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Still years away from market approval if proven effective. The trial in Canada is dose ranging trying to find a therapeutic dose level. They plan to start trials in the US next year. If approved it will be covered by insurance so hopefully not incredibly expensive ... but who knows. It' too early to talk about approval until we see some data in DMD boys, drugs worked in animals but the results did not translate in DMD boys by now, we need to wait and see. With trials starting in 2011 I don't think it can hit the market earlier than 3 years or so from now.
Ofelia - You indicate that the "drug works in animals but results did not translate in DMD"...I do know that the drug was tested in menopasal women in Canada with good results - albeit, it's not the DMD boys. So, yes it still has to do through many years of testing. Just happy there is something to look forward to. It sure beats nothing - if your son does not have the exon 51 deletion that seems to be the target of the exon skipping. Char
Also, I do not know how certain Shire is that this can work. Both Biomarin and GSK made similar deals and paid money upfront, this is the way these deals are made, no guarantee that the durgs will work though.
It is interesting that Shire is putting in $45 million in upfront money, and they're only getting US/Canada commercialization rights for that. Link.
The GSK / Prosensa deal was $25 million upfront.
Does that indicate anything about Shire's confidence level? Don't know. Maybe they just want to be first to market, so they put more money in up front.
Ofelia Marin said:Still years away from market approval if proven effective. The trial in Canada is dose ranging trying to find a therapeutic dose level. They plan to start trials in the US next year. If approved it will be covered by insurance so hopefully not incredibly expensive ... but who knows. It' too early to talk about approval until we see some data in DMD boys, drugs worked in animals but the results did not translate in DMD boys by now, we need to wait and see. With trials starting in 2011 I don't think it can hit the market earlier than 3 years or so from now.
Ofelia - You indicate that the "drug works in animals but results did not translate in DMD"...I do know that the drug was tested in menopasal women in Canada with good results - albeit, it's not the DMD boys. So, yes it still has to do through many years of testing. Just happy there is something to look forward to. It sure beats nothing - if your son does not have the exon 51 deletion that seems to be the target of the exon skipping. Char Also, I do not know how certain Shire is that this can work. Both Biomarin and GSK made similar deals and paid money upfront, this is the way these deals are made, no guarantee that the durgs will work though.
In the first site listed, they say how the ACE-031 trial can benefit more than just DMD. They say, "Over the past few years, pharmaceutical companies have been racing to develop ways to mimic myostatin gene mutations in the hope of treating everything from the muscle loss that accompanies muscular dystrophy, cancer, and aging to obesity and other metabolic disorders." This is probably why there are others investing in this. It is able to serve more than one medical purpose.
http://www.technologyreview.com/biomedicine/19589/?a=f
http://www.checkorphan.org/grid/news/treatment/acceleron-pharma-ini...
http://www.medicalnewstoday.com/articles/187960.php
http://www.acceleronpharma.com/content/products/ace-031/duchenne.jsp
Hi,
I always read about concerns that ACE 031 may use up the storage of stem cells to reach a point that there are no stem cells left.
Do you have any idea about this,please!
Yes, there is concern about stem cell depletion ... no one knows exactly what will happen at this time.
Peter Novak said:
Hi,
I always read about concerns that ACE 031 may use up the storage of stem cells to reach a point that there are no stem cells left.
Do you have any idea about this,please!
Ofelia and Peter - I read a huge article on stem cells. I read about a researcher that developed stem cells from other sources because of the current administration at the time (Bush) banned usage of them. What was interesting about the scientist was that he has two kids that are type 1 diabetics and he did indeed figure out a way by developing another type of stem cell to use. It seems to me that scientists are coming up with more and more ways of reproducing stem cells from different areas of the body. I guess, I don't share your concern of stem cell depletion at this time. Further, I thought that the drug that Acceleron is developing isn't coming from stem cells. I thought it is something that is manufactured from chemicals much like the exon skipping ones. Char Burke
Char, stem cell depletion is a concern for any myostatin inhibitor. Only time will tell, they have not been tested/used long term so no one knows what's going to happen...
http://en.wikipedia.org/wiki/Myostatin
"It remains unclear whether long term treatment of muscular dystrophy with myostatin inhibitors is beneficial: the depletion of muscle stem cells could worsen the disease later on."
Char Burke said:
Ofelia and Peter - I read a huge article on stem cells. I read about a researcher that developed stem cells from other sources because of the current administration at the time (Bush) banned usage of them. What was interesting about the scientist was that he has two kids that are type 1 diabetics and he did indeed figure out a way by developing another type of stem cell to use. It seems to me that scientists are coming up with more and more ways of reproducing stem cells from different areas of the body. I guess, I don't share your concern of stem cell depletion at this time. Further, I thought that the drug that Acceleron is developing isn't coming from stem cells. I thought it is something that is manufactured from chemicals much like the exon skipping ones. Char Burke
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