MSA with Ambroxol HCL - a tentative succ... - Cure Parkinson's

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MSA with Ambroxol HCL - a tentative success story!

Robb48 profile image

I'm a little hesitant to post this, as it is still VERY early days, but if there is any chance that what I am seeing is caused by ambroxol, and if it might be replicated by other people, then I think I have a moral duty to put it out and give people the option to test for themselves, or their loved ones.

So, MSA is a rare form of Parkinsonism, very nasty, with an average time to death of around 8 years - or less in the case of older individuals, such as my mother who is almost 70. Very hard to diagnose with certainty, confirmation is often only via autopsy. Which brings me to my first caveat - my mother has a number of atypical symptoms, although MSA can cause a spectrum of effects and the neuro specialists do believe it to be the most likely diagnoses. Still, it is perhaps a note of caution for others with MSA.

That said, I (her son) have been slowly ramping up the dose in accordance with the only official study for Parkinsons, right now she is only a few days into week 3 and on a dose of 180mg x3 per day, taken with food. And this is another reason why I'm so tentative, she's not been taking it that long, yet her condition was improving even after just a few days. I really didnt believe it could be ambroxol at first, because my understanding was that it would take months before benefits were seen. However, the improvements have been (mostly) steady and consistent, and I'm struggling to think of anything else that could cause it. I did previous ramp up her dose of ubiquinol (co-q10) to 900mg, and increase her high dose thiamine to 7g/day from 5g and later 6g/day, but that was weeks prior to starting ambroxol, and I doubt these could cause such a significant improvement.

OK so all those caveats out the way! Her symptoms. A month ago, I would often only get a dozen words or so out of her a day. It was looking extremely bleak. She was socially very disconnected from us, barely taking part in conversations, and her voice was a constant near whisper. She was unable to take part in activities, feeling a sort of sickness or unwellness almost constantly - she couldnt describe it, just said she felt "horrible" all the time, and it seemed to be related to her gut. She might read for a bit, but after a while it would make her feel sick. Even excluding those feelings, her drive was entirely gone. Reduced to watching TV most the day, except for the Herculean efforts of my father to make sure she was getting out for walks daily. Her balance was bad (lots of furniture walking), physically weak, needing help getting out of chairs, and her condition was rapidly deteriorating - faster than MSA should, we thought. For someone who was constantly doing things before, always sorting out the house, cooking, cleaning, finding activities, socially engaged, and maintaining an extremely active lifestyle for someone her age, this was heart breaking. All of these symptoms have decreased, she it now taking part in conversations, engaging with us, being self directed, genuinely SMILING without it being forced, her voice is louder, she is moving easier - not just on her daily walks, but getting out of chairs, cars etc. The differences are striking. However, she also had insomnia before, although intermittent. And that has not improved so far, if anything it is worst, though given how badly it fluctuated before we cannot say with any certainty it is related to ambroxol, and insomnia was not a noted side effect in the ambroxol / Parkinsons safety study. And she is still having occasional bad days in terms of feeling "terrible", but I would not say worst than before, it is rarer and it comes back mostly in relation to when she's had especially bad sleep for a few days. But even when she is feeling rough, the psychological differences are still there, if maybe a little diminished at times - she's still engaging, actively doing things (self-directed), her facial expressions are normalising, more eye contact, voice louder etc. It is hard to say how much better she is in % terms, comparing her now to prior to onset a couple of years ago, but perhaps up to 50%?! At least in terms of her character, psychology, social interactions etc - brain stuff relating to the dopamine systems I guess, her physical issues are improving more slowly. Which seems bizarre from a physiological point of view, since the dopamine (DaT?) scan of the brain showed significantly reduced dopamine uptake, which I presumed meant damaged. So, are they better now?! Were they just "gummed up" before, but the effects on her musculature/mitochondria are more difficult to improve?! We really understand so little about these conditions, but hey, results are results!

Aside, I also have the recently discussed fancy probiotic PS128 incoming from Singapore (both Active and Mind options, will test each), which I am very much looking forward to trying on her given how her symptoms started with and seem to be heavily related to the gut. I'll update on this thread, for good or bad, in coming weeks or months re ambroxol, and note if PS128 does anything.

So! Thats my story. Honestly just writing this is bringing me to tears, I cannot believe I am saying it but if progress continues, could it even lead to a "cure"?! If only partial..... with a condition that is so horrible, and previously so unstoppable... well.... Of course without proper clinical trials there is no way to know if this is truly the ambroxol, or if what we are seeing is in any way reproducible, and who knows -her condition could nose dive again tomorrow, or progress might simply stop..... but.... now there is reason for hope. My heart goes out to anyone else suffering from this brutal condition, or with relatives who are suffering. I wish you all the best of luck!

----

Addendum - 17 tumultuous days later! Soon after writing the above, I stopped my Mum on her regimen of other supplements, because we ran out and I wasnt sure if she needed them any more. This turned out to be a bad bad horrible terrible decision, and she tumbled back to her old state over a period of a few days. I then bought more and tried to re-start her on them, but there were issues with the dosing of thiamine that complicated things further. However