Is it too soon for mesenchymal stem cell trials in people with ALS?

[wildwilly]

I do not have acess to full text (but I did order the article). If anyone has the article please post a comment on results of the preclinical data. If not I will post a comment when I recieve the article.


Amyotroph Lateral Scler. 2008 Dec;9(6):321-2.

Is it too soon for mesenchymal stem cell trials in people with ALS?

Badayan I, Cudkowicz ME.

Neurology Clinical Trials Unit, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA 02129, USA. ibadayan@partners.org

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease affecting primarily the motor neurons. Stem cell therapy is under development as a possible treatment approach. A pilot study of intraspinal injections of mesenchymal stem cells (MSC) was conducted in 9 participants. We review this paper, the rationale, preclinical data and study design.

[wildwilly]

This was a pilot study of autologous intraspinal injections of mesenchymal stem cells into 9
individuals with ALS.

There was one injection and individuals were followed for 4 years. MRI sans, respiratory function, and psychiatric outcome measures were evaluated pre and post injection. Four individuals died from disease progression. Their disease pathology was not presented.

Efficacy of dosage/treatment not reliable secondary to multiple experimental design weakness/flaw (participant variability/disease heterogeneity, no control, sample size…..). No information what happened to the cells. Poor follow up on several patients.

There were no adverse events reported to the injection and or treatment.

Conclusion: If additional preclinical trials are to be held, experts in ALS and experimental design should be consulted. Based on the present study additional open label study’s in ALS patients is not warranted.

Not exactly the review I was hopping for.