In a collaborative project, scientists have found that an existing cancer drug may offer potential to treat Huntington's disease. The work was published yesterday in Science Translational Medicine from researchers at Duke University, UC San Diego and the Salk Institute.
"It's not just the response from the drugs, but the mechanistic pathways these drugs are targeting," said senior author Al La Spada, M.D., Ph.D., director of the forthcoming Duke Center for Neurodegeneration and Neurotherapeutics.
In this study, mice with the equivalent of Huntington's disease became more mobile, recovered from neurodegeneration and lived longer after being treated with bexarotene. The researchers found that bexarotene functions by activating PPARδ, a transcription factor that keeps neurons functional by keeping mitochondria healthy and removing dysfunctional proteins. Mice and humans with this disease have a hard time activating PPARδ, so when the researchers treated their mice with bexarotene, they the mitochondria health improve in the neurons and an increased removal of damaging misfolded proteins.
Further research still needs to completed to address dosage sizes or how to even use this drug in human patients when treating Huntington's disease. However, the work does provide hope for a potential therapy against Huntington's disease.