In the US all the babies are tested in order to diagnose cystic fibroid causing genes. For cystic fibroid, there is no cure but new medications are giving hope to people as they improve the symptoms.
The three-drug combination can prove to be life-saving for many patients with cystic fibroid, researchers reported on Thursday. “Within 24 hours of taking the pill combination, patients were puffing less, their sinuses were clearer, they had more energy,” one of the researchers said.
Adding a third drug to the existing combination of two drugs played an important role in improving the patient’s symptoms. It has also raised the expectations of many patients to live longer.
The two existing drugs already cost $300,000 a year and adding a third drug will further increase its price. However, the doctors are very excited to treat cystic fibrosis which is genetically transferred disease. It causes lungs and digestive issues, plus also decreases the lifespan of a person.
Dr. Fernando Holguin of the University of Colorado said in the New England Journal of Medicine: “These results represent a major breakthrough in cystic fibrosis therapeutics, with the potential of improving health and possibly survival in all patients who carry the most common CFTR mutation.”
The New England Journal of Medicine is talking about the two experiments done on cystic fibrosis by Vertex Pharmaceuticals. The outcomes are also now being shared at the North American Cystic Fibrosis Conference in Denver.
Dr. Jennifer Taylor-Cousar of National Jewish Health in Denver said: “Within 24 hours of taking the pill combination, patients were puffing less. Their sinuses were clearer. They had more energy.”
Cystic fibrosis is one of the rare diseases and has only targeted 80,000 people around the world. One can only be attacked by this condition if there are more than 1,000 mutations in the CFTR gene. It affects the certain type of type cells like those in the digestive tract and lungs.
People who are the residents of Northern Europe have a higher risk of carrying these mutations. If both the parents are the carriers of this mutation then there are 25% chances that the child will develop the disease.
It is a life-threatening disease, patients experience different types of lung infections and excessive mucus which makes difficult for them to breath. This new combination of the drugs can treat some of the abnormalities of cells but the efficiency of this drug totally depends on the particular genetic mutation that has caused the disease.
The third drug in this pairing has a great importance as it helps to overcome the genetic malfunction by adding stimulating effect on the dual combination. The Taylor-Cousar and his colleagues said: “This approach has the potential to treat the underlying cause of cystic fibrosis in approximately 90% of patients.”
The two teams reported that a positive result was seen in the trials of two experimental Vertex drugs. The lung infection in the patients got improved roughly by 13%. The expert says this result does not seem to be very beneficial, but still, it is.
The company is now working on more drugs which can treat other mutations too, and has been conducting several trials in order to get approval to use these drugs on more people with cystic fibrosis.