It's Good, but It's Not THAT Good

I've heard a lot of different reactions to my blog a few weeks back about the clinical trial I'm currently on which will remain nameless. One particular reaction or thought that I wanted to address goes some like this,

"Oh, that's awesome!! I can't wait until it's available because I hate CF and I hate treatments."

I guess I'll just be the one to put it out there - You can take any med currently being studied in the pipeline, and if approved, it will still not take CF away. Now this can mean different things to different people. Obviously, there will be people who get on the right "miracle" drug and it totally flips their life upside down and they start living as though CF didn't exist. I have a feeling that will be rare.

I think it's safe to say that most of us will still feel some if not all the effects of CF daily, but hopefully, less often and to a lesser degree. Many of us will still have to do treatments if we want to be the best version of ourselves.

Case in point: Today's workout was extremely hard and I was dragging booty the entire class. It was hard to catch my breath. I felt low on energy. I was doing more resting than usual and I was light-headed most of the class.

I thought for a second, "Where are you now _______ (name of study drug)?" You see, just because I've had fantastic results from the drug, or the placebo, doesn't mean that CF, or in most cases, just regular life and human reactions, go bye-bye.

I got to bed later than normal yesterday. I didn't sleep as well. I ran/walked 4 miles yesterday and I'm pretty sure my legs remembered. I ate like a 20 year-old college student this past weekend. I'm sure I could have given better effort during my treatments while in San Diego. I was out of my regular routine for 3 days. I think it just all added up.

Here's my point, no matter what drug comes out next, it won't change the fact that we're going to have to work hard. We're still going to have to make good decisions and treat our bodies with respect. The hope of course is that when we do screw up (as I am king at this) the penalty to pay maybe won't be so steep. Maybe, and Lord willing, the next generation will have no clue what "our CF" feels like. All of this of course is only speculation.

The last thing we can afford to do is wait on a med that may or may not be a "game changer". We all need to keep our nose to the grindstone and kick some CF booty each and everyday. This looks like doing our treatments, living an active lifestyle and putting our health first. If we can do that, there is no doubt that we will be the best version of ourself.

I'll leave you with this quote from the great Larry Bird which I think nicely ties up this blog with a nice ribbon. He said, "I find that the harder I work, the luckier I get."

Couldn't have said it better myself.

Top Ten Tuesday: Popping The Pills

Man, it's been quite some time since I've done a Top Ten Tuesday!

Recently, or to be more specific, just over a month ago, I started a new clinical trial. I'm not able to tell you which drug is being researched, but I would bet good money that you have heard of it. It requires me to take a few pills in the morning and a few pills at night. Other than some trips to see the research coordinator for PFTs, weight, height, blood work and EKGs, it's super easy peasy. I've never been involved with a clinical trial that just required swallowing some pills. Usually they have involved inhaling medications which of course is more time consuming.

Anyway, here are ten things that I've noticed while taking the drug, or placebo, over the past handful of weeks...

1. Almost immediately, I felt like I had a bit more energy for everyday activities.

2. I've been able to push harder with increased stamina during my 45 minute "throw-up" class at the gym.

3. I usually run on the treadmill at a max of 5.0. Recently, I've been pushing it to 6.5 or 7.

4. I was unusually nauseous the first week of the trial. It has subsided some, but still present most days.

5. Could be a figment of my imagination, but I seem to sweat more (maybe because I can work harder).

6. My cough has decreased.

7. My sputum production increased the first 2 weeks and has now decreased from my baseline.

8. My lungs feel clearer.

9. I don't know my PFT results, but it feels like I'm able to take a bigger breath and blast out with less obstruction.

10. I'm more excited for the future. I've always been excited, but this could change some things.

There you have it. I'm sure there are more both good and bad things that I've experienced, but I wrote down the first 10 to come to mind. Keep your fingers crossed that others are experiencing the same positive effects that I have while taking this drug or placebo. Because, even if it's placebo, I'm very, very thankful for the results.

Placebo Effect Powerful in Asthma

This article was originally posted on Medpagetoday.com

Asthma inhalers have a real impact on airways, but the symptom relief isn't any greater than that achieved with placebos, researchers found.

