Wednesday, February 9, 2011

February 9, 2011: Back in the saddle...


Well, it was Ali's first day of pre-school, and daddy's first day with my own new rhythm.  Ali practically RAN all the way to school.  It was so cute.  She is so unafraid of new social situations, it is just awesome to watch.  I am so proud of that little girl.  That is such a gift to have.

With Ali's new school hours, I started new hours in my office as well.  I can walk to a neighborhood gym from her school, so I have gone back to my gym-rat origins and given up the not-working living room workouts.  I wasn't enjoying it and not effective at all in maintaining a workout regimen.  Now, I start in the office a little later, but only after taking care of my self first.  I work late at night every night in my office anyway, so I don't feel bad about starting the day at 10; come 10 pm, I am almost always still on my computer.

I also quit an hour earlier because of preschool hours.  Today, Ali and I walked to the gym to fill out come paperwork, then went to the bank to deposit some checks.  I am excited to show her the bank as she has some seed money from the tooth fairy and grandma.  I told her that she could spend it, but if she saved it, we (or a bank) would actually pay her money in return for not spending it.  She thought that was a pretty great idea, so the incentive for saving hopefully took its first roots in her brain.

Thanks for all the questions over the past two nights.  Here are a few more quick answers to questions...

The age range for the study is I believe under 18.  They still only have had 15 patients in the study, which by research numbers is nothing.  It is too soon to generalize any specifics about what the sweet spot might be for best age.  I would appear anecdotally that 4-6 would be the optimal age range for this population, but again, that is based on far too few to hold any real weight.

Some insurance companies (like ours - Anthem BC/BS) will pay for the clinical trial, some won't.  It's up to the individual insurance provider.  The U has someone dedicated to working with insurance companies to help and assist with coverage.

Transplant doesn't require a sibling donor.  An unrelated donor's cod blood can work as well.

Yes, unfortunately, there is a point of no return for EB kids.  Transplant can't undo what's already been done in regards to webbing of hands.

Last night, there were a few rather negative questions that I'd like to answer.  It is clear the reader has a unfavorable opinion of the study, which is fine, but I think there is a piece missing.

This is research.

So far, the results - meaning that stem cells are leaving the blood supply and turning themselves into skin cells for the first time in medical history - are worthy enough for publication in the New England Journal.  The spectrum of success is as broad as it could possibly be... from amazing success with several patients now, to tragic failure with several patients, to side effects as bad as EB, to graft failure.  This treatment in its current form is a part of a process.  It was never meant to be a cure or the end.  It has always been merely a stepping stone.

Yes, the doctors are SURE this is the best route to follow.  No other treatment to date has produced the systematic improvement on a patient like we have witnessed with Keric or Payton or Charlie.  This treats the whole body, inside and out, which is necessary for RDEB And JEB kids.  Again, this isn't the finish line at all.  This is their least elegant solution, but it is where they need to begin to learn enough to progress.  The docs would have never understood this mystery of Collagen VII being expressed elsewhere in the body unless they took this head on.  On a personal note, Ang and I KNEW what the risks were; we also remember that 4 EB children died in the U.S. last December without going through BMT.  The potential to die with or without trying BMT is a daily issue.

Please make no mistake: the doctors are EXTREMELY up front about how intense and dangerous bone marrow transplant is.  They encourage patients and their families to avoid it if possible.  That is why they are hoping for additional research funds to move past bone marrow transplant as we know it as the sole systemic treatment option.  There is no way to predict outcomes on something that has never been done before.  Like I said, no one has looked at EB as a systemic disease.  Up till now, all research and treatment has been localized.  This is how it was with leukemia for a long time.  Of course, none of the kids survived when Leukemia was treated this way.  Until they asked a different question about how to treat childhood leukemia, there was no success, and no hope.  Now, not only is there great hope for kids with leukemia, but every treatment option available for adult cancer today (which claims 1 in 3 adults) resulted from that ONE breakthrough when the doctors began treating leukemia as a systemic disease, not a localized one.

Alright, I hope to carve out a few more tomorrow.  Please keep them coming!

God night.


  1. Dear Tim

    i am SO SORRY if my questions sounded negative, that was in no way intendended!!!

    I am here to support, as i always have, and i truly apologize if you were offended by my questions.

    I believe though that people watching the documentary for the first time might ask themselves those very same questions.

    Doctors have to be ready to find people DOUBTING about this -or any other, for that matter!- line of research, and be ready to give sincere answers to solve those doubts.

    i know i have expressed my doubts in the past, i may have been rude insisting on it.

    Sorry again, NO OFFENSE INTENDED!!!!

    Love and hugs

  2. Tim,

    Thanks for your post! It is interesting and informative. Let's hope the holistic approach gains good results soon!

    Ali is a wonderful girl - wish her many good friends in the pre-school and nice time there!


  3. Ali sounds just like Noah. New situations and new people he just embraces...nothing like his mommy. I am so glad he has that quality.

    One more question...what happens after the bmt? What kind of follow up is required?

    Sending my love to all and sweet kisses for Ali.

    Denise WI

  4. Tim,
    I have read your blog for a while now, but never commented before (I know, I know.. ) Because I have always struggled with what to say to you. I want to comment, but then end up thinking too much about it and end up to concerned with what I would say. So I hope this makes sence, because I am trying not to think too much about it :).

    You and your family are such an amazing inspiration and I find myself drawing on that inspiration in many areas of my life. You have also taught me about appreciation. I am blessed to be mommy to a beautiful 15 month old girl with healthy skin. I thank God for all the small things about her with your help. Many things that I admittedly took for granted before I have begun to sing His praises for.
    Mercy Me's song 'Bring the Rain' has become one of my favorites and it makes me think of and pray for your family each time I hear it. The lyrics describe the kind of faith all Christians desire to have but hope never to have to exercise. Your family epitomizes those lyrics. Seeing pictures of each of your (Ang, Ali, and you) faces with a smile on it reminds me that He truly is much greater than our pain. He is the strong deliverer and He IS mighty to save.

    I will close by saying this; your tenacity and drive for your cause is wonderful. I know that Bella is so proud of her Daddy.

    Christy TX

  5. Tim,

    I've tried posting this question three times now, so I'm going to make it short:

    Is it common to have kidney issues after bone marrow transplant and/or chemo? Is this unique to the EB kids?

    Can't wait to hear about Ali's school adventures!

    Karen Steiner
    Denver, CO

  6. Hi Tim-

    Thanks so much for all the great answers! And I have no doubt that Ali had a wonderful day at school - can't wait to hear about it!

    Continuing to pray!!

    Love from TX,
    Laura (for Team A)

  7. I am fascinated with how far this research team has come and the strides they are making for children with EB. I was wondering if you were able to notice any improvements in Bella's skin soon after the transplant? Also, how long do the doctors/researchers anticipate it will take to make drastic improvements in a child's skin following a transplant?

  8. Hi Tim,
    Witht eh iPhone launch I have had so little time to get here - I have to catch up. My supportis with you all and this study - looks like teh best hope out there!

    My question is I saw and have inferred from your blog and a few of the other kids blogs that as the doc's learn each day they adjust the trial. How quickly do they adjust the protocol?

    Another question is for those kids that it does not seem to have worked for - they seem to be worse then ever - is there anything the doc's can do for kids like EJ, Sam and McKenzie?

    Sending you all my best - Tina