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Prion Disease: Researchers Discover New Treatments for Neurodegenerative Diseases | Health News

Washington DC: Possible effective treatment strategies for patients with prion disease have been suggested by a new study published by Oxford University Press. The results of the new study were published in the journal Nucleic Acids Research.

Prion disease is a rapidly destructive and currently untreatable neurodegenerative disease. Prion disease is very rare but usually causes rapid neurodegeneration. About 300 prion diseases are reported each year in the United States.

The most common prion disease affecting humans is Creutzfeldt-Jakob disease. Bovine spongiform encephalopathy, commonly known as mad cow disease, is another prion disease.

Prion disease is caused by disrupting the structure of the normal human prion protein, creating a toxic mass in the brain. Lowering prion protein levels in patients is a promising therapeutic approach, as prion proteins are central to the disease.

Senior author Sonia Varab learned to have a mutated prion protein gene before switching careers to become a patient scientist and advocating for treatment.

She and her colleagues have previously observed that antisense oligonucleotides that reduce levels of prion proteins can prolong the survival of animals infected with misfolded prions. While these initial data were promising, many important questions remained before the development of treatments became possible.

Research teams led by Broadlab of Harvard and Vallabh of MIT, Holly Kordasiewicz of Ionis Pharmaceuticals, and Deb Cabin of McLaughlin Research Institute report the results of a preclinical study of antisense therapy for prion disease.

In this new study, the authors laid the foundation for full-scale clinical development using an extended set of antisense oligonucleotides targeting prion proteins.

This study shows that reducing prion protein levels in animals infected with prions significantly extends survival across multiple treatment paradigms.

Researchers here have shown that reducing prion protein levels triples the survival of prion-infected animals. Even slightly lowering the level of prion protein should have been clinically easier to achieve, but it did bring important survival benefits.

Reduction of prion protein is effective throughout the prion strain and over a range of different treatment time points. Researchers have shown that reducing prion protein before symptoms appear can be effective. They also show for the first time that a single dose of prion protein lowering therapy can reverse the markers of disease, even after the formation of toxic clots in the brain.

These data provide optimism that gene-targeted drugs designed to reduce prion protein levels in the brain may be effective in the clinic by looking straight at the genetic center of prion disease. It shows the perspective. ”

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