Huntington’s disease is a neurodegenerative pathology that is characterized by the destruction of a type of brain neurons, which affects movement function and leads to dementia in advanced stages. There is no cure, but medications are used to reduce symptoms and improve the patient’s quality of life, and now a team of scientists from the Karolinska Institute in Sweden has discovered that clofazimine, a drug used in the treatment of leprosy, could be effective in treating Huntington’s disease.
These researchers conducted a preclinical study to evaluate whether available medications could decrease the toxicity of proteins known as polyQ. These proteins are found in patients with certain inherited neurodegenerative diseases, including Huntington’s disease.
By analyzing hundreds of drugs, they found that clofazimine reduces the toxicity of polyQ proteins and restores mitochondrial function in zebrafish and worms. This finding supports the previous hypothesis that polyQ diseases are associated with dysfunction of mitochondria, organelles responsible for producing energy within cells. The findings have been published in eBioMedicine.
Treat neurodegenerative diseases with an antimicrobial drug
“Our work not only suggests the interest of a specific drug for the treatment of polyQ neurodegenerative diseases, but also helps us better understand the causes of these diseases. “It is possible to find new uses for old drugs, which reduces the time needed to find new therapies,” said Óscar Fernández-Capetillo, professor and research group leader at the Department of Medical Biochemistry and Biophysics at the Karolinska Institute.
“Our work also helps us better understand the causes of polyQ neurodegenerative diseases”
The authors of the work conclude in their article that their results “support the potential of reusing the antimicrobial drug clofazimine for the treatment of polyQ diseases.” One problem, however, is that clofazimine is not very efficient at penetrating the nervous system, so the team is currently looking for solutions to this limitation, testing the effectiveness of clofazimine in mammalian models of neurodegenerative disease.
“We hope that our discovery can become a new medicine, but there are still some obstacles to overcome,” said Óscar Fernández-Capetillo. Researchers are also conducting similar tests with other drugs in pathologies associated with aging, such as cancer and other neurodegenerative disorders.
