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[Littlesister]

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Graham

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Graham. Thank you for your reply. It makes absolute sense, to me, to have as much information as possible although I hadn't thought about it in terms of unknowns and prognosis. I will add it to my list of requests and discussions with my brothers consultant.
Again thank you for your time.
Little Sister

[Graham]

It is true that there are a few interesting trials going on in the NHS currently and knowledge about MND is approaching a tipping point, another year or five or more...

The DNA test is the gene test and the whole genome test will pick up all bad genes, including currently unknown MND types.

It also informs of prognosis and care needs, eg C9ORF72 may lead to dementia.

[Littlesister]

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Graham

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Gordan1111

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Ellie

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Ellie I hope this reply reaches you all. I was looking for a reply button to each of you, but it doesn't seem to work like that.
So firstly, thank you all so much for taking the time and making the effort to reply. It means alot to me.

Graham

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Graham your reply
You definitely need to figure out what MND bro has, because there are trials going on now that bro may be able to enrol for. A whole genome test is the best.
I have fathomed out about the various types of MND and that certain trials target certain types, but I don't know what my brother has and I don't think he does either. It's on the list as a question for the consultant. My brother said he was having some kind of DNA test I think but I don't think it was a "whole genome test". Can you tell me what the reason for needing it would be? I can understand if you want to find out if you're likely to get a disease, but for someone with MND what could the results be used for?
As for 15+ years - astonishing! And good to hear.

Gordan1111

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Gordan1111 your reply
I agree any final decision about taking or testing a drug or treatment should be yours. The illegal I'm guessing could be cannabis which you read about for medical use. That's another line of research.

Ellie

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Ellie your reply
Thank you for that insight. It's really helpful to have cons as well as any pros. I can't believe how hard it is to find information. I have written to both MND Association and the MND Scotland Association and suggested they compile a fact sheet for people like me considering the option to seek services overseas or to import medicines.

Can I share with you some information that I saw last night on my local news channel? I live in the south west so I receive BBC Points West. There was an interview with a doctor called Steven Gill a neurosurgeon at Southmead Hospital in Bristol. It relates to a drug called GDNF and the rights to the drug have been purchased by Parkinsons UK. The doctor is going to run a trial next year for people with MND using this drug. There was footage of a man who has Parkinsons and had been on a trial of it. The trial was stopped as researchers said it didn't stop tremors, but as time has gone on some people in the trial seem to have improved and there is a thought that maybe the GDNF is more of a long term drug. I have tried to contact the doctor today for more information (his secretary is on holiday so I have left a message) and I have also emailed Parkinsons UK asking for more information. I have tried Googling it and there is information about the old trial but nothing about the new. Again I'm not trying to give false hope but I do believe that sharing information is the way forward.

Kind regards
Little Sister

[matthew55]

I know hope is all we have but realism must be considered at some point. 😉☹️😘😍x

[Ellie]

Littlesister

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Littlesister A warm welcome to the forum.

I know of two people who used Edaravone - one bought it from India without a prescription, one bought it in Europe, with a prescription. Both had personal contacts administer the meds through an IV. Unfortunately neither saw any benefits, both eventually stopped, however, neither were in the early stages of progression.

I wish your brother the very best.

Love Ellie.

[Gordan1111]

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Littlesister

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Littlesister I have sympathy from where you're from. It feels too often as if we're left to scrabble in the dark desperate for any treatment from snake-oil merchants. I personally feel if there's they nothing they can do then let it be a real Wild-West. Let us have dispensation to be able to access any drug, legal or not.

[Graham]

Hi Little Sis,

My fingers tire and so I am brief.

Well done supporting big bro.

Only currently authorised drug is Riluzole and it is rubbish.

You definitely need to figure out what MND bro has, because there are trials going on now that bro may be able to enrol for. A whole genome test is the best.

I've not tried any drugs but I have survived 15+ years. That is why it is essential to know what MND bro has.

Our MNDA won't / can't comment and we are left to our own devices for experimental drugs but if any of them worked, the companies would be sure to let us know.

