While it can’t 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 effects are “spectacular”, said Tuesday David Fiant, president of the association Vaincre la Mucoviscidose, who was one of the first to test it.
Kaftrio, from the American laboratory Vertex, is part of an innovative class of drugs once morest this disease of genetic origin, which inexorably deteriorates the respiratory and digestive systems and was 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 pathology.
In the form of tablets
Delivered in the form of tablets for life, this triple therapy (a combination of three molecules) clearly reduces the effects of the disease, in particular particularly disabling lung conditions.
“Just before starting Kaftrio, I was on oxygen therapy, waiting for a lung transplant,” said David Fiant, 40. “Six hours of daily care (physiotherapy, aerosols…), three-four weeks of infusions per year: I no longer knew if I was living to treat myself or if I was taking care of myself 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 was able to “take a shower alone”, to “climb 15 steps at once” and above all to “accompany (his) daughter to buy comic books”.
When his doctor examines him shortly following, “he hears the air circulating” in his lungs. This had never happened to him before.
Since then, he has considerably reduced the physio and the 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 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 be able to benefit from it: for some, including most children, there is not yet a marketing authorization; 15% of patients also have a genetic profile rendering this treatment inoperative. And some 900 patients who have 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 are not entitled to it. But when you have received a transplant, you live with a sword of Damocles above your head, rejection is always possible “.
“We ask not to be forgotten, research must progress to provide solutions to all patients,” she pleads. On Sunday, the “Virades de l’espoir”, organized for more than 30 years by the association Vaincre la mucoviscidose, should make it possible to raise funds for the fight once morest the disease.
Several research projects for patients with rare mutations are currently at different stages of development.
There are also questions: “Will Kaftrio block the progression of the disease or only slow down its progression?”, Launches Pierre Foucaud, vice-president of the association.
From 2019 to 2021, the number of transplant patients fell from 21 per quarter to two per quarter, no doubt thanks to the drug. But “will these transplants be deferred for ten or fifteen years? We don’t know,” adds Mr. Foucaud. “For all the patients, a tremendous hope has opened up, but they remain insufficient respiratory,” he says.
