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View Full Version : Kalydeco gets approval for eight more mutations



CFParent2
02-21-2014, 04:54 PM
Good news for approx. 150 people with CF 6 and older.

a supplemental New Drug Application (sNDA) for KALYDECOTM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KALYDECO was first approved in January 2012 for people with CF ages 6 and older who have at least one copy of the G551D mutation. With the approval of the sNDA, KALYDECO is now approved for use in people with CF with the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In the United States , approximately 150 people ages 6 and older have one of the additional eight mutations for which KALYDECO is now approved.

welshwitch
02-21-2014, 09:58 PM
Wow! Great news!!!

scrapper1264
02-22-2014, 10:29 AM
So happy for those 150 people!

cheerfulpuppies
02-22-2014, 11:17 AM
Hopefully that means it will get approved for more later on too.

mom24dodd
03-29-2014, 08:40 PM
I totally think my son is one of those 150 people - I about drained every ounce of liquid in my body with tears when I talked to his pulmonologist about this. Of course I was the one who had to inform HIM - but I don't really care. Does anyone know anyone who is on this? Does it mean an end to the enzymes and lung therapy? That would just be the best news ever if it does!

2roses
03-30-2014, 11:08 AM
Mom24dodd: My 2 daughters with G551D (as one mutation) have been on Kalydeco for 2 years. They have not stopped any routine meds including enzymes. What it has done is make them more stable and neither had needed hospitalizations since. My one daughter was in the hospital every few months for several years prior to that, so we credit the Kalydeco. They both stopped taking oral antibiotics all the time too. PFTs were good prior and are staying good.

I know some have said they could stop enzymes and back way off on breathing treatments, but that hasn't been our experience.

This is still AMAZING news that your CFer can get this groundbreaking drug! I hope you'll see a huge improvement.

mom24dodd
03-31-2014, 08:34 AM
2roses: Wow - ok - seems like your results are very good. My son will be 9 this year and has only had to be on anti-biotic one time - thankfully - and never has been hospitalized for being sick. He has autism too so we really cannot do PFTs - we just get the respiratory therapist to do deep throat culture and every so ofter we get bronchoscopy done.
Thanks so much for your input!

Aboveallislove
03-31-2014, 08:45 AM
Mom24dodd this blog gives you another before after look and might be what your son could experience given his current status...no treatments would be heaven, but even no hospitalization is an amazing thing...so happy for all benefitting from K!
http://luckycfmom.blogspot.com/

FrancisB
04-01-2014, 07:24 PM
Aboveallislove: thank you for the blog link from "luckycfmom". I was so inspired and moved with pure joy at reading her son's results. I'm with the many,waiting with great anticipation, for the combination of Kalydeco and VX809 to complete trials (expected in June 2014) and get FDA approval (expected December 2014). We all need to keep supporting the CF Foundation.

Allansarmy
04-02-2014, 02:49 PM
This is exciting news indeed. 150 people is 150 people who do not have to suffer as much or possibly even extend their lives longer. Love to hear news like this. Not to sound too self indulgent but any news on the Delta mutation which if I am not mistaken is the most popular of all mutations for CF? Also, if a person has the double Delta mutation like my son, does that change things with this drug if it gets approved. Or I should say "WHEN" I must stay positive.

mom24dodd
04-23-2014, 07:35 PM
Thank you Aboveallislove (http://forum.cysticfibrosis.com/members/13361-Aboveallislove) -I need to find a way to contact that mom who wrote that blog to see how she got her very young son to start taking that pill. My son has Autism - he will just not take a pill that does not dissolve or does not taste good. We still put the enzyme beads in applesauce and he is 8 years old turning 9 next month. Our doctor will not start the prescribing process for Kalydeco until we can get my son to swallow a pill - any pill. We are going to try to start with the whole enzyme capsule - but even that he is refusing...

Aboveallislove
04-23-2014, 07:46 PM
Mom maybe start a new post asking that...I've seen others talk about getting small empty capsules from pharmacy and working up...there might be ther good Ida's too!

Angelo
04-24-2014, 11:37 AM
Seriously, did I read that right, 150. Out of the tens of thousands with CF. Happy for those 150. But I am not HOLDING MY BREATH waiting for the mainstream medical community. Going that way, I would have expired 3 years ago. And even if the med works for me, it does nothing to reverse existing damage.

Everyone needs to know about the adult stem cell therapies. Had 2 so far and alive because to them. The CF doctors do not want to hear about it. In fact if you talk to your doctors, they might even retaliate against you. My CF docs said adult stem cells do not work. They were confident about that. Not sure how they could be confident without any proof or clinical trials one way or the other. As intelligent doctors and scientists, the only unbiased, intelligent thing they could have said is, "we don't know"! Lack of proof does not mean that something is not true, it simple means that you don't know with certainty. And the definition of proof is not exclusive to the concept of "clinical trials". There are other ways to prove something.