[Littlesister]

Hello All.
I'm using the name Little Sister as my brother is newly diagnosed and he hasn't yet told our extended family. I hope you don't mind me joining in as your comments already ring a bell with me. The standard response from doctors seems to be there's very little we can do but make you comfortable whereas the evidence from forums such as this says otherwise and while subjective I think should hold greater sway as you are the experts.

I'm looking for some first hand information from anyone who has travelled abroad to try the treatment called Edaravone/Radicava made by Mitsubishi Tanabe Pharma GmbH. Have any of you tried it? It's licensed in Canada and the USA and I think Japan. I understand it's not a cure, but I wonder if anyone on here has first hand experience of it. Additionally has anyone managed to get support from a consultant to use it as a treatment in the UK and imported it? My brother has authorised his consultant to discuss it with me and I think I have already upset the consultant by just asking such questions.

I fully understand how upsetting it could be to go off on a wild goose chase, but as someone has commented there needs to be a more centralised way of sharing this sort of information. I have discovered many leads to information and discussions about trials, supplements, self medication, overseas work etc but it seems to me that people with MND and their families are left to make their own enquiries for anything that is "unproven" or from overseas.

Thank you
Little Sister

[bilvers]

The American website https://healingals.org is very active in positive work for reversals. There is a Neurologist, Richard Bedlack who has recorded 49 which he says are genuine. He has not been involved in actual reversals, but he is doing research into supplements that have been involved in the reversals.

[SallyAnnB]

As a postscript to this discussion, I asked my consultant about TruNiagen & Pterostilbene, which he confessed he hadn't heard of. I sent him some of the information posted here and he thought it was a reasonable concept. However, he warned against taking them at the same time as co-enzyme Q10. He said it's 'either/or' as they both work on oxidative mitochondrial metabolism (whatever that is!).

[redbeak]

Apologies for my delay in responding!

The report of the Valencia trials that I accessed reads as follows:

To explore EH301 as a potential treatment for ALS, first author José de la Rubia, of the Catholic University San Vicente Mártir in Valencia, and colleagues conducted a placebo-controlled, randomized trial between February and June 2017. Upon recruitment, Estrela or de la Rubia assigned 15 participants to receive 1200mg of EH301 daily, and 17 to receive placebo. In other words, the trial was blinded with the exception of its two directors.

The trial continued for four months, with clinical evaluations at baseline, two, and four months. The modified ALS functional rating scale (ALSFRS-R) served as the primary endpoint. Secondary outcomes included measures of pulmonary function, muscle strength, and skeletal muscle weight and activity.

Twelve of the 32 randomized participants dropped out of the trial. Five withdrew before starting treatment, and the other seven—four in the placebo and three in the treatment group—discontinued between their two- and four-month appointments. Of the four dropouts from the placebo group, two were related to trauma, one to gastroenteritis, and one to bronchitis. In the treatment group, one dropout was related to trauma, one to hepatitis, and one to depression. The researchers presented findings only from the 20 participants who finished the trial.

The groups were not exactly matched at baseline. Compared with completers on placebo, those on treatment had lower ALSFRS-R scores, weaker muscles, lower activity in some muscles, and had had symptoms for a shorter duration of time.

These baseline differences raised the possibility that the treatment group might have had, on average, a slightly more aggressive form of the disease.

Seven of the 10 completers on EH301 improved significantly on the ALSFRS-R. The group posted an average improvement of 3.4 points by two months, and 2.5 points by four months. Conversely, nine of the 10 completers in the placebo group declined, by an average of 3.0 and 5.5 points at two and four months, respectively.

The EH301 group also improved on secondary measures, according to the paper. Nine participants gained muscle strength, as measured by the Medical Research Council grading scale index, while six gained pulmonary function, as gauged by forced vital capacity. Using surface electromyograms, the researchers found that participants on EH301 had a boost in activity in several muscle groups. They also gained an average of 0.5 kg of skeletal muscle weight, while the placebo group lost an average of 1.3 kg.

As expected, everyone in the placebo group declined on every clinical endpoint throughout the trial, with the exception of one participant who improved by one point on the ALSFRS-R. Conversely, the entire EH301 group showed improvement on at least one endpoint, with only one participant improving on less than two endpoints.

Adverse events were reported by four participants in the placebo group and five in the treatment group. They included mild diarrhea, moderate stomachache, and mild headache. They were short-lived and deemed unrelated to treatment.

