Cystic Fibrosis Drug Offers Hope to Patients

Breakthrough for Cystic Fibrosis 

Dr Bonnie Ramsey, professor of paediatrics at the University of Washington School of Medicine and the Centre for Clinical and Translational Research at Seattle Children’s informs that there is a breakthrough for cystic fibrosis, Ramsey is one of the four lead authors of two Phase 3 clinical trials, who confirmed that Orkambi which is a new drug from Vertex Pharmaceutical has the potential to help people from 12 years and older, with 2 copies of the most common mutation, F508del and Vertex aided in funding the trials. A ground-breaking cystic fibrosis therapy could help in improving the quality of life of the patients according to doctors.

People often tend to die before the age of 40 since mucus tends to clog and damage the lungs making them vulnerable to infection. Major trial on around 1,108 patients in New England Journal of Medicine, indicated a combination of drugs can bypass the genetic fault which causes the disease and could increase life expectancy. The Cystic Fibrosis Trust has informed that it could improve the lives of several people. Faults in sufferer’s DNA are inherited from their parents causing damage of the microscopic machinery which controls the salt and water levels in the linings of the lungs. This leads to thick mucus which inevitably damages the lungs.

Antibiotic – Prevention of Infection, Drugs – Loosen Mucus

The use of antibiotics helps the prevention of infection and drugs tend to loosen the mucus though the fundamental problem for most of the patients is not dealt with. The combination of lumacaftor and ivacaftor drugs were designed for repairing the microscopic machinery and the trial indicated that the patients who were given the combination for a period of 24 weeks,showed better lung function and also gained weight in the trial.

Prof Stuart Elborn, who had led the European part of the trail from Queen’s University Belfast, informed BBC News website that it is very exciting and really demonstrates that they can correct the basic defects in cystic fibrosis and is likely to become a fundamental treatment for cystic fibrosis. Beginning with the children, it could prevent the developing process of the disease if the basic defect is corrected at the initial stage of life.

There are several types of error in the DNA which could end in cystic fibrosis. Combination of treatment could work on around half of the patients while one of the drugs on its own could correct a small amount of errors.

Ground-Breaking Findings 

Professor of paediatrics at North-western University, Susanna McColley, informed that these are `ground-breaking findings’, which showed the future of treating cystic fibrosis. She informed BBC that - for subjects she cared for, they felt better in ways that are not necessarily measurable.

Jane Allen, director of research at the Cystic Fibrosis Trust charity has informed that `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.

As this leading edge of science continues to be explored and understood better, we are hopeful that a future of personalised medicines is increasingly within reach’. Regulators around the world have been examining this therapy.