CuATSM Therapy May Slow ALS Progression, Improving Cognition and Respiration in Patients, Phase 1 Trial Shows

CuATSM Therapy May Slow ALS Progression, Improving Cognition and Respiration in Patients, Phase 1 Trial Shows

CuATSM, one of the lead investigational therapies of Collaborative Medicinal Development (CMD), may slow disease progression and improve the respiratory and cognitive function of patients with amyotrophic lateral sclerosis (ALS), a Phase 1 trial shows.

The findings of the multicenter, open-label, dose-finding study (NCT02870634) were announced by the company’s CEO, Craig Rosenfeld, during a presentation at the 29th International Symposium on ALS/MND, held from Dec. 7 to 9, 2018, in Glasgow, Scotland.

CuATSM is a small artificial molecule that is able to deliver copper to cells containing damaged mitochondria, the cell compartments responsible for the production of energy. Damaged mitochondria are considered a hallmark of several neurodegenerative diseases, including ALS, Parkinson’s disease, Huntington’s disease, and Alzheimer’s disease.

Since CuATSM only delivers copper to damaged cells, leaving healthy cells unharmed, the idea is to use this molecule to target only unhealthy cells and reduce the damage caused by the disease.

In October 2016, CMD launched the first-in-man clinical trial for CuATSM in patients with sporadic and familial ALS to determine the best dose, and study its pharmacokinetic properties.

During the first two phases of the study, patients were divided into several groups to determine the best and safest dose for CuATSM (recommended Phase 2 dose). In the third phase, the goal was to confirm patients’ tolerability to treatment and evaluate its efficacy.

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According to a press release, data from the third phase of the study showed that disease progression slowed significantly in patients with sporadic ALS after 24 weeks of treatment with CuATSM at the recommended Phase 2 dose, as measured by the Revised ALS Functional Rating Scale (ALSFRS-R) score (-0.29 points/month vs the expected -1.02 points/month).

In addition, patients’ respiratory function assessed by forced vital capacity (FVC) and cognitive function by the Edinburgh Cognitive and Behavioral ALS Screen (ECAS) score also showed a significant improvement after 24 weeks of treatment with CuATSM (+1.1 % predicted/month vs -2.24 % predicted/month for FVC, and +10 points vs no changes for ECAS).

As expected, patients treated with lower doses of CuATSM experienced minor improvements in ALSFRS-R score and FVC compared with those treated with higher doses over the 24 weeks. This observation is in line with data from preclinical studies in mouse models of ALS, showing that CuATSM acted in a dose-dependent manner when administered to the animals.

CMD is now planning to launch a randomized, placebo-controlled clinical trial for CuATSM to confirm these results.

CuATSM treatment should not be confused with taking copper supplements, which can be toxic at even moderate doses, and do not help people with ALS, scientists say.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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    • Joana Carvalho says:

      Hi Bibi. More clinical trials are needed before we reach that point, I am afraid. Hopefully more news about these upcoming studies will be available soon.

      • Robert Benjamin says:

        Hey. I’ve been on Radicava since May 2018 with some improvement. Will this be used in conjunction with Radacava infusion?

        • Joana Carvalho says:

          Hi Robert,
          I did not find any information regarding the use of CuATSM as a combination therapy, so I am not sure if that will be an option later on.

    • Joana Carvalho says:

      Hi Seamus. Not yet, only that CMD is planning to go ahead and launch a new clinical trial to confirm these data. Hopefully it will happen soon.

  1. Terry says:

    I have a friend diagnosed with MND. How do I go about to see if he can participate in the trial or when will access to this drug be available on the market?

  2. Maryse Gauthier says:

    J’espère dans un avenir prochain que cette nouvelle recherche fera une différence significative pour tous ceux qui ont cette maladie dévastatrice la SLA et je remercie de tout cœur tous ceux qui comme mon mari ont participe a une recherche quelconque et ceux qui comme lui ont perdu la pire bataille de leur vie mais qui malgré tout ont garde espoir jusqu’à la fin dans la recherche.

  3. Paul says:

    Hi Joana, If the phase 1 trial had proven success why wouldn’t this be available under the “right to try” law or at a minimum, do some more trials in the US? For many diagnosed with ALS, another couple of years to study this may be too late. I’d be happy to take it now with just the hope that it could help.

