Masitinib Improves ALS Patients’ Functioning, Clinical Trial Results Show

Masitinib Improves ALS Patients’ Functioning, Clinical Trial Results Show

Masitinib met its main Phase 2 and 3 clinical trial goal of improving the functioning of amyotrophic lateral sclerosis (ALS) patients, according to its maker, AB Science SA.

The randomized, double-blind AB10015 (NCT02588677) trial compared the effectiveness and safety of a combination of masitinib and riluzole with a combination of a placebo and riluzole. Riluzole is marketed as Rilutek or Teglutik.

Masitinib was administered for 48 weeks. The 394 patients were randomized to get either a combination of 4.5 mg/kg/day of mastinib and riluzole, a combination of 3 mg/kg/day of mastinib and riluzole, or a combination of a placebo and riluzole.

The primary endpoint, or measuring stick, of the trial was the change in the patients’ revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) over the 48 weeks. The changes that the ALSFRS-R tracks over time correlate with patients’ quality of life and survival.

Progression free survival (PFS) was a secondary endpoint of the trial. Researchers defined it as a deterioration of more than nine points in a patient’s ALSFRS-R score, or the patient’s death.

Researchers did their analysis in two phases. In the first, they tested the effectiveness of 4.5 mg/kg/day of masitinib against a placebo. In the second, they compared the effectiveness of 3 mg/kg/day of masitinib with a placebo.

Patients who received 4.5 mg/kg/day of masitinib had a statistically significant change in their ALSFRS-R score by the end of the trial, compared with placebo-treated patients. That dose level also improved patients’ PFS and quality of life.

Researchers assessed quality of life by the change in patients’ Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ) score between the start and end of the trial. The self-report is a subjective patient measure of well-being.

Patients who received 3 mg/kg/day of masitinib also improved their ALSFRS, PFS, and quality of life scores, compared with placebo-treated patients, the trial results showed.

There were no new safety issues with the treatment or major adverse reactions.

The results will be presented at the European Network for the Cure of ALS (ENCALS) conference in Ljubljana, Slovenia, May 18 – 20.

“This is a very good news for the patients. These final data confirm findings from the study’s interim analysis and proves that masitinib is capable of slowing down motoneuron degenerative disease such as ALS, which is a devastating condition with an urgent unmet medical need,” AB Science’s CEO, Alain Moussy, said in a press release:

Professor Olivier Hermine, president of AB Science’s scientific committee, said that “perhaps the most impressive finding from this study is that masitinib has generated a significant difference in progression free survival with respect to the placebo treatment arm. Similar to cancer studies when PFS is significantly improved, this indicates a clear clinical benefit in favor of masitinib.”

Masitinib is a tyrosine kinase inhibitor. It targets mast cells and macrophages — which are important to immunity — by inhibiting a limited number of kinases. Masitinib is being developed to treat ALS and similar conditions, and as an inflammatory disease therapy.

In October 2016, AB Science announced that the European Union had accepted a conditional marketing authorization application for masitinib as a treatment for ALS.


  1. Janice Boone says:

    When and where can we get this. We have just been diagnosed with ALS for the 5th time and it is destroying my family.

    • Tim Bossie says:

      This drug is in clinical trial now and is not yet approved by FDA or available to the general public. Please contact your doctor about this and if your father is a candidate for the medication.

  2. April says:

    Hurry up USA !!! What is the hold up? Our family is dying with our sisters diagnoses of ALS along with many other families in this world!! I see a new drug on market for MS. It’s called ” Ocrevus “.
    Why can’t this be given to patients with ALS?? We are in medical crisis in this country with this disease. It shouldn’t happen! We have known about this horrific disease for many decades. What is going on in our medical facilities “Scripts” that receive hundreds of thousands of dollars every year to research the Brain and neurology of this disease? Please help us someone!

  3. Karen says:

    I want this drug where is it available it has now passed 3 phases of testing in Europe so it should be available for use.

  4. don ng says:

    understand that masitinib is also in the from of masivet masivet is actually for vet wonder is it safe if i take this masivet for masitini thanks si have no choice tks singapore do not have but i have a friend who is in portugal he can buy for me tks

  5. Robert Green says:

    I to was diagnosed 4 months ago. What the heck, I see my neorulgist at Barrows in the 6th of Sept. Hopefully I can get some straight talk

    • Karen says:

      I would like some straight answers too because I hear patients say it has helped and the ALS Society says it isn’t promising. Who is telling the truth?

    • Karen says:

      Check with als worldwide, the last time I talked to them, they did not have much hope in it but was promoting Radicava which from what I understand does not have much of a success rate, 7%. There is so much misinformation about these drugs who knows what to believe. I think we are at the mercy of big pharma and neurologist who don’t know anymore than they did 150 years ago. I wish they could sit in our bodies for one day.

      • Steven says:

        I agree it feels like I’m stuck in quick sand and no help coming to help me out of it. But it seems like I’m waiting to see if nurown or tirasemtiv can help me out this quick sand.

  6. cheryl l ferguson says:

    3/4 of a year later……………..nothing. ask God to heal you, thats the only way.. he said I am the way, the truth and the life, he so seeks me will never die

  7. Peter_from_Poland says:

    I’m assuming that if you are reading this you have read up on the
    research and trials with Masitinib. The drug is available as a prescription veterinary drug – Masivet, so if you find a doctor who is willing to experiment and a cooperative vet…. I mean what do we have to lose, right?

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