Alex Parker sits in her chair with a cup of tea in her hands, cosy in a navy blue jacket. She coughs, but it’s not because winter is coming. She has fought this cough from the day she was born. And for the rest of her life, she will rely on small boxes of hope.
Alex has cystic fibrosis.
Kalydeco is a prescription medicine designed to treat the cause of cystic fibrosis in patients who hold the genotype G551D mutation. In Australia, an estimated 250 people, or eight percent of those with cystic fibrosis, have the G551D mutation.
Kalydeco is the first medicine to target the genetic defect making it the most important development in the treatment of the disease since the discovery of the cystic fibrosis gene by Vertex, a global biotechnology company that aims to discover, develop and commercialise new medicines for rare diseases, founded in 1989.
Alex, 25, was diagnosed with cystic fibrosis at six weeks old. She has pursued her career as a freelance commercial photographer, based in Victoria, Australia. Alex has also produced a blog called ‘Kalydeco for Cystic Fibrosis Diary’ to record her experience of taking Kalydeco.
“It’s hard to explain to other people how cystic fibrosis affects a person. Obviously, you can explain that it clogs the lungs and pancreas with mucus, but the problems lay so much deeper than this,” she says.
Before she started taking Kalydeco, Alex’s life revolved around three to four tune-ups at the hospital per year, which in the cystic fibrosis community refers to a preventive course of antibiotics and respiratory therapy. Alex also experienced continuous coughing and a lung function of only 65 percent.
“I used to get up at 6:30am to get ready for university and I start the morning with a coughing fit. I cough so much that I vomit, as there’s so much pressure on my whole body.
“Later I’d go for a run and basically feel like I’ve rolled around in a sea of salt after. Cystic fibrosis people sweat out a lot of salt! Sometimes the coughing fit happens during the run, which is absolutely lovely for strangers to witness as they stand by and wonder whether I’m about to hyperventilate.”
The hope sets in her eyes as she adds, “But now, Kalydeco has helped loosen up my lungs.”
Before Kalydeco’s listing on the Pharmaceutical Benefits Scheme (PBS) in December 2014, the average cost of the treatment was $500,000 a year per patient, meaning the drug was out of reach for most Australian families.
Alex’s lifestyle before taking Kalydeco involved 11 different medications and antibiotics that aimed to fight lung infections, assist in breaking down mucus in her lungs, reduce reflux symptoms, help digest food and assist with asthma-related issues.
Since Kalydeco’s subsidy, it has helped patients and their families regain their hope from this life-threatening condition.
According to Professor Felix Ratjen, at the 2013 Australasian Cystic Fibrosis Conference in Auckland, New Zealand, Kalydeco has significantly increased the life span of patients since 2010. From a previous life span of only 14 years old in 1969, patients can now expect to survive until 37 years old.
“Doctors made the observation that nutrition is really key for survival. We need to learn more about this because this can help us to better understand which factors we actually need to focus on in our treatment,” says Professor Ratjen.
Michelle Adair, the CEO of Cystic Fibrosis Federation NSW, also expects Kalydeco’s performance to increase the life expectancy of cystic fibrosis patients.
“Physically, it is providing extraordinary improvements in their lung function, their weight gain, and their digestive system.
“This profoundly impacts their social and mental health, their motivation and their confidence. It’s the first drug that really has given hope to the cystic fibrosis community,” says Adair.
Back at Alex’s home, she looks at empty space and says, “To say the least, sometimes I feel as if I’m a 25-year-old living in a 90-year-old incontinent body.
“But I have so much to look forward to now that Kalydeco has given me a better chance.”
Alex Parker at a commercial shoot in Melbourne. Photo: Alex Parker.
Noelle Reyes 