Neurosteroids, like allopregnanolone (ALLO), are natural signaling molecules made in the brain from cholesterol and are essential for normal brain biochemistry. They can induce potent anxiolytic, antidepressant, anticonvulsant, and analgesic effects, mainly through interaction with the GABA channel, the main inhibitory system in the brain.

In addition to these psychopharmacological effects, neurosteroids also demonstrate neuroprotective, neurotrophic, and anti-inflammatory effects in several animal models of neurodegenerative diseases, mainly by shutting down overactive immune cells (like microglia). Chronic activation of the brain’s resident immune cells results in neuroinflammation, a common feature of many neurodegenerative diseases which is implicated in their onset and progression.

Altered levels of neurosteroids such as ALLO have been measured in many neuropsychiatric and neurological disorders (anxiety, depression, post-partum, post-traumatic stress disorder, schizophrenia, alcohol dependence, epilepsy, Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, Niemann-Pick type C disease, diabetic neuropathy, traumatic brain injury, stroke and retinal degenerative disorders) both in humans and animal models.

Both preclinical and clinical studies demonstrate a therapeutic potential for neuroactive steroids to both alleviate symptomology, and/or modify the disease state in a variety of CNS disorders.