ALJAZEERA: …Many of these crippling diseases start with the buildup of rogue, scrunched-up proteins in the brain. The brain’s response to this is to switch on a defense mechanism called the unfolded protein response, or UPR.
The mechanism orders cells to stop producing new proteins so that the problem is not worsened. But the buildup of misshapen proteins prevents the UPR mechanism from being switched off.
As a result, the misshapen proteins are no longer made – but nor are normal proteins that are essential for brain-cell survival. Neurons start to die, are not replenished and the disease progresses.
The new drug works on a key point in this switching pathway, an enzyme called PERK, to keep protein production open… (more)
