Researchers have been studying an experimental treatment for cystic fibrosis that is thought to be effective regardless of a patient’s genetics. The research is still in the early stages, but the effects on mice and cell cultures were positive. The full article can be found here, at Oregon State University Newsroom.
About Cystic Fibrosis
Cystic fibrosis (CF) is a condition that causes sticky mucus to build up in certain areas – particularly the lungs and digestive system. As a result, people can experience more frequent infections, difficulties digesting food, breathing issues (such as shortness of breath, coughing, and airway damage), and reduced growth and weight gain, amongst other possible symptoms. It is an inherited condition that is estimated to affect about 30,000 people in the US.
About the Experimental Treatment
The researchers from Oregon State University and Oregon Health and Science University Hospital have developed an experimental treatment for CF that could be inhaled at home. It is a molecular medicine that contained chemically modified CFTR messenger RNA that is believed to help cells to produce a functional form of a protein involved in cell chloride and water transport. In this way, it is hypothesised to support respiratory function in patients with CF.
The possible treatment is still in very early stages of development. However, a study published in the journal Molecular Therapy found that the therapy was successful in cultures of cells taken from patients, and in mice that had been genetically engineered to not have a functioning CFTR gene (a gene implicated in CF).
The Potential Benefits of the Experimental Therapy
Scientists claim that this possible therapy may overcome some of the issues around treatments that patients with CF currently face.
For example, it works on all patients irrespective of the specific genetic alteration that causes their condition. Over 1,000 different genetic alterations have been linked to CF, and this diversity can cause researchers difficulty when developing treatments. However, the therapy tested in the study is expected to work on all patients.
A second issue brought up by Ema Robinson, a study author and research assistant at the College of Pharmacy, is that it can be difficult for patients and their families to consistently comply with treatments. Patients often need to visit a hospital several times a week for long periods, and logistically this can often be very difficult to always manage, and sometimes impossible. In contrast, the new experimental therapy could likely be taken at home making consistent treatment much easier for patients.
