Stem Cell Treatment Targeting Spinal Cord Won’t Slow ALS, Researchers Say

Stem Cell Treatment Targeting Spinal Cord Won’t Slow ALS, Researchers Say

Using stem cell treatments to protect motor neurons is not enough to prevent the loss of nerve-muscle connections and disease progression in a mouse model of amyotrophic lateral sclerosis (ALS), researchers from Italy found.

The study, published in the journal Stem Cell Research, had exposed mice to repeated brain injections of so-called mesenchymal stem cells, gathered from the umbilical cord. But, while the cells lessened inflammation in the spinal cord, it became apparent that more is needed to slow disease progression.

Earlier studies had noted that treatment with mesenchymal stem cells could improve survival in genetic ALS mouse models if started at birth or before symptoms appeared. But since human patients do not receive treatment until the disease is apparent, researchers at the Mario Negri Institute for Pharmacological Research in Italy wanted to test the approach in mice with obvious disease signs.

For the study, “Multiple intracerebroventricular injections of human umbilical cord mesenchymal stem cells delay motor neurons loss but not disease progression of SOD1G93A mice,” they gathered mesenchymal stem cells from human umbilical cords.

They injected them into the mice brain ventricles, which are natural fluid-filled cavities in the brain. The animals received injections once every two weeks — getting a total of three injections.

The team had hoped that the cells would travel to the spinal cord, but results were disappointing. Nonetheless, it became apparent that the cells did impact the environment in the spinal cord even from their position in the brain ventricles.

Researchers noted that the environment in the spinal cord shifted from a pro-inflammatory to an anti-inflammatory state — evident by changes in immune mediators called cytokines. Changes also were seen in the activity of glial cells involved in spinal cord inflammation.

Meanwhile, protective molecular pathways, acting to prevent cell death, had been activated in the mice spinal cords. While the cells did not reach the spinal cord, researchers said the effects indicated they secreted factors that could impact processes in other parts of the central nervous system.

Despite this protective environment, with more motor neurons surviving in the spinal cord, motor neuron axons were disconnected from the muscles and the team noted no slowing of disease progression. While not statistically significant, they did note a slight increase in survival time, however.

The findings suggest that potential stem cell or other neuro-protective treatments — delivered into the central nervous system —need to be combined with approaches that protect the connection between motor neurons and muscles to prevent disease progression.


  1. Charlie says:

    ‘The findings suggest that potential stem cell or other neuro-protective treatments — delivered into the central nervous system —need to be combined with approaches that protect the connection between motor neurons and muscles to prevent disease progression.’

    I wonder what Brainstorm’s ‘NurOwn’ project researchers will take from this announcement. I am guessing….. ‘frustration.’

  2. Charlie says:

    Brainstorm have said on their website “Our cells can protect neurons from toxins and have been effective in animal models of ALS, (sciatic nerve injury, Parkinson’s disease, multiple sclerosis, optic nerve ischemia and Huntington’s disease).

    Yet this report from Italy says efficacy is lacking.
    I think we need an independent researcher’s view.

  3. Charlie says:

    The ALS Therapy Development Institute says on its website (Re;NurOwn)
    “While this was an important phase 2 study, it is important to note that neither the overall study, nor the post hoc analysis were powered to measure for efficacy using the ALSFRS-R score. This is not uncommon, as phase 2 studies are typically designed to provide additional safety and tolerability data only, while at the same time informing an investigator how to best design a larger pivotal phase 3 study to measure efficacy as its primary endpoint. The principal investigators reported promising efficacy data through their post hoc analysis, when excluding “slow progressors”, but stopped short of making claims that NurOwn was effective in treating a person’s ALS. ”

    One step forward, one step back.

      • Charlie says:

        Efficacy means it works for all patients in a trial. If it works just for some (how many did it work well for?) then it lacks efficacy.

  4. Lisa says:

    I’m in Brainstorm’s Phase 3 clinical trial with NurOwn and this was not the news I wanted to read about this morning!!! My clinic appointment is tomorrow and my doctor was in Boston so I’m looking forward to hearing her thoughts on this.

  5. Dan says:

    This is taken from umbilical cord not bone marrow hip. Two completely different procedures.Both studies at Mass and Mayo are using bone marrow from hip.I would hope before we put negative information on this subject at least get it right.I am here in South Korea with a family member getting treatment today for 8 weeks and these first 10 days have shown great improvement with just radicava. Took the stem cell out 8 days ago and get it back in the 26th. I can say he looks 10 yrs younger but obvious the radicava is not a cure nor is the stem cell but a cocktail of these will hopefully slow down or even stop progression. I don’t know much about this awful disease and I’m resigned to the fact that I’m best served by listening to docs from Mass and or Mayo as my questions really don’t matter cause if this dont work the end result is not good. I write this to say that this so far has been a much better experience then I thought so please don’t listen to above article as they are two different things. My prayers are with all and I share this to keep hope alive.

    • Charlie says:

      ‘This is taken from umbilical cord not bone marrow hip. Two completely different procedures.’
      Are you saying that the two harvest sites will result in significantly differing outcomes?
      I had thought that a stem cell was a stem cell, was a stem cell.

  6. Charlie says:

    Here’s an example of how quoted company prices can increase dramatically on thin evidence.
    Whilst we can all wish this company ‘science-speed’ with its research, we can understand why a company shouts loudly on paper-thin results. We have apparently wonderful results based on only THREE patients’ response. Share price up 77%. It is clear who benefits from the razzmatazz – shareholders, and this ‘breakthrough’ relates only to there being no adverse effects. What about efficacy?
    “Cellect Biotechnology Ltd. shares APOP, +76.97% rocketed 79% in premarket trade Thursday, after the Israeli company said it has made a breakthrough in a stem cell trial. The company said it has successfully completed transplantation of stem cells in the first group of three patients using its ApoGraft technology in a mid-stage trial. After a month, all three patients had demonstrated full acceptance of the transplant with no adverse events or reactions. The company is planning to recruit another three patients once the data has been reviewed. “The company believes that these interim results of ApoGraft present the first signs of a breakthrough in stem cell transplantation,” it said in a statement. “The product is transplantable within less than 12 hours from donation through a simple process performed on the bedside after selective physiological elimination of immune reaction-causing cells.” Shares have gained 117% in the last 12 months, while the S&P 500 SPX, +0.25% has gained 19%.”

  7. Charlie says:

    Mayo Clinic in Rochester just beginning a Phase 2 trial of stem cells intrathecally. 60 patients, results published in 2020 after finishing work December 2019.

    Horrifyingly long wait time for us. Urgency is a given lots of lip-service, but nothing, repeat nothing, speeds up phase 1/2/3 trialling.

    • syd says:

      sorry, i obviously jumped info gap from january to june 2018. i’m in contact with some patients in phase III. it is obviosly who is placebo, who is treated, ’cause results are clinicaly significant after 1st transplant. dont care what is written above, but results and price share tell a lot. it jumped from less than 3usd/share to 4,5/share. ms and autism in praeclinical status are marketing tricks, but nurown obviously isnt.

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