The fight against cystic fibrosis (CF) has taken a major step forward, with pioneering research showing that cells causing the debilitating genetic disorder could be successfully replaced with healthy ones.
What Is Cystic Fibrosis?
Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system. This causes lung infections and problems with digesting food.
In the UK, most cases of cystic fibrosis are picked up at birth using the newborn screening heel prick test.
Symptoms usually start in early childhood and vary from child to child, but the condition gets slowly worse over time, with the lungs and digestive system becoming increasingly damaged.
Treatments are available to help reduce the problems caused by the condition and make it easier to live with, but sadly life expectancy is shortened.
What Cystic Fibrosis Stem Cell Therapies and Treatments Are Being Investigated?
Research Study 1
Research into CF stem cell treatment was recently published in the journal Stem Cell Research and Therapy applies cell transplantation therapy by Dr Nigel Farrow, a Post-Doctoral Research Fellow from the University of Adelaide’s Robinson Research Institute alongside Associate Professor David Parsons head of the research team based in the Women’s and Children’s Hospital.
“Our research which applies stem cell transplantation, involves harvesting adult stem cells from the lungs of CF patients, correcting them with gene therapy, and then reintroducing those cells back into the patient,” says Dr Farrow.
“The newly transplanted adult stem cells pass on their healthy genes to their ‘daughter cells’ providing a constant means to replenish the airways with healthy cells, and thereby combatting the onset of cystic fibrosis airway disease,” he said.
Research Study 2
The Phase 1, open-label study (CEASE-CF, NCT02866721), supported by the Cystic Fibrosis Foundation, will enrol 15 adult CF patients at an Ohio centre. All will be given a single infusion of human mesenchymal stem cells (hMSC), which are found in the bone marrow.
James Chmiel, MD, of University Hospitals Rainbow Babies & Children’s Hospital 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.”