Wednesday, March 10, 2021

NIAID Scientists Use Cerebral Organoid to Test Drug for CJD

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Wednesday, March 10, 2021

NIAID Scientists Use Cerebral Organoid to Test Drug for CJD

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A cerebral organoid shown overlaid with test results from prion infected organoids that were left untreated or treated with PPS. The results show that treatment reduces disease-associated protein. (NIAID)

 

NIAID scientists who two years ago developed a human cerebral organoid model of Creutzfeldt-Jakob disease (CJD) for studies have advanced the model to screen drugs that could treat the deadly prion disease. Their new study published in Scientific Reports explains how the scientists tested pentosan polysulfate (PPS) to determine its potential preventive and therapeutic benefits. In the experiments, PPS treatment reduced the disease indicators by 10-fold or more without causing tissue death. While it may extend a patient's life, PPS has not been shown to improve quality of life. Human cerebral organoids are small balls of human brain cells ranging in size from a poppy seed to a pea; scientists use human skin cells to create them. CJD, a fatal neurodegenerative brain disease of humans caused by infectious prion proteins, affects about 1 in 1 million people each year. The scientists are working to expand the organoid model for screening larger numbers of novel drug candidates.

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