Cystic Fibrosis: This new treatment that is changing the lives of some patients

Although it cannot cure them, a new treatment for cystic fibrosis has literally changed patients’ lives by reducing the effects of the disease. But not everyone can benefit from it.

Its effect is “spectacular,” said Tuesday David Fiant, president of the Vaincre la Mucoviscidose association, who was one of the first to test it.

Kaftrio, from the American laboratory Vertex, belongs to an innovative class of drugs against this genetic disease, which inexorably damages the respiratory and digestive systems and was once fatal to children and adolescents.

Reimbursed in France for a year, it is considered by associations as a revolution capable of turning cystic fibrosis into a chronic and stabilized pathology for some patients.

In the form of tablets

This triple therapy (a combination of three molecules), administered in the form of lifelong tablets, significantly reduces the effects of the disease, particularly debilitating lung diseases.

“Just before I started Kaftrio, I was on oxygen therapy awaiting a lung transplant,” said David Fiant, 40. “Six hours of care a day (physiotherapy, aerosols…), three-four weeks of IV fluids a year: I didn’t know more whether I live to treat myself or care to live.”

“I took a first dose of medicine one morning; at 3 p.m. I felt the first effects,” he continues. For the first time in years, he could “take a shower alone”, “climb 15 steps at once” and, above all, “accompany (his) daughter to the comic purchase”.

When his doctor examines him shortly afterwards, he “hears the air circulating” in his lungs. That had never happened to him before.

Since then he has significantly reduced the physio and treatments.

Rather than acting on the symptoms, Kaftrio acts on the underlying causes of the disease by repairing defects in a protein, CFTR, caused by a genetic mutation.

“Sword of Damocles”

Last March, it was approved for children aged 6 to 11 with certain genetic profiles. But be careful, “this is not a miracle treatment, there is no cure for cystic fibrosis yet,” warns David Fiant.

Most importantly, only 40% of the 7,500 patients in France will be able to benefit: some, including most children, are not yet marketed; 15% of patients also have a genetic profile that makes this treatment ineffective. And around 900 patients who received a transplant cannot claim it either.

Transplanted 15 years ago, Sabrina Perquis, 41, saw the arrival of this new treatment with “a lot of hope”. But for her it was also “a hard blow because people like me have no right to it. But when you’ve had a transplant, you live with a sword of Damocles over your head, cancellation is always possible.”

“We ask not to be forgotten, research must move forward to provide solutions for all patients,” she pleads. On Sunday, the “Virades de l’espoir”, which has been organized by the Vaincre la mucoviscidose association for over 30 years, should make it possible to collect donations for the fight against the disease.

Several research projects for patients with rare mutations are currently in different stages of development.

Questions also arise: “Will Kaftrio block the progression of the disease or just slow it down?” launches Pierre Foucaud, vice-president of the association.

From 2019 to 2021, the number of transplant patients has dropped from 21 per quarter to two per quarter, no doubt thanks to the drug. But “are these transplants going to be delayed by ten or fifteen years? We don’t know,” adds Mr Foucaud. “Great hope has opened up for all patients, but they remain inadequately respiratory,” he says.

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