A small molecule agent like methylene blue that has been grandfathered into approved use as a diagnostic tool in humans can be studied further as possible treatment for the neurodegenerative illness

By Kathleen Raven

Methylene blue Image: Flickr/amandabhslater

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FromNature Medicine’s”Spoonful of Medicine” blog:

A compound already sitting on the shelves of biomedical laboratories and emergency room supply closets seems to interrupt the formation of neurodegenerative protein clumps found in Huntingtons disease, according to a preliminary animalstudypublished August 7 in theJournal of Neuroscience.

This versatile agent, called methylene blue, gets a mention in medical literature asearlyas 1897 and was used to treat, at one time or another, ailments ranging frommalariato cyanidepoisoning. The U.S. Food and Drug Administration has never formally approved it as a therapy for any illnesses. But that fact hasnt stopped biomedical researchers from tinkering with the agents apparent ability toimprovecognitive function. And although the new paper out today relies on a Huntingtons disease model in flies and mice, scientists are hopeful. “Because of existing knowledge of methylene blue and the fact that its not harmful to humans, I would hope that progress toward clinical trials could go relatively quickly,” saysLeslie Thompson, a neurobiologist at University of CaliforniaIrvine and lead author on the new study.

Huntingtonsdiseaseoccurs when the C-A-G sequence of DNA base pairs repeat too often on theHTTgene, resulting in an abnormally long version of the huntingtin protein, that therefore folds incorrectly and forms clumps in the brain. The illness usually begins to affect people in their 30s and 40s, causing movement problems and early death. No drug is currently available to stop the disease from progressing.

For their experiment, researchers fed methylene blue mixed with food for a week toDrosophilaflies engineered to carry a mutated copy of theHTTgene. An examination of the flies brains showed that protein clumps had been reduced by 87 percent compared with a control group. Meanwhile, mice designed to carry the mutated gene were given methylene blue and underwent several tests to assess mobility. At two months of age, the treated mice showed abnormal clasping of their hind claws only 20 percent of the time in a reflex test, whereas their untreated counterparts clasped at a 60 percent rate. (Less clasping meant healthier mice.) While the number of mice used was not sufficient to provide statistically significant results and the difference in the test quickly dropped off at nine weeks of age, Thompson still views the data as hopeful, because even a delay in Huntingtons symptoms would be very helpful. Thompson quickly points out that more research is needed. “The early steps of aggregation [protein clumping] are getting altered in a test tube, in flies and in miceand thats significant,” she says, and speculates that methylene blue may possibly prevent the mutantHttprotein from sticking to itself.

See the article here: