"This is a major finding," said Karl Johe, Ph.D., Neuralstem Chairman of the Board and Chief Scientific Officer. "There is currently no way to confirm the survival of the cells in patients while they are alive. Levels of functional recovery, or a slowdown in the progression of the disease in various patients, have given us reason to believe the cells have survived. Now, cell survival has been demonstrated by definitive evidence.
Among the six patients autopsied (five died of ALS disease progression and one, of unrelated heart failure), the survival period, from stem cell transplantation to death, ranged from 196 – 921 days. Five of these patients had discontinued all immune suppression medications for 57 – 638 days prior to death, but showed the stem cell DNA content in the range of 0.67% - 5.4% of total DNA in some spots of cord treated with the stem cells. There was no correlation of DNA content to survival period without immune suppression medication. These data, therefore, suggest that long-term immuno suppression of patients is not required for long-term survival of our cells, which points towards the feasibility of needing only transient immune suppression in future ALS trials," Dr. Johe concluded.
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. Neuralstem has recently treated the last patient in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in spinal cord injury.