In a recently published study in the New England Journal Of Medicine, Dr. Raksha Jain, the author of the study has found a combination of medicine made of three drugs.
Dr. Jain has examined and come to the conclusion that the three-drug combination is capable of not only improve the conditions of Lungs but also can be used to treat cystic fibrosis patients. A disease caused due to genetic mutations.
The good news is, this is not just a study anymore the Food and Drug Administration has already approved of the treatment based on the study.
Cystic Fibrosis is a fatal disease that shortens the life of a person by affecting the lungs (respiratory system), digestive system and reproductive system along with sweat glands.
The three-drug combination is a treatment that can be done to the most common form of mutation behind the disease.
Phe508del CFTR protein is the most common mutation found in the CF patients that gets encoded by two genes, one from each parent.
About 90% of people suffering from cystic fibrosis, that is approximately 80,000 people worldwide have the CFTR protein (Cystic Fibrosis Transmembrane Conductance Regulator).
The trial that was conducted through June to April 2018-2019 was co-sponsored by Vertex Pharmaceuticals, in 115 sites spread across 13 countries. For the experiment, 403 patients of 12 years and above were randomly given placebo or the three-drug combination treatment.
In comparison with the placebo, patients receiving the three-drug combination elexacaftor-tezacaftor-ivacaftor combination therapy did well. The effects of the treatment were noted through 4 to 24 weeks. Patients receiving the treatment showed symptoms, 63% lower than general.
Cystic Fibrosis patients are known to have a high amount of salt in their sweat. The treatment group was identified to have less amount of salt compared to the placebo group, which indicated the success.
Despite the success of the experiment, and safe the combination of drugs need to be tested more on different patients for safety and reactions.
Also, Dr. Jain reaffirmed that the CF community is actively at work and research to find out and come up with a treatment for the disease with different mutations, people who are not eligible for the three-drug treatment.