Can we give CuATSM a try without waiting for the Australian trial completion (5 years from now)

[Farooqji]

Collaborative Medicinal Development LLC ("CMD"), a privately-held biopharmaceutical company developing innovative therapies for neurodegenerative diseases, In a presentation, Dr. Craig Rosenfeld, CMD's CEO, reported the results of 24 weeks of treatment with CuATSM at the recommended phase 2 dose. Patients with idiopathic PD showed a marked improvement in disease severity evidenced by a decrease in total UPDRS score of 7 points over 24 weeks. Patients also showed a marked improvement in quality of life evidenced by a decrease in PDQ-39 score of 15 points over 24 weeks; a 5 point change in PDQ-39 is considered the threshold for a clinically meaningful change. In the natural history of PD, both UPDRS and PDQ-39 scores increase (worsen) in correlated fashion over time.

biospace.com/article/releas...

CuATSM is a small synthetic molecule that contains copper.

Synonyms

copper-ATSM

CuII(atsm)

The metallo-protein Cu/Zn-superoxide dismutase (SOD1) is a ubiquitous enzyme responsible for scavenging superoxide radicals. Mutations in SOD1, which alter its metal binding capacity and can result in protein misfolding and aggregation, have been linked to familial amyotrophic lateral sclerosis (ALS). Cu-ATSM is an orally bioavailable, blood-brain barrier permeable complex that has traditionally been used in cellular imaging experiments to selectively label hypoxic tissue via its susceptibility to reduction by oxygen-depleted mitochondria.

It is available for sale from here

caymanchem.com/product/17122

The starting dose was 12 mg/day, which has been shown to be well tolerated in an ongoing phase 1 pharmacokinetic and dose-finding study of Cu(II)ATSM in patients with ALS (ClinicalTrials.gov identifier NCT02870634).

clinicaltrials.gov/ct2/show...

[Farooqji]

19 Patients were enrolled in three dose cohorts at CuATSM doses of 12, 36 and 72 mg/day. Treat-

ment was well tolerated, with most (13/19) patients completing six 28-day cycles of treatment. Over 24

weeks, dose-related changes in efficacy parameters were observed. At the RD of 72 mg/day, patients

showed improvement in disease severity by UPDRS (mean decrease of 7 points) and improvement in QoL

by PDQ-39 (mean decrease of 15 points).

[WinnieThePoo]

Always good to have promising new therapies being trialled. Short phase 2 non-placebo controlled trial is one of dozens - most of which have turned out to be a placebo effect. It needs more testing, and so its not available for sale to the public. But one day, one of these therapies will be effective, and this might be the one

[Dehlia]

Did I read correctly, that this drug is FDA approved?

[park_bear in reply to Dehlia]

No. It has to pass phase III testing to receive FDA approval.

[SilentEchoes in reply to park_bear]

CuASTM is already FDA approved as a PET imaging agent. It is also in phase 2/3 trials for ALS.

[SilentEchoes in reply to Dehlia]

Cu-ASTM is FDA approved, being repurposed for NDD.

[smv123 in reply to SilentEchoes]

What is NND and FDA approved so we can get it to use???

[park_bear]

Kinda pricey to buy at the link - >$100/dose.

[Coling]

I was on a three month trial in Melbourne, it was not double blind, mainly for safety. I did not take up the offer of another 3 months. Although I did not notice any difference in my symptoms I am keen to try a 12 month trial for efficacy.

[Farooqji in reply to Coling]

What dose were you taking? And why you didn't opt for continuing the trial?

[Coling]

Sorry, I am afraid that because it was Feb 2018, I did not keep a record of drug strenght only that it was important to take it 3 times a day. I quit after 3 months because I developed shingles which was not considered as a side effect. If I can remember or find out my dosage I will let you know

[smv123]

If this seems to work, I ran across something the other day that said that people were signing a petition asking for emergency use for CuATSUM to help with ALS. Couldn't Parkinson's people do the same thing to try to be able to use this???

[smv123]

Here is that article on the petition.change.org/p/allow-people-w...

[Farooqji in reply to smv123]

I think Exenetide is more potent in the current "On horizon" therapies for Parkinson's. PD community should launch a campaign for expediting the phase 3 trial

[smv123 in reply to Farooqji]

How do we do that????