ACE-031 and UTROPHIN Levels
I was going through the acceleron website for details on ACE-031. As per the information available on their website, ACE-031 is shown to help grow muscles which have HIGHER UTROPHIN Levels.
So it mean that ACE-031 can help in growing STRONG MUSCLES WHICH HAVE HIGHER UTROPHIN LEVELS ALSO.
To quote from the information available at http://www.acceleronpharma.com/content/products/ace-031/duchenne.jsp
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Importantly, the composition of muscle produced by ACE-031 treatment is distinct from that of dystrophin-deficient muscle. Utrophin is a protein that is related and serves a function similar to normal dystrophin, but unlike dystrophin, it is only produced during fetal development. Increasing adult production of utrophin could potentially compensate for loss of strength and function due to defective dystrophin. In adult mdx mice, ACE-031 treatment increases the expression of utrophin, which may contribute to the significantly increased muscle strength observed in these animals.
ACE-031 Treatment Increases Utrophin Expression in MDX Mice".
As such this is very encouraging news but I was not able to understand how is that achieved. I was under the impression that ACE-031 is protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB).How it is able to increase UTROPHIN levels also? Has anyone got any clarifications here.
Replies to This Discussion
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Permalink Reply by Ofelia Marin on
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Very good question Raktim. I would also like to know how much upregulation was obtained. Have you tried emailing Dr. Diana Escolar? She might have some answers. She is also in FB.
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Permalink Reply by RAKTIM SINGH on
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I will try to contact Dr. Diana. Will update if I get some info here.
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Permalink Reply by Lori Ware on
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I know that Pat Furlong has some infor on ACE-031....at conference I found out that it: 1. increases muscle 2. decreases fibrosis 3. decreases affects of steroids and 4. increases utrophin. Phase 2 trial begins in Canada by the end of this year.
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Thanks Lori for this update.
Wow!!! That's amazing. So as such it provides all the things we are looking for.Also, ACE-031 will help bigger MD population ( apart from helping all DMD kids, my guess is that it will help BMD kids also).
I think PH2 has already started in Canada and enrollment will be completed by AUG END.
Raktim
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Yep, it does seem amazing...I sure hope it lives up to it. THe other good thing is that they don't need $...they have a HUGE population for this drug...atheletes, older folks losing muscle, MD, probably even spinal cord injury to help keep muscles in shape...who knows!!! Certainly a good one to be excited about.
RAKTIM SINGH said:Thanks Lori for this update.
Wow!!! That's amazing. So as such it provides all the things we are looking for.Also, ACE-031 will help bigger MD population ( apart from helping all DMD kids, my guess is that it will help BMD kids also).
I think PH2 has already started in Canada and enrollment will be completed by AUG END.
Raktim
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Permalink Reply by Joshua's mom on
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We start in August! Joshua is going to be a part of it!
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Keep us posted!!!!! Good luck!
Joshua's mom said:We start in August! Joshua is going to be a part of it!
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Of course I'll keep you posted! It's going to be an experience I wish all of our boys & girls could be part of.
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Permalink Reply by tom evans on
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Are they planning on any non-ambulatory studies,if so,how can we get on their list?
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This trial is asking for ambulant boys... not even girls are allowed. :( I'll give you the link to the sight and contacts etc.
http://clinicaltrials.gov/ct2/show/NCT01099761?term=DMD+Canada&...
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