Peakman's Phase-I Results

Peakman's Phase-I Results

Years ago, when I first started tracking research to cure type-1 diabetes, Peakman's clinical trial was one of the first ones that I added to my list and started following. And it was very frustrating, because nothing happened. Years went by and nothing happened. But now something has happened. The phase-I clinical trial ended, and the results have been published.

This research is based on the idea of using a modified protein to teach the body's immune system to not attack it's own beta cells. The clinical trial gave some people no protein, some a little protein, and some a lot. The patients were all people with established type-1 diabetes.

You can see the abstract here. The full study is pay-for-view:
http://www3.interscience.wiley.com/journal/121536033/abstract
And JDRF (which helped to fund the work) has a good description of it here:
http://www.jdrf.org/index.cfm?fuseaction=home.viewPage&page_id=DD5ADF6D-110A-9BB5-F8684C43208F87D2
Here is a great quote from JDRF's description:

Peptide immunotherapy is a type of treatment that uses small proteins to "reset" the immune system to a healthy state, much like the common allergy shot. In the case of proinsulin peptide therapy, the goal is to train the immune system to tolerate the insulin-producing beta cells that are the target of the immune response that causes type 1 diabetes.

From a safety point of view, this study was completely successful: no safety issues were found. So they can move on to phase-II trials. They hope to start those in less than 6 months. (That will be interesting in itself. My experience is that the time between ending a phase-I and starting a phase-II is always more than 9 months, so it will be interesting to see if these guys can do it quicker.)

Safety is the official goal of every phase-I clinical trial. However, most of them also try to generate some data on effectiveness of the treatment. Since they are testing minimal doses (for safety), expectations are set low, but it is still common to see some improvement. But I don't see that for this trial. I don't see any record that they looked for or found lower BG rates, lower A1Cs or less insulin usage. They did see some small changes for a 6 month period in the patient's immune system, but it is not clear what impact those changes would have.

So my overall summary for the research right now, is that their results from phase-I definitely get them to phase-II testing, but there is no data to evaluate if it is working or not. We'll need to wait for the phase-II trial to finish for that.

Joshua Levy