CuATSM ?

[Dis1960]

First the tabloid take

https://www.3aw.com.au/melbourne-medical-breakthrough-finds-new-drug-slows-progression-of-mnd/

https://www.dailymail.co.uk/news/article-6579727/Motor-Neurone-Disease-breakthrough-Remarkable-new-drug-slow-disease-70-cent.html

This link suggests CuATSM was discussed at the Glasgow symposium:

https://www.scimex.org/newsfeed/motor-neurone-disease-breakthrough-patient-trial-shows-impressive-clinical-results

[Montana Tony]

Sounds Promising

Sounds promising but it’s early days ,but “ pick me” email on the way to Melbourne just in case

[Kayleigh]

Many thanks for sharing that very interesting information with us Douglas.

I agree with Montana Tony, it does sound very promising.

Hopefully, there is hope for all of us with this drug, in the very near future. It's about time we had a viable alternative to Riluzole on the NHS! - and maybe this drug will be the one?

Best wishes,
Kayleigh x

[Jer788]

Hi folks, I’ve read a few articles regarding this drug and it appears they are looking for trial it’s at any stage of the disease. Apparently you need a referral from an Aussie practitioner/neurologist to be considered. Are MNDA involved ? Can they assist ? Please post if anyone hears anything positive from the UK authorities as I’ve yet to get any positive feedback from them.
Best Jerry x

[Doug Carpenter]

Thanks Dis for the links.

Yes, very interesting results. I visited one of the research groups in Sydney last July and it was clear that many bright people were doing amazing things.

There’s a brief summary of the trial here:

https://alsnewstoday.com/2019/01/10/cuatsm-may-slow-als-progression-improve-cognition-respiration-phase-1-trial/

And more technical details, including selection criteria, here:

https://clinicaltrials.gov/ct2/show/NCT02870634

There are interviews with Prof Dominic Rowe and Prof Peter Crouch on an Australian radio station here:

https://www.2gb.com/hope-for-those-living-with-motor-neurone-disease-breakthrough-drug-could-slow-progression/

Apparently, CuATSM was originally used to deliver copper to hypoxic cells for imaging studies of cancer tissue. It’s taken around fifteen years to get this far, i.e. the first stages of human trials.

I’m going to attempt to attach a pdf of the Glasgow conference abstract to this post. But this may exceed my Forum technical skills!

Doug

[Doug Carpenter]

CuATSM abstract jpeg.jpg

Not sure if this will work.

I tried to upload and attach the abstract as a .pdf but was told that is an invalid file. So I'm trying it as a .jpeg

[Ellie]

MND Research Blog on CuATSM:

https://mndresearch.blog/2019/01/15/whats-the-story-with-cuatsm/

[Jer788]

Thanks Ellie for getting the nuts and bolts of this treatment. These trials are very frustrating really a case of “ while Nero fiddled Rome burned “. I hope we’re all still about to get this or alternative treatment.
Jerry x

[Fru]

Hi Ellie,
I too am so glad you posted that.
Love Fru

[Admin_MND]

Good afternoon all,

Thank you to Ellie for sharing the research teams recent blog regarding CuATSM.. We also wanted to give a brief overview to all users of the forum.

Recent press coverage of results of the Phase 1 trial of Copper ATSM (CuATSM) have reported that the compound produced a 70% reduction in rate of progression and an improvement patients who took the drug at 72mg/day.

As an Association we recognise this trial as an important first step in the drug development process. However, we must remain realistic when interpreting and reporting data from this small, Phase 1 study. Phase 1 studies are designed to determine what dose of a new drug is safe for people to take. It is too early to confidently report on the effectiveness of the drug although passing this safety milestone is an important development.

CuATSM is not yet at a stage where a clinician can prescribe it as a treatment. Drug development is a long journey, where any drug has to pass important rigorous checks before approval as a medicine. Historically, many promising drugs have failed at this first (Phase 1) stage.
We are hopeful that CuATSM will prove to be an effective therapy in the future. The next and important stage – Phase 2 - is to perform a robust trial on a larger number of people to really see if it can alter the disease progression. This randomised, blinded, placebo-controlled trial, is planned for later this year in Australia. We will keep the MND community updated on any news from this study.

It is positive to see such a clear example of how the money raised by charity supporters can effectively drive research to the point of a 1 clinical Phase trial. All involved should be congratulated on this achievement.

[Jaxx]

What have sufferers got to lose ?

[Kayleigh]

Good point Jaxx. For many of us, our lives are slipping through our fingers like sand.

[Dis1960]

It is positive to see such a clear example of how the money raised by charity supporters can effectively drive research to the point of a 1 clinical Phase trial. All involved should be congratulated on this achievement.

Sadly it shows Australia can be congratulated but what part did MNDA play ? Nothing apparently

It’s a worldwide disease so shouldn’t there be more international cooperation

[Onein300]

Whenever we get a phase 1 reporting there is always a call to make drug available now.

But the harsh reality is it is not proven yet.

We have to wait for phase 2.

It can’t be just made available. It’s a derived compound, and vastly expensive to make.

Who would pay for the vast distribution?

Why should we divert funds from other ventures etc?

Such calls can actually delay the ultimate cure. That is the harm it can do.

And it may be a complete false lead.

All the trial has shown is it is safe for 24 weeks. The efficacy data is totally meaningless because there was no placebo arm.

But it is an interesting drug.

The Mnda are contributing to many major projects. Worldwide collaboration is happening. But we don’t need duplication.

I want a therapy as much as anyone with mnd, but sadly we have to be rational and quite focused.

[Kayleigh]

Many thanks for your interesting post Lee, and for putting foward the rational and scientific side of things.

I appreciate what you have said about needing to stay focused on the harsh reality of situation.

However, it is not always easy to keep a rational view of things when a diagnosis of MND can put such a strain on us emotionally.

I find that even if my head is saying 'stay rational' about things, my heart is often breaking because of the devastation felt by not having an effective treatment or cure.

I suppose that even if the drug is cleared for use in Australia, it will still have to go through further testing and approval for it to become available here on the NHS. These things take time - but feelings of desperation often arise, as time is not particularly on our side.

I know that if my perspective on some things becomes 'too emotional', then the more scientific and rational folk here will soon ground me with the realities of the situation - and that is good - otherwise sometimes I could be 'clutching at straws' a bit to tightly, and pinning my hopes on things that realistically might not happen in my lifetime.

We are all here, sadly, because our lives are affected by MND - but it is good that we all have different viewpoints and knowledge to offer - and I do love a good debate now and then!

Take care everyone (especially if you are going out in the cold and frosty weather this week).

Sending love to you all,
Kayleigh x