There is new hope for treating one of the most common types of cystic fibrosis, as researchers discovered an effective combination of lumacaftor and ivacaftor. Results showed the new cystic fibrosis therapy proved to boost lung function and decreased the rate of pulmonary aggravation.
The combination treatment has successfully passed the Phase III of an international clinical trial, and its results were made public online via the New England Journal of Medicine.
Almost half of the cystic fibrosis patients taking part in the trial presented two copies of the cystic fibrosis gene mutation, which the combined lumacaftor-ivacaftor medication seemed to effectively and safely fight against.
Cystic fibrosis is a genetic disorder also known as mucoviscidosis, and it mostly affects the lungs, but sometimes the pancreas, too. The body of a patient suffering from this disease miscalculates the amount of mucus it should produce, so a lot of it invades various organs.
If it forms inside the lungs, the mucus causes chronic infections and lung function is gradually decreasing. When the pancreas is targeted, it stops enzymes from travelling to the gut where they should help digest food.
Patients who suffer from cystic fibrosis have an average life expectancy of 37 years, and there are currently no treatments aiming to cure the disease. Instead, most of the available therapies are attempting to manage and restrict symptoms and damages caused by the disease.
One of the study’s lead authors, Susanna McColley, MD, a Professor of Pediatrics at Northwestern University Feinberg School of Medicine, explained that in spite of the major progress registered in developing complementary therapies for cystic fibrosis, the biggest challenge of treating the underlying genetic cause has yet to be tackled.
Even though ivacaftor has received FDA authorization for a few years now, it can only help a small number of ago cystic fibrosis patients, because it only targets a rare genetic deformity in cystic fibrosis.
McColley has expressed enthusiasm, given the results of their study; combining ivacaftor with lumacaftor in the same treatment seems to be fighting against the most common cystic fibrosis anomaly.
Multiple medical centers took part in the study for 24 weeks; more than 1,100 patients over 12 years old were included in the placebo-controlled clinical.
Even though the results are so far promising, McColley said there is need for longer-term data in order to make sure the combo treatment is able to change the course of the disease and prolong life expectancy.
On May 12, the FDA Pulmonary-Allergy Drugs Advisory Committee got together in order to review the final results of this study; FDA approval is still pending.
Image Source: Brunet
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