Posted on 05/04/2024 12:15:40 PM PDT by ConservativeMind
A preclinical study from Karolinska Institutet offers hope for treating severe neurodegenerative diseases with an existing drug. The study suggests that the leprosy drug clofazimine may be effective in the treatment of Huntington's disease.
The research group examined whether existing drugs could reduce the toxicity of so-called polyQ proteins. These proteins are found in patients with certain hereditary neurodegenerative diseases, including Huntington's disease, for which there is no cure.
Screening hundreds of drugs, they found that the leprosy drug clofazimine reduces the toxicity of polyQ proteins and restores mitochondrial function in zebrafish and worms. The finding supports the previous hypothesis that polyQ diseases are associated with the dysfunction of mitochondria, the organelles in charge of producing energy within cells.
"Our work not only suggests the interest of a specific drug for the treatment of polyQ neurodegenerative diseases, but also helps us to better understand what causes these diseases.
"It is possible to find new uses for old drugs, which reduces the time needed to find novel therapies," says Oscar Fernandez-Capetillo.
A problem is that clofazimine is not very efficient in entering the nervous system. The research group is now trying to figure out solutions to this limitation, by testing the efficacy of clofazimine in mammalian models of neurodegenerative disease.
"We hope that our discovery can be developed into a new medicine, but there are still some hurdles that need to be overcome," says Oscar Fernandez-Capetillo.
The researchers are also conducting similar drug screens in other age-related pathologies such as cancer and other neurodegenerative disorders.
(Excerpt) Read more at medicalxpress.com ...
They found mitochondria are a major part of the problem, and clofazimine worked on mitochondria in this disease.
There are other mitochondria boosting options (drugs and supplements) out there, but the status of these options is not yet known.
What is Huntington’s disease? Sixty years ago, my family lived in Huntington, Connecticut. Am I at risk of getting it?
:^) It may be the hereditary one, not sure. There’s a Wilson’s syndrome, which is not the same thing as Wilson’s disease, for example.
Dreadful…..a friend died from it…….several other cases. In her family. .
…..
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