Following the blinded portion of the trial, participants were offered an open-label extension to continue EH301. All 20 participants joined, but the researchers only reported one-year data from the 10 participants who had initially been randomized to the treatment group. By one year, they still had not declined relative to baseline on the ALSFRS-R, nor in muscle strength or activity in six out of eight muscle groups tested. The authors note that people with ALS typically decline by one point per month on the ALSFRS-R. That said, forced vital capacity did weaken by 11.5 percent relative to baseline, a dip that was still less than what the placebo group lost during the first four months of the trial

There have apparently been trials in Israel and more recently by the University of Aahrus in Denmark who looked at effect of it in the ageing process! I haven't seen the report but the impression that I got was that it was positive.

Of course the trials are limited and apparently Elysium is in a Commercial legal battle in the US at the moment!

I started feeling benefit after around a week although with MND there are always good and bad days!!

Thanks Julian that is really helpful

[Julian50]

Apologies for my delay in responding!

The report of the Valencia trials that I accessed reads as follows:

To explore EH301 as a potential treatment for ALS, first author José de la Rubia, of the Catholic University San Vicente Mártir in Valencia, and colleagues conducted a placebo-controlled, randomized trial between February and June 2017. Upon recruitment, Estrela or de la Rubia assigned 15 participants to receive 1200mg of EH301 daily, and 17 to receive placebo. In other words, the trial was blinded with the exception of its two directors.

The trial continued for four months, with clinical evaluations at baseline, two, and four months. The modified ALS functional rating scale (ALSFRS-R) served as the primary endpoint. Secondary outcomes included measures of pulmonary function, muscle strength, and skeletal muscle weight and activity.

Twelve of the 32 randomized participants dropped out of the trial. Five withdrew before starting treatment, and the other seven—four in the placebo and three in the treatment group—discontinued between their two- and four-month appointments. Of the four dropouts from the placebo group, two were related to trauma, one to gastroenteritis, and one to bronchitis. In the treatment group, one dropout was related to trauma, one to hepatitis, and one to depression. The researchers presented findings only from the 20 participants who finished the trial.

The groups were not exactly matched at baseline. Compared with completers on placebo, those on treatment had lower ALSFRS-R scores, weaker muscles, lower activity in some muscles, and had had symptoms for a shorter duration of time.

These baseline differences raised the possibility that the treatment group might have had, on average, a slightly more aggressive form of the disease.

Seven of the 10 completers on EH301 improved significantly on the ALSFRS-R. The group posted an average improvement of 3.4 points by two months, and 2.5 points by four months. Conversely, nine of the 10 completers in the placebo group declined, by an average of 3.0 and 5.5 points at two and four months, respectively.

The EH301 group also improved on secondary measures, according to the paper. Nine participants gained muscle strength, as measured by the Medical Research Council grading scale index, while six gained pulmonary function, as gauged by forced vital capacity. Using surface electromyograms, the researchers found that participants on EH301 had a boost in activity in several muscle groups. They also gained an average of 0.5 kg of skeletal muscle weight, while the placebo group lost an average of 1.3 kg.

As expected, everyone in the placebo group declined on every clinical endpoint throughout the trial, with the exception of one participant who improved by one point on the ALSFRS-R. Conversely, the entire EH301 group showed improvement on at least one endpoint, with only one participant improving on less than two endpoints.

Adverse events were reported by four participants in the placebo group and five in the treatment group. They included mild diarrhea, moderate stomachache, and mild headache. They were short-lived and deemed unrelated to treatment.

Following the blinded portion of the trial, participants were offered an open-label extension to continue EH301. All 20 participants joined, but the researchers only reported one-year data from the 10 participants who had initially been randomized to the treatment group. By one year, they still had not declined relative to baseline on the ALSFRS-R, nor in muscle strength or activity in six out of eight muscle groups tested. The authors note that people with ALS typically decline by one point per month on the ALSFRS-R. That said, forced vital capacity did weaken by 11.5 percent relative to baseline, a dip that was still less than what the placebo group lost during the first four months of the trial

There have apparently been trials in Israel and more recently by the University of Aahrus in Denmark who looked at effect of it in the ageing process! I haven't seen the report but the impression that I got was that it was positive.

