Wednesday, June 3, 2015

Orkambi

As most of the CF world is probably aware of by now, the vertex drug developed for DDF508 has a good chance of becoming available to patients this July. Everyone in the CF community was hopeful this drug targeting DDF508 would be a life changing miracle drug just like Kalydeco was for people with the G551D mutation. Unfortunately, the results of the study quickly dashed many of our dreams that this drug would be our miracle drug.

As much as I am disappointed that Orkambi, which will work on my mutation, is not the life changing and life extending drug that Kalydeco is for a select few with CF. I can say I am beyond excited, despite having all the facts, that we may have another medication that could potentially help slow the progression of my disease. When you live with a progressive disease new treatments are never developed fast enough. In fact, discoveries to treat CF are far and few between. And even when therapies are developed I have found myself allergic and unable to take advantage of many of these medications and treatments. The progression of the disease is always three steps ahead of the treatment and watching your lungs and body deteriorate is heartbreaking at best. It development of treatments are very much a matter life or death for so many with CF. This is why I have to roll my eyes at the naysayers that complain about the humble 3% increase in lung function that were seen in the Orkambi study.

What the naysayers don't realize is that 3% may not seem clinically significant, the truth is that the drug is so much more than 3% increase (on average) in lung function. It is the fact that is can slow the guaranteed progression of this disease. That is something that can not be taken lightly. To me, it  means I may have a few more years added to the end of my life to watch my daughter turn into a woman. It may mean one or two more anniversaries I spend with my husband. It may mean another birthday and a few more grey hairs. So yes, 3% may not seem like a big deal to someone who hasn't had an expiration date stamped to their lungs. It may not seem like 3% could change a life, but pessimists are stuck on a low percentage and not the potential for what slowing, even minutely, can do to a life. How a few years can mean so much to a mother, a wife, a sister, a friend who would do anything to hold on to their life for just a little longer.

So yes, lets celebrate the accomplishment of the researchers, the foundation, the  CF community that we may (as long as the FDA gives the okay this July) have one more tool in our fight again CF, we may have a little more hope for tomorrow, and we may have a little more time on this Earth.

7 comments:

  1. Someone to celebrate - http://www.nytimes.com/2015/06/03/science/irwin-rose-nobel-winning-biochemist-dies-at-88.html?hpw&rref=obituaries&action=click&pgtype=Homepage&module=well-region&region=bottom-well&WT.nav=bottom-well

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  2. I couldn't agree more!! I would take anything that would give me a few more years of life with my loved ones, and hopefully it will make that quality of life just a little better if it slows the rates of acute illnesses and exacerbations. I have only one copy of DF508 so I don't think Orkambi will be for me, but I'm hoping us one copy-ers are next ;)

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    1. I know they are working really hard of DF508 correctors which will intimately help both doubles and single 508ers. Things are moving slower for all of us than I would like, but I am hoping we will see good things for all mutations in the near future!

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  3. it's so nice to see someone else who has a positive look on the med! 3% is a lot for someone who can't get that increase by themselves.
    I never really looked at the med like it would fix the damage that has been done, but it is going to help prevent new damage.
    I try to explain it to people like a pause button for CF progression, it's not going to be perfect but every little bit help!
    Stay positive and keep moving forward!

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    1. I agree 100% that every bit helps! I am currently impatiently waiting for my insurance to agree to cover it ;)

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  4. I believe individual perspective has a lot to do with it too. Everyone has had a different life and different life experiences. I myself am lucky enough to have just turned 36, having almost died many times due to CF. I was lucky enough to be able to start on orkambi in February 2016 and lucky for me have had a great experience so far. For the past 5 years with my health declining and my symptoms so violently hard to deal with I was in hospital every 3-4 weeks for an average of 2-3 weeks at a time some times 1.5 months at a time.... It's now the end of may and I have only be in once this year...unheard-of for me! :) my symptoms have decreased by about 30% and I have not only reach my goal weight of which I hadn't been able to do for the past 5 years even with have a PEG for stomach feeding but I have exceeded it by 3kgs! And that was achieved only with eating food, not the assistance of bag feeding.. No additional supplements either! I have been able to run (not for long) which I haven't been able to do for years and my overall strength in my body has vastly improved. Without as many physical symptoms, I'm not burning of fat as fast :) my lung function hasn't improved and is still very low, but I haven't needed to be stuck on oxygen 24/7.... In fact at all! I feel myself coming back, my partner looks at me with so much hope and happiness cause I'm going to be here a lot longer than it was going to be...and I am fighting hard to make that happen...

    Unfortunately it's not the same for everyone and I'm so grateful to have a mutation that has been addressed with this wonderful medication that has stopped my progression.. I'm no longer existing.... I'm living! :) thank-you for shining positive light on this. Christie

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    1. Wow, I am so happy that Orkambi is helping you so much! It is such a huge blessing for so many people. Thank you for sharing your experience! I am sure it gives so many people hope!

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