Drug Target For Prion Diseases?

Researchers at St Mary's Hospital Medical School in London, UK, have uncovered new clues to the mechanism of neurodegeneration in prion diseases such as Creutzfeld-Jakob disease. Prion diseases are neurodegenerative conditions, transmissible by inoculation and in some cases inherited. The prion consists largely of a modified form of host glycoprotein (PrPc), called PrPsc.

They have discovered that the normal form of the prion protein seems to be essential for synaptic transmission. Mice which are unable to make their own prion protein show abnormalities in brain tissue function, and in particular the uptake of the neurotransmitter gamma amino butyric acid (GABA) by its receptors is impaired. The researchers suggest that an increased concentration of PrPsc may lead to a reduction in PrPc and this may contribute at least in part to the early synaptic loss and neuronal degeneration seen in CJD and other diseases.

The research, described in Nature (July 28), may form a starting point for the design of drugs which would interrupt the degenerative process, for example by binding to PrPsc and slowing or preventing its accumulation.