Anti-Inflammation Treatment Masitinib Protects Against ALS Damage, Researchers Say

Anti-Inflammation Treatment Masitinib Protects Against ALS Damage, Researchers Say

AB Science SA‘s anti-inflammation treatment masitinib protects muscles and nerves against damage from amyotrophic lateral sclerosis (ALS), researchers have concluded.

Professor Luis Barbeito of the Institut Pasteur in Montevideo, Uruguay, presented the findings at the 27th International Symposium on ALS/MND (Motor Neuron Disease) in Dublin, Ireland, in December of 2016.

Masitinib is an oral treatment that inhibits proteins known as tyrosine kinases that play important roles in cells. Preventing the proteins’ activation helps fight cancer, inflammatory diseases and disorders of the central nervous system (CNS).

Researchers have learned that masitinib reduces inflammation in immune-system cells such as mast cells and microglia that can promote the development of ALS.

“Beyond our recently published findings, we have acquired additional preclinical data showing neuroprotective effects of masitinib in ALS,” Barbeito said in a news release. “We have now shown that masitinib generates its observed neuroprotective effect in ALS by regulating neuroinflammation in the peripheral nervous system [the part of the nervous system that regulates muscles] as well as the central nervous system and that it also penetrates the blood-brain barrier to a greater extent than previously thought. Overall, these data provide further compelling pharmacological rationale for the recently reported positive phase 3 interim analysis.”

A previous study of rats carrying a genetic mutation that leads to ALS showed that masitinib inhibited microglia and prolonged the lives of animals that developed the paralysis associated with the disease.

Meanwhile, interim data from a Phase 2/3 trial (NCT02588677) showed that masitinib and the ALS treatment riluzole improved patients’ quality of life and ability to survive. Researchers used the Amyotrophic Lateral Sclerosis functional rating scale, which measures progression of the disability, quality of life and survival, to assess patients who had taken 48 weeks of treatment.

Masitinib was granted Orphan Drug status in 2016 by the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products.


  1. Deanne says:

    So when do ALS patients get access to this drug????? Our loved ones are becoming paralyzed and dying while we continually read about treatments that are effective and yet we have no access to. The height of cruelty.

    • Tim Bossie says:

      This treatment is in phase 2 and 3 trials now and not available to the general public. We do not know at what point we may see the approved product.

    • A fellow PALS says:

      My suggestion is to bombard the FDA with calls from every friend, relative, and colleague you can think of. Email the FDA contact info. to everyone you know and ask them to call the FDA…weekly if needed. I met with my Congressman this morning and I urge you to do the same. If our collective voice gets loud enough, surely they will hear us and hopefully it will lead to action! The 21st Century Cures Act has provisions to allow the FDA to move things to market much more quickly than in the past. PLEASE CALL YOUR CONGRESSMAN!!

  2. Hossein Oshrieh says:

    Wouldn’t it make sense to allow this drug be made available to any ALS patient who wants it. The risk will remain with patient while the pharma and medical community would benefit from large scale results. It’s a win win for all sides.

  3. John says:

    Sorry for my frankness but you certainly do not have many answers . If patients agree to try at own risk how can there be lawsuits? There is so much momentum building for als treatment but it’s like wind that isn’t allowed to touch our sails. .cant exceptions be made sometimes? Especially that this drug is already being used in cancer treatment. What do als patients have to lose? If you could be in our or caretakers shoes for just one day…just one day…you would know.

    • Tim Bossie says:

      I wish we made the rules, but we don’t. There is a lot of momentum right now and we hope that it continues so that many people will be able to benefit, and possibly one day soon be cured of this awful disease.

    • A fellow PALS says:

      If it is already in use for cancer treatments in another country, let’s find out what country and import it. If it is the US, then drugs here can be used for off-label treatments of other conditions. If someone knows where this drug is available, please post here and I will research a way for us to get it. Does anyone know how it is administered…IV, oral, injection, intrathecally, etc.?

  4. Kim Persaud says:

    It’s seems all you can do is tell us what is happening with research. What is the point of posting your research, which means nothing to Als patients. It’s like given false hope. It will be nice if you can get one treatment available. Then it will show that you are actually working for Als patients.

    • Tim Bossie says:

      We are a digital publishing website that publishes the latest research and news surrounding ALS. We do not create, nor do we sell, any type of treatment. We only publish news.

      • Gavin Gibson says:

        Let’s get enough names and signatures together on a formal petition to go to governments in all our countries asking for legislation that will allow patients with diagnosed ALS (MND in Australia) to voluntarily agree to use this drug now! Will ALS News today be prepared to coordinate such an undertaking? I’ll sign it!!!

        • Tim Bossie says:

          Hi Gavin… we can certainly understand your passion for this. ALS is such a terrible illness that wrecks families and leaves brokenness in its wake. Your idea is not out of the realm of possibilities, but would have to be citizen led and be coordinated by someone (an organization) much larger and more influential than ALSNews.

          • Loriann Grimes says:

            I have ALS and have survived 6 years so far but my symptoms have started to speed up. We have to be given a choice we do not have the choice if we are going to die. So I do not understand the law suit issue?

  5. Anne says:

    Is there the possibility of use in “Right to Try” states? Sorry for my ignorance but by father was only recently diagnosed so we are just learning as we go.

  6. Indira manyam says:

    Hi All,
    My husband is suffering with MND, we tried all the options but there is no improvement and decease is progressive.
    We are literally waiting for medicine, but still it is in R&D stage for getting FDA approval.
    we are ready to provide any written letter to use this medicine.
    Can somebody advice us to get this medicine my mail ID is

  7. Donna Stephens says:

    My husband was diagnosed with ALS in January and since then I have been researching, it feels endlessly. I came across Masitnib earlier and found that it has been used in veterinary medicine for years. Look up Masivet. Thank you Tim for your effort and information.

    Masitinib is a tyrosine-kinase inhibitor used in the treatment of mast cell tumors in animals, specifically dogs. Since its introduction in November 2008 it has been distributed under the commercial name Masivet. Wikipedia

    • M. Gauthier says:

      Massitinib /massivet is a drug Given to dog since 2008 as anti inflammatory
      Why FDA or Sante Canada don’t put that medecine on the market for peolple whose got ALS
      why why ? It’s important …
      it’S a question of Life or dead

  8. John says:

    I’m thinking about to use Masivet or Kinavet(Masitinib), it’s a vet drug, although the same substance. To long to wait. For the record these news have been reposting for years, although it’s hard to say about efficacy even from news.

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