Stable asthma patients reported the same degree of symptom improvement with inhalation of albuterol as occurred with a placebo inhaler or sham acupuncture (50%, 45%, and 46% improvement, respectively, P=NS) in a blinded pilot study led by Michael E. Wechsler, MD, of Harvard and Brigham and Women's Hospital in Boston.

All three were equally better than no treatment at all (21% improvement, P<0.001), they reported in the July 14 issue of the New England Journal of Medicine.

"Placebo effects can be clinically meaningful and can rival the effects of active medication in patients with asthma," the group concluded in the paper.

Yet the results also suggested that physicians need to keep a close eye on objective measures of asthma control, Wechsler and colleagues noted.

Albuterol had a strong objective effect on airflow, with a mean 20% improvement in forced expiratory volume in 1 second (FEV₁) -- nearly three times the 7% boost seen in all three other groups (P<0.001).

But "patients could not reliably detect the difference between this robust effect of the active drug and the effects of inhaled placebo and sham acupuncture," the researchers warned.

So "subjective improvement in asthma should be interpreted with caution and objective outcomes should be more heavily relied on for optimal asthma care," they recommended.

However, patients' subjective experiences are not wrong simply because they don't fit the objective facts of FEV₁ and actually should trump physician judgment in such symptom-defined conditions, Daniel E. Moerman, PhD, of the University of Michigan in Dearborn, argued in an accompanying editorial.

"It is the subjective symptoms that brought these patients to medical care in the first place," he wrote. "They came because they were wheezing and felt suffocated, not because they had a reduced FEV₁."

A treatment should be acceptable as long as it yields significant improvement for patients, has a reasonable cost, and doesn't have negative effects over the short or long term, Moerman suggested.

The study included 46 patients randomized to double-blind treatment with an albuterol inhaler, a placebo inhaler, sham acupuncture, or no intervention administered in a crossover design during a total of 12 sequential office visits three to seven days apart after a washout period for short- and long-acting bronchodilators.

The no-intervention group acted as a control for the placebo groups, which few prospective studies have tried, the researchers noted.

The most likely explanation for the greater efficacy of placebo was expectation of improvement as "the mere ritual of treatment may affect patients' self-monitoring and subjective experience of their disease," they wrote.

The study was limited by use of a nonvalidated subjective measure of asthma symptom improvement that didn't include a measure of worsening as well as by unknown generalizability to chronic asthma, the group cautioned.

Original article can be found here

Why don't you do clinical drug trials?

I know I've talked about it before on RSBR, but I just have to talk about it again: Clinical Drug Trials.

Today was my first visit for the new trial that I'm on which will be studying the effectiveness of inhaled Levaquin. I'm super excited for this one, because last year I did an inhaled Cipro study and Levaquin is the "cousin" of Cipro. Why am I excited? Because, and this is "off the record" and completely my opinion, I felt like a million bucks and my lung function increased (mind you, I very well could have been on placebo...doubt it.) Anyway, I'm hoping to have the same great experience with this inhaled antibiotic and I figure if I don't, somebody else is, so it's a win-win in my mind!

I thought I'd list a few reasons you should be doing clinical drug trials:

If not you, then who? If you're one of those peeps who champions a "cure" and does not actively search out drug trials, then let me be frank, you're talking out both sides of your mouth. We will never have a cure without clinical trials. And more immediate, we'll never have better medicines to help control the symptoms. So no more cure talk if you're not willing to put your money where your mouth is.

Speaking of money, you get paid. It's almost too good to be true. You can get paid to do medicines that can potentially increase your lung function? Yes. At the very worst, you get cash money to puff on some saline or pop a sugar pill. This is easy money if there ever was some.

You learn more about CF. Now maybe I've just been blessed with a great research coordinator or maybe she's just wicked smart, but I've learned so much about how drugs work and the anatomy of CF. And to be honest with you, the more you learn about CF, the more you realize that we have a lot more control over this thing than we give ourselves credit for.

You're taking control of your health. Doing a clinical drug trial is just another way of taking control of your health. It's another way of being active and not settling for "the cure" to come to you (when I speak of "a cure", I'm not speaking of our genes being corrected and our cells working properly, I speaking about us being our own darn cure by what choices we make in our lives). It's up to each and every one of us do to everything possible to put ourselves in the best position to succeed.

