Amyotrophic lateral sclerosis is a cruel disease. It causes the nerves that control muscles to die. When these upper and lower motoneurons degenerate, it causes weakness, muscle atrophy, muscle cramps, and twitching. Patients with progressed ALS lose the ability to walk and to move, and ultimately lose the ability to swallow and to breathe.

Unfortunately, there is no cure for this disease. Two drugs, riluzole, and edavarone, can help slow the progression of amyotrophic lateral sclerosis; however, these treatments cannot stop the disease.

Stem cells are an intriguing potential option for those wanting to manage symptoms from neurodegenerative diseases like ALS. The hypothesis is simple. Scientists are aggressively pursuing stem cell research to treat amyotrophic lateral sclerosis to study the use of stem cells to potentially restore diseased nerve cells in ALS which may help to restore muscle function. Drs. Gugliandolo, Bramanti, and Mazzon recently reviewed the potential use of mesenchymal stem cells for the treatment of ALS.

Mesenchymal stem cells can be gathered from many different sites in the body including bone marrow, umbilical cord, or adipose (fat) cells. The stem cells can then become several different cells in the body, including nerve cells (i.e. neurons). Mesenchymal stem cells also produce and release (e.g. through exosomes) an astounding number of molecules that help other cells grow and develop. Thus, mesenchymal stem cells can not only become new nerve cells, they can support other nerve cells’ growth and development.

The authors describe in detail the potential for mesenchymal stem cells to help treat ALS. The review shows the many successful uses of stem cells in animals (mice) that have experimental ALS. In short, stem cells slowed the loss of motor function (muscle activity), delayed the progression of ALS, and increased length of survival.

Clinical trials of mesenchymal stem cells to treat ALS are in Phase I and Phase II, however initial results are encouraging. First of all, treatment with these stem cells is safe in patients with ALS—no serious adverse events have been reported in any of the trials reviewed in the journal article. In at least 9 clinical trials, mesenchymal stem cells slowed disease progression in patients with ALS to some degree.

Patients with ALS and those who care for them should note that while these trials have shown that mesenchymal stem cells are safe and at least partially effective in the treatment of ALS, Phase I and Phase II clinical trials only have a relatively small number of patients compared to Phase III trials. Nonetheless, the clinical trial results thus far look promising, certainly promising enough for certain types of stem cells to advance to Phase III pivotal clinical trials.

Reference: Gugliandolo, A., et al. Mesenchymal Stem Cells: A Potential Therapeutic Approach for Amyotrophic Lateral Sclerosis? Stem Cells International. Vol. 2019, Article ID 3675627, 16 pages, 2019. https://doi.org/10.1155/2019/3675627

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