Science, Not Statistics

 

I’m alive today because I relied on scientific research to find a treatment for my rare disease. Without the drug I used off-label, I wouldn’t be alive today. My internal bleeding was becoming progressively worse, and it was only a matter of time before I faced a situation that could have had a terrible outcome.

So why am I writing about science? Because today the rare disease community, our families and friends need to unite to send a message to the FDA. Next Friday is an important meeting for the Duchenne Muscular Dystrophy (DMD) community as Sarepta’s Eteplirsen is up for review. This drug has the potential to save many lives.

I have seen first hand via social media just how powerful Eteplirsen is. With DMD, boys are not able to walk once they reach their teenage years. Rare family members have gifted us with videos and photos of their sons who are participating in the Eteplirsen clinical trials walking, playing, and just being normal kids. They’re not losing abilities like the children who aren’t receiving the drug. While it is heartwarming to see improvements, I am constantly reminded that other families are waiting while their children are losing strength. Time is of the essence, Duchenne Muscular Dystrophy is not waiting for FDA approval of Eteplirsen. Just about every week my timeline lights up in remembrance of another son lost. How many more of our boys have to die before the FDA approves Eteplirsen?

There are no safety issues with Eteplirsen, I want to highlight that. The FDA has taken issue with statistical data. Let me explain to you why this enrages me, and why I see this as an attack on our entire rare family, all 30 million of us.

In October 2014, there was no statistical data to tell me that Rituxan would put my ITP in remission. In fact, if I just analyzed raw data, I had less than a 50% chance of it working. Why? Because Rituxan was being used on an entire ITP population, not those who had platelet destruction issues which the CD-20 inhibitor would address. Instead, I used patient data- meaning I analyzed what those who had successful Rituxan treatments had in common. I found the common links on our blood work, how we responded to other treatments, what our doctors thought, even how we bled. I researched the science behind how Rituxan works, what exactly it does to the body and how that relates to the destruction of my platelets. I went in to my doctor and I asked for Rituxan. I knew it was my only hope, I knew my life depended on it. And I was certain that with my research, I had an excellent chance of success.

Rituxan saved my life, but it was NOT a miracle, it was science. 

Eteplirsen is to Duchenne Muscular Dystrophy what Rituxan is to Immune Thrombocytopenia. It isn’t about statistics because these treatments aren’t meant for everyone and you cannot apply mass-market measures to orphan drugs for a complete analysis. You cannot expect small patient populations to produce statistic data sets that rival those of drug trials for diseases like diabetes or COPD.

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Success for orphan drugs lies in science. It is in the photos and videos of 15 year old Billy (above with Pluto), who still has the ability to walk. This wouldn’t be possible without Eteplirsen.

Billy is living proof that Eteplirsen works. He is the walking scientific evidence of a safe and effective treatment for Duchenne Muscular Dystrophy.

So friends, I ask that you take a few seconds to fill out this short form that will request members of Congress to sign a letter urging the FDA to apply the FDASIA tools to Duchenne which would allow for an accelerated approval of Eteplirsen. This historic moment will set the stage for future orphan drug development, so your participation is critical.

Parent Project Muscular Dystrophy has a short form you fill out with your name and address, and they will send the letter to your representative for you.

Let’s end Duchenne.

Tis The Season for Action!

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Kona wanted to make a special holiday appearance in honor of our call to action for the Senate over the 21st Century Cures initiative. I sincerely hope that when I arrive in DC in a few weeks, progress will have been made. There is no time to waste when 30 million Americans depend on Congress to take action.

My blog is a road map of my rare disease journey, starting last year when I decided to take a chance and use Rituxan off label. It is not approved for ITP patients. Why? Because we don’t have enough information and research on ITP to conduct a comprehensive clinical trial. One of my goals for 2016 is to change that. Through my research, I have been able to isolate a population of ITP patients who can and will benefit from use of the drug. Simply put, this drug is a life saver and the people who achieve remission from it enjoy a few years of a fairly normal life. There is absolutely no reason why this should be denied to people like myself.

Tomorrow is December 18th, which happens to be the one year anniversary of my remission. I truly believe this gift was bestowed upon me to give me a chance to fight for others. I have lost a few friends in the past year to ITP, it doesn’t get any easier and it makes me realize just how fortunate I am.

I’ve spent the last 365 building my new normal, now it is time to change medicine.

Chronic Illness Gift Guide 2015

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Since being diagnosed with fibromyalgia right before my birthday, conserving spoons has become a priority. About this time last year, I wrote a gift guide for prednisone. Now I want to compile a list of gift ideas for people who suffer from chronic illnesses and fatigue. These gifts will help out that spoonie in your life!  Continue reading

Prednisone-Induced Hatred of My Body

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Two weeks before I was hospitalized and began the fight of my life.

I admit I hate my body. I hate that it fights with itself, I hate that is destroys platelets and my joints. I hate the way I look after almost eight months of heavy steroids. I can’t fit into my clothing, I feel disgusting and I’m ashamed of what has happened because of those evil white pills. I lost every sense of my ‘healthy’ self, and the process of getting back to where I use to be is extremely slow. Continue reading

Win a Signed Copy of How to Heal a Bruise!

A wonderful platelet pal, the owner of ITPandMe.com has published a book! I am so excited to be giving away three signed copies of the book.

Rules:

Contest begins on Wednesday, September 23, 2015 at 11am EST.

Three (3) people will be selected to win a signed copy of How To Heal A Bruise by sharing a social media campaign message to raise awareness of ITP.

#ITPAware day, Sept 25! Check out How To Heal A Bruise: http://itpandme.com/itp-awareness-day-coming-soon/ #raredisease @RareCandace @meghan_brewster

**Make sure you put Meg & I in the tweets so I can see them!**

Also, I will accept bonus submissions if you tweet using #ITPAware and link to How To Heal A Bruise on Amazon!

BUT WAIT!

Take a photo holding a sign discussing life with ITP, the 21st Century Cures Initiative or how much you hate Prednisone and you will receive TWO (2) bonus entries!

Note: you’re giving me consent to use your photo on social media to continue to raise awareness about ITP. Get creative and have fun! Make sure you tag me on Instagram and Twitter: @RareCandace 

Winners will be announced Thursday, October 1st at 5pm EST (if I am late, forgive me as law school and my new autoimmune issues have been kicking my butt lately).

This isn’t About Passion

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In the last few months as I slowly get to know professors and administrators in my school, I have noticed a trend. I wouldn’t say it is an academic thing, or law school related, rather it is just ignorance. When I express my motivation (or insanity) behind deciding to go to law school such a short time after being diagnosed with my rare disease, I’m met with a strange reaction. Everyone says the same thing except in different ways. It boils down to, be passionate but focus on your studiesContinue reading

Stay Out of My Bubble

With two weeks of law school completed, I am physically drained. It isn’t due to the work, I have worked longer and harder hours doing physical and mental labor that was nothing short of torture at times. The difference now is my health. I cannot sustain myself for extended periods of time because my body has to try and fight off germs from the hundreds of people I encounter at close proximity every day. I almost exploded this week because people can’t respect personal space and therefore were making me worry that I might become sick. Up with a scratchy throat at 8am on a Saturday, I’m afraid that my concerns were justified. Continue reading

Five Things I Learned Living With ITP

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September is ITP awareness month, so it is only fitting to start off with a list of the things that I have come to discover about life with low platelets. It has been a challenging, difficult and frustrating journey. However, I would be lying if I said I wasn’t a better person because of it. Continue reading