Cystic fibrosis: a new treatment changes the lives of some patients

(AFP) – Although it cannot cure them, a new treatment for cystic fibrosis has literally changed the lives of patients by reducing the effects of the disease. But not everyone can take advantage of it.

Its effects are “spectacular,” said on 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, is part of an innovative class of drugs against this disease of genetic origin, which inexorably deteriorates the respiratory and digestive systems and once often fatal to children and adolescents.

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

Delivered in tablet form for life, this triple therapy (a combination of three molecules) clearly reduces the effects of the disease, especially particularly crippling lung conditions.

“Shortly before starting Kaftrio, I was on oxygen therapy, awaiting a lung transplant,” said David Fiant, 40. “Six hours of daily care (physiotherapy, aerosol, etc.), three to four weeks of infusions a year: I no longer knew if I lived to be treated or if I took care of myself to live”.

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

When his doctor visits him shortly thereafter, he “feels the air circulating” in his lungs. He had never happened to him before.

Since then he has greatly reduced his physiology and treatment.

Instead of acting on 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 ages 6 to 11 with certain genetic profiles.

But beware, “this is not a miracle treatment, there is still no cure for cystic fibrosis,” warns David Fiant.

Above all, only 40% of the 7,500 patients in France will benefit from it: for some, including most children, there is still no marketing authorization; 15% of patients also have a genetic profile that makes this treatment ineffective. And even about 900 patients who have received a transplant cannot claim it.

Transplanted 15 years ago, 41-year-old Sabrina Perquis saw the arrival of this new treatment with “much hope”. But for her it was also «a hard blow because people like me don’t have the right. But when you have received a transplant, you live with a sword of Damocles over your head, refusal is always possible ».

“We ask not to be forgotten, research must progress to provide solutions for all patients,” he says.

On Sunday, the “Virades de l’Espoir”, organized for over 30 years by the Vaincre la mucoviscidose association, should make it possible to raise funds for the fight against the disease.

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

There are also questions: “Will Kaftrio stop the progression of the disease or will he only slow down its progression?”, Launched Pierre Foucaud, vice president of the association.

From 2019 to 2021, the number of transplant patients dropped from 21 per quarter to two per quarter, no doubt thanks to the drug. But “will these transplants be postponed for ten or fifteen years? We don’t know,” adds Foucaud. “Great hope has opened up for all patients, but there is still insufficient breathing,” he says.

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