U.S. Food and Drug Administration Approves Cayston(R) (Aztreonam for Inhalation Solution) for the Improvement of Respiratory Symptoms in Cystic Fibrosis Patients with Pseudomonas Aeruginosa

http://www.gilead.com/pr_1393831

For all my buddies who couldn’t qualify for the study or the ‘compassionate care’ program, it’s your time now!!!

(now for the fight for insurance coverage… fun fun!)

Hi y’all!

I survived the swine flu. I honestly have no idea how I did though. It was horrendous. I was so weak, I couldn’t even lift a bottle of Poweraide Zero to my lips and needed a straw. Since then it has been hard to get back on my feet again.  I’ve had two rounds of IV antibiotics with about 3 or 4 weeks in between. I’ve never needed IVs so close together before. If any of you cfers are debating on getting the shot, my advice is to GO GET IT!  :)  Just make sure it’s the shot and not the nose spray.

Slowly I will be getting back in the swing of things, more blog posts, replying to email, updating the format of the site like I promised long ago.

More to come!!!

Actelion – Miglustat (Actelion is a Swiss drug company developing/testing Miglustat)

Miglustat Shows Promise in Treatment for Cystic Fibrosis (article from bio-itworld.com)

Pretty interesting!  It is easy to live in this little bubble of a life I have, sometimes it we forget there may be other things going on.  For the most part I only know of cystic fibrosis studies, in the US, and that are sponsored by the CFF(Cystic Fibrosis Foundation). I have forgotten there is a whole-nother world out there! Thanks so much for this info (and hope) Nathan.  I wonder what in the world are other countries studying.

Nathan has me wondering, what other treatments are being studied in the rest of the world, outside the US, outside the realm of the CFF.  Surely there must be some drug companies studying CF aside from those receiving money from the CFF. I wonder what is going on outside this little bubble I live in…

Here’s the comment:

Dear Salty:

I think you’re being a little unfair to the FDA. They made a decision the CF community didn’t like — but that doesn’t mean they’ve been unethical as you suggest. And “foot dragging” would have meant no decision at all.

Still, I share your irritation because nobody — not even the CF Foundation — seems inclined to to explain exactly why the FDA decided that further clinical study was necessary. It always gets my dander up when the experts won’t let the regular folks in on the secrets, especially when their work is of such importance to CF patients like yourself.

The decision about which you complain was actually made last September and, as best I can tell, did not result in all access to inhaled astreonam being cut off. From the CFF web site:

“Because the FDA did not raise any safety issues related to the drug in their ruling, Gilead [the manufacturer] will continue to provide early access to aztreonam lysine for inhalation for those CF patients with limited treatment options and a serious risk of disease progression. Individuals with CF should contact their care centers to determine their eligibility to participate in the EAP.”

Presumably you’ve done that . . .?

My reply:

Although I appreciate your comment, it appears you aren’t completely up to speed on the study I speak of. The FDA has made clear their reasoning… the new board is not up to speed (pardon the pun) with the needs of cystic fibrosis patients, nor understands the need of getting drugs to market quickly to help save people’s lives.  As far as “foot dragging” I don’t know exactly how to address that.  The drug was up for review, they asked for more study results, Gilead studied, got more results, appealed the decision, the FDA now asks for another study, which will be reviewed at yet a later date… despite ”the FDA did not raise any safety issues related to the drug in their ruling” as you quoted.  I don’t know if there are better words than to say they are dragging their feet …as I have stated before, numbers are my game, not words so I apologise for my lack of vocabulary. …Yet this entire time  If there are no safety issues are related to the drug, then why on earth are they not approving it? I just don’t get it, and I guess that is what I was trying to describe in my previous post.

The inability to receive proper treatment for one month, may shave YEARS off a cystic’s life, no exaggeration there my dear. Speed means lives being saved. An ounce of prevention is worth a pound of cure my mother always told me. A good solution now is better than the best solution ten years from now.

For example, the year previous to my start on inhaled aztreonam I suffered horribly from lung infections, three rounds of IV antibiotics, at least three weeks in each round. And guess what drug I was infusing in my veins????? – AZTREONAM! Now if I happened to be lucky enough to have enrolled in the expanded access program at that time, I could have saved my insurance company quite a bit of money for the hospitalization, picc line placement, home nursing… the list goes on and on. Plus my employer would have benefited, not only from my ability to work instead of being sick (considering I was sick for weeks prior to each IV treatment), but maybe it would have kept their insurance premiums from going up astronomically (but that’s just speculation).  Not to mention that since starting the study I have not had a single round of IVs, and my need for oral antibiotics (which are pretty much useless to me anymore) has almost deminished.  I have yet to miss a day of work since starting on this program due to lung issues.

