Cystic Fibrosis

Researchers at the Wake Forest Institute for Regenerative Medicine (WFIRM) are investigating a potential new therapy for cystic fibrosis (CF).

About 1,000 new cases of CF are diagnosed each year. People with the disease have a gene defect that causes the body to produce thick, sticky mucus that builds up in the lungs and digestive tract. The mucus can lead to life-threatening lung infections and disrupt the normal breakdown and absorption of food. According to the Cystic Fibrosis Foundation, the median life expectancy for people with the disease is the late 30s.

Research Significance

While the genetic cause of CF has been known for more than 20 years, a curative treatment has not yet been developed. The disease is often diagnosed before or shortly after birth. An effective therapeutic intervention at an early stage could prevent the onset of disease.

Research Approach and Goals

Early work in animal models showed that cell therapy reversed or prevented lung disease from occurring and was safe to use. As a result, an ongoing Phase I clinical trial is being conducted in Australia to evaluate cell replacement therapy for the treatment of bronchopulmonary dysplasia in pre-term infants with hopes of an additional clinical trial in the U.S.

In tandem with that study, researchers are collecting immune cells directly from CF patients to test the therapy on the patient’s cells. This is a more personalized approach and demonstrates that not all CF patients are the same, with some whose cells respond better than others. Now researchers are trying to understand why.

Research Highlights and Innovation

A potential therapy for the disease may be a type of stem cell found in placenta (afterbirth) and amniotic fluid that was discovered by researchers at WFIRM. These cells have been shown to have the ability to form normal lung cells and to produce lung-specific genes and proteins, including the gene that is defective in CF patients.

The scientists will first explore the use of these cells as an injectable therapy in animals with a CF-like disease. Magnetic resonance imaging (MRI) will be used to track the location of the cells. The ultimate goal is to develop a cell replacement therapy to restore normal gene function in patients with CF.