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A Research Study On Cystic Fibrosis

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INTRODUCTION Imagine going to the mechanic with squeaky brakes. After careful analysis of the car, the mechanic confidently concludes that the vehicle has a condition called “squeaky brake-itis” and prescribes a pair of earplugs to drown out the irritating noise. (Sabatier) Treating the symptoms instead of the cause of an issue would be totally unacceptable in mechanics, yet it happens in healthcare quite often. Cystic Fibrosis serves as an excellent example of treating symptoms because for a long time that was the only kind of treatment healthcare provided for these patients. To combat this issue, a new medicine called Ivacaftor was developed. This drug was designed to treat the underlying cause of Cystic Fibrosis by binding…show more content…
Like placing one’s hand on a heated burner, numbing the pain does not solve the issue. In 1980, the average life expectancy for a CF patient was less than 20 years. (New) Recently, scientists realized that in order to treat this complicated disease they would have to investigate a more complex cellular level. In 1989, the CFTR gene was discovered. (Discovery) This gene carries the instructions for the CFTR protein, which sits on the cell surface transporting chloride ions across the cell wall. When the CFTR gene is mutated, the CFTR protein is made incorrectly, and it is unable to exchange chloride ions. The high chlorine concentration inside of the cell is not optimal for normal cell function, so the lung cells naturally begin to pull in water from the mucus to dilute the thick chlorine concentration inside the cells. The result is thick, sticky mucus on the outside of the cells that mats down the cilia in lung airways. Clearing the throat and absorbing nutrients thus becomes extremely difficult and the symptoms of CF arise. (May) Shortly after the discovery of the CFTR gene, efforts were made to develop gene therapy for all CF patients. The intention was to insert healthy DNA so that the protein would be made correctly, but single cells have too short a life span to make a significant difference. (Learning) The next, more practical idea, was to invent a small molecule that would
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