An excellent description of why the FDA has not approved the use of inhaled aztreonam, and has denied appeals since September 2007, is spelled out here by the Cystic Fibrosis Research Institute.  I found it with a simple google search, yes, I do my homework. APPEAL TO FDA FOR APPROVAL OF INHALED AZTREONAM TALKING POINTS FOR ADVOCACY (pdf file)

Perhaps the CFF has been keeping the reasoning quiet as to not step on the FDA’s new board member’s toes? You really don’t want to go pissing off someone saying they aren’t knowledgeable of a topic when they have the authority of denying what you are desperately trying to get approved.  Especially when people’s lives are at stake. -makes sense to me.

Not just anyone can be on the expanded access program (link to details on ClinicalTrials.gov), unfortunately. There are 115 CFF approved care centers and only 65 are participating in the study. Also the study is limited to those who have a lung function with an FEV1 LESS than 50%.  Hence, the Expanded Access Program is not open to those with a lung function above 50%, which rules out most children, adolescents and young adults who definitely could use this drug as a preventative measure, allowing them to keep their highly valued high lung function until a cure comes along.  This program does not account for other people who may not have cf, but still culture pseudomonas, or other bugs that aztreonam could possibly treat.

Yes, I am on this expanded access program. I am getting my inhaled aztreonam for free.  If I didn’t think it could help others, I sure as hell wouldn’t be wishing the FDA would hurry up and approve this drug because then I’d have to open up my wallet.  BUT, out of the goodness in my heart, I wish others could get on this drug, the ones who do not qualify to be in the study, because I know for a fact it has extended my life in years, and improved my quality of life greatly.  I wish the same for my co-cystics, my transplanted buddies, and others who must deal with the nasty vicious Pseudomonas Aeruginosa maybe due to severe lung disease like COPD, PCD, and the list goes on.

I must say again, I just don’t “get it”.

Neither drug is a “cure” for CF.  To me a cure would mean that the CF mutation would be completely fixed with no further treatments needed, nor would the CF mutation be passed on through the future generations.  The Vertex drugs do neither of that.  Regardless, both drugs are hoped to be almost a miracle for us with CF if the drugs actually do what they are predicted to do.

Before I go on about these drugs (in a future post), I want to air a frustration I am having with the CF community specifically regarding the excitement and hope that this drug will be “the one” to fix all our problems.  Not trying to be Debbie Downer, and don’t get me wrong because yes I am excited about these drugs too, but it seems like everyone is getting wrapped up in the hype and is forgetting the reality that we have been here before specifically with the gene therapy studies of both Copernicus and Targeted Genetics.  Both companies developed a gene therapy (like early to mid ’90’s? maybe) that would also “fix” CF at the mutation/cellular level (but just in the lungs where most of the life shortening damage is done), both showed great promise, but both companies struggled to actually make things happen mostly because of funding and then some other issues… I feel right now the same excitement of the Copernicus and Targeted Genetics studies started up many years ago, but this time I have some reservations to throw all my hope into one drug.  To me, the Vertex drug it is not YET considered to be “the biggest breakthrough since finding the gene in 1989.”

The biggest fear I have is that if for any reason these drugs don’t make it to market or don’t work the way they hoped, or have some horrible side affect that does more harm than good, what is going to be the affect on CF research overall?  I fear that with the excitement and energy put into this one drug we are putting all our hope in one basket, all our effort in one place, that if this doesn’t work, we are screwed because we all assumed it would work.  I wonder if there are other drug companies that may have great ideas, who may be putting their ideas on hold waiting to see if this Vertex thing works out.

This really concerns me because I’m one of the adult Cystics who didn’t grow up with some of the major life extending drug therapies that the kids have now, like Pulmozyme and Hyper Tonic Saline.  I have no extra time to spare. I don’t have time to waste putting all hope and energy in one place.  So please don’t blame me for not getting all psyched up about the Vertex studies.  I will get pissed off when the media claims there is a new “cure” for CF, because if I have to take a pill twice a day (along with the others I am already taking due to permanent lung damage and because I have absolutely no pancreas left), yet if I have children of my own, or grandchildren if I am ever so lucky, there is still a chance they may have to do the same. That, to me, is no cure. So the fight for a cure must go on.

