Precision medicine, also known as personalized medication, is now surfacing more than ever, but could not have happened without the years of research and billions of dollars in funding to discover that one’s genetic code is composed on billions of letters. PM Live reported that this discovery back in 2000 changed medicine for everyone and has led us to where we are today.
Unlike many other diseases that have multiple causes, cystic fibrosis is caused by a small mutation in one single gene. Simply said, three letters changed in that billion letter genetic code can lead to the devastating results of the rare disease. Some symptoms include lung damage, yet many experience other forms of organ damage as well.
It took 15 years to find a treatment that actually targets the root of the problem, and today this treatment is being used to treat just 5% of CF patients in England. Continued testings progress with the potential of treating half of these CF patients battling in the United Kingdom. Additionally, researchers believe the potential with new medication could lead to treatment for somewhere closer to 90% of these patients.
The research they’ve done and transformative medicines they’ve come across has serious potential to help not only CF patients, but other diseases like sickle cell diseases. Vertex, a small program that later headed the CF drug development, has been relying on their ability to culture cells in their lab for possible treatment.
Yet, while Vertex has made huge strides, they are developing medications faster than the NHS systems and processes can keep up with, which then makes it difficult to make the medications available for patients that need it in a timely manner.
Due to this unique situation, a new proposal has been submitted to the government in the UK to speed up the process of getting CF patients current medications, as well as those coming in the near future. Genomics are here, and they are here to stay. Now it’s time to shift gears, and get everyone ready to handle the requirements of genomics so patients can benefit from its advantages sooner than later.