New medication for cystic fibrosis is saving people's lives.

Sadly, only 40% of France's 7,500 people with cystic fibrosis can benefit from this therapy.

Man with cystic fibrosis and walking difficulties, as captured in this photograph.

PARIS: Cystic fibrosis sufferer David Fiant claims he "could no longer identify if I was living to heal myself or healing myself to live" due to the gruelling treatment he had to endure.

He required oxygen therapy, a six-hour daily care regime, and three to four weeks of antibiotic infusions annually while awaiting a lung transplant.

Then, he was among the first in France to try out a novel, triple-drug treatment for the rare, potentially fatal illness.

"One morning, I decided to take my first dose of the medication. The symptoms had begun by 3 p.m "What he had to say was.

The 40-year-old said he had never been able to do things like "accompany my daughter to buy comic books," climb 15 steps at once or take a shower without help in years.

After starting medication, Fiant's doctor could hear air moving through Fiant's lungs during an assessment. He claimed that had never happened before.

Subsequently, Fiant's treatment plan was substantially scaled back, and he assumed leadership of the French advocacy group Vaincre la Mucoviscidose (Defeat Cystic Fibrosis).

The treatment results were "amazing," he said, although he was careful to point out that they were not miraculous.

He emphasised that "there is currently no cure for cystic fibrosis."

Increases in life expectancy

Somewhere in the neighbourhood of 70,000 people around the world are thought to be living with this genetic condition, which is brought on by a faulty gene and manifests itself in the form of mucus buildup in the lungs, digestive system, and elsewhere.

It shortens people's lives by making it hard for them to breathe, and long-term problems like infection further reduce their life expectancy.

However, since it was initially licenced in the United States in 2019, the triple-pill medication taken by Fiant—known as Kaftrio in Europe and Trikafta in the United States—has had a significant impact.

According to the Cystic Fibrosis Foundation, life expectancy for those born in the United States with cystic fibrosis between 2017 and 2021 has increased to 53 from 38 in the previous decade.

To treat cystic fibrosis, Kaftrio repairs a mutant flaw in the CFTR gene, which has been heralded as a significant medical advance.

Both the European Medicines Agency and the British government approved the treatment in August 2020.

The problem is that only 40% of France's 7,500 people with cystic fibrosis can benefit from it.

Children under six are not eligible for treatment, and 15% of people have an ineffective genetic profile.

About 900 people who have transplants of significant organs like lungs or livers are also ineligible.

Ahead of a "sword of Damocles," 

41-year-old Sabrina Perquis waited with "great anticipation" for the new medication to arrive in France.

However, she discovered 15 years ago that she could not take Kaftrio because of her transplant.

It was a hard punch, she said.

She continued, "Rejection is always a possibility after a transplant, so you are constantly living with a sword of Damocles over your head."

"The favour we're asking for is to be remembered. The hunt for a treatment that works for every patient must continue."

Several research projects, each at a slightly different level of development, are looking for a treatment for people with extremely rare genetic abnormalities.

Vaincre la Mucoviscidose vice president Pierre Foucaud said that other questions remain, such as whether the treatment completely blocks the progression of the disease or merely slows it down.

From 2019 to 2021, the number of French people with cystic fibrosis undergoing an organ transplant dropped from 21 every quarter to only two. Doctors hoped Kaftrio would eliminate the need for transplants.

But will the delay of these transplants be simply 10–15 years? The question came from Foucault. Our best guess is "no."

Even while Kaftrio "opened up a tremendous hope for all patients," he said, further research is still needed.