But now 3 years later and 2 treatments later, I AM PROOF! Got my life back, immune system strengthened (have not caught cold in over 2 years), osteoporosis reversed without meds. I can breathe better, whereas before I could speak a complete sentence because I was gasping for breath even at rest. Less than a year after the second treatment, I came off oxygen and was able to get out of the house everyday -- without oxygen. Before I could not leave the house and had trouble crossing the living room, on oxygen. And ready for this -- Have not had or needed a tuneup since Spring of 2011. That is 3 years without a tuneup. Before that, I was getting tuneups every 3 months. And early 2011 had 3 tuneups back to back which did not improve my condition and almost killed me. We had to leave Nashville for my safety.

Now in S. FL. and my new CF doctor does not think I have any proof and that the stem cells don't work. But at least he talks to me like an adult. At the last appt in December 2013, he said I was doing well and do not need a tuneup. He cannot explain why I am doing so well, but still thinks the stem cells don't work, because I have no proof. I told him, "I AM THE PROOF". My stem cell doctor (Near Naples, FL) has treated over 700 patients with heart problems, COPD, pulmonary hypertension, MS, spinal cord injuries. Over 700 proofs walking around partially or completely healed through adult stem cells.

I still do my inhalers and vest to clear out my lungs. I do that huffing breathing exercise to cough and push up the mucous. My FEV1 is better, it went from about 10% to nearly 30%. But I have learned that the PFT's do not reveal everything that is going on in the lungs. They do not explain the difference in my body between almost dead, and able to walk around all day with out oxygen. They don't explain the difference between not being able to drive anymore (due to a brain fog from lack of O2 to the brain and excess CO2) to driving from Delray Beach to Naples FL by myself and spending a couple of days there to attend a luncheon (recognizing the team of people pioneering the adult stem cell therapies).

I for one, am looking forward to more adult stem cell treatments for further reversal of the damage in my lungs. But I have not proof.

CFParent2
04-24-2014, 11:57 AM
Seriously, did I read that right, 150. Out of the tens of thousands with CF. Happy for those 150. But I am not HOLDING MY BREATH waiting for the mainstream medical community. Going that way, I would have expired 3 years ago. And even if the med works for me, it does nothing to reverse existing damage.

Everyone needs to know about the adult stem cell therapies. Had 2 so far and alive because to them. The CF doctors do not want to hear about it. In fact if you talk to your doctors, they might even retaliate against you. My CF docs said adult stem cells do not work. They were confident about that. Not sure how they could be confident without any proof or clinical trials one way or the other. As intelligent doctors and scientists, the only unbiased, intelligent thing they could have said is, "we don't know"! Lack of proof does not mean that something is not true, it simple means that you don't know with certainty. And the definition of proof is not exclusive to the concept of "clinical trials". There are other ways to prove something.

But now 3 years later and 2 treatments later, I AM PROOF! Got my life back, immune system strengthened (have not caught cold in over 2 years), osteoporosis reversed without meds. I can breathe better, whereas before I could speak a complete sentence because I was gasping for breath even at rest. Less than a year after the second treatment, I came off oxygen and was able to get out of the house everyday -- without oxygen. Before I could not leave the house and had trouble crossing the living room, on oxygen. And ready for this -- Have not had or needed a tuneup since Spring of 2011. That is 3 years without a tuneup. Before that, I was getting tuneups every 3 months. And early 2011 had 3 tuneups back to back which did not improve my condition and almost killed me. We had to leave Nashville for my safety.

Now in S. FL. and my new CF doctor does not think I have any proof and that the stem cells don't work. But at least he talks to me like an adult. At the last appt in December 2013, he said I was doing well and do not need a tuneup. He cannot explain why I am doing so well, but still thinks the stem cells don't work, because I have no proof. I told him, "I AM THE PROOF". My stem cell doctor (Near Naples, FL) has treated over 700 patients with heart problems, COPD, pulmonary hypertension, MS, spinal cord injuries. Over 700 proofs walking around partially or completely healed through adult stem cells.

I still do my inhalers and vest to clear out my lungs. I do that huffing breathing exercise to cough and push up the mucous. My FEV1 is better, it went from about 10% to nearly 30%. But I have learned that the PFT's do not reveal everything that is going on in the lungs. They do not explain the difference in my body between almost dead, and able to walk around all day with out oxygen. They don't explain the difference between not being able to drive anymore (due to a brain fog from lack of O2 to the brain and excess CO2) to driving from Delray Beach to Naples FL by myself and spending a couple of days there to attend a luncheon (recognizing the team of people pioneering the adult stem cell therapies).

I for one, am looking forward to more adult stem cell treatments for further reversal of the damage in my lungs. But I have not proof.

A few comments, about the following. Long term damage reversal after using Kalydeco has not really been tested since it is relatively new. I would like to hear about the people using Kalydeco to see if anyone has increased their FEV1 after the initial improvement. The lungs are pretty powerful when it comes to recovering, after damage from smoking and other toxins after the cause is removed. It is certainly a possibility for those who already are seeing benefits from the drug.

