Scientists are testing a new drug combination designed for cystic fibrosis patients. The therapy seems to both significantly extend the life of patients and improve its quality. What drugs are they talking about?
Cystic fibrosis (cystic fibrosis(CF) is a genetically determined disease in which the function of the endocrine glands is disturbed. Symptoms mainly concern the respiratory system (recurrent infections that lead to lung damage and respiratory failure) and the digestive system (including pancreatic damage, sometimes with secondary diabetes). On average, 1 in 2500 babies are born with cystic fibrosis.
The study, the results of which was published in the New England Journal of Medicine, included 1108 patients with cystic fibrosis. They were given a combination of two drugs for 24 weeks: lumacaftor and ivacaftor. Compared to the control group, patients receiving both drugs were less likely to develop respiratory infections and gained weight.
– The results are very promising and prove that we are able to correct the effects of the genetic defects underlying cystic fibrosis – says Prof. Stuart Elborn of Queen’s University Belfast, who led the European part of the international research. – Probably in the near future the method we are testing will become a standard of care in cystic fibrosis. If implemented earlier, it can stop the development of the disease at an early stage and prevent its devastating effects.
Although scientists talk about a breakthrough, they make a reservation that cystic fibrosis has many genetic variations and estimate that the combination of lumacaftor and ivacaftor may be effective in up to every second sick child.
The dizzying costs of therapy must also be taken into account. Ivacaftor is one of the most expensive medical products in the world and the annual supply of the drug costs over PLN 1,2 million. Lumacaftor is not yet available on the market and it is difficult to estimate what the price will be.