    • Joana Carvalho says:

      Hi Paul. I understand where you are coming from, but unfortunately clinical trials are not that straightforward. Even for investigational drugs to be available under the “right to try” law there are is a number of criteria that need to be met before patients are allowed to have access to them:
      I am not saying there won’t be any future trials in the U.S. On the contrary, based on these promising Phase 1 results, I would say new trials should happen in the future. As for the when that will happen, it is still unclear at this point. I am sorry, I know that is not what you and everyone else here would like to hear, but that’s all we can say for now.

      • Eric says:

        The “Right to Try” law has been so watered down by Big Pharma and the FDA that it would more accurately be called the “Right to Beg” law…

  4. Janet Brisky says:

    I would like to be notify when stage 2 starts and if I could be in the trial. I was dx in 2018 with ALS and have foot drop, some balance problems and slight weakness in 1 finger. I have no problem with my breathing.

    Thank You

  5. Lauren says:

    I understand the trials are only recruiting in Australia for now. In your opinion would have any hope of joining the trial if I were able to relocate to Australia?

  6. Stephanie Snapp says:

    Has there been any updates on the trials in Australia? Now that President Trump has passed the right to try how can someone get find the CuASTM?

  7. Janet Brisky says:

    I was not accepted into the trial because you can not have ALS more than 24 months and I had it 28 months. This did upset me and still does. My question is if it does help will it only be allowed to patients with ALS 24 months or less? The only hope to keep us going are trials and if they have such stipulations like age or months had ALS it’s really something that gets me down . When I see, trials that find out people Newley diagnosed with ALS don’t sleep well, I can scream, what a waste.

  8. Ann says:

    Would this potentially help with SLONM? I was diagnosed and told this was a progressive neurological disease. Only treatment offered was IVIG, and this did not help so was stopped. Looking for any other possible intervention.

  9. Mark Raskin says:

    I’m at 21 months with ALS and need a drug like this now. Here in the U.S. it feels like cancer gets all the money, insurance does not want to understand ALS and we’re all stuck with Riluzole and Radicava, both doing very little at best. Because the majority of us now have a much shortened life span there certainly seems to be NO URGENCY to try and save us. Yea, I’m angry.

    • Billy Lee Roth says:

      You are so correct. The ALS Association needs to go on an all out long term media blitz until enough funds are procured through private and public funds. This disease needs to be defeated. We need much more help to see significant progress.

    • Bobbie says:

      What happened to the hundred or hundreds of millions of dollars from the bucket challenge. Where did that money go?

  10. Lee Cumbie says:

    Seems to me the real barrier is getting the manufacturer to agree to allow patients to utilize the drug not that the FDA is out of the loop.

    Right to Try removes government red tape so that you can work directly with your physician and a drug manufacturer to pursue an investigational treatment. However, the law has important eligibility criteria that you must meet in order to access a new treatment.

    An eligible patient is a patient who has:

    Been diagnosed with a life-threatening disease or condition
    Exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (this must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying)
    And has provided, or their legally authorized representative has provided written informed consent regarding the eligible investigational drug to the treating physician

    If you meet these criteria, you’ll need to ask your doctor to help you access a treatment. If the manufacturer agrees, the process is straightforward from there. Here’s how it works:

    Your physician contacts manufacturer of investigational treatment in FDA Phase 2 or 3 trial and asks for treatment under the Right to Try law.
    If the manufacturer agrees, your doctor and manufacturer will develop a treatment protocol for your specific situation and arrange for the treatment to be provided to the physician.
    Manufacturer determines if cost-recovery is necessary and provides you with the cost. Companies are allowed to charge patients for certain items, but there are strict guidelines in federal law that must be followed, and they cannot make a profit.
    You’ll sign an informed consent document that explains that you understand you are taking an investigational treatment with unknown side effects. Other contents of the document are determined by the laws in the state where your treatment will take place.
    Your physician may have additional notifications to make, but that will be something he or she already knows is required if it applies to them.
    Treatment begins at a time and place that works for you and your doctor.
    The manufacturer will notify FDA within a year that a treatment was made available and report outcome data.
    Here’s a letter to get your doctor started on a request.

    You can learn more about treatments that are in FDA Phase 2 or 3 trials at

    If you or your doctor have questions about the process and steps required, please contact Naomi Lopez-Bauman at (602) 462-5000.

  11. Raj says:

    I found this article today.. and looking at the clinicaltrials link saw this..

    Actual Study Start Date : November 16, 2016
    Estimated Primary Completion Date : December 30, 2019
    Estimated Study Completion Date : March 30, 2020

    Seems like they are coming close to something.. is that right??

    Would be nice to know what Phase this is.

    Any feedback is appreciated.

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