New Anti-Prion Compound Provides New Hope For Treating Brain-Wasting Disease

New drug compound can bind to prions and prevent it from replicating. Herrmann/Science Translational Medicine

Prions have previously been described as the “world’s most indestructible biological entity,” and for good reason. These infectious proteins are able to reproduce with deadly consequences. While you may not have heard of prion disease, you may know some of its various forms – bovine spongiform encephalopathy (BSE) and Creutzfeldt-Jakob disease, for example. There’s no effective medication for the treatment of animals and humans infected with prion disease, but a new drug compound could lead to a breakthrough.

As a type of fluorescent compound, the promising agent could do what others have failed to do so far: block prions from growing. The findings, published in the journal Science Transitional Medicine, could lead to new treatment for the fatal disease. 

Prion disease occurs when misfolded prion proteins induce others to fold into this abnormal shape. This sets a chain reaction where the misfolded proteins build up, leading to large clusters that cause brain damage. Scientists are still unclear exactly how prions destroy brain cells. They have previously tried destroying prions by freezing, boiling and even using radiation, but nothing has worked because the molecules are tightly bound.

Researchers were therefore surprised when certain compounds, known as luminescent conjugated polythiophenes, increased the survival of mice and rabbits infected with prion disease. Researchers tested a range of polythiophenes and the most promising candidate was found to prolong survival in mice by 80%. Researchers suggest the drug compound was able to stabilize and lock clusters of prions, which prevented them from replicating. Computer simulations showed that the polythiophenes were able to bind with prions and prevent them from attaching to another prion.

As Live Science explains, mice given polythiophenes before they were given prion disease survived up to 140 days. Prion mice given polythiophenes after infection lived up to 100 days. The rabbits and mice given polythiophenes were found to have fewer prion clusters and less severe brain damage. 

Researchers hope to further develop polythiophene as a treatment for prion disease. Creutzfeldt-Jakob disease is the most common form of prion disease in humans and symptoms range from loss of memory, change in personality and slurred speech. Most people infected with the fatal condition die within a year. 

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