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What in the world?

Just a few days ago I received a comment from Nathan on my post called Putting all our hope in one basket.  Nathan was so kind to inform me/us of a trial of a drug, Miglustat, being studied outside of the US.  Here are a few links he gave me:

Actelion – Miglustat (Actelion is a Swiss drug company developing/testing Miglustat)

Miglustat Shows Promise in Treatment for Cystic Fibrosis (article from bio-itworld.com)

Pretty interesting!  It is easy to live in this little bubble of a life I have, sometimes it we forget there may be other things going on.  For the most part I only know of cystic fibrosis studies, in the US, and that are sponsored by the CFF(Cystic Fibrosis Foundation). I have forgotten there is a whole-nother world out there! Thanks so much for this info (and hope) Nathan.  I wonder what in the world are other countries studying.

Nathan has me wondering, what other treatments are being studied in the rest of the world, outside the US, outside the realm of the CFF.  Surely there must be some drug companies studying CF aside from those receiving money from the CFF. I wonder what is going on outside this little bubble I live in…

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This entry was written by Salty and posted on August 24, 2009 and filed under Cystic Fibrosis, treatments.
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One Comment

  1. Well, not so great news on Miglustat. I’ve been checking for updates on the Miglustat clinical trial, and noticed this morning that the Bio-IT World story has changed. When the story was first posted July 22nd, the story said that the study had concluded and that Actelion was “currently reviewing the results”. Now the story says, “After reviewing the results of the Phase IIa study, Actelion decided not to continue clinical development for the time being. But in light of the French team’s results (the study of Miglustat in CF respiratory cells), Actelion has decided to conduct further preclinical evaluation of the drug in CF before making any final decisions”.

    This is both disappointing and frustrating. There is no mention in the Bio-IT World story of an update to the original posting. After digging around more on the Actelion website, I found in the press release, “Actelion announces Half Year financial results 2009″ the same line, “The recently concluded Phase IIa proof-of-concept study does not support further clinical development at this time…”. This seems to be the only information they give. They do not include any information about the clinical trial data that leads them to this decision. I will look into this more and send along any interesting information I find.

    I apologize for getting anyone’s hopes up over this trial. As someone with CF, I was very hopeful myself that this would be a new and revolutionary treatment option. I am still hopeful that someone (maybe Vertex, maybe someone else) will develop a successful CFTR corrector that will be that next big step in CF treatment.

    1. Nathan on August 27th, 2009 at 6:28 am

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