Why I’m excited for Vertex Pharmaceuticals, Inc.

Yesterday, I subbed in a 5th grade classroom and I realized, yet again, how much I miss working. Due to the economy, I have not had the option to work, but the underlying reason I have yet to be employed is far deeper.  When I was working full-time last year, my lung function was not doing as well, I was missing treatments, and I was getting sick. . . often.  David and I realized at that point in time that my life-long dream of being a full-time teacher is, currently, unobtainable. My lung function is incredibly high and I am grateful for that, but if I continue to work full-time, my lung function will not be high for long.  It’s impossible for me to include a full-time teaching job (plus coaching/activities, grading, lesson planning, and other take-home work), maintain my rigorous treatment/exercise schedule, makee dinners, and help keep the house clean.  It’s overwhelming and there simply are not enough hours in the day.

Facing this fact is depressing, but I’ve had almost a year to get used to it now.  I keep looking for jobs that will be flexible enough to make my treatment schedule work, and would be away from enough germs to keep me healthier. I can tell you that it is nearly impossible.  I’m currently subbing as much as possible which keeps my dream alive, but still exposes me to a tremendous amount of germs.  Sadly, I came home yesterday from subbing feeling beyond wiped out and still had to make time to cook dinner and do my treatment. It was another reminder that my health is not as good as it used to be even 2 years ago.

With that said, David and I have a goal: keep me as healthy as possible so that I can fully enjoy the benefits of a cure for CF.  Enter Vertex Pharmaceuticals! Vertex has been in the process of working on two therapies which treat the underlying cause of cystic fibrosis. Up until now, every drug on the market has been about treating the symptoms, so this new drug would be groundbreaking.  Today, Vertex has announced that it has officially submitted its drug, VX-770 for FDA approval.  VX-770 would not treat my CF mutation (DDF508), but it would be a miracle drug. It is being seen as the “cure” for CF right now, and the best part about the drug is that it’s a simple pill to swallow.

Vertex is also actively working on a combination drug which is specifically for my CF mutation (the most common mutation in the CF community).  They are currently in clinical trials throughout the United States and so far it seems the drug has shown very promising results.

Here is the text taken from the Vertex site which further explains these new drug therapies.

“VX-770
(Cystic Fibrosis)

Cystic Fibrosis (CF) affects about 30,000 people in the United States and approximately 70,000 people worldwide. Cystic fibrosis is caused by defective or missing CFTR proteins, which result in poor ion flow across cell membranes, including in the lung, and the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. In people with the G551D mutation, CFTR proteins do not function normally at the cell surface. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR at the cell surface. In people with the F508del mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809, known as a CFTR corrector, aims to increase CFTR function by increasing the trafficking, or movement, of CFTR to the cell surface.

A Phase 3 clinical development program for VX-770 is complete, and Vertex submitted a New Drug Application for VX-770 to the U.S. Food and Drug Administration (FDA) in October 2011. Vertex is seeking approval of VX-770 for use in people six years of age and older who have at least one copy of the G551D mutation in the CFTR gene. Vertex also plans to start the registration process for VX-770 with the European Medicines Agency (EMA) by the end of October 2011.

Vertex is also conducting an exploratory Phase 2 clinical trial to evaluate combination regimens of VX-770 and VX-809 in people with the most common mutation in CF, known as F508del.

VX-809 is the second investigational oral drug candidate for the treatment of cystic fibrosis (CF) to be advanced into development as part of a successful collaboration with the Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT).

Cystic fibrosis is caused by defective or missing CFTR proteins, which result in poor ion flow across cell membranes, including in the lung, and the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. In people with the F508del mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809, known as a CFTR corrector, aims to increase CFTR function by increasing the trafficking, or movement, of CFTR to the cell surface. In people with the G551D mutation, CFTR proteins do not function normally at the cell surface. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR at the cell surface.

Vertex is conducting an exploratory Phase 2 clinical trial to evaluate combination regimens of VX-770 and VX-809 in people with the most common mutation in CF, known as F508del. Vertex recently completed the first part of the trial and is on track to initiate the second part of the trial in September 2011.”

 

The news makes me want to cry. It’s impressive, incredible, and miraculous. I’m unsure of whether the CF daily treatment schedule would be altered at all, but regardless if it makes me healthier, it’s one step closer to getting into a classroom for real. It’s one step closer to being able to work full-time again. I know many reading this will think something along the lines of, “Trust me, you don’t want to work.” To those people, please try to understand the emotional/mental toll that unemployment can take on you.  I am failing to help provide for our family, and there are days when I feel incredibly worthless. David fights me to the end on these feelings, but regardless, they’re still present. I can not wait to be able to work again.

Above all, if this drug goes through the rest of its clinical trials and becomes FDA-approved, it is one step closer to seeing my 75th birthday and beyond. It’s a step toward a longer, healthier, and more enjoyable life, and it will provide extra years with my husband. I don’t think anyone can blame me for being just a TAD bit excited! 🙂

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