Dr. Fallon's work is all over the web today:

http://www.futurity.org/top-stories/drug-may-block-muscular-dystrop...

http://www.physorg.com/news/2010-12-human-protein-muscle-function-m...

 

This looks like good news, but we know that the MDX mouse model does not translate to humans - plus Stanford's recent stem cell work has me wondering if this would even be a good idea.  

PPMD gave Dr. Fallon an End Duchenne grant for this research - any thoughts?

 

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I think that they should test it also with the Blau mice and maybe also to combine it with a tellomerase inhibitor.
When do they plan to file for IND? The article says that the next step is testing in humans, will this start next year, 2 years from now, ever?

I lost interest in MDX results unfortunately. I remember the "good" results reported by Biomarin's utrophin upregulator.

Steve,

 

I have no medical background but it would seem to me since Utrophin appears to have some capabilities similar to Utrophin it would help protect the muscle cells from injury.   My understanding is this would reduce the need for the body to rebuild the cells and therefore preserve the Chromosome tips (telomer length) for subsequent muscle generation.

 

I believe Dr. Brown, PTC, and Genzyme (post BioMarin) are all working on Utrophin up-regulation.   Hopefully we'll see a combination of Utrophin, Exon Skipping, Ataluren, PGD2/HQL-79 *, mini-dystrophin, and other options in the 'near' future. 

 

My entire, extended family remains very motivated to help the cause and the researchers.   I hope all the parents and families keep donating, fund raising, and slugging away at DMD.   I think we are collectively getting our hands near the throat of DMD and I just can't wait to see us start squeezing - until someone researcher discovers the 'silver bullet' and takes out DMD.

 

Squeeze!

 

-Bob

Dad of Kyle, age 6.

* HQL-79  http://mdn.mainichi.jp/mdnnews/news/20101211p2a00m0na013000c.html

Hi Bob,

Thanks for your input - I couldn't agree with you more in terms of not letting up, raising money, pushing both research and treatments for all of our boys. I love the metaphor - and I'll gladly join in the squeezing when the time comes!

At the same time, it feels like there has been a substantial shift in the past few weeks with some of the research coming out, and I'm trying to get my head around all of the implications of that.

I agree with Ofelia that MDX seems like such a poor predictor that I wonder if it's even worth pursuing or getting excited about that research at all...

MDX is quite an inexpensive model and since we need to start somewhere we definitely need to start with it...

However, in the last 3 years since my son was diagnosed I saw/read tens of papers claiming that this or that drug/treatment "works" in MDX that I am not getting excited anymore. Some do not replicate in humans, some are so far from clinical trials in humas that it doesn't even matter, and hopefully some start trials soon. At this point, when I read about the MDX results my first question is how far they are from clinical trials in humans...we all know that only after YEARS of trials in humans something will get approved. Unfortunately, looking at the slow progress of the existing trials, I focus on things that will enter trials in the next couple of years.

 

Biglycan might be close, I read updates about it for the past few years, so I am hoping that they are close to testing it in humans.

Of course, the more things we have in the pipeline, they higher the probability to get something approved.


Steve Dreher said:

Hi Bob,

Thanks for your input - I couldn't agree with you more in terms of not letting up, raising money, pushing both research and treatments for all of our boys. I love the metaphor - and I'll gladly join in the squeezing when the time comes!

At the same time, it feels like there has been a substantial shift in the past few weeks with some of the research coming out, and I'm trying to get my head around all of the implications of that.

I agree with Ofelia that MDX seems like such a poor predictor that I wonder if it's even worth pursuing or getting excited about that research at all...

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