I could go on and on about the reasons you should be doing a clinical drug trial, but I'll step off the soapbox and save you from my diatribe. On a serious note though, get involved!! Stop thinking about all of the reasons you won't do one, and just do it all ready. That is, unless you can come up with a reason that doesn't sound like a total lame-o excuse, which I am more than willing to listen to.

Which leads me to my next question, why don't you do clinical drug trials?

Clinical Trials Act

'IMPROVING ACCESS TO CLINICAL TRIALS ACT' PASSES U.S. SENATE IN VICTORY FOR CF ADVOCATES

The U.S. Senate last night passed the "Improving Access to Clinical Trials Act" (I-ACT), a bipartisan piece of legislation championed by the Cystic Fibrosis Foundation, its advocates and 120 other health advocacy organizations.

The legislation enables patients with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits.

"We are one step closer to breaking down a serious barrier to participation in clinical trials, which one day could deliver a cure for cystic fibrosis," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "This legislation represents an important opportunity for people with CF to take part in groundbreaking research that was previously out of their reach. We are elated that this bill has been approved by the Senate."

The legislation was introduced by Senator Ron Wyden, D-Ore., with Senators Chris Dodd, D-Conn., James Inhofe, R-Okla., Richard Shelby, R-Ala., Dick Durbin, D-Ill. as original co-sponsors and an additional 14 co-sponsors also signed on.

Current law prevents many people who receive Supplemental Security Income (SSI) from accepting research compensation because it makes them ineligible to receive government medical benefits. This penalty has stopped significant numbers of people with rare diseases from participating in clinical studies.

Following Senate approval, the bill now awaits consideration by the U.S. House of Representatives. Reps. Edward Markey, D-Mass., and Cliff Stearns, R-Fla., are leading the effort to pass the bill in the House. The legislation, HR 2866, is co-sponsored by 135 members.

Passage of this legislation is particularly important for people with CF, a rare genetic disease that affects 30,000 people in the United States. A limited patient population makes it challenging to find enough people to participate in research studies evaluating the effectiveness of promising new drugs.

Source: http://pr-canada.net/index.php?option=com_content&task=view&id=241611&Itemid=58

Another Day, Another Drug Study

Can I just say, I love me some drug studies! I'm pretty much on a schedule now of doing drug studies back-to-back-to-back and so on and I'm already plotting the next one I'd like to do. I figured though that I'd fill you guys in on the one that I started today. Actually, I misspoke, I sort of started it today. I went in to give blood and urine so they could establish a baseline to compare the rest of the data with. Friday is the big day in which I actually get to take one of two doses of the drug or a placebo. I of course hope I get the real deal, but hey, somebodies got to take the sugar pill right?

Ok, so what is this drug? Since I'm no medical mind and although I have a pretty good idea of what it is supposed to do according to my doc, I still won't attempt to answer. I will however let Clinical Trials Feeds give you their answer:

Official Title: “A Randomized, Double Blind, Parallel Group, Placebo Controlled 28 Day Study to Investigate the Safety, Tolerability and Pharmacodynamics of SB-656933 in Patients With Cystic Fibrosis”

Study Drug, SB-656933, is a selective CXCR2 antagonist in development as a novel, once-daily oral anti-inflammatory agent for the maintenance treatment of Cystic Fibrosis (CF) and Chronic Obstructive Pulmonary Disease (COPD). We want to find out if this experimental drug will help decrease inflammation and slow the progression of lung disease. This study compares how well different doses of the experimental study drug (SB-656933) control inflammation in patients with CF. Two doses of the study drug will be assessed against placebo to see which dose works best.

