Fenretinide Gets Orphan Drug Status
Jun 2nd, 2010 by Tom

There were a couple published studies in the last few years about ceramide levels in people with CF being either too high or too low.  I didn’t know what to make of it but I now see that the drug Fenretinide is being moved forward and has received orphan drug status.  This drug increases the number of ceramide species, thought to be deficient in CF, and this in turn boosts the ability to fight off pseudomonas infections in the CF lung.  In theory anyway.

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It looks very promising based on the mice studies but we all know mice are not the best model for CF.

‘Orphan Drug’ Research Offers Hope.

By combining molecules that potentiate the channel activity independently, it is possible to attain therapeutic results in Cystic Fibrosis
Apr 21st, 2010 by Tom

Every time I read an article like this (Potentiation of disease-associated CFTR mutants by hydrolyzable ATP analogs) on PubMed, I realize how far we have to go still.  Trying to keep a positive attitude, it is exciting to see that it is possible to combine multiple compounds and get up to a 36 fold improvement in gating for the G551D defect and almost a 20 fold improvement in gating for the DF508 defect.  For all of those dealing with rarer defects such as A455E that do not have much of a problem functioning once at the cell surface, it would be nice to see how these compounds behave.

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