Sharon...I noticed at the top of the graph you posted ace-031 is there any news on what Shire is doing with this drug or when and if trials will ever resume?

Views: 659

Reply to This

Replies to This Discussion

Good question, I would asked the same..............!?

the latest news on their site (

A Single Ascending-Dose Study of Muscle Regulator ACE-031 in Healthy Volunteers. Muscle & Nerve. November 21 2012

Link to abstract:

Hi Chris--the news is that they are working on it... (so, no, not really anything new); however, the company remains committed to Duchenne and are still sending staff to Duchenne-related meetings so I think that's a good sign. 

Hi Sharon,any updates on SMT C 1100?it was supposed to start phase 2 in 2012,but.................!!!!

Thank you

Sharon Hesterlee said:

Hi Chris--the news is that they are working on it... (so, no, not really anything new); however, the company remains committed to Duchenne and are still sending staff to Duchenne-related meetings so I think that's a good sign. 

Hi Moein--I spoke with Summit recently.  I don't have the exact time line but they definitely are planning to start the phase II this year (they are just finalizing the protocol and pulling together resources now).  Summit will be reviewed at the next TREAT-NMD Advisory Committee on Therapeutics in April --this is the mechanism that PPMD uses to make funding decisions about preclinical and clinical therapeutic projects.

Thanks Sharon for giving us updates on ACE-031 and a host of other issues along the way.Kindly assist on this one too.Have you ever come across info on this research on Decorin,an anti-fibrotic agent going on at University of Pittsburg?I have tried getting in touch with the team carrying out the research but i have not had any luck.Thank you.

Using decorin, an anti-fibrosis agent, to improve muscle recovery after injury

De-activating TGF-beta (fibrosis)
Fortunately, researchers at the University of Pittsburgh have been working hard to develop ways to control fibrosis. The Pittsburgh scientists realised that fibrosis is stimulated by the overproduction of a chemical called transforming growth factor-beta (TGF-beta). TGF-beta is produced by tissues in response to injury and disease and is the major cause of fibrosis in humans and other animals.
The Pittsburgh researchers also knew that decorin - a proteoglycan (a protein with sugar attached) naturally found in the human body - has the ability to de-activate TGF-beta. Decorin has been shown to inhibit fibrosis in the kidney, liver, and lung, so why - the Pennsylvania scientists reasoned - would it not also stop fibres from taking over muscle tissue?
In recent research, the Pittsburgh medical team has in fact been able to show that decorin is a powerful anti-fibrotic agent in muscle tissue - and thus that decorin administration has the potential to be an outstandingly effective treatment for muscle injury (Kazumasa, F. et al., 'The Use of An Antifibrosis Agent to Improve Muscle Recovery after Laceration,' The American Journal of Sports Medicine, Vol. 29 (4), pp. 394-402, 2001).
vito is offline   Reply With Quote


Hi Wainaina:

Decorin is a small proteoglycan (like biglycan, which is in testing now as an indirect utrophin upregulator) that investigators have been looking at for a long time due to its potential antifibrotic properties.  It's never really gained a lot of momentum although you can find publications on decorin and the mdx mouse as late as 2012.  It's possible that it may be a challenging molecule to use as a therapeutic because proteoglycans are little proteins covered with short sugar chains and they can be very difficult to recreate in the lab in large and precise quantities.  They were able to get around this poduction problem with biglycan because it turns out that a short version of the protein without most of the sugar chains is just as effective as the original version.  I don't know if this would be true for decorin.  It is, however, officially on the my internal "target" list--just not much activity towards making it a realistic therapy.  Hope this helps!

Hi Sharon Thank you for informing me on this one.I have come across it on several occasions and didn't know what to make of it.

Just curious:If we were to find a way of stopping fibrosis from occurring in the muscles of our sons as the disease progresses,would it likely mean that a cure in the future would make it possible for the muscles to replenish themselves(since a muscle tissue with fibrosis is unlikely to rejuvenate)?If i am not wrong i think i also came across an article on this site on a scientist trying to find a a way to reverse fibrosis?I am convinced soon a cure will be available.I am now focusing on what it will mean for the boys whose muscles will be too  compromised by the time the cure arrives.But again may be am being a bit too optimistic.Any way thanks Sharon and gd day.  

Hi Wainaina:

So, the drug that Halo Therapeutics is working on (halofuginone) is a strong anti-fibrotic and that's supposed to be in clinical testing this year for Duchenne.  You are right that the idea is to slow or block fibrosis so that muscles can better replenish themselves.  I would envision combining an antifibrotic medication with something that directly strengthens the muscles like a utrophin upregulator or myostatin inhibitor or exon-skipping agent.


Charley's Fund has put a lot of resources into the Halo drug and PPMD has contributed $100,000 to it as well (along with many other groups).




Hi Sharon

Great to hear PPMD is covering all bases.I didn't know what Halo are working on is an anti-fibrosis agent.We are with u guys all the way,and we know one day we will have the cure that we so desperately seek.Thank you and have a great week.

Reply to Discussion


Need help using this community site? Visit Ning's Help Page.



© 2022   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service