Are their any open clinical trials for deletion 22-41?

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About dystrophin and the immune response...

 

So, Jerry Mendell's group identified a T cell response to the minidystrophin gene using a very sensitive assay called an ELISpot--this assay allows you to tell exactly what "epitope" (part of a protein) is being recognized by the T cells.  They were also able to show that some of the boys had a pre-existing T cell response to dystrophin, probably produced by so-called "revertant fibers."   My understanding is that AVI, Prosensa and PTC Therapeutics didn't look for T cell responses with the ELISpot, but rather just looked for antibodies to dystrophin and didn't see any.  So they probably don't know if there are pre-existing T cell responses that could be amplified by newly expressed dystrophin due to skipping etc.

 

BUT, all of that being said....what we don't really know is what the T cell response means.  Based on my discussions with Carrie Miceli, an immunologist at UCLA, it might actually be relatively benign.  It's a concern right now, but not a show-stopper neccessarily, which is probably why the other companies didn't do the more sensitive ELISpot assay.

 

As for Mendell's microdystrophin project, the group is definitely gearing up to do another phase I, this time they wil probably exclude boys who have pre-existing T cell responses to dystrophin just to be on the safe side.  They'll start with another intramuscular injection using higher doses of vector than they used in the first study.  If they can demonstrate that they are able to get substantial expression of dystrophin without signs of an immune response (whether antibodies or T cells or whatever) then they will move on to more systemic delivery for phase II.

 

Hope this helps!

 

Sharon

To save time and rescue our sons,I suggest to work on Utrophin Gene Delivery to avoid the immune response.If successful and our sons are rescued,then researchers will study and work on Dystrophin Replacement without hurry over a cup of coffee.Do you agree?



Moein said:

To save time and rescue our sons,I suggest to work on Utrophin Gene Delivery to avoid the immune response.If successful and our sons are rescued,then researchers will study and work on Dystrophin Replacement without hurry over a cup of coffee.Do you agree?

I do not have hope that Mendell's project will get here before my son becomes non-ambulatory. I do however believe that the fact that dystrophin was produced by all boys in the higher dose groups of AVI and GSK trials is promissing. I am not concerned about the T-cell assay in this case. Ataluren's data analysis should also be helpful in this case, if some boys do have pre-existing T cell response, they were part of the Ataluren trials as well.

Moein said:

To save time and rescue our sons,I suggest to work on Utrophin Gene Delivery to avoid the immune response.If successful and our sons are rescued,then researchers will study and work on Dystrophin Replacement without hurry over a cup of coffee.Do you agree?

I found one of the widgets for predicting which exons need to be skipped to put the dystrophin back in to frame:

 

http://www.dmdregistry.org/reports/predictor/

I see that the status for ACE 031 on www.clinicaltrials.gov was changed from(SUSPENDED) to (TERMINATED)!


Does this mean that the whole trial was cancelled?


Moein, Pat made a response to this a while back. Its somewhere on the site, maybe her blog?

Moein said:

I see that the status for ACE 031 on www.clinicaltrials.gov was changed from(SUSPENDED) to (TERMINATED)!


Does this mean that the whole trial was cancelled?

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