And how will they know if this stuff works? Well, we have to go to ClinicalTrials.gov for that:

Primary Outcome Measures:

• Safety and tolerability of SB-656933 in subjects with cystic fibrosis, including, adverse events, vital signs, clinical laboratory assessments (hematology, chemistry, urinalysis, and VB-1),electrocardiographic (ECG)parameters,and exacerbation of CF [ Time Frame: 28 days and followup ]
  

Secondary Outcome Measures:

• Sputum microbiology at 28 days as measured by bacterial colony counts of Pseudomonas aeruginosa and Staphylococcus aureus [ Time Frame: 28 days ]
  
• Induced sputum neutrophil number (cells/mL) and percentage (%) [ Time Frame: 28 days ]
  
• Induced sputum inflammatory markers: (e.g. but not limited to: NE, MPO, total protein). [ Time Frame: 28 days ]
  
• FEV1 and FVC [ Time Frame: 28 days and followup ]
  
• Serum and plasma markers of inflammation: (e.g. but not limited to: fibrinogen, CRP, CC-16, MMP8, MMP9, SP-D, CXCL8 (IL-8)) [ Time Frame: 28 days ]
  
• Daily Respiratory Symptom Diary for Cystic Fibrosis (Self Reported Version (Exploratory)) [ Time Frame: 28 days ]
  
• Plasma SB-656933 concentrations and pharmacokinetic parameters including area under the plasma drug concentration vs time curve (AUC(0-24), AUC(0-infinity)),maximum observed plasma drug concentration (Cmax)and time to maximum observed plasma drug consent [ Time Frame: 28 days ]

So needless to say, I'm hoping this sucker works cause inflammation is definitely something I battle. I will keep you guys posted on how it's going and let's all hope that this can be another gun in our arsenal in the next handful of years. I guess I better go read the disclosure clause in the contract now :)

Great Results for Fellow Cyster on Denufosol

I asked Katie if I could share her encouraging blog from CysticLife about her experience with the clinical drug Denufosol. Check out what she had to say!!

So I headed to the Adult CF clinic at Northwestern/Children's Memorial today for my first study visit after starting on the actual drug in the second phase of the denufosol trial. I was so psyched . . . after hearing about the fantastic PFTs people were having on the drug . . .

So I blow and blow and blow (I have the "honor" of being one of the patients who can blow out ALL of the candles on the computer screen during my PFTs. I swear it's from years of blowing up balloons and practicing with so many PFTs and really has nothing to do with my lung capacity . . . ha ha. . .). And look at my numbers . . . and my FEV is exactly the same . . . and my FEV1 is 3 % predicted higher - which is actually within my "range."

My reaction: "You've got to be freakin' kidding me . . . .a whole year of blind study and now three times a day for the last three months with the real thing, and this is all I've got?" Granted, my PFTs are relatively normal, but still . . . I wanted some bang for my buck . . . The research team really didn't know how to respond . . .

So I saunter upstairs for my clinic appointment, my disappointment apparent . . .

And then my doctor comes in and shows me a chart of just my FEV1 in actual numbers, not percent predicted, for the last two years . . . it looked like this:

That big jump being today's FEV1 in actual numbers!!!! Two docs were confering as to why such a jump in actual numbers only calculated out to a 3% difference in predicted? They decided they were going to have to look into how predicted is calculated for this study . . . it's based on height, age, as well as "results" so they wondered if something in my combination was making the predicted "off." We may never know.

Needless to say, I was much happier seeing the actual numbers! Screw predicted . . .

So after about 30 minutes of saying denufosol was not worth my time, I changed my tune and will stay on it. It's obviously doing something good. If nothing else, it is containing any exacerbations . . . I've haven't been on oral antibiotics for about 9 months and I'm usually on Cipro 3-4 times a year for three weeks at a time.

It does irriate my throat -but my throat gets irritated if a piece of dust is in the air. The stuff I cough up is undeniably more slippery - so thinner. I still find it to be a pain for 15 minutes three times a day, and quite honestly, once I'm off study and am prescribed it for real, I'll likely do it twice a day more often than three times . . . I've even proposed that to be a study after it gets initial FDA approval. But the proof was in the numbers . . .so I'm a believer.

To be continued . . .

You can find the original post here.

Clinical Trials for Cystic Fibrosis

So first thing that I want you guys to notice is that I changed my shirt. Second, thing you should notice is that Mandi hijacked my bed. Finally, I'd love some feedback on this blog post and some answers to my questions at the end. Thanks guys!