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 amyotrophic lateral sclerosis (ALS) in April 2013. The company’s Phase I trial was the first FDA-approved neural stem cell trial to treat ALS. Neuralstem's lead cell therapy candidate NSI-566 has been awarded orphan drug designation by the FDA for the treatment of ALS.
The company received FDA approval to commence a Phase I NSI-566 clinical trial for chronic spinal cord injury in January 2013. The Phase I
cSCI trial will use the same cells and procedure as its ALS trial. Neuralstem is targeting additional major central nervous system 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 central nervous system conditions.
The company is in an FDA-approved Phase Ib safety trial evaluating NSI-189, its lead novel proprietary, neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). The trial advanced to its third and final cohort in April 2013 and is expected to conclude in the third quarter of 2013. NSI-189 has been shown to stimulate new neuron growth in the hippocampus and increase hippocampal volume in preclinical studies. Additional indications could include traumatic brain injury, Alzheimer’s disease, and post-traumatic stress disorder (PTSD).