CuATSM Trial Participants

Dvholmes

New member
Joined
Mar 22, 2021
Messages
4
Reason
PALS
Diagnosis
07/2020
Country
US
State
MI
The CuATSM trial is fully enrolled and we can expect results later this year. Has anyone spoke with anyone in this trial? I have only found 2 individuals in the trial and they’ve both passed. They were not SOD1 carriers and I wonder if that had anything to do with it. I have not found any individuals claiming any benefit from CuATSM. I know there is a 1:1 drug to placebo in this trial but I would expect hearing some benefit from someone on the open label by now. It’s giving me concern about the effectiveness of CuATSM despite all the media hype.
 

KevinM

Distinguished member
Joined
Mar 30, 2019
Messages
306
Reason
PALS
Diagnosis
06/2019
Country
US
State
FL
City
Tallahassee
I have found that hype far outpaces actual results with most proposed therapies, even those that have made it this far. I am pessimistic about CUATSM, but that comes from only one anecdotal result.

I recall reading about an Australian barrister that was diagnosed in late 2016 and in 2017 started on CUATSM. Unfortunately the drug did not slow his progression, and he died three years later. In that case, we knew he was given the compound so there was no question about a placebo.

If you subscribe to the speculation that ALS is an umbrella description for a number of motor neuron diseases, then it is extremely unlikely that any one therapy will work for all of us. CUATSM may in fact show efficacy in a subgroup, but as we saw with NurOwn Ph. 3 results, that alone is no guarantee it will ever be approved by the FDA. Kevin
 

Samkl

Distinguished member
Joined
Nov 1, 2019
Messages
114
Reason
Loved one DX
Diagnosis
08/2019
Country
AU
State
NS
City
Wollongong
I read an interview of the barrister, who said he believed his progression had slowed whilst on cuatsm, and that he was grateful he’d had the chance to be on it, for the time that he was.
 

KevinM

Distinguished member
Joined
Mar 30, 2019
Messages
306
Reason
PALS
Diagnosis
06/2019
Country
US
State
FL
City
Tallahassee
That’s interesting, Samki. I did not see his interview. I wonder why the treatment was stopped?

Also, I know that even if he felt it did help, his overall progression was typical of many, so I believe—as with AMX35—that the results, if positive, will be modest. Trust me, I’m rooting with all my might that it dramatically slows progression, but I’ve become a bit cynical. K
 

Dvholmes

New member
Joined
Mar 22, 2021
Messages
4
Reason
PALS
Diagnosis
07/2020
Country
US
State
MI
Samkl, Do you know if he was SOD1? All the trials up till Phase II focused on SOD1. I believe (could be wrong) Phase-II intended to include those without SOD1. So maybe there is a SOD1 subgroup in this trial that it does work for and for others it does not. I did find 2 more that were on the trial but cannot get a response from either. I believe they may have passed. It’s possible the drug is still effective but the dosage was just set at a low level to show efficacy but no drastic change. I too hope this drug works. If that’s the case we’ll probably need a phase-III trial.
 

Samkl

Distinguished member
Joined
Nov 1, 2019
Messages
114
Reason
Loved one DX
Diagnosis
08/2019
Country
AU
State
NS
City
Wollongong
Kevin, I found the Sydney Morning Herald article on Peter Ginter. He was on a cuatsm trial in 2017. Below is a quote from it.

“The assaulting weakness slowed considerably after August, when he was given a slot on the Cu-ATSM trial, and slowed further still after February this year, when his dose was upped to 72 milligrams a day. Every morning he swallows six of these shiny blue pills before breakfast, the dosage soon to double, having received an all-clear in liver tests”.
 

KevinM

Distinguished member
Joined
Mar 30, 2019
Messages
306
Reason
PALS
Diagnosis
06/2019
Country
US
State
FL
City
Tallahassee
Thanks for pulling up the article, Samki. Though it wasn’t stated, I presume at some point (completion of the trial?) the treatment was stopped. The issue of open label extension for trial participants couldn’t be more pertinent if that was the case. Nothing would be harder mentally than seeing positive results, then being unable to continue treatment.
 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
10,180
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
OLE fortunately has become more of an expectation. I believe mgh will no longer participate in trials without them. I know for sure they advocate very strongly for that and also expanded access.

There was one trial that ultimately failed where a subset felt they strongly benefited and when the trial ended without ole they crashed - feeling that they progressed very quickly to where they would have been if never treated. NP001.
 
Top