Seattle researchers have found out a new therapy that can eventually improve lives of patients suffering from cystic fibrosis. This could not be considered a cure till now because it wasn’t known if the treatment can increase life expectancy among patients, but this cystic fibrosis treatment does a bit of correction in the disease and can make the patients live a better life.
Cystic fibrosis is a disease where more patients tend to perish before the age of 40 because their lungs get damaged due the thick mucus. They are more prone to infections and all these start from an early age. There were no proper cystic fibrosis drugs that could solve the core of the problem.
Two large clinical trials were conducted. The new drug can successfully treat the protein that creates the problem and can reduce the cause of death by 40 percent.
Doctors and researchers were trying for a long time to find a cure to the deadly disease but till today the drugs were just curing the symptoms, not the underlying causes. Many types of errors in DNA can cause the problem and this time it has targeted one of them.
More than 1,000 patients were tested in New England Journal of Medicine with a combination of drugs created using lumacaftor and ivacaftor being given to patients for 24 weeks. The result was better lung functioning.
The disease also creates problem with the mucus lining in the guts, which does not allow one to gain weight. Doctors were pleased to see that some patients actually gained weight during the treatment.
One out of 2,500 newborns in America is born with cystic fibrosis, and the life is not a happy one as problems related to mucus and breathing stays for a long time. It is still not known if the new medicine will increase the life expectancy, but it would surely give a better life to half of the patients. For the other half, there still needs to be something groundbreaking.
Dr. Pamela Davis, dean of the school of Medicine at Case Western University, wrote on New England Journal of Medicine, “This is the beginning of effective therapy for cystic fibrosis associated with the most common mutant form of CFTR.
“The road to success has been long, despite diligence, enthusiasm, and excellent collaborative efforts among academics, industry, and patients.”
The director of research at the Cystic Fibrosis Trust charity, Janet Allen, said: “These results open up a new front in the fight against cystic fibrosis and this combination therapy looks set to be an important additional treatment option that could improve the lives of many.”