About stem cell usage, I always thought this might have some application. The first time I heard about its use was in revitalizing old dogs. They took blood from the dogs and reactivated the stem cells. The stem cells helped the prolong mobility. Too bad some of the best methods are limited to dogs and off label uses. Younger acting cells always seem to help on many levels. Glad to hear it has helped you. I am guessing the CF community has not pursued it since it does not directly fix the malfunctioning CF cells.

Angelo
04-24-2014, 12:07 PM
I am not a medical expert or a scientist, so reading all the technical stuff is often over my head. But after reading some about the different classes of mutations, the malfunctioning process of each class is different. And the process that Kalydeco addresses is not the same process that goes wrong in class 2 and class 1. I have class 2 and 1, so I don't hold much hope for this drug to make a huge difference. I think it is wrong to give people false hope that this drug will eventually work for all CF.

A few years ago, I started researching stem cells and believed that adult stem cell treatments would be the way of the future for CF and many other illnesses. While I know that are many scams out there, (found those also in my research), there are some amazing advancements and treatments with amazing results elsewhere than the U.S. My own stem cell doctor is in the U.S., but has to do the procedures off shore. What I ran into is the financial and political juggernaut that we call big pharma. They do not want us (YOU) doing these treatments because they have $millions to lose. I have not needed a tune in 3 years.

Adult stem cell treatments is full of hope for all CF warriors. I know because I am one of you who decided to step outside the convention medical routine box. After all, I had nothing to lose. I was weeks away from dying. I apologize if anything I post shows signs of bitterness or anger, but I am angry at the medical system who puts their own profits ahead of our health and lives. I have found another way. And great for those who go for a lung transplant and survive, but how many do not make it. The stem cell treatment has so little risk and no negative side effects compared to lung transplant and other treatments. I was skeptical going into the first treatment, but again, nothing to lose. Each treatment reverses some of the damage. Now I have no reservations about getting these treatments and trust the doctor and his team.

By now, your CF docs may know about him. And trust me, they will say all kinds of nasty things about him. But those same docs who were so negative gave up on me and had no other options. They even told my wife that she needed to get over it and start making arrangements. Code for your husband is a dead man. Alive and healthier 3 years later, do you think I care about all their nasty and unfounded comments.

I write all this out of my passion to get help for all CF patients, but it is a seriously uphill battle, and against all odds. But I and most you are no strangers to beating the odds. God has rescued me off my deathbed, against all odds many times. One of the other CF warriors just lost the battle last week. He was hoping to get the treatments and waiting for the funds. I was in tears all day after reading the email from his mother earlier this week. His doctor, too, were bashing Dr. Grekos, but they could not save his life. Correction: I am not angry, I am furious. There is a great solution, but our government who is owned by the drug companies, will not allow it. All so a few wealthy people can get even wealthier.

There is one problem. Because it is not allowed in the U.S., insurance does not cover the cost. So I have to do my own fundraising. Life could be worse.... Oh, It was! Now its better. Yeah. I thank God every day for Dr. Grekos and his team. I am now 3 years past my expiration date.

Angelo
04-24-2014, 12:14 PM
A few comments, about the following. Long term damage reversal after using Kalydeco has not really been tested since it is relatively new. I would like to hear about the people using Kalydeco to see if anyone has increased their FEV1 after the initial improvement. The lungs are pretty powerful when it comes to recovering, after damage from smoking and other toxins after the cause is removed. It is certainly a possibility for those who already are seeing benefits from the drug.

About stem cell usage, I always thought this might have some application. The first time I heard about its use was in revitalizing old dogs. They took blood from the dogs and reactivated the stem cells. The stem cells helped the prolong mobility. Too bad some of the best methods are limited to dogs and off label uses. Younger acting cells always seem to help on many levels. Glad to hear it has helped you. I am guessing the CF community has not pursued it since it does not directly fix the malfunctioning CF cells.

correct, I am in the face of the director of 2 CFF chapters. Palm Beach County and Broward County(Ft. Lauderdale). One has even contributed financially. They know my story and tried to run it up the ladder with HQ. CFF does not want anything to do with me. Their purpose is to raise money for the research to develop more drugs. They are a fundraising arm for the drug companies.

the treatment for dogs is similar. but because of the genetic mutation that will produce the same CF affected lung tissue, we need a close genetic donor. My Mom has been my donor twice now. They take bone marrow from her. Mix with my own marrow, and will get some body fat from me. The stem cells extracted from the fat are very nurturing and the marrow stem cells are more action oriented. My own in the mix avoids rejection. Then they activate the stem cells with growth factors. next day, stem cells into my arm, IV. Next day I can go home. No side effects, no meds, no anti-rejection meds needed. Then it takes a few months and gradually my health improves.