History of Exsulin and AAT

Based on my last couple of posts, I got the two following questions:

How is the phase-II Exsulin study that is currently underway, different from the phase-II Exsulin study that was reported on in 2009?

The first trial gave one day's dose in one injection.  In the current trial, one day's dose is spread over three injections over the course of the day.  On one hand, the researchers think this will lead to better results, because they think that Exsulin does not stay in the system very long, so putting it in repeatedly over the day should lead to better results.  The drug will be more consistently in the system the whole day through.  On the other hand, they also think this will lead to fewer "injection site side effects".  In the earlier trial, some patients complained about pain, redness, itching, and other problems right around the injection site.  Since the second trial will only be injecting 1/3 of the dose at once, they are hoping there will be far fewer of these side effects.  They are also changing the exact formulation to try to minimize this discomfort.

Also, the first study lists the doses as 600 and 300, while the second lists them as 200 and 100.  I'm assuming that is per injection, and the daily dose was the same for each study.  But it is possible that is not true and the second trial is using a much lower dose.  If so, this would be another big difference.

The purpose of most phase-II studies is to find the best dose, the best format for that dose, and to test the treatment on a larger population than in phase-I.  So this study is testing improvements to the dosing and formulation; just what you would expect in phase-II studies.

What is AAT used for today?  Is that disease like type-1 diabetes?

AAT (Alpha 1-antitrypsin) which is also sometimes called A1AT, is approved for treating Alpha 1-antitrypsin deficiency.  Makes sense: your body doesn't produce enough AAT, so AAT is approved as a treatment.  This disease is not an autoimmune disease, and is nothing like type-1 diabetes.  It is genetic, and comes in different severities depending on if you have one or two of the bad genes .  It is estimated to affects 1 out every 2,500 people in the US, although only about 10% of the people affected are actually diagnosed.  Here is a note on the most common symptoms from the Alpha-1 Association:

The most common indicators of Alpha-1 include shortness of breath, a chronic cough, and abnormal liver test results. If you have any of these symptoms there is a simple blood test that can detect alpha-1 antitrypsin levels. This test is also recommended if you have relatives, especially siblings, who have been diagnosed with alpha-1, or if there is a family history of early emphysema, with or without smoking.

You can read about it here:
http://en.wikipedia.org/wiki/Alpha_1-antitrypsin_deficiency

And here are some support groups:
http://www.alpha1.org
http://www.alpha-1foundation.org


Joshua Levy
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions.
Blog: http://cureresearch4type1diabetes.blogspot.com
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