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?"