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NIH Record - 75th Anniversary - National Institutes of Health

Scientists Use Human Cerebral Organoid to Test Drug for CJD

Two years after establishing a human cerebral organoid system to study Creutzfeldt-Jakob disease (CJD), NIH researchers have further developed the model to screen drugs for potential CJD treatment. The NIAID scientists describe their work in Scientific Reports.

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 caused by infectious prion proteins, affects about 1 in 1 million people each year. It can arise spontaneously, result from a hereditary mutation within the prion gene, or arise due to infection, for example, from eating contaminated meat products. There are no preventive or therapeutic treatments for CJD.

Cerebral organoids are ideal for studying nervous system diseases over long periods of time because they can survive in a controlled environment for months to years. They have been used as models to study Zika virus infection, Alzheimer’s disease and Down syndrome.

The CJD study was conducted at NIAID’s Rocky Mountain Laboratories. 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. PPS is a benchmark anti-prion compound in laboratory experiments, but it is rarely used clinically because it requires direct administration into the brain.

While it may extend a patient’s life, PPS has not been shown to improve quality of life. However, using the anti-prion properties of PPS with the new human organoid CJD model allowed researchers to assess the value of this model system for drug discovery. The human organoid model can be used to screen compounds that may be useful for preventive treatment as well as screening drugs against established CJD. With this fully human model of disease, scientists are optimistic about identifying compounds that would benefit patients with CJD.

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