breck076 - 16-2-2012 at 07:07
What is special about Ampicillin and its structure versus other beta-lactam antibiotics? I have dystonia and there is currently a trial being held to
use Ampicillin to activate TorsinA and return cell function back to normal. I'm more interested in how an antibiotic, of all things, would be able to
do this better than any other drugs currently available. Please feel free to move this if I have posted in the wrong area. TY.
phlogiston - 16-2-2012 at 12:33
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2860854/
I read the relevant part of the paper and, briefly, the authors test several possibilities for the mechanism and show that ampicilin is able to act as
a so called molecular 'chaperone'. Such molecules can help proteins to achieve their proper folding, which is critical to protein function. Mutated,
improperly folded torsinA tends to form aggregates, but in the presence of ampicilin, less of the torsinA ends up in aggregated form. They also
describe that in the presence of ampicilin, the total level of torsinA is increased, but they do not show why (and I think it may possible happen
because of its chaperone activity).
Other drugs based on the same principle are being tested for other diseases, such as cystic fibrosis.
In the paper, they also find other drugs that improve torsinA function, and they point out that some of these have the same core structure (fig 3),
which is apparently important.
breck076 - 16-2-2012 at 18:10
Thanks. I'll have to read through this again tomorrow evening. I had found it before, but lost the URL. I finally got the OK just a few minutes ago
from my neuro to try this. Doesn't help much that I'm allergic to Penicillin, but that's why I'll be taking the first few doses in the emergency room.