Stem Cell Transplantation Shows Potential as ALS Therapy, Study Suggests

Stem Cell Transplantation Shows Potential as ALS Therapy, Study Suggests

Stem cell transplantation in patients with amyotrophic lateral sclerosis (ALS) has the potential to be “an important alternative strategy” in treating the disease, a new study suggests.

In recent decades, many advances in identifying and understanding the underlying mechanisms of ALS have been made. But those advances have not resulted in more or better therapies available to stop the disease commonly referred to as Lou Gehrig’s disease.

The study, titled “The use of mesenchymal stem cells (MSCs) for amyotrophic lateral sclerosis (ALS) therapy – a perspective on cell biological mechanisms” and published in Reviews in the Neurosciences, provides an overview of the therapeutic potential of stem cell transplantation as therapy for ALS. It was authored by Bor Luen Tang, associate professor in the Yong Loo Lin School of Medicine, National University Health System, Singapore.

Several potential therapeutic agents and disease modifiers have been tested in preclinical studies using animal models, and some have reached human trials. However, many fail to meet safety or efficacy requirements, with no real breakthroughs on the horizon.

In the past 20 years, only two disease-modifying drugs have been approved by the U.S. Food and Drug Administration (FDA), Sanofi’s Rilutek (riluzole) and MT Pharma America’s Radicava (edaravone).

Stem cell transplantation represents a new therapeutic approach. By transplanting multipotent mesenchymal stromal cells or mesenchymal stem cells (MSCs), it would be possible to replace degenerating or dysfunctional neurons. This approach could provide a way to overcome the main hallmarks of ALS, potentially improving patients’ symptoms and stopping disease progression.

MSCs can be isolated and expanded from several neonatal and adult sources, including umbilical cord blood, bone marrow, adipose tissue, and dental tissue. Importantly, these cells can be genetically manipulated to enhance their ALS-targeted therapeutic activities.

“MSCs could be an easily obtained and manipulated autologous source platform upon which combinatorial therapeutics could be built,” Tang wrote.

Stem cells have a great capacity to produce several signaling molecules that can modulate their surrounding environment. If stem cells could be manipulated to send anti-inflammatory and pro-survival signals, they could potentially prevent neuron degeneration and death seen in ALS.

“While replacement therapy remained unrealistic, the clinical efficacy of this therapeutic option could be potentially enhanced if we could better decipher the mechanisms underlying some of the beneficial effects of transplanted cells, and work toward augmenting or combining these in a strategic manner,” Tang wrote.

So far, preclinical studies with animal models of ALS have demonstrated the beneficial effects of MSCs transplantation, which improved motor function and prolonged the animals’ lifespan.

More recently, Phase 1 clinical trials have demonstrated the safety of several MSC delivery routes, providing support for further developments into stem cell treatment.

“The recent indication (albeit modest and limited) of procedural safety within typical disease duration and the moderate beneficial effect of MSC transplantation into patients with ALS should provide sufficient impetus for larger Phase 2b-3 trials,” the researcher wrote.

Preliminary results of Phase 2 trials suggest that that stem cell transplantation can have therapeutic potential for ALS. Although the results were not striking, some improvements on patients’ respiratory capacity and reduction of disease progression rates, compared to control groups, were reported.

“It is clear from the discouraging results of drug trials and other recent advances that merely targeting the intrinsic survivability of motor neurons would be ineffective,” Tang wrote. “Therefore, exploring the ability of MSCs (or engineered MSCs) to work on modifying the disease environment would be an important alternative strategy.”

26 comments

  1. My husband is 40 yrs old n suffering with ALS since 5 yrs….he cannot walk, or rather there is no function of his hands n legs….he can’t speak…he can eat n by God’s grace no breathing issue so far. So do u think stem cell can help him… he is too young doc n I m desperate to help him out…plus help

  2. Charlie says:

    ‘….modest and limited…. procedural safety…’
    ‘…the results were not striking,…’
    ‘the moderate beneficial effect of MSC transplantation..’

    Clearly this is of very limited effect and Tang should set his sights a bit higher and perhaps aim for something more substantive.

    We must all be very careful of stem cell claims that are not tested in Western Europe or North America. The lure of the banknote is stronger than the lure of the cure.

  3. Maximo says:

    Charlie, Singapore is one of the most advanced countries in the world. With life expectancy well above USA. Your unfunded mistrust, shared by many, doesnt help us find a solution to our problem.
    We all have to work TOGETHER to beat this thing.
    Im 40 years old, with a 3 year old child. Now i can barely walk, or hold a fork. I know what to expect.

    Regards from Argentina.

    • Charlie says:

      Maximo, I trust and hope that you can travel to Singapore and get effective stem cell treatment. In North America there is no stem cell treatment that has approval from the healthcare system. The reason for that, my ALS consultant tells me, is that it has not proved effective. To my knowledge, no Singaporean clinic offers any guarantees on anything involving stem cell treatment, excepting of course, a much slimmer bank account. My ‘unfunded mistrust’ as you put it, has had no , and never will have, any, practical impact on the success or failure of stem-cell treatment trials. I call it as I see it.

    • Avi says:

      El 23 de junio ALS News Today publicó un artículo sobre las investigaciones de BrainStorm en la Universidad de Tel Aviv. Y en el hospital Hadasa de Jerusalén está trabajando en el mismo tema el profesor griego Dimitrios Karussis: “Stem cell therapy trial for ALS and MS patients ….”, The Jerusalem Post, Paul Alster, 08/05/2016.