Random glucose test – This is usually a fingerprick, or done while draining your arm/body of blood for other tests.  They test the amount of glucose (sugar) in your blood at the given moment.  Two random tests above 200 mg/dl is a diagnosis for diabetes.  That is kind of tricky for us with CFRD since illness is common and so is steroid use, which will give you higher than usual blood glucose levels.

C-Peptide test – I was told there is no real ability to measure the amount of insulin your body produces.  But when your body produces insulin it also produces a protein, c-peptide.  In theory if your body does not make enough insulin, you won’t make much c-peptide either.  Usually this means that your pancreas has given up the ghost and is no longer working properly.  I had this test done, as required to get insurance coverage (my crappy old policy) for my insulin pump.  Welllll… I failed the test… or did I pass it?? Turns out that my body makes enough c-peptide to qualify me as “normal” and not needing insulin.  Well… that is by far, not true.  The exact cause of CFRD is truely unknown to this date.  Of course our lil pancreases are on their way to complete self-distruction (which mine showed via a CT scan), but there is a study out there that claims the creation or use or something about the natural made insulin doesn’t work correctly.  Which is why I apparently have insulin dependent diabetes, but still have a normal amount of c-peptide (or at least I did last year).

Antibody test -  This is where Type 1 diabetics differ from all other types and exactly why CFRD is NOT Type 1 (although treatment is similar which is not to be confused with cause).  Type 1 diabetes is an autoimmune disease which is caused by antibodies that attack the insulin producing cells, the beta cells, in the pancreas.  If you have diabetes and have antibodies, you have Type 1.  If you have diabetes and do not have antibodies, you have a Type Other than Type 1.  A cure for “Type 1″ diabetes will not cure my diabetes and that is why I am such an advocate for the “Type Other”s.  It is possible for someone with CF to have Type 1, it is the same rate as for the “normal” population, but just because you have cystic fibrosis related diabetes and you are insulin dependent, this does not make you Type 1.

Urine Sugar tests – If you have too much sugar in your blood, it will spill over in your kidneys (which is not good) into your urine.  Usually one of the first tests they do when they suspect diabetes.

Liver function tests – For folks with CF our livers are at risk of damage from all the drug coctails we are required to take.  I am only mentioning this test because I know a few CF docs who try putting their CFRD patients on pills for treatment instead of insulin shots.  I guess pills are ok, but I would be hesitant because most diabetes pills have a high risk of causing liver issues…. Just one more thing I do not want to add to my list of things I need to deal with.

I am sure there are plenty of other tests I am not noting in this post.  Anyone else want to chime in?

side note: I have wondered since the extra glucose messes up the red blood cells, I wonder if it affects how oxygen, and how much of it, is carried through the blood stream (since it is also carried by the hemoglobin) which is kind of an issue for some of us cystics with messed up lungs.  I’m going to look more into that someday. But if I find that is true I am going to get even more pissed at the CF docs who put off CFRD testing (not mine luckily). *sigh*

Apparently a “normal” person would have an A1c level at about 5.0%.  My doctors suggest an A1c of 6.5% or a little lower, normal would be ideal, but that is darned hard to reach.  My docs find that above 6.5% it seems to really affect the CF lungs.  To me I really couldn’t understand what that number meant until I found a calculator online that could translate the number into something I was familiar seeing on my little meter tester thingy I use every day.  Here’s one I like, it goes both ways from A1c% to mg/dl.
http://www.accu-chek.com/us/rewrite/content/en_US/4.1.2:20/article/ACCM_general_article_2422.htm
Here’s a little chart on dLife:
http://www.dlife.com/dLife/do/ShowContent/blood_sugar_management/testing/a1c_conversion.html

154 mg/dl is the magic average number.  Personally, what I try to do is make sure I am at a safe, but lower number (preferably 85-95) through out the night since I am spending 8-9 hours at that level.  Recently with my Continuous Monitor I have found I am having a strange spike at 2:30-3am which means I am going to need to change my basal insulin rate at that time… but that is a whole different story.

