Neurodegenerative disorders such as Huntington's disease are characterized by the accumulation of misfolded, defective proteins in brain cells. Certain drugs can stimulate a process for clearing such proteins, called autophagy, in some cells, but not in neurons. Steven Finkbeiner and his colleagues at the University of California, San Francisco, have identified a small molecule, a phenoxazine known as 10-NCP, that induces autophagy in neurons isolated from rodents.
In neurons expressing the mutant huntingtin protein, 10-NCP improved survival. The drug also decreased levels of mutant protein in neurons isolated from a mouse model of Huntington's disease. Moreover, the researchers found that several structurally similar drugs, already approved by the US Food and Drug Administration, could trigger neuronal autophagy.
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Neurobiology: Neuronal housekeeping. Nature 467, 255 (2010). https://doi.org/10.1038/467255c
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DOI: https://doi.org/10.1038/467255c