making muscle wasting history

I just read this post [below] on investorvillage. I think the impression of anybody reading Pat's post would be to think the clinicians and AVI had made the error of not assessing efficacy in the trial.

I then remembered that the clinical trial was entitled "Safety and Efficacy Study of Antisense Oligonucleotides in [DMD]".

http://clinicaltrials.gov/ct2/results?term=Duchenne+exon

Further more, the key paragraph at the top of the page says ...

"The specific aim of this phase I/II study is to assess efficacy (dystrophin production) and safety of intramuscular administered morpholino oligomer directed against exon 51"

After reading this post several times I think any reasonable person could not help but reach the conclusion that this post gives a very bad impression of the clinicians and AVI - which simply isn't true.

I get a strong impression after reading this post, that this post was unhelpful, and that the left hand doesn't know what the right hand is doing when it comes to lobbying for exan skipping as a platform drug.

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Comment by Pat Furlong on exon skipping platform

http://www.investorvillage.com/smbd.asp?mb=412&mn=4228&pt=msg&mid=7717187

Msg 4228 of 4228 at 8/7/2009 2:49:47 PM
by themicrokid

PPMD comment by Pat Furlong

Comment by Pat Furlong on July 29, 2009 at 5:54pm

Hi Paul, We are working with Ed Connor from Children's National. Ed's career was focused around regulatory issues and vaccines, so he is quite well versed on seeking approval for a backbone chemistry and (in the case of flu) annual modifications based on the anticipated virus (threat). Timing is an important issue. As you know, to date, there is no proof of efficacy. While Prosensa and AVI have completed (prosensa) or are current invovled in (avi) systemic trials on exon 51, none of the trials will provide efficacy data. So, at the moment, going to fda to lobby about expediting additional chemistries for additional exons, is not useful. Once we have proof of concept on one more perhaps two exons, I think we will be able to make the case to streamline the process. The meeting in September is to initiate the conversation and the rationale, hopefully open minds and opinions. In rare diseases, these conversations will someday become common as these are often progressive conditions with few patients. In addition, as approaches such as antisense and stop codon suppression (ataluren) have the potential to treat, streamlining the process, saving time, preserving function and saving lives will mean regulatory agencies have to change. Duchenne is in the lead (thankfully) and these conversations will be frustrating at times I am certain. But, at the end of the day, it is a new day in Duchenne, we have amazing and potentially life-changing therapies on the horizon. As things move forward, we will work with Dr. Conners and others to organize, develop our agenda/messages and lobby to accelerate drug development/clinical trials and of course ACCESS to therapies for all.