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Thanks, guys. If I also find something I’ll post it here

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All of them. I maintain an exhaustive list of potential ALS meds on weekly basis.

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I was just informed yesterday that Phase III Trial of AMX0035 is expected to start within a month in Europe (not certain, but expected).

I was asked by my doctor would I consider participating. However, I don’t know what would be the best decision –> if I choose to participate I can’t start with TUDCA now and there’s a chance of getting a placebo during the trial. So in that case a full year without any meaningful therapy.

Alternatively, I could ignore the trial and just take TUDCA on my own, perhaps with ibudilast (ketas) if I can obtain it via SocialMedwork. I’m in an early stage of ALS. Does anyone have a suggestion what to do?

As for the 2nd component of AMX0035 (SPB), although it is available in NL I can’t get a prescription from my doctor for it (as it is not proven for ALS). So without prescription is both unavailable and too expensive for me.

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It seems that Nuticost brand is not available in EU. I have access to Amazon.com (US site) and could buy it there but I’m afraid that it’s not allowed to get them imported into The Netherlands (EU). They may be withheld by customs..

I did find some alternative TUDCA brands though but none of them seems too legit (Brawn nutrition, Hepatica / DG Supplements, ALLMAX Nutrition).

I could on the other hand join the TUDCA clinical trial via TRICALS, but only in 3 months from now as I’ve just started with Riluzole..

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I would like to take Nutricost TUDCA and Theracurmin HP as in the trials, but having difficulties purchasing them in Europe. Does anyone know of any alternatives that are available in EU / Netherlands? Or ways to purchase and ship these from US in bulk? Where do you get your supplements from?

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TUDCA is a molecule that helps to protect motor neurons by camouflaging a stress chemical released within the motor neuron that starts a process that results in cell death.

TUDCA may act by:
-Increasing glucose-induced insulin release via the cAMP/PKA pathway, increases insulin sensitivity
-Relieving endoplasmic reticulum (ER) stress. The ER makes sure proteins are folded properly
-Reducing programmed cell death (apoptosis) in healthy cells. TUDCA prevents the molecule BAX from reaching the mitochondria. BAX causes mitochondria to release cytochrome C, which causes enzymes (caspases) to initiate apoptosis
-Inactivating Bcl-2-associated death promoter (BAD), a molecule involved in apoptosis
-Removing toxic bile acids from the liver and preventing them from damaging liver cells

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From what I read the use of iPSC lines is mainly discussed in terms of drug discovery and not so much in the context of clinical trials (e.g. for disease heterogeneity consideration purposes or for deriving scientifically justified eligibility criteria). Also, iPSC-based drug discovery / repurposing seems to be a growing trend. Some examples that I have found are:

• Ropinirole, Retigabine, Bosutinib: New ALS Drug Candidate Developed Using iPSCs
• CRISPR/Cas9 screen in human iPSC-derived cortical neurons identifies NEK6 as a novel disease modifier of C9orf72 poly(PR) toxicity
• Effect of complement C3 inhibition with pegcetacoplan in an iPSC-derived ALS neuromuscular junction model of neuroinflammation

Also, there are some interesting results from clinicaltrials.gov: iPSC | ALS

From my point of view, your proposal of using iPSC lines to identify subpopulations for whom a drug may work could make sense. I just don’t know how willing would the trial sponsors be to do this up front as opposed to using this approach to better understand the drug efficacy results in a post-hoc analysis, thereby motivating potential follow-up trials.

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Thanks, I am the sole creator

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Hi Katie. Thanks for sharing the info. Great that you’re able to participate the trial. I hope that you’ll be able to experience a greater positive effect soon.

I was just wondering —  given that Pridopidine works by binding and activating the sigma-1 receptor, do you perhaps know if this drug would also work for PALS with SIGMAR1 gene mutation?

 

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Indeed, I might’ve acted a bit naively there. Still young though 🙂 I’ve also joined Patients Like Me sometime ago, trying to find answers wherever I can. None of them really helped so far, but in the future, who knows.

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Hi Lisa. I’ve just joined ‘stuffthatworks’ on your suggestion. I also think that it’s a good website that we could all benefit from. So I share your recommendation for everybody to join in.