Your Very Own Drug: Pitfalls and Promises of Personalised Medication
In the month of May during the year two thousand and one, a scientist aged only thirty three by the name of Frederick Van Goor turned up at a biotech firm that had recently been purchased by a rival firm, for his first day of work. Upon his arrival, Goors new boss proceeded to inquire of him whether or not he held any information about the genetic factors that lead to the development of cystic fibrosis, an extremely fatal lung disease. Frederick knew little about this but nonetheless he was deeply intrigued by the notion of formulating a cure for this, so he decided to simply respond in the affirmative to his new boss's enquiry.
Frederick's considerable impudence was the start of a 10 year long path of discovery that involved a great number of scientists that culminated in the production of the very first drug known to treat cystic fibrosis right at its genetic origins. This drug may be approved within the coming year and if approved would generate vast sums of money for Goor's employers, the firm Vertex Pharmaceuticals. In stark contrast to other medication which seem to merely decrease the speed at which affected individuals perish from the disease, which harms individuals by resulting in the filling of their lungs with very thick mucus, hindering breathing, the drug formulated by Vertex which has been dubbed VX-770 shows improvements in the function and performance of patients lungs of almost ten percent. This performance increase was maintained for an entire year while clinical trials where taking place. This was named as an incredible 'milestone' discovery by the New England Journal of Medicine.
But this drug is not without its adverse effects. The drug VX-770 has been shown to work exclusively for individuals whose cystic fibrosis is as a result of a specific genetic mutation. This means that VX-770 will only be applicable to one thousand two hundred of the thirty thousand cases of children and adults ailed with cystic fibrosis. Not many patients suffering from cystic fibrosis live past the age of forty. Nonetheless, Frederick has aided in the invention of a second drug that when administered jointly with VX-770 may enable VX-770 to help a larger number of people, although current clinical trials have not yielded promising results. To top that of there is also the price of the drug to be considered, it is thought by analysts from Wall Street that Vertex will charge a lump sum of two hundred thousand dollars or even more just to treat a single patient over a one year period. Nonetheless Vertex has assured the general public that no one will miss out on the chance to use this drug.
This may herald the beginning of the era of personalised medicine, not just for rare diseases such as CF but also for more common ailments such cancer. These personalised medications may prove effective for few select individuals but the vast majority of society will require the use of a number of new drugs simultaneously in order to achieve the desired effects. This is something that both drug companies and the Federal Drug Agency are wary of. Nonetheless, pharmaceutical companies has demonstrated a new eagerness to develop drugs for rare diseases, this is mostly because the pharmaceutical firms can charge exorbitant sums of money for the successful drugs. Goor has admitted that these problems must be solved.