A new therapy could profoundly improve cystic fibrosis patients’ quality of life, say doctors.
Patients suffering from cystic fibrosis often die before their 40s as mucus clogs and damages their lungs and leaves them prone to infection.
A major international trial on 1,108 patients, in the New England Journal of Medicine, showed a combination of drugs could bypass the genetic errors that cause the disease and may increase life expectancy.
Errors in sufferers’ DNA – inherited from their parents – damage the microscopic machinery that controls salt and water levels in the linings of the lungs.
The result is a thick mucus that inexorably damages the lungs.
Antibiotics help prevent infection and drugs can loosen the mucus, but nothing deals with the fundamental problem for most patients.
The combination of drugs – lumacaftor and ivacaftor – were designed to repair that microscopic machinery.
The trial showed that those patients given the cocktail for 24 weeks had better lung function.
Cystic fibrosis also affects the mucus lining in the gut so the doctors were pleased to see the patients also gained weight in the trial.
There are however, many types of error in the DNA that can culminate in cystic fibrosis.
This treatment combination should work on around half of patients, while one of the drugs on its own corrects a small proportion of errors.
New treatments are still required for the remaining patients.
The therapy is being examined by regulators around the world.
AstraZeneca, the second largest drugs company in UK, has announced it is buying California-based Pearl Therapeutics in a deal worth up to $1.15 billion.
Privately-owned Pearl specializes in drugs used in the treatment of lung disease and asthma.
AstraZeneca said it will pay $560 million initially, then $450 million subject to drug development milestones being reached.
It could also make sales-related payments of up to $140 million in addition.
The acquisition will give AstraZeneca a foothold in the emerging market for a new class of lung treatments known as LABA/LAMA drugs, administered via an inhaler.
AstraZeneca has announced it is buying California-based Pearl Therapeutics in a deal worth up to $1.15 billion
These drugs are used in the treatment of chronic obstructive pulmonary disease, which affects about 210 million people worldwide.
Anglo-Swedish AstraZeneca, under its new chief executive Pascal Soriot, who took over in October 2012, has identified respiratory treatments as one of its core businesses.
AstraZeneca has been suffering falling profits and sales as several of its blockbuster drugs, such as the anti-psychotic Seroquel and anti-cholesterol Crestor, have lost their patent protection, allowing cheaper generic drugs to enter the market.
It was also forced to write off $140 million relating to a failed arthritis drug.
AstraZeneca is simplifying its drug development programme, cutting 5,000 jobs by 2016, and is looking to expand through acquisitions instead.