem cells then respond to the environment, and hopefully reverse some of the abnormalities. We hope in future studies to demonstrate that the stem cells reduce the infection and inflammation and return the lungs to a more normal state.”

According to Chmiel, the treatment can reduce lung inflammation without completely eliminating it. (Inflammation is the body’s protective mechanism against bacterial infections.) The goal is to reduce patients’ symptoms while keeping bacterial proliferation at bay.

“One of the issues in CF is that people with the disease get bacterial infections in their lungs, and these bacteria incite a vigorous and excessive inflammatory response,” Chmiel said. “It’s actually the body’s inflammatory response that damages the lungs. The inflammatory response tries to eliminate the bacteria, but it’s not successful. Instead, the inflammatory system releases molecules that damage the individual’s own airways. The lung disease causes much of the illness and is responsible for the majority of the mortality of the disease.”

If proven safe, the therapy could help improve CF patients’ life expectancy.

“While there’s been a tremendous increase in survival for people with CF from when I entered the field in the 1990s, that’s still not good enough,” Chmiel said. “While we’ve made great progress, we still have a long way to go.”