Progressive supranuclear palsy (PSP) is a brain disease that belongs to a group of neurological diseases referred to as tauopathies. PSP impairs eye movements, locomotion, balance control, and speech, and is currently incurable. Now scientists have discovered a molecular mechanism that may help in the search for effective treatments for PSP and potentially other tauopathies. Their study, which focuses on a protein called PERK (protein kinase RNA-like endoplasmic reticulum kinase), was published in EMBO Molecular Medicine.

In tauopathies, a molecule called tau forms clumps rather than stabilizing the cytoskeleton as it normally does. Affected neurons can degenerate or even perish. To prevent such events, pathological molecules are normally repaired or disposed of by the organism. The protein PERK is part of such a maintenance system. However, in PSP, this mechanism appears to be defective. In previous studies, the researchers had found that the risk for PSP is associated with variants of the PERK gene, and that loss of PERK function induces tau pathology in humans. For the current study, they examined the functioning of this protein more closely, to see how its effects could be positively influenced. To this end, they investigated samples of brain tissue from deceased patients, cell cultures and mice with a genetic disposition for PSP.

They found that the disease sequelae decrease when PERK is activated with pharmaceuticals. Their findings, the researchers say, show that PERK is an important part of the disease mechanism.

The scientists see potential for tackling other brain diseases because PERK helps eliminate the abnormal tau molecules that also occur in other diseases such as Alzheimer’s disease.

Paper: „A protein called PERK may be a target for treating progressive supranuclear palsy: Acting upon the maintenance system of neurons alleviates disease sequelae in laboratory experiments“
Reprinted from materials provided by DZNE- German Center for Neurodegenerative Disease.

април 7, 2017