From Arthritis to Zoster

Maryland-based NeuralStem, a biotherapeutics company engaged in the development of neural stem cells for treating diseases of the central nervous system, has just begun patient trials to determine the safety of spinal stem cell injections. This represents the first such trial in the United States and marks a major milestone in the use of stem cells for treating serious human conditions.

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, is a progressive degenerative illness that eventually destroys the nerves that control voluntary muscle movement. Without nervous impulses to control muscle movement and maintain muscle integrity, individuals with ALS eventually lose the ability to initiate and control all voluntary movement, including efficient breathing activity. Death from ALS, which typically occurs within two to five years of diagnosis, is usually due to respiratory failure or pneumonia.

The specific cause of ALS is unknown, although immune dysfunction may play a role. Mitochondrial disorders, DNA abnormalities, exposure to toxic or infectious agents, and cellular oxidative stress have all been proposed as contributing factors to this disease.

Scientists at NeuralStem have demonstrated that “neuro-adherent” stem cells can survive, integrate, and differentiate when placed in the central nervous systems of animals. These cells, when used in animals with ALS, exert a protective effect – essentially forming functional “patches” over damaged nerves – that delays the progression of the disease.

Now, with approval from the FDA, NeuralStem has injected its neural stem cells into the spinal cord of a 60-year-old man with advanced ALS. The company, in conjunction with researchers at Emory University, has chosen to begin trials in people with more serious disease, because these subjects are less likely to lose additional function if the stem cell transplants cause unanticipated adverse effects.

In short, this initial trial is not designed to test the treatment’s efficacy; it is meant to evaluate the safety of spinal stem cell injections. Up to 18 patients will be recruited for this phase of the investigation.

With time – assuming that NeuralStem’s cell lines don’t precipitate untoward side effects – this therapy will be directed toward alleviating or even curing a devastating disease. Additionally, other serious disorders of the central nervous system will lend themselves to similar approaches.

In the end, stem cell therapy (regardless of cell line origin) has the potential to allay a great deal of human suffering, improve the quality of life for countless individuals, and reduce the financial burden of disability that weighs on our economy.  

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