'Tremendous hope': New cystic fibrosis treatment changing lives

1 day ago 33

PARIS - Mr David Fiant says his cystic fibrosis and the arduous therapy it required was so bad he "could no longer tell if I was living to heal myself or healing myself to live".

While waiting for a lung transplant, he was on oxygen therapy and had a six-hour daily care regime, as well as three to four weeks of antibiotic infusions a year.

Then, he became one of the first people in France to take a new triple-drug treatment that has been hailed as a gamechanger for people with the rare life-threatening disorder.

"I took my first dose of the medicine one morning. By 3pm, I was feeling the first effects," he said.

For the first time in years, he was able to shower by himself, climb 15 steps at once and above all "accompany my daughter to buy comic books", the 40-year-old said.

When Mr Fiant's doctor examined him after he started the treatment, he could hear air circulating in his lungs. That had never happened before, he said.

Mr Fiant's regime of care has since been drastically cut back - and he has become the president of the French non-profit Vaincre la Mucoviscidose (Defeat Cystic Fibrosis).

While the treatment's results were "spectacular", he emphasised that it was no miracle.

"There is still no cure for cystic fibrosis," he stressed.

Life expectancy rising

More than 70,000 people worldwide are estimated to suffer from the inherited disease, in which a defective gene causes sticky mucus to build up in the lungs, digestive tract and other parts of the body. It causes difficulty breathing, as well as long-term complications such as infection that leads patients to have significantly shortened life expectancies.

However, the triple-pill treatment taken by Mr Fiant, which is sold as Kaftrio in Europe and Trikafta in the United States, has had a major impact since the US first approved it in 2019.

The life expectancy of people with cystic fibrosis born between 2017 and 2021 in the US is now 53, up from 38 a decade ago, according to US non-profit organisation the Cystic Fibrosis Foundation.

Kaftrio has been hailed as a breakthrough because it targets the underlying cause of the disease, repairing a mutated defect in the CFTR gene. The treatment was approved by the European Union's medicines agency and by Britain in August 2020.

However, out of the 7,500 cystic fibrosis patients in France, only 40 per cent are able to use the treatmen...

Read Entire Article