The most important Type 1 trial you probably never heard of
Suppose I told you that there was a way to predict who will get type 1 diabetes? Suppose I told you that if you were predicted to have type 1 diabetes, you might be able to stop the disease or at least put it off for 10 years or more? Suppose I told you that this is possible today? And that in order to take advantage of this possibility you could enroll in a simple study that would give you the prediction information free of charge and provide potential treatment also free of charge if you are predicted to have the necessary gene structure? Would you enroll? I would and had it been offered to me before I was diagnosed I believe my parents would have enrolled me. This is the story of the Type 1 Diabetes TrialNet study currently underway and recruiting new members across the US and in other part so the world. You can visit their web site at:
The genesis of the study is many fold but one benchmark is a Finnish study from 2001 which found a correlation between the incidence of the presence of a gene derivation and being diagnosed with type 1 diabetes ("The First Signs of β-Cell Autoimmunity Appear in Infancy in Genetically Susceptible Children from the General Population: The Finnish Type 1 Diabetes Prediction and Prevention Study," 2001). The study found that children of first generation type 1 diabetics who tested positive for this gene derivation were more likely to develop type 1 diabetes by age 15 than the general population ("The First Signs of β-Cell Autoimmunity Appear in Infancy in Genetically Susceptible Children from the General Population: The Finnish Type 1 Diabetes Prediction and Prevention Study," 2001). As of today there are 10 gene derivations thought to have similar high probabilities of predicting type 1 diabetes (it used to be 5). These derivations are thought to predict the occurrence of type 1 diabetes.
Is this is a cure? No. No one knows at this time if any of the interventions work and if they do who they work for. What is known is that if you are at high risk for developing type 1 diabetes, there may be way to delay its onset (buy time) or better yet stop the disease from developing. Yes for those of us who have type 1 there is no cure, insulin is still our best means of control. But we are moving closer and closer, ever so slowly to perhaps understanding the triggers for the disease process and ultimately stopping its development.
So who qualifies? TrialNet offers the following two descriptive sentences of who qualifies for the trial:
“Anyone between the ages of 1 and 45 years with a sibling, child or parent with type 1 diabetes.
Anyone between the ages of 1 and 20 with a sibling, child, parent, cousin, uncle, aunt, niece, nephew, grandparent or half-sibling with type 1 diabetes.” ("TrialNet pathway to prevention," 2014).
How does it work? Any qualified person who enrolls is sent a blood test kit. You take it to a lab of your choosing. Then you wait for the results. If you age are 20 or more and you are tested negative for the derivation you get a letter and you go on. Only about 5% of people will test positive (A pathway to prevention participant handout, 2014).
If you are less than age 20 you can be enrolled in annual or bi annual testing to determine the presence of antibodies until age 20 (A pathway to prevention participant handout, 2014).
If you test positive depending on the results of the test and your age you may be placed in one of three trails. These trails are testing methods to delay or stop the development of type 1 diabetes. But a caution here, these are trails you will never know if you got the real drug or a placebo. Obviously to know if a method or drug is effective it must be double blind tested. Yes these drugs are thought to help. No it is not known if they do. That is what the trial is for.
My take is that this is our best way forward at the moment. It is not an all for one idea and it is not what we want yet, a cure. But no one can deny this is one heck of a leap forward over the primitive testing that was available in the 1960’s. Some of us remember that when our parents were diagnosed we entered a regiment of annual glucose tolerance tests. They were truly awful ordeal of drinking a concoction of nastiness so sweet it nearly chocked you, followed by blood work at 1, 2, 3 and 4 hours to see if your blood sugar rose over a certain level. It was what I termed my annual misery. Incidentally my glucose tolerance test was negative in the spring of 1974 and 6 weeks later I was in a hospital room with a blood sugar of over 500. Yeah so much for that test. Will TrialNet be thought of as useless in 40 years? Maybe. No one knows for sure. But I like the odds much better than the odds of glucose tolerance test predicting anything but an upset stomach.
Is it for everyone? No of course not. A child or adult may be told they have the derivation but never develop type 1 even without treatment. I mean let’s face it no one knows for certain these are the genes or that this is the treatment. Or it might be? Like I said no one knows for certain. But if you were told you had a chance and this was a way forward for your child or grand children would you take it?
I have spoken to one of my sons and he and his wife have chosen to apply and enroll their son. I have not spoken to my other son (I am waiting to do it in person) but I hope he and his wife chose the same for he and his children. If they don’t that is fine. I understand the personal nature of the choice.
One thing I do know, and firmly believe, research is our collective way forward. Those who participate will be moving us forward, and goodness knows we need to move forward, even if forward is a dead end alley.
The First Signs of β-Cell Autoimmunity Appear in Infancy in Genetically Susceptible Children from the General Population: The Finnish Type 1 Diabetes Prediction and Prevention Study. (2001). The Journal of Clinical Endocrinology & Metabolism, 86(10), 4782-4788. doi: doi:10.1210/jcem.86.10.7907
A pathway to prevention participant handout. (2014). Washington DC: Retrieved from www.DiabetesTrialNet.org.
TrialNet pathway to prevention. (2014). Natural History Study of the development of type 1 diabetes Retrieved June 12, 2014, from http://www.pathway2prevention.org/