According to a story from the Daily Record, cystic fibrosis patient Kelli Gallacher found that she was able to stabilize her disease using the drug Symkevi. Now that her own disease is under control, Kelli and her mother Maggie both plan to return to their work with Flutterby FUNdraisers, a charity that they started in 2007 that focuses on raising funds for cystic fibrosis gene therapy.
About Cystic Fibrosis (CF)
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
Finding a Gene Therapy for Cystic Fibrosis
The charity has been focused on the work of Dr. Arash Saleh, a scientist working at the UK CF gene therapy consortium. Symkevi has the potential to be an effective therapy for as many as 90 percent of cystic fibrosis patients, but for the remaining ten percent, gene therapy is probably the best option. Dr. Saleh provides regular updates on progress from the research so that donors have a good understanding of where their money is going and the impact that it is having.
The consortium recently partnered with a pharmaceutical company that will help facilitate the continued development of a gene therapy for cystic fibrosis. It will be tailored to the small number of patients with the rarest variants that can’t be effectively treated with Symkevi.