  4. kal says:

    From what I have seen youtube you can get stability. Face it there is nothing else in the pipeline. There may be nay-sayers out there trying to discourage stem cells because they would rather keep the drug pipeline open to keep big pharma getting rich by using patients. Remember this is essentially a turf war. This is why God says the cure is in the backyard but you people rather make it an industry.

    • Charlie says:

      Kal,
      I doubt very much that God has any interest in ALS, because if he did it would have been beaten a long time ago.
      I think we could make a good case in court that Satan has quite a lot of interest in this disease.

      • Janet says:

        Amen. Do something research! Talk does nothing! There is a cure out there and it takes risk to find it! God has not left us hopeless!

        • Cynthia Dixon says:

          From your statement, you obviously aren’t a Christian. To say that God does not have any interest in ALS is cruel and completely untrue. If you truly believe in God, you have faith.
          Some of the greatest blessings in this world come from God’s power in the midst of trials. God sometimes changes us, molds us, strengthens us, and builds us through hardship. But it’s in his time. And, yes, Satan is alive and well, as we can see by many things (and people) on this planet.

    • Charlie says:

      This penicillin/hydro cortisone treatment must surely get some research time, based on these findings. Big Pharma will have a crazy pink fit if something comes of it because these two drugs are pretty inexpensive.The longevity of the improvements would be looked at very closely. I’d guess that a patient cannot keep taking these two drugs forever, but what do I know…..

  5. Hallo Leidgenossen und Leidgenossinnen,
    ich wurde von Dr. Jason Williams in Bogota operiert. Die Stammzellen bekam ich Anfang November 2013. ALS wurde bei mir im November 2011 festgestellt.
    Ich bin stark gehbehindert. Das war ich aber auch schon nach meiner Knieoperation im Februar 2007.
    In 2013 vor der OP in Bogota war ich kaum noch zu verstehen.
    Jetzt kann ich mich sprachlich gut verständlich machen, obwohl mein Sprachbild sehr schlecht ist. Kopf arbeitet noch
    einwandfrei.
    Die Beweglichkeit der Hände wird langsam schlechter.
    Ich denke, dass die OP eine Menge Zeit bisher gebracht hat.
    Jeder Zeit würde ich mich noch einmal operieren lassen.
    Viele Grüsse an Alle aus dem schönen Osnabrück in Germany.

    • Alice Melão says:

      Stem cell therapy has the potential to benefit all of those with disorders affecting motor neurons. Still, the clinical effects of this treatment strategy are not fully recognized. More studies are necessary.

    • don ng says:

      i do not think that singapore has any stemcell therapy m living in singapore how come i have not heard about anything about stem cell tks sir

    • don ng says:

      can anyone here confirm to me that in sngapore has stemcell treatment i m living in singapore and has not heard anything about stemcell pls avise and confirm tks

  6. Charlie says:

    Lots of research projects have ‘potential.’ All of them perhaps.
    One small problem… once trials start, they fail in efficacy. nothing -NOTHING- is proving effective when examined closely.

  7. Cynthia Dixon says:

    I feel this article is very negative and misleading. On this exact site in December 2016, Brainstorm was listed with the following information on stem cell trials. If you signup for their newsletters, they are very informative.

    Brainstorm-
    “This Phase 2 trial demonstrated clinical meaningful improvements in disease symptoms as measured by the well-established ALSFRS-R scale,” said Berry, unit chief of the Massachusetts General Hospital ALS Multidisciplinary Clinic, in a press release. “Importantly, there is evidence that NurOwn may be halting disease progression or improving symptoms in some patients. The CSF biomarker profiles were also encouraging,” Berry said. “The significant increases in neurotrophic factors and decrease in inflammatory markers observed in the treated group post-transplant provide a biological mechanism supporting the observed clinical effect,” he added.

  8. Charlie says:

    ..”no stem cell therapy has received Health Canada or U.S. Food and Drug Administration approval for ALS treatment at this time. Patients who are researching their options may come across companies with Web sites or materials that say otherwise and offer fee-based stem cell treatments for curing this disease. Many of these claims are not supported by sound scientific evidence ..’

  9. Cynthia Dixon says:

    Correct, no one has received approval for stem cell theraphy in the USA from FDA. And, I would never promote trying any drug that isn’t FDA approved. But to say NOTHING is proving efficacy isn’t true. Brainstorm is a legitimate company that’s referenced by the ALS Association – U.S. Food and Drug Administration Approves Start of BrainStorm Stem Cell Trial for People with ALS. Their clinical trials have shown clinical improvements. To indicate that that is “nothing” just because it hasn’t been tested yet in USA, is giving no credit to advancements. When you are a person with ALS you hang on to every bit of information. And I encourage people to be very careful where they get that information. These are facts of Brainstorm that are supported by the ALS Association. There are many promising drugs right around the corner, including tirasemtiv. Dr. Bedlack at Duke university has many videos of upcoming treatments that are very close. This site, in my opinion is for information. This is correct information that I’m stating. It’s more than promising.

  10. Mauro says:

    In Germany I found “ANOVA IRM STEM CELL” what makes “SECRETOME”
    very interesting !! Clinic and highly regarded doctors !! It is worth taking a look.

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