First Trial of Potential ALS Gene Therapy VM202 Shows Treatment’s Safety

First Trial of Potential ALS Gene Therapy VM202 Shows Treatment’s Safety

The investigational gene therapy VM202, developed by VM BioPharma and intended for the treatment of amyotrophic lateral sclerosis (ALS), has been shown to be safe and well-tolerated in a small Phase 1/2 clinical trial, according to data published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.

While the study, sponsored by Korean biotech company ViroMed and its U.S. division VM BioPharma, was not designed to assess the therapy’s effectiveness, researchers said they observed promising results.

“The results of this study demonstrated that multiple intramuscular injections of VM202 were safely administered and well tolerated by patients with ALS,” Jack Kessler, MD, professor of neurology at Northwestern University‘s Feinberg School of Medicine and an investigator of the study, said in a press release.

Meanwhile, ViroMed was recently granted a South Korean patent for the use of hepatocyte growth factor (HGF) protein — the mechanism by which VM202 exerts its action — for the treatment of ALS.

The report, “Open Label Study to Assess the Safety of VM202 in Subjects with Amyotrophic Lateral Sclerosis,” showed that 17 of the 18 participants completed the study (NCT02039401), with none experiencing serious adverse events related to the drug.

Mild or moderate injection site reactions were common, with 26 reactions in 12 participants. Three patients had severe adverse events during the study that were unrelated to the treatment. One patient also died of breathing difficulties caused by ALS progression.

“While the size and design of this study do not allow us to make conclusions about treatment effects, some stabilization of function was observed during the first three months following treatment with VM202, and these encouraging and rare study findings in the previous ALS studies lay the groundwork for future studies to determine the potential of VM202 to alter the long-term course of ALS,” Kessler said.

“The results from this trial will help inform our future research efforts surrounding the potential clinical effectiveness of VM202 in the treatment of ALS,” added Sunyoung Kim, chief scientific officer at ViroMed.

VM202 is a gene therapy that acts by delivering a gene that produces the HGF protein. This factor is a trigger of blood vessel formation and nerve growth. In ALS, researchers inject the therapy into selected muscles with the intent of regenerating both muscles and damaged motor neurons.

In animal models, researchers have observed neuroprotective and regenerative effects of the gene therapy. In ALS patients, reports state that HGF acts directly on motor neuron cells or modulates the activity of nearby neurons, which researchers also believe contribute to the therapeutic effect.

VM BioPharma hopes that the approach can halt ALS disease progression.

The FDA has granted VM202 orphan drug designation and fast track status for the treatment of ALS. In February, the agency also approved an investigational new drug application for a Phase 2 study of the approach.

VM202 is also in development for diabetic neuropathy, critical limb ischemia, non-healing ischemic foot ulcer in diabetic patients, and coronary artery disease.


  1. Steven says:

    So what does this mean a therapy for ALS patient’s that’s absolutely going to work or is this another wait and see new research?

    • Magdalena Kegel says:

      Hi Steven,
      At this point it is not possible to say that this is absolutely going to work or not. The clinical trials program will have to answer these questions.

        • Magdalena Kegel says:


          The treatment is only now tested in clinical trials, so there is no information available about the cost if it becomes approved.

          • Nedham says:

            I have a patient with this disease (ALS) for five years. Is it possible to come to treatment in this way and with whom to communicate if possible?

          • Magdalena Kegel says:

            The study described in the article is not recruiting more participants, but the company is planning further trials. Our newsletter frequently includes information about available trials, as the information becomes available to us. You can also search the trials registry for suitable studies.

          • Nedham says:

            Although I know that this method of treatment is under clinical trails and not approved but is it possible to treat it under clinical trails

  2. Charlie says:

    “…some stabilization of function was observed during the first three months following treatment with VM202, ”
    “VM BioPharma hopes that the approach can halt ALS disease progression.”

    Very interesting.
    It seems very clear that this treatment is a possible ‘stop order’ for ALS thus trapping the pALS at the stage they have reached during decline. Those with decline at he advanced stage would, at best, be held within their torture. Again, we see a possible treatment that would be of most benefit to those newly diagnosed.
    Reversal seems not to be on this agenda.

  3. Charlie says:

    “In animal models, researchers have observed neuroprotective and regenerative effects of the gene therapy.”
    So in animals there is regeneration, but this is not observed in humans at Phase 1/2 Clinical Trial.
    Researchers might usefully devote a little more time to see why animals can regenerate neurons with this treatment, but humans cannot.

  4. Charlie says:

    “…some stabilization of function was observed during the first three months following treatment…’
    So we can conclude that, after three months, stability is lost and the decline continues?
    I think we should be told.

    • Magdalena Kegel says:

      Hi Charlie,
      I understand your frustration with the slow pace of research and treatment development. But at this point, it is premature to claim either success or failure with this approach.

      Phase 1/2 trials are primarily designed to study the safety of a treatment and investigate if the treatment does what it is intended, on a mechanistic level. It is not possible to conclude that stability is lost or remains as the study did not analyze such data.
      It is also at this stage impossible to determine if the treatment triggers regeneration also in humans. While this is easy to establish in mice by studying neurons and muscles under a microscope, the process is a bit more complicated in humans. Again, the study was not designed to study if regeneration takes place.

      • Charlie says:

        Magdalena, one person’s ‘frustration’ is another person’s death.

        Wouldn’t you agree that as research is ‘scatter-gun’ it clearly indicates that researchers have no clue what the cause is or what treatments can hit back effectively at this horrendous, barbaric disease.

        As research is so clearly scatter-gun, all parties are doubtless relying on a purely serendipitous trip over an effective medical treatment.

        A bit like Fleming’s carelessness in tidying up the lab and then finding penicillin had killed the bugs.

        Luck appears our only likely friend and no researcher is getting very lucky. Riluzole and Radicava ? Aspirin for brain tumours.

  5. Willy says:

    I have had 11 family members die from this terrible disease. We are a family with the familial type. My father died about 18 years ago from this, and now my older brother has it and is at end stages, and another cousin in the same situation. Would be wonderful to find something to halt this disease or a cure, I am now 52 years old with a wife and two kids hoping I do not carry the gene.Is there no way to run clinical trials faster, over these last 18 years there seems to be nothing new to slow this disease down.Sorry for the venting, but would hope we could do better as a society to better achieve a brighter outcome. Worried in Michigan.

  6. Maria V Bonino says:

    I’m satisfied after your comments, I know it is difficult to know at this time how medication works. Are necessary more trials.I have son 46 years old suffering this illness, I am asking for help.
    Thanks, Maria V. Bonino

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