Of course the trials are limited and apparently Elysium is in a Commercial legal battle in the US at the moment!

I started feeling benefit after around a week although with MND there are always good and bad days!!

[redbeak]

redbeak

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redbeak If it's just the Valencia trial details you want, they are on that clinical trials site too under the identifier NCT03489200.

There is a trial summary on general release and you could try and request a full copy from the author. It's important to note that it was a tiny sample - 32 participants, out of which 12 dropped out - and the trial was short at only 4 months.

Summary:
Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study.
https://doi.org/10.1080/21678421.2018.1536152

Background: Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved to treat ALS remains unclear. Novel disease-modifying therapeutics that are able to modulate the disease course are desperately needed. Our objective was to evaluate the efficacy and tolerability of Elysium Health’s candidate drug EH301 in people with ALS (PALS). Methods: This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study.

Thirty-two PALS were recruited thanks to the collaboration of the Spanish Foundation for ALS Research (FUNDELA). Study participants were randomized to receive either EH301 or placebo and underwent evaluation for 4 months. Differences between EH301 and placebo-treated participants were evaluated based on standard clinical endpoints, including the revised ALS functional rating scale (ALSFRS-R), forced vital capacity (FVC), and the Medical Research Council (MRC) grading scale. Results: Compared to placebo, participants treated with EH301 demonstrated significant improvements in the ALSFRS-R score, pulmonary function, muscular strength, and in skeletal muscle/fat weight ratio. EH301 was shown to significantly slow the progression of ALS relative to placebo, and even showed improvements in several key outcome measures compared with baseline. Conclusions: This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.

Thanks Ellie, I will have a look. Noted on the trial's limitations, hopefully the Bergen trial will provide some further back up in the next year or two.

Personally, the initial results along with Julian's anecdotal evidence, the relative low cost/easy availability and the lack of current opportunity to participate in MND Smart and other trials means I will give it a try and hope for some benefit. Will feedback here.

[Ellie]

redbeak

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redbeak If it's just the Valencia trial details you want, they are on that clinical trials site too under the identifier NCT03489200.

There is a trial summary on general release and you could try and request a full copy from the author. It's important to note that it was a tiny sample - 32 participants, out of which 12 dropped out - and the trial was short at only 4 months.

Summary:
Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study.
https://doi.org/10.1080/21678421.2018.1536152

Background: Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved to treat ALS remains unclear. Novel disease-modifying therapeutics that are able to modulate the disease course are desperately needed. Our objective was to evaluate the efficacy and tolerability of Elysium Health’s candidate drug EH301 in people with ALS (PALS). Methods: This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study.

Thirty-two PALS were recruited thanks to the collaboration of the Spanish Foundation for ALS Research (FUNDELA). Study participants were randomized to receive either EH301 or placebo and underwent evaluation for 4 months. Differences between EH301 and placebo-treated participants were evaluated based on standard clinical endpoints, including the revised ALS functional rating scale (ALSFRS-R), forced vital capacity (FVC), and the Medical Research Council (MRC) grading scale. Results: Compared to placebo, participants treated with EH301 demonstrated significant improvements in the ALSFRS-R score, pulmonary function, muscular strength, and in skeletal muscle/fat weight ratio. EH301 was shown to significantly slow the progression of ALS relative to placebo, and even showed improvements in several key outcome measures compared with baseline. Conclusions: This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.

[redbeak]

SallyAnn, I just bought the Nicotinamide in the form of TruNiagen over the counter from Superdrug and Pterosilbene on line and currently take 1200mg of Nicotinamide with 50/100 mg of Pterosilbene which, you will see from the Elysium website, are the two core constituents! The dose is as close as I can get to the trial quantity. But all this is done without any advice and this is where the Association could come in!
I am happy to post the Valencia trial report if you would find it helpful. I also understand that there is an ongoing trial at the University of Bergen in Norway.

Hi Julian - could you post the Valencia trial report, that would be really good as I have a medically knowledgeable friend who will look at it for me (beyond my comprehension!)

Found the Bergen Trial - details here:

The NO-ALS Study: A Trial of Nicotinamide/Pterostilbene Supplement in ALS. - Full Text View - ClinicalTrials.gov