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Thread: Kalydeco

  1. #1

    Arrow Kalydeco

    So what exactly does it do? Is it true it cures 1/2 of CF? How does it make your liver produce more enzymes? How long do you have to use it until you can stop and you're fixed? Why is it so expensive?


    My doctor said I can be in phase 2 of the studies, but is it safe? I'm worried I'm gonna like, die or something. Does it cost money to participate in the studies? How can I be apart of the studies? What are the benefits of doing the studies?

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    Last edited by Enzo2311; 07-31-2013 at 07:14 AM.

  2. #2
    It is not a "cure" from my understanding, but a very effective therapy currently only working for a specific defect. BUT it is being studied with an additional drug to see if it will have the same beneficial results for other mutations, particularly DeltaF508, the most common. From what I understand it helps improve lung function and weight gain. I don't know that all the other things that CF patients have to do are entirely eliminated, but through taking this simple pill they see an improvement to their lives.

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  3. #3
    Hi my daughter is on Kalydeco right now and all I can say is I would jump through any hoop to get in on the trial they are offering you. Unfortunately she is too young. The Kalydeco alone is helping our daughter so much I can not wait to get her on the 809 with the Kalydeco. My advice....get in on the trial! You won't regret it!! And as far as possible side effects and cost...I would ask your research coordinator. The only side effect that I am aware of for Kalydeco (and yall jump in if I'm wrong) is possible rise in liver enzymes. Luckily Abby did not have this side effect.

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    --Jenny
    Mom to Clayton 11 no CF
    and Abby 8 diagnosed at age 2, mutations are DF508 & 2585delT
    multiple sinus surgeries and currently on Kalydeco
    use to culture: MRSA

  4. #4
    Kalydeco is an in-order-of-magnitude game-changer therapy and the type that was unheard of in CF and probably unthinkable after the fall-out of failed gene therapy hopes circa 94-95'ish.

    It is what's known as a 'potentiator,' it allows those with CF who have some level of functioning protein that exists on the surface of certain cells to provide NaCl exchange. The lack of this NaCl exchange contributes to the sticky mucus problem which is at the core of fatality curve associated with CF. It's not a cure. Those with access to Kalydeco have to take it everyday forever (unless something better comes along; if there are parts of the body that don't like Kalydeco (liver being the most culpable reading the papers filed w the FDA).

    If one has a chance to get into a trial with Kalydeco, if you demonstrate efficacy, it will be unlike anything you have ever taken / exercise program / deity praying -wise. If it works on you, it will work in a matter of days, not weeks or months. It is like a like switch being turned on in a dark, dark room.

    It cannot reverse scarring that is on your lungs already but it can prevent future scarring.

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  5. #5
    i think you mean phase 3 for the 809 and kalydeco trial since you have two copies of df508 if i remember correctly. if you do the trial you will get the drugs about one year before everyone else. i would fight to get in. they have already passed many safety hurddles and they watch you ckosely while on. id call dr today and say i want in and asap.

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  6. #6
    Quote Originally Posted by stranger View Post
    Kalydeco is an in-order-of-magnitude game-changer therapy and the type that was unheard of in CF and probably unthinkable after the fall-out of failed gene therapy hopes circa 94-95'ish.

    It is what's known as a 'potentiator,' it allows those with CF who have some level of functioning protein that exists on the surface of certain cells to provide NaCl exchange. The lack of this NaCl exchange contributes to the sticky mucus problem which is at the core of fatality curve associated with CF. It's not a cure. Those with access to Kalydeco have to take it everyday forever (unless something better comes along; if there are parts of the body that don't like Kalydeco (liver being the most culpable reading the papers filed w the FDA).

    If one has a chance to get into a trial with Kalydeco, if you demonstrate efficacy, it will be unlike anything you have ever taken / exercise program / deity praying -wise. If it works on you, it will work in a matter of days, not weeks or months. It is like a like switch being turned on in a dark, dark room.

    It cannot reverse scarring that is on your lungs already but it can prevent future scarring.
    Quote Originally Posted by JENNYC View Post
    Hi my daughter is on Kalydeco right now and all I can say is I would jump through any hoop to get in on the trial they are offering you. Unfortunately she is too young. The Kalydeco alone is helping our daughter so much I can not wait to get her on the 809 with the Kalydeco. My advice....get in on the trial! You won't regret it!! And as far as possible side effects and cost...I would ask your research coordinator. The only side effect that I am aware of for Kalydeco (and yall jump in if I'm wrong) is possible rise in liver enzymes. Luckily Abby did not have this side effect.
    for like 120 pils it costs $30,000, how can anyone afford to take it forever? No one could afford that.

    How often must it be take? One a day? One a week? What did the doctor say to your daughter, like when she can stop taking it? Like can she slowly withdraw from it at a certain point?

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  7. #7
    Quote Originally Posted by Aboveallislove View Post
    i think you mean phase 3 for the 809 and kalydeco trial since you have two copies of df508 if i remember correctly. if you do the trial you will get the drugs about one year before everyone else. i would fight to get in. they have already passed many safety hurddles and they watch you ckosely while on. id call dr today and say i want in and asap.
    i have cf day Friday so I'm gonna say I want in. Especially since its free (I'm assuming), he mentioned it to me a few months ago so I hope I can still participate.

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  8. #8

    taking kalydeco for 18 months

    My son has been taking Kalydeco for about 18 months now and all that I can say is he is a completely different person. He is 9 years old and in the past year alone he has gained 16 pounds, grown about 5 inches, FEV1 has gone from 92 - 102, hardly needs to take enzymes, no more aerosol treatments and no more vest. It is literally like he does not have CF. Of course, he still has CF but the change has been dramatic and it has completely changed our family.

    He did have elevated liver enzymes but they resolved within a few months. I truly hope and pray that the kalydeco vx809 or kalydeco vx661 combinations help the entire CF population because everyone DESERVES this chance.

    To comment on an above post about scarring... My son had what appeared to be scarring in his left lower lobe (not confirmed by bronchoscopy) that completely resolved. He also now cultures only normal flora while before he has cultured staph aureus, haemophilus and pseudomonas.

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    Last edited by Tom; 08-01-2013 at 08:12 PM.

  9. #9
    Quote Originally Posted by Enzo2311 View Post
    for like 120 pils it costs $30,000, how can anyone afford to take it forever? No one could afford that.

    How often must it be take? One a day? One a week? What did the doctor say to your daughter, like when she can stop taking it? Like can she slowly withdraw from it at a certain point?
    The dosage is twice a day. It is not a "cure" and the improvements made will only remain as long as the drug is in your system. So if you stop taking it, your CF symptoms will return to pre drug conditions.

    But, the potential is amazing. We are currently trying to get my daughter enrolled in the study.

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    Teri
    mom to
    17 year old with CF (diagnosed at 8 year)
    15 year old no CF

  10. #10
    Tom,

    Thanks so much for the uplifting post. How tremendously wonderful!!! Love the line about changing your family!!! We can't wait. It's just so great to hear how significant the impact is for the CFer and their family. I know we are closer than ever but it really helps to hear first hand reports. Keeps the light shining in for me.

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