Thursday, September 15, 2011

September 15, 2011: Mission Accomplished and Home Again...


I am writing this on the plane ride home from D.C. since I won’t get in till late tonight.  We are bouncing through the skies above Virginia trying to get above some bad weather apparently.  Boy, I hate turbulence… as if anyone actually likes it, hahaha.

Well, mission accomplished...

... But before I go on about today, none of this would be possible without Ang and her mom holding down the fort back home so that I could be not only physically present but focused and mentally present as well.  Thank you, ladies!  

Today, Dr. Tolar presented the results of the 17 patients that have enrolled in the EB Bone Marrow Transplant Clinical Trial at the University of Minnesota, and I gave the beginning and ending remarks to the panel discussion on regenerative medicine and cell-based therapies. 

As Dr. Tolar described it, “It was a 1-2 punch.”

He hit them in he head and I hit them in the heart. 

Here is my opening statement.  Unfortunately, my awesome camera person thought my camera died when it auto-shut off, so the closing statement wasn’t recorded.  It was my fault for not telling her about that feature!

Here is the transcript of my closing statement…

For those of you that regulate the doctors, help me understand, who regulates you?  You claim to stand for protecting the vulnerable, but how do you rectify all those that die waiting in line?

Parents of children with rare diseases LIVE their children’s disease.  They ARE, in many cases – as was stated – THE experts.  Why?  Because no one is more responsible for your child than you.  To the Academics, Government, and Industry: you get to go home at the end of your day.  Parents don’t punch a clock. Don’t dismiss parental consent.

If the reward of all this is progress, the path to progress is unfortunately sometimes through failure, and always involves some level of risk. No risk, no reward. No risk = No progress.

Lastly, this has been a stimulating day and a half conversation, but will disappear into history without action items.  So, what one thing can you do this week to address the legitimate challenges unearthed here?  Ideas are king, but nothing without implementation.  Your presence here indicates your passion and your commitment.  What will you bring home with you?  What difference will you, yes, the person in YOUR seat, what difference will you make?

Go make it.

Turns out some of them got the message.  After the workshop ended, I had 3 FDA regulators and a bioethicist come up to me and not only tell me how much my statements moved them, but two of them got into a very spirited brainstorm with myself and Dr. Tolar as to how to take the clinical trial to the next phase of approval based on our combined testimony today, including how to apply for more funding immediately through one particular channel.  My heart delighted as I watched them exchange business cards and talk about next steps. 

In addition, the head of the entire event, who works for the NIH, came up to me afterwards to thank me for coming and speaking.  I told her that I was accountable to the entire EB community for what comes out of this meeting, so how could I follow up with her to make sure there is outside accountability in the processes that were identified at this event, and she gave me her email address and invited me to reach out to her personally when the first draft of the white paper from this event comes out (which will be in a month according to Dr. Wagner) and she will keep me involved. 

I have to tell you how intimidated I was to give the audience both barrels, because there is a large part of me that wants to be liked by everyone and is also non-confrontational.  However, Drs. Wagner and Tolar were counting on me, and I know I’ve written about this before (a while ago), but what is magical about the transformation that occurred when I set foot behind the podium was the same thing that gave me strength with all the docs along the way… it was all for Bella.  

Being Bella’s daddy and essentially (still) fighting for her has given me a level of purpose and - not confidence - but courage that I simply don’t possess when it comes to fighting for me.  Who knows, maybe it is rubbing off a little.  I talk a good game, but inside, I’m still just a kid who got beat up for talking back as kid on the playground, and watched my 5 best friends murdered for fighting back with their landlord as a young adult.  There is a deep vein inside of me that says standing up for yourself and fighting back can get you hurt or killed, and I have to overcome that totally irrational feeling.  Doing it for Bella seems to make it much easier. 

I have to say that I felt so privileged to speak with the caliber of researchers that attended this workshop.  The best researchers across the country in gene therapy and cell therapy came and presented the amazing groundbreaking research they are doing, coupled with the obstacles and barriers they face along the way.  Holy cow; what a devoted, and really, really smart group of people!  So many great people are working so hard to help kids of so many different rare diseases.  It was very heartwarming.

Dr. Wagner did an awesome job taking notes over the two days and put together an initial white paper of all the topics, challenges, and opportunities covered which will go out to all of us participants this week for review, revision, or addition in case he missed something.  I’m really privileged to support him.  He has a huge heart and endless passion as well as leadership.  He and Dr. Tolar just continue to inspire me to do what I can on my end to realize this collective vision of EB becoming treatable. 

