Japanese researchers have been developing a new kind of drug treatment for dementia, and after years of preclinical work, they’re ready to begin human clinical trials in the next year. The treatment candidate, a novel therapeutic molecule called SAK3, was declared safe by Japan’s governing board. In experiments with mice, it appears to halt neurodegenerative symptoms in animal models of dementia and Alzheimer’s disease and reverse the disorders’ effects.
SAK3 is a T-type calcium channel enhancer. It’s designed to promote neuronal activity by increasing releases of the two neurotransmitters, acetylcholine, and dopamine, which become less active in neurodegenerative diseases.
Initial tests in Alzheimer’s mouse models showed SAK3 improved cognitive deficits and minimized the build-up of the amyloid proteins typically implicated in Alzheimer’s progression. Furthermore, d SAK3 could potentially be effective in other neurodegenerative diseases beyond just Alzheimer’s.
The team’s most recent study focused on Lewy Body Dementia, a condition characterized by the build-up of misfolded alpha-synuclein proteins. It’s the second most prevalent type of dementia following Alzheimer’s.
Kohji Fukunaga, an author of the study, explained:
“We found that chronic administration of SAK3 significantly inhibited the accumulation of alpha-synuclein in the mice. Even after the onset of cognitive impairment, SAK3 administration significantly prevented the progression of neurodegenerative behaviors in both motor dysfunction and cognition.”
SAK3 appears to effectively improve the brain’s natural ability to locate and destroy the misfolded proteins that characterize several neurodegenerative diseases. The therapy could also improve cognitive dysfunction.
Fukunaga said:
“There are currently no disease-modifying therapeutics for neurodegenerative disorders such as Alzheimer’s disease, Lewy body dementia, Huntington disease, and frontotemporal dementia in the world. We discovered the novel, disease-modifying therapeutic candidate SAK3, which, in our studies, rescued neurons in most protein-misfolding, neurodegenerative diseases.”
However, we’ll only really know how promising SAK3 is for people with trials investigating the drug’s efficacy in humans.
