Researchers have discovered a drug that can boost the production of brain-repairing stem cells in the nervous system.
University of Texas Southwestern Medical Center scientist Andrew Pieper and his colleagues, writing in Cell, describe the discovery of the agent P7C3, which they identified by screening through 200,000 candidate drugs molecules.
Selecting the most promising-looking 1000 agents, these were divided up into groups of ten that were injected as a cocktail into the brains of mice. The mice were also given a drug called BrdU, which labels dividing cells, enabling the scientists to count the rates at which new nerve cells were being produced in the brain region known as the hippocampus of the treated animals. This enabled the team to home in on one compound in particular that had the most potent action, which was P7C3, an aminopropylcarbazole.
In subsequent experiments they were able to show in animals lacking a gene called npas3, which triggers a preponderance to brain degeneration, that treated animals maintained normal nerve cell architecture in the hippocampus and that the cells were correctly wiring themselves up electrically.
Even more convincingly, the team then tested the effect of giving the compound to elderly rats which, just like humans, are prone to forgetfulness. Compared with controls, the treated animals performed significantly better at memory and navigation tasks.
The researchers think that the new agent is working by preventing newborn nerve cells that are produced in some parts of the nervous system from undergoing a process called apoptosis, in which the cells kill themselves.
The next step will be to look at safety and possible efficacy in humans, since this agent could prove highly effective in restoring or at least preserving brain function in patients with degenerative diseases like Alzheimer's.