World’s First Stem-Cell Study for Cystic Fibrosis

Cleveland medical center treats 39-year-old man

A 39-year-old man with cystic fibrosis (CF) has made history by becoming the first person in the world to receive human adult stem cells in a new study that researchers hope will some day lead to the development of a therapy to reduce the inflammation and infection caused by CF. The study is being conducted at University Hospitals Rainbow Babies & Children’s Hospital in Cleveland, Ohio.

Bob Held from Alliance, Ohio, received an infusion of allogeneic human mesenchymal stem cells (hMSCs) collected from the bone marrow of a healthy adult volunteer. Held was diagnosed with CF when he was 16 months old.

Currently, there is no cure for CF, and the life expectancy for patients who survive into adulthood is approximately 41 years of age.

The phase 1 study will assess the safety and tolerability of hMSCs in adults with CF.

“This is an early-phase trial, and the most important thing is to ensure safety,” said lead investigator James Chmiel, MD. “This study consists of a single infusion of stem cells. We will follow the study participants for a year to make sure it’s safe.”

A total of 15 clinically stable adults with CF will be enrolled in the study. Financial support was provided by the Cystic Fibrosis Foundation.

CF mainly affects the lungs, which fill with sticky mucus as the body’s immune system overreacts to bacteria. The lungs are the source for much of the illness and shortened lifespan seen in CF.

“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 explained. “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.”

The stem cells are donated by adult volunteers who go through a rigorous screening process. In the current study, patients with CF will receive the cells through intravenous infusions.

“Once in the patient’s body, the stem cells track to the area where there is a significant amount of inflammation, and they take up residence there. The stem cells then respond to the environment and hopefully reverse some of the abnormalities,” Chmiel said. “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.”

“This therapy aims to turn down the inflammatory response, not eliminate it, because we still have to keep the bacteria in check. We want to reduce inflammation and the subsequent lung damage caused by inflammation without allowing the bacteria to proliferate,” Chmiel said.

Source: MedicalXpress; January 31, 2017.