Once last year I scored a 6.0% on my A1c test (yes, it went on the refrigerator)… I really doubt I am there again, it took a lot of hard work and discipline, but here is some tricks of how I did it…  Having a lower level for many hours while I am asleep made it easier to get to my goal A1c level.  When I spike at meals, I try to keep it under 200 mg/dl at the two hour mark by counting my carb intake as close as I can (IMPOSSIBLE when on evil evil prednisone), but I make corrections at the 2 hour mark if I have to (my pump figures out the calculation taking in consideration how much active insulin I already have in my system, which is why I am such an advocate of the pump, it does all the brainpower).  I have played with my numbers and found at my of 1 unit of insulin (which is an unbelievably small amount, it’s like a few drops!) for every 8 grams of carbs and my correction or sensitivity rate of 1 unit to 25 mg/dl (how much my bg number will drop with one unit of insulin), If I miss calculate my carb intake by 20 (two macroon cookies, who ever eats just two cookies?!) my blood sugar level will go up 62.5 points (mg/dl). HOLY!  That means I need to keep quite a close eye on things.  It never used to be that way when I was on shots, I was on a 1:15 ratio…. but then again, I was never in control of things on shots.

Back when I was out of control, my A1c level (at least the first one I really paid attention to) was 9.3%.  That number alone wasn’t enough to scare me.  But it should have.  The toll it took on my poor body was too much for it to handle and I have not escaped a few complications.  Nothing too scary, but annoying enough to get put on a few extra medications.  I wish I would have known then what I know now.

The Oral Glucose Tolerance Test, OGTT, has been used as the primary diagnostic of Type 2 diabetes as well as some of the Type Others of diabetes like CFRD, (once again, CFRD is not Type 1 OR Type 2 we are Type Other go look it up on the wikipedia!).  The OGTT is time consuming and fairly painless.  Like it says, the test is done by intaking glucose oraly and they test your tolerance to it.  In other words they take a few blood draws (or fingersticks) before and after you drink something like orange soda and see how your body reacts to the sugars.  If it doesn’t react properly, then the finger points to diabetes.

Getting ready for the test… Don’t eat or drink from midnight the night before, they say 8-14 hours.  I am not sure if black coffee is ok, but it seems like caffeine has different reactions to blood sugar levels in different people, so if I were you having this test I’d drink water and only water.

Next, you show up at the doctor where they sit you down hopefully in front of a TV because this test takes two hours of basically just sitting there.  Some doctors (or nurses) use a small IV to draw blood, so they don’t have to stick you mulitple times.  Others use a finger stick blood glucose meter, which you just better learn to deal with if the test comes up positive (I have to use my fingerstick thingy 4-8 times a day, sometimes more).  I had my OGT test 7 years ago, and I think they used the fingerstick method… but my memory escapes me.  …The nurse sits you down and draws a fasting level which will show what your blood sugar level is when you have no food in your system.  This alone can be enough to diagnose someone with diabetes (as it was for me).  If your pancreas does not make enough insulin to regulate the sugars normally produced in your body, even without food, more than likely you have da ‘betes, they call that fasting hyperglycemia.  Pretty much, if you have fasting hyperglycemia, you have diabetes.

As a quick note, to explain how insulin works simply, it is like a key that unlocks the cells and allows them to take in the sugar (ie glucose) and turn it into or store it as energy to use in the future.  Without insulin (made by your pancreas or by supplemental shots) it is not possible to change the nutrients into energy.  Basically put.  Without insulin, sugar builds up in your blood which wrecks your blood vessels and does some other nasty stuff.

So… back to the test.  After they draw a fasting blood glucose level, they hand you a bottle of this sickening sweet drink that is like a flat orange soda with a little extra sugar added.  If they are nice enough, they will have refridgerated it for you which helps it go down a lot easier.  (Ooh! If you are going for this test you could make a funny out of it and bring in a swirly straw and a drink umbrella!!!  Bet the nurses would be a little nicer when they stick you for blood! Wish I would have thought about that before! hehe!)  Back to the matter…  I have heard a lot of folks complain about how sweet that drink is.  But to me, when my body wasn’t absorbing enough energy from (not enough insulin to do so) I craved sweets and other carbs night and day, the warm flat sugary soda tasted so yummy.  For those of you number crunchers, the “soda” has 75 grams of carbs in it.  Regular orange soda has 35 grams in one cup… so it really isn’t much sweeter than two cups of the real thing.

