SAK3, a new drug that could treat Alzheimer’s disease, has been developed by a Japanese research group led by Tohoku University Professor Kohji Fukunaga. After years of successful preclinical work, the drug is now ready to enter clinical trials.

A few years ago, a team of researchers developed a molecule they called SAK3. The molecule is designed to increase the release of acetylcholine and dopamine — two neurotransmitters that slow down in neurodegenerative diseases. By ensuring that these two chemicals are released in sufficient quantities, researchers can promote neuronal activity and fight neurodegenerative diseases.

The plan sounded good enough, so researchers took to testing the abilities of SAK3 using model organisms. The initial studies on mice turned out very promising. SAK3 not only improved cognitive deficits but also reduced the production of amyloid beta protein, the main component of the amyloid plaques found in the brains of people with Alzheimer’s disease. This suggests that the molecule could work against mild to severe Alzheimer’s disease, and, researchers note, SAK3 could be effective beyond Alzheimer’s and also work against other neurodegenerative diseases.

A recently published study analyzed the effectiveness of the molecule against Lewy Body Dementia, the second most common type of progressive dementia after Alzheimer’s disease. The condition is characterized by a build-up of misfolded proteins. The study found that the administration of SAK3 significantly inhibited the accumulation of these proteins, and even after the onset of cognitive impairment, it prevented the progression of neurodegenerative behaviors.

It appears that SAK3 can improve the brain’s natural ability to destroy the misbehaving proteins that are responsible for so many neurodegenerative diseases. If the findings carry over to humans, then the molecule could open up treatment avenues for a number of conditions.

But whether or not this is the case is still unclear. We need clinical trials to be certain of its positive impacts on humans, and that won’t happen overnight.

While the drug seems promising, it will likely take years before clinical trials are concluded, and (if everything goes according to plan) actual treatment can commence. But it’s promising to see more treatments for Alzheimer’s moving closer to clinics. In June 2021, the FDA approved the first new Alzheimer’s drug in almost 20 years.

The study “Pharmacological properties of SAK3, a novel T-type voltage-gated Ca²⁺ channel enhancer” has been published in the journal Neuropharmacology.