Researchers at the Salk Institute for Biological Studies in US developed the drug, known as J147, which reverses memory deficits and slows Alzheimer's disease in aged mice following short-term treatment.
The study builds on earlier research finding that the drug preserves brain function when given to Alzheimer's mice model. The previous research was published in 2011 in the journal PLoS One.
"J147 is an exciting new compound because it really has strong potential to be an Alzheimer's disease therapeutic by slowing disease progression and reversing memory deficits following short-term treatment," said lead study author Marguerite Prior, a research associate in Salk's Cellular Neurobiology Laboratory.
From the test results of each chemical iteration of the lead compound, they were able to alter their chemical structures to make them much more potent.
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Although J147 appears to be safe in mice, the next step will require clinical trials to determine whether the compound will prove safe and effective in humans.
To test the efficacy of J147 in a much more rigorous preclinical Alzheimer's model, the Salk team treated mice using a therapeutic strategy that they said more accurately reflects the human symptomatic stage of Alzheimer's.
In a different experiment, the scientists tested J147 directly against Aricept, the most widely prescribed Alzheimer's drug, and found that it performed as well or better in several memory tests.
Prior and her colleagues said that several cellular processes known to be associated with Alzheimer's pathology are affected by J147, including an increase in a protein called brain-derived neurotrophic factor (BDNF), which protects neurons from toxic insults, helps new neurons grow and connect with other brain cells, and is involved in memory formation.
The study was published in the journal Alzheimer's Research and Therapy.