Stem Cells for ALS

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Put simply, some human cells can regenerate:

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Regeneration of a human toe nail

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However, many human cell lines, including central nervous tissue, can not regenerate:

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Human stem cells can go through numerous cycles of cell division while maintaining the undifferentiated state, but can still differentiate into specialized tissues like nerve cells:

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When the appropriate stem cells are injected into injured tissues they will multiply, develop, and repair CNS injuries – at least, that’s the theory:

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So far, human Phase I (safety) trials have shown that stem cells can be injected directly into the spinal cords of ALS patients:


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The Phase II (dose escalation) trials began in September 2013 in Michigan and Atlanta, and aims to recruit 15 patients for 5 different dosing protocols, and will look at efficacy:

Click here to find out more, and watch this space for results.

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3 thoughts on “Stem Cells for ALS

  1. The two block diagrams in this article are very helpful in understanding the basic principles. As you know, Children’s Hospital in Ohio is doing a gene therapy trial for IBM patients. Is the basic principle the same as applicable to ALS? In both case it is the weakening of the muscle tissues that is often the underlying cause.
    Kindly comment.

      • Thanks, Dr Holland. I thought that ALS also required muscle growth, but I have a lot to learn about stem cells. More importantly, the commonality of Ohio trial and BYM338 really intrigues me. Your recent excellent blog on BYM 338 had some question marks for the associated trial. Would those apply to the Ohio trial also?
        On a separate and broader issue: why does it take so long to start recruiting for a trial? Patients for serious illnesses like IBM and Cancer have a limited lifetime left and even a few months can mean a good fraction of that spent just waiting!
        Keep up the good work of educating patients!

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