By Dr Peter Crouch, University of Melbourne
Clinical outcomes for copper-ATSM as a treatment for MND have very recently been reported in the media, with some of the reports describing exciting results for efficacy.
We all desperately hope for the day when any person diagnosed with MND can be prescribed a drug that will stop the disease in its tracks. Copper-ATSM may one day prove to be that drug, but we’re not there yet. Here, we provide a brief update on copper-ATSM.
The development of copper-ATSM as a treatment option for MND started around 15 years ago when it and related compounds were tested in animal models of neurodegenerative disease. Over the years, the laboratory-based evidences supportive of copper- ATSM being an effective treatment accumulated to the extent where clinical testing in MND patients became a legitimate possibility. It was given to MND patients as a therapeutic for the first time in 2016 when the phase 1 testing commenced. In December 2018, outcomes from the phase 1 trial were announced at the International ALS/MND Symposium in Glasgow, and in January 2019 the same information was circulated as a press release from the company supporting the clinical testing, Collaborative Medicinal Development.
First and foremost, the phase 1 trial was a success. The primary objective, as per any phase 1 trial, was to assess safety and tolerability of the drug and, if possible, identify a dose that could be taken to the next round of testing. By assessing the drug at a number of different doses the trial successfully identified a safe and recommended dose for phase 2 testing. Many potential drugs that show promise in the research laboratory ultimately fail to get through phase 1 testing, so for copper-ATSM to get past this important milestone is an excellent achievement.
In addition to confirming that the drug is safe, reporting on the phase 1 trial also included descriptions of efficacy, such as improved respiratory function and a slowing of disease progression as assessed by the ALSFRS-R scoring system. These are very exciting indications that copper-ATSM may prove to be an effective drug for MND. However, the number of patients included in phase 1 testing (for any drug) is kept small by necessity, and phase 1 trials are not designed to demonstrate efficacy. For example, all patients in the phase 1 trial knowingly receive the drug and there is no placebo control group for direct comparison. So, while the phase 1 results for copper- ATSM are very encouraging and are good cause for excitement, there is still a considerable amount of work to do before it can be confirmed that copper- ATSM is an effective treatment for MND.
It is therefore very welcome news to hear that planning for copper-ATSM to enter phase 2 testing is already underway. Phase 2 trials are specifically designed to assess efficacy. It is reported that the phase 2 testing will start in the second half of 2019 and that the trial will include 80 MND patients. Details of the phase 2 trial, including the participating clinics and the patient recruitment criteria, are yet to be announced.
Upon completion of the phase 2 testing we all hope that the promising indications from the phase 1 trial are confirmed as a reality.
Further discussion of the phase 1 trial for copper- ATSM can be found here: www.mndresearch.blog/2019/01/15/whats-the-story-with-cuatsm
Further information is on the MND Australia website at: https://www.mndaust.asn.au/News-and-media/MND-in-the-news.aspx