Transplanted neural stem cells treat ALS in mouse model

By Heather Buschman, Ph.D.
December 19, 2012

In 11 independent studies, a consortium of ALS researchers shows that transplanting neural stem cells into the spinal cord of an ALS mouse model slows disease onset and progression, improves motor function, and significantly prolongs survival.

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is untreatable and fatal. Nerve cells in the spinal cord die, eventually taking away a person’s ability to move or even breathe. A consortium of ALS researchers at multiple institutions, including Sanford-Burnham Medical Research Institute, Brigham and Women’s Hospital, and the University of Massachusetts Medical School, tested transplanted neural stem cells as a treatment for the disease. In 11 independent studies, they found that transplanting neural stem cells into the spinal cord of a mouse model of ALS slows disease onset and progression. This treatment also improves host motor function and significantly prolongs survival.

Surprisingly, the transplanted neural stem cells did not benefit ALS mice by replacing deteriorating nerve cells. Instead, neural stem cells help by producing factors that preserve the health and function of the host’s remaining nerve cells. They also reduce inflammation and suppress the number of disease-causing cells in the host’s spinal cord. These findings, published December 19 in Science Translational Medicine, demonstrate the potential neural stem cells hold for treating ALS and other nervous system disorders.

“While not a cure for human ALS, we believe that the careful transplantation of neural stem cells, particularly into areas that can best sustain life—respiratory control centers, for example—may be ready for clinical trials,” Evan Y. Snyder, M.D., Ph.D., director of Sanford-Burnham’s Stem Cell and Regenerative Biology Program and senior author of the study.

Neural stem cells

In this study, researchers at multiple institutions conducted 11 independent studies to test neural stem cell transplantation in a well-established mouse model of ALS. They all found that this cell therapy reduced the symptoms and course of the ALS-like disease. They observed improved motor performance and respiratory function in treated mice. Neural stem cell transplant also slowed the disease’s progression. What’s more, 25 percent of the treated ALS mice in this study survived for one year or more—roughly three to four times longer than untreated mice.

Neural stem cells are the precursors of all brain cells. They can self-renew, making more neural stem cells, and differentiate, becoming nerve cells or other brain cells. These cells can also rescue malfunctioning nerve cells and help preserve and regenerate host brain tissue. But they’ve never before been studied extensively in a good model of adult ALS.

How neural stem cells benefit ALS mice

Transplanted neural stem cells helped the ALS mice, but not for the obvious reason—not because they became nerve cells, replacing those missing in the ALS spinal cord. The biggest impact actually came from a series of other beneficial neural stem cell activities. It turns out neural stem cells produce protective molecules. They also trigger host cells to produce their own protective molecules. In turn, these factors help spare host nerve cells from further destruction.

Evan Snyder, M.D., Ph.D.

Then a number of other positive events take place in treated mice. The transplanted normal neural stem cells change the fate of the host’s own diseased neural stem cells—for the better. This change decreases the number of toxin-producing, disease-promoting cells in the host’s spinal cord. Transplanted neural stem cells also reduce inflammation.

“We discovered that cell replacement plays a surprisingly small role in these impressive clinical benefits. Rather, the stem cells change the host environment for the better and protect the endangered nerve cells,” said Snyder. “This realization is important because most diseases are now being recognized as multifaceted in their cause and their symptoms—they don’t involve just one cell type or one malfunctioning process. We are coming to recognize that the multifaceted actions of the stem cell may address a number of these disease processes.”


This research was funded by Project ALS, California Institute for Regenerative Medicine, the U.S. National Institutes of Health (National Institute of Neurological Disorders and Stroke grants R21NS053935, 1RC2NS070342-01, 1RC1NS068391-01, R01NS050557-05, U01NS05225-03), U.S. Department of Veterans Affairs, Christopher Reeve Foundation/American Paralysis Association, Sanford Children’s Health Research Center, Zinberg Foundation, ALS Therapy Alliance, ALS Association, Angel Fund, Al-Athel Foundation, Pierre L. deBourgknect ALS Research Foundation, P2ALS, and HeadNorth.

Original paper:

Teng YD, Benn SC, Kalkanis SN, Shefner JM, Onario RC, Cheng B, Lachyankar MB, Marconi M, Li J, Yu D, Han I, Maragakis NJ, Lládo J, Erkmen K, Redmond DE Jr, Sidman RL, Przedborski S, Rothstein JD, Brown RH Jr, & Snyder EY (2012). Multimodal Actions of Neural Stem Cells in a Mouse Model of ALS: A Meta-Analysis. Science translational medicine, 4 (165) PMID: 23253611

Facebook Comments

About Author

Heather Buschman, Ph.D.

Heather was an SBP Communications staff member.



  1. Pingback: ‘Insulating’ brain cells appear to play a critical role in brain cell survival and may contribute to neurodegenerative diseases such as ALS |

  2. my wife has ALS and will be willing to have stemcell treatment.she is losing her breath more and more.i fear she will pass before I can get her help.we just heard about the stem cell treatments being used.if you know of any centers that are treating this ALS,even in another country please please let me know.and thank you DH

    • james fisher on

      Did you ever hear back from anyone after you sent your email concerning your wife? My sister is in the same position. She has ALS.

    • Patrick Bartosch on

      Hi David,

      My apologies for the late response. Since we are a basic medical research institute – most of our scientists are biologists and chemists, not medical doctors – I cannot really offer recommendations on treatment center. What you could do, is google ALS patient advocacy groups and contact them directly. They may know of centers and doctors who are specialized in ALS. If you’re interested in clinical trials, please visit



  3. james fisher on

    How do you go about becoming a patient in any upcoming clinical studies? We are in a position where we would like to participate. Do I contact Dr. Snyder directly, or Sanford-Burnham Medical Research Institute?

    • Patrick Bartosch on

      Hi James,

      Thank you for reading Beaker and your comment. Sanford-Burnham is a basic medical research institute, which means that we are generally not seeing patient. If you’re interested in clinical studies, we recommend you visit

      All the best!


  4. Its like you read my mind! You seem to know a lot about this, like you wrote the book in it or something. I think that you can do with a few pics to drive the message home a bit, but instead of that, this is fantastic blog. A fantastic read. I will definitely be back. dgekacdedcbf

Leave A Reply



* Copy This Password *

* Type Or Paste Password Here *

45,189 Spam Comments Blocked so far by Spam Free Wordpress