Data From A Phase-I Clinical Trial of Polyclonal Tregs

18 Month Data from a Phase-I Trial of Polyclonal Tregs

I previously blogged on this research here:
http://cureresearch4type1diabetes.blogspot.com/search/label/Polyclonal%20Tregs
so please read that blog posting for details of what is being tested, and who it is being tested on.

A quick summary is this: Dr. Trzonkowski and co-researchers at the Medical University of Gdańsk  remove one specific type of T regulator cell (called "CD3(+)CD4(+)CD25(high)CD127(-)") from a person with type-1 diabetes.  They use these cells to grow a lot more of them, and then put them back in the body.  Since regulatory T cells naturally regulate the body's immune system, the hope is that they will prevent the autoimmune attack which causes type-1 diabetes. 

The previous data was from 10 children, who were treated within 2 months of diagnosis.  But this new data covers 12 children (aged 8-16), also treated within 2 months, and were followed for 18 months.  They were compared to a placebo (untreated) group.

The big news is simple: two of the treated children did not need to inject any insulin at all for 11 months.  The treated patients in general generated statistically significantly more C-peptide than the untreated group, which means they were generating more of their own insulin.  And obviously, two of the treated kids were generating a lot more insulin.  It is important to remember, that some honeymooners do not require insulin for some part of their honeymoon time.  However, I think that 2 out of 10 is far more than would be seen randomly, and I think that 11 months is longer than one would expect naturally.

A pessimist might say "It only works for honeymooners, it worked for less than 20% of the people, and it only worked for 11 months."  But I am an optimist.  I say "If it works for honeymooners now, maybe it will work for established type-1 diabetics in the future, or might work when combined with a beta cell growth factor.  Previously we had a prevention for no one; if this work pans out, we will have something that works for some people, and if we start out with 11 months, maybe we can stretch it longer with more research, or repeated doses.

So I think these results are very interesting, and well worth following.  I'm very happy that at least one other group (Drs. Gitelman, Bluestone, and Herold) also have a 14 person, phase-I trial going into this treatment.  However, that group is not expected to finish their trial until 2016. Hopefully Dr. Trzonkowski and his co-researchers will have a phase-II trial well underway by then; maybe even finished (if their phase-II trial gets funded quickly).   

Abstract: http://app.core-apps.com/tristar_ada13/abstract/9b942b88cb23351f1c62fdf808057354

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. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog. 
Clinical Trials Blog: http://cureresearch4type1diabetes.blogspot.com