0
0
Karolinska Institutet’s Research Offers Glimmer of Hope in Neurodegenerative Disease Treatment
In a groundbreaking preclinical study, researchers from the Karolinska Institutet have identified a potential treatment avenue for severe neurodegenerative diseases, particularly Huntington’s disease, utilizing an existing drug typically prescribed for leprosy. The study, published in the esteemed journal eBioMedicine, sheds light on the therapeutic potential of clofazimine in mitigating the toxicity associated with polyQ proteins, often implicated in various hereditary neurodegenerative conditions.
PolyQ proteins, prevalent in patients afflicted with certain hereditary neurodegenerative diseases like Huntington’s disease, have long posed a significant challenge due to the lack of effective treatment options. However, the research team, led by Professor Oscar Fernandez-Capetillo from the Department of Medical Biochemistry and Biophysics at Karolinska Institutet, embarked on a mission to explore the efficacy of existing drugs in ameliorating polyQ protein toxicity.
After meticulously screening hundreds of drugs, the researchers stumbled upon a promising candidate: clofazimine. This leprosy drug demonstrated remarkable potential in reducing the toxicity associated with polyQ proteins and reinstating mitochondrial function, a critical aspect compromised in neurodegenerative conditions, in zebrafish and worms. These findings not only underscore the therapeutic promise of clofazimine but also shed light on the underlying mechanisms driving polyQ diseases, linking them to mitochondrial dysfunction.
Professor Fernandez-Capetillo highlighted the significance of repurposing existing drugs, emphasizing that such an approach significantly expedites the quest for novel therapies. However, a notable hurdle lies in clofazimine’s limited efficacy in penetrating the nervous system. Nevertheless, the research team remains undeterred, actively exploring strategies to overcome this limitation, including assessing the drug’s efficacy in mammalian models of neurodegenerative diseases.
“While our discovery holds immense promise for the development of a new medicine, there are undoubtedly hurdles that necessitate diligent navigation,” remarked Professor Fernandez-Capetillo, acknowledging the challenges ahead.
Furthermore, the researchers are extending their drug screening endeavors beyond neurodegenerative diseases, delving into age-related pathologies such as cancer. This multifaceted approach underscores their commitment to unraveling the therapeutic potential of existing drugs across diverse disease spectrums, offering renewed hope for patients worldwide.
The study, titled “The anti-leprosy drug clofazimine reduces polyQ toxicity through activation of PPARγ,” represents a significant milestone in the pursuit of effective treatments for debilitating neurodegenerative diseases, heralding a new era of therapeutic possibilities rooted in innovative research endeavors.
For more information on the study, refer to Xuexin Li et al’s publication in eBioMedicine (2024).