At 30 minute intervals they test the level of sugar in your blood until the 2-hour mark.  They should be able to tell you right then and there what the number is.  I stole the following data off of the American Diabetes Association website it is in reference for ANYone testing for diabetes, no matter what Type (and are also used for the diagnosis of CFRD):

Fasting Levels:
Normal Fasting <100 mg/dl (mg/dl is the unit measure used in the US and what would be shown on your fingerstick meter if you are using one)
Pre-diabetes Fasting <125 mg/dl
Diabetes diagnosis Fasting >126 mg/dl
For me, I was above 126 fasting, so we already knew at that point I was screwed.

At the two-hour mark:
Normal <140 mg/dl
Pre-diabetes 140 to 199 mg/dl
Diabetes diagnosis 200+ mg/dl  (which I hit at the first 30 minute mark)

If you really are concerned and your doctor is not listening to you, you could try the test at home if you have a meter and some decaffeinated soda (doesn’t have to be orange flavor, I would prefer grape or strawberry… ooh! or Squirt!).  Make sure you do the test in the morning though because your body makes natural steroids throughout the day which will affect your numbers.  But still, if you do the test at home, it is not an official diagnosis, but it may put your mind at ease if it’s negative and you have been worrying.  Oh! And make sure you are not taking steroids or sick.  Those two can really throw off your results.

I have been quite frustrated and disillusioned by what some of my fellow cystics have said to me.  They have shown interest in getting the test done because they are showing signs of CFRD and they want to take a proactive role in taking care of their health.  Some of their doctors say, “nah, you don’t really need it” or “go to your primary care physicain”… do these doctors have no clue in how untreated diabetes affects your health? especially your CF health?!!  I just want to shake those doctors.  It’s a simple test which could save someone a lot of pain and suffering.  Untreated and uncontrolled diabetes makes you MUCH more prone to infection, which is a cystic’s worst nightmare!  Not to mention all the other crud that comes along with it….  Come on doc’s get with the program and TEST YOUR PATIENTS!  Personally, I go with the CFF (Cystic Fibrosis Foundation) guidelines as a baseline, more can be done, but less is just irresponsible. The CFF suggests that anyone (with CF of course) who is pancreatic insufficient (takes enzymes) be tested (via OGTT) every year after the age of 12 if they show signs or symptoms.  Some docs think the A1C test is enough… but I’ll get into that later.  Next post will be about the A1C test.

Hmmm… well I think that sums up the oral glucose tolerance test.  Any questions?

I’ve had a weird tingly feeling in my legs in the evenings for years now, and finally brought it up to my CFRD doc.  Turns out it could be neuropathy (nerve damage pain) which for me was caused by years of not really caring about my diabetes.  Apparently a simple treatment for the discomfort exists, over-the-counter capsaicin cream.  Yep, it is a cream made with the burnin’ hot oil that comes from hot peppers!  Who would have guessed!  I have been a bit nervous to try it.  I still have flashbacks of an old guy friend of mine who… ummmm… wasn’t so careful after cutting up peppers for salsa then went to the little boy’s room… let’s just say it wasn’t his eyes that were hurting, but many tears fell that night.  DO NOT TOUCH SENSITIVE SPOTS AFTER TOUCHING HOT PEPPERS!

My legs were super creepy crawly, driving me crazy.   The feeling is always worse when my blood sugars are bad, which they are right now thanks to the lovely Prednisone and a lack of continuous glucose monitor sensors (my refill order has been sitting on someone’s desk for the past few months, no explaination why except it is not the fault of my insurance).  I finally talked myself into giving the capsaicin cream a try.  Tried a little dab… nothing…. rubbed more in… eh, it kind of took the tingling away, a little… my feet felt like they were lathered in regular ol’ body lotion.  No cooling sensation, no burning… nothing special.  Reading the lable it sounded like it was supposed to feel like icy’hot.  Brodie, his little inquisitive doggy mind, oh boy… When I wasn’t paying attention I felt the slightest lick of his tongue on my feet.  I would have loved to see how big my eyes got when I felt that!  I was afraid his little tongue would fall off or something… I mean, what would I do if it burnt his tongue?!  then… nothing.  lick… lick…  Didn’t even faze him.  Strange… was I just duped into buying into a placebo?

The next morning when i woke up my feet were on FIRE!! Holy smoke!  Turns out that it was a bit much for my super sensitive skin.  At least I know it wasn’t just plain lotion now.  I think I will give it another try sometime possibly soon and let you know how it goes.  Definitely, I will be using much less next time.