Friday, May 7, 2010

It's Official-the Clinical Trial is ON!

If you had told me three months ago that I would be voluntarily signing my son up for a clinical trial, where he would receive a drug that was used for organ transplant rejection and chemotherapy I would have told you to take a flying leap from a very tall building without a parachute. I would have said "not unless hell freezes over". I would have said NEVER. Just like the time Sam wanted to live on 30 thousand-some-odd acres and take care of a bunch of wild cows (which we did) and just like when he decided that we should move to California (which we did). I am going to stop saying NEVER.

Over the years we have worked hard to teach the kids about how to stay in tune with their bodies. We have stressed how important it is to take care of themselves and have educated them to be responsible for their own health care. Doctor's and medicine don't heal people, they support the body to heal itself. We have focused on teaching them to question, research and advocate for themselves. To always trust their own instinct and follow their gut feelings.

While T was still in the hospital dealing with complications of his diagnosis with diabetes I was all ready researching. The last day of our hospital stay we were presented with clinical trials that were available for kids newly diagnosed with type 1. They presented the options to us and a lot of paperwork to read over.

Deciding to do a clinical trial is a process. T is 13 and the ultimate decision is his. This is his body and his choice. We talked it over and he was very interested, so the research and questions began. There were three separate steps to take to be included in the trial. The first was a series of blood tests, the second a test for tuberculosis and the third a Mixed Meal Tolerance Test (MMTT). The MMTT test is to help determine if T's body is still producing any insulin on it's own. T met all the criteria of the trial. The medicine must also be administered within 100 days of diagnosis.

In order for any of this to make sense you will need some background info. Type 1 diabetes is an autoimmune disease. T cells are a part of your immune system that help fight viruses, infections etc. and sometimes these cells go rogue-they don't switch "off" when they should and they attack the cells in the pancreas that produce insulin. When this happens and enough cells that produce insulin are killed, symptoms of diabetes occur. Insulin is the bridge between your cells and the sugar that they need to function. After someone is diagnosed and starts to get insulin they often have what is called a "honeymoon" period. The rogue t-cells haven't destroyed all the insulin producing islet cells yet and the pancreas still produces some insulin. Some people still have up to 40-60% of the islet cells still functioning. This makes diabetes easier to manage.

The drug used in the trial is called thymoglobulin. There will be 66 people in the United States enrolled in this trial before it ends. T will be number 23. This is a double blind placebo trial. The doctors and patients won't know if the patient is getting the medicine or a placebo. 2/3 of the participants get the medicine and 1/3 get a placebo. Thymoglobulin has been used since the 1980's and is a pretty safe medicine, however it does have side effects that make you feel like you have a bad flu. They are temporary and go away when you stop taking it. The medicine is given in the hospital over several days into a special IV called a PIC line. Thymoglobulin is being studied because scientists and doctors believe that it may "reset" those rogue t cells of the immune system and turn them "off" so they stop attacking the pancreas. Hopefully this will extend the "honeymoon" period.

Scientists and doctors recently announced that they were able to create human pancreatic islet cells that produced insulin in mice. This is a huge break through. They need to study thymoglobulin to see if it prevents the immune system from attacking these pancreatic islets cells. It won't do any good to replace the cells if the body destroys them again. The trial is designed to compare people receiving the medicine to people receiving a placebo during their honeymoon phase. The hope is that it will stop the t cells from attacking the remaining insulin producing cells, preserving them and making diabetes easier to manage.

Thymo is a safe, but serious drug. Participants who recieve the drug also get steriods and antibiotics to manage the side effects. Participants who get the placebo get identical appearing medicines that contain no active ingredients. Most participants who get thymo feel like they have the flu for the first few days of getting it and then 10-14 days later they feel like they have the flu again.

T will be admitted to UCSF on Monday at 10 am to start the trial. The decision has been agonizing at times, but I feel more comfortable with it than I ever thought I would.

Deciding to participate in this trial has been a challenging decision. Probably the most difficult one I have ever made. We have talked and researched and questioned endlessly. T has been very involved and has asked educated, poignant questions. I am hesitant to speak for him because he has had his own journey in making his decision. His reasoning for participating is sound and difficult to argue. I have "what if'ed" myself to misery on many occasions. I have always told the kids to trust their instincts and I am proud to say that he is. Deep down, I knew he would participate and that we would support him. Every time the word "NEVER" screams in my head I know it is time to check in with myself. Some of "my stuff" is coming up. When it happens it usually means that I am afraid to follow my gut instinct. Eventually, through processing, soul searching and introspection I come around and then I am all for it.

Now when the fear creeps in and the "what if's" start I always end it with "What if- it works?"


  1. I cannot tell you how impressed I am with your family. I know this is the right thing to do. I know you are not going into this lightly. I know that it's going to be a long week, but in the end you will be able to say, "We gave him the best shot." And that is a gift to be able to say that. ((HUGS)) to you all. Call me anytime, night or day. I'm here to help...believe it or not I can actually help with NON D kids too! (I know! Who woulda thunk?)

  2. This is awesome. It's so exciting to hear about each new step forward in curing diabetes, and I'm so impressed your son has chosen to participate. Big hugs to all of you. - Mo

  3. What an AMAZING opportunity! Thank you for participating in this study that can not only help YOUR son, but possibly ALL of our D-kids down the line!

    I think you made the right decision for you! Don't second guess yourself. You did the research and are as prepared as you can be.

    Good luck! I cannot wait to hear how things go!

  4. WOOT WOOT!!! You and your family are amazing. Thanks to you and your son! You are making a difference for all. What an inspiration you and your family are to all of us.

    Please don't second guess are making a difference.

    Love you and please update us on the outcome!!!

    xoxo...PS Happy Mother's Day!

  5. Yeah..that "Never" should "never" say it!! I've said it all to often, only to have it backfire! :)
    This is simply amazing. I can imagine the fears you must have, but seem to be very much at peace. THANK you...this is really amazing! I can't wait wait to hear how things go!

  6. I agree with Tracy... dont second guess yourself. You have a very brave little man on your hands :) that must make you so proud.

    Please keep us posted on how its going.

  7. I can't wait to hear all about this journey. I can only imagine the decision making process your family has been going through...but can honestly say that I'm grateful for your willingness to forge ahead. The information this study my unveil could be paramount in helping future T1 dx...and, potentially, help the road to a cure.

    I hope you will journal this experience here so that we can share it firsthand.

    You are amazing! Much love and support to your family!!!!

  8. Although the decision has been a hard one for you I am sure your heart led you in the right direction. I think it is amazing that your son wants to participate. What a strong and amazing man you are raising.

    I will be here following your blog and cheering you on.

    Many prayers coming your way!!

  9. What an incredible family you have! I admire your choice to do this trial. Your son/family are incredibly generous to this world.
    You are making the right decision and trusting your instinct is THE right thing to do.
    What if's seem to get in my way too. Sometimes I can be my biggest obstacle.
    Please keep us updated.
    Your family is in our prayers!

  10. Good luck - we will say prayers for you!


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