One thing we uncovered today which is REALLY EXCITING is that a treatment moves from “experimental” (read: almost no insurance reimbursement) to “standard of care” (read: insurance reimbursement!) after a certain number of hoops have been jumped through.  Well, that guy from the FDA that was talking to Dr. Tolar and me felt very strongly that Dr. Tolar has demonstrated sufficient data to move the treatment at the U of M to that standard of care level.  This is really exciting, because it means the FDA will give its stamp of approval, which will mean the U will be able to bill insurance companies to cover the treatment!  Now… of course he was careful not to promise anything in stone, much less a timeline for this, much less that insurance companies will actually reimburse, but they won’t be able to hide behind the “experimental” defense and say no to coverage!  Progress, progress, progress.
Now onto the follow up.  That is the key to all of this.  Too bad I am not an implementation specialist… I am an idea guy and a connector… front end stuff.  Good thing my amazing wife is the queen of implementation!  Honey?  Will you follow up on me following up? LOL.  Hey, play to your strengths, right?

After the workshop, I had 5 hours to kill before my flight, so me and my bags hopped on the metro (D.C. subway), went to the capitol district, hopped on one of those double decker buses, and spent 90 minutes in awe of our beautiful capitol.  Then, I hopped off the bus, used my smart phone to find the nearest subway station, and hopped over to the airport on the subway with oodles of time to spare.  Man, public transportation… it’s amazing!  Enjoy my tourist pics!

National Archives.

U.S. Capitol.

Smithsonian Institute.

Jefferson Memorial.

Lincoln Memorial.

Washington Monument.

The White House.

Lastly, I am excited to be on my way home to my family.  I can’t wait to see each one of them.  I love being a family man.  Earlier in my life, that statement would never have come out of my mouth, and even through the tragedy we have endured, the sublime joy of loving four other people as much as I do has totally been worth all the pain of loss along the way.  I’d do it again EVERY TIME.

God night.

Peaceful Bella...


  1. Great job, Tim!

    I am sorry that my English is not so good to understand everything from the video, but your closing words are very impressive!

    The news that the trial will be financed by the insurance (fingers crossed) is inspirational not only for you, Americans, but also for the EB community abroad. I means the cure is coming nearer.

    I'll send you and e-mail with some questions. I hope you can help me with some info.


  2. I'm so proud of you, Tim...for your passion, your eloquence, and the love you show to your family and the families of others who are suffering. And if I who am a stranger feel this way, I can't even imagine how proud your family is of you...especially Bella who must be saying from above, "That's my dad!"

    Let's hope this is the beginning of greater treatment opportunities for EB kids and ultimately a cure.

    Be blessed, sweet family, and have a great weekend!

    A friend in NC

  3. That was amazing! There was no doubt in my mind that you would do a wonderful job and you nailed it!

    You are so right...Bella is remembered never to be forgotten and also so loved. As I listened to you speak I watched the photos on the right...the tears flowed.

    Sending my love to all, sweet kisses for Ali and Julian. Also Julians package (with a little something for Ali) is on the way today.

    Denise WI

  4. Tim, your speech was inspiring and brought tears to my eyes. I would love nothing more than to work on clinical trials without spending years looking for the funding to do it.

    It has been nearly one year, but the passion I feel for Bella and perfecting EB treatment is the same. Thank you, and your whole family, for your incredible strength.

    Sending my love,

  5. Well done! I am so prowd of you! I'm sure Bella is even more so!

  6. That was amazing. You should be so proud. I knew you would do a great job, just like you did caring for her. Great speech, and great feedback, wow!

    So good. MUCH LOVE

  7. The things you are accomplishing to cure this disease are AH-Mazing!! After following Bella's, Jonah's & Tripp's stories, I really hope a cure is found soon! Bless you for all you do for all the EB families.

    Side note-your comment about turbulence made me laugh. My first & only flight was 15 hours to Germany. I could not sleep UNTIL we hit turbulence & then I was out like a baby. :) It made me feel like I was riding in a car, which I am totally capable of sleeping in. Is it weird that I like turbulence? lol

    P.S. I can't get my google account to load so I will be "anonymous" instead of "Brenda". Or if this shows up 1/2 dozen times, that's why.

  8. Tim,

    Extraordinary speech. Breathtakingly good.

    She is remembered because you speak so beautifully and powerfully of her.

    Take a bow, Tim. That was simply brilliant,

    Fondly, to all of you,


  9. Way to go Tim! God knew what he was doing when he chose you and Ang for Bella's parents. Bella and all EB babies need a voice... a loud, resolute, compassionate, dedicated mouthpiece. He knew how much you would both love her and how you would be empowered by your love for her to help change the world.