New cystic fibrosis treatment may end suffering for patients
Trikafta/Kaftrio pill treatments are the new breakthrough in cystic fibrosis treatments as it may restore the patients' abilities to live normally again without organ transplants.
While waiting for a lung transplant, cystic fibrosis patient David Fiant says 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 as part of his treatment that was so exhaustive he "could no longer tell if I was living to heal myself or healing myself to live."
He later became one of the first participants in the new triple-drug treatment praised as a pivotal moment for those suffering from the rare disorder, which was life-changing that when his doctor examined him after he started the therapy, he could hear air circulating in his lungs for the first time.
Fiant described his trial with the new drug treatment, saying, "I took my first dose of the medicine one morning. By 3 pm I was feeling first effects," adding that he was able to shower by himself, climb 15 steps at once, and take his daughter "to buy comic books".
Following his triumph over his life-threatening condition, Fiant has since then become the president of the French non-profit Vaincre la Mucoviscidose (Defeat Cystic Fibrosis). Although he stated that the results were "spectacular", he underlined that a complete cure has still not been found for cystic fibrosis.
Life expectancies at risk
The condition, which is transmitted by genetic inheritance, builds up sticky mucus in the lungs, digestive tract, and other organs leading to difficulty breathing and long-term complications, such as infection which can often be fatal.
As a result, life expectancies are significantly impacted and become shorter than expected. More than 70,000 people worldwide suffer from this disease.
Sold on the market as Trikafta in the US after its approval in 2019, its effects have proven to be major with the life expectancy of cystic fibrosis patients born between 2017 and 2021 in the US now being 53, as opposed to 38 a decade ago, according to the US non-profit organization the Cystic Fibrosis Foundation.
The triple-pill treatment was then approved by the European Union's medicines agency and by the UK in August 2020, where it was sold as Kaftrio. Both varieties have been applauded for targeting the underlying cause of the disease and repairing the mutation in the CFTR gene.
Its demand does have a downside. Out of the 7,500 cystic fibrosis patients in France, only 40% are able to use the treatment.
The reason is that the treatment has not been approved for children under the age of six, and 15% of patients have a genetic profile that the treatment won't work for. Moreover, approximately 900 individuals who received transplants of lungs or livers are also deemed ineligible.
"Do not forget us"
Sabrina Perquis, a cystic fibrosis patient in France, was excited to take the treatment until she found out that she could not use Kaftrio because of a transplant she had 15 years ago, calling it "a tough blow."
"When you have had a transplant you live with a sword of Damocles hanging over your head because rejection is always possible," she said, adding, "We are asking not to be forgotten. Research must continue to find a solution for all patients."
Although several research studies are taking place and are at varying stages of development, questions still remain, such as whether the treatment completely blocks the disease's development or if it solely impedes it, according to Vaincre la Mucoviscidose Vice-President Pierre Foucaud.
Cystic fibrosis patients who had an organ transplant fell from 21 to just 2 per quarter in France from the years 2019 to 2021, fueled by hopes that the Kaftrio treatment would make the transplant operation unnecessary. "But will these transplants merely be delayed by 10 to 15 years?" Foucaud asked. "We don't know," adding that Kaftrio has "opened up a tremendous hope for all patients" but more work needs to be conducted.