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Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. The company received FDA approval to commence a Phase II NSI-566 clinical trial for ALS in April 2013, and was approved in January 2013 to commence a Phase I NSI-566 safety clinical trial for chronic spinal cord injury (cSCI). Neuralstem is targeting additional major CNS conditions with its cell therapy platform, including ischemic stroke, glioblastoma (brain cancer), and Huntington’s disease.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some CNS conditions. The company is in an FDA-approved Phase Ib safety trial evaluating NSI-189, its first of four neurogenic small molecule compounds, for the treatment of major depressive disorder. The FDA approved dosing of the third and final cohort of depression patients in April 2013. Additional indications could include traumatic brain injury, Alzheimer's disease, and post-traumatic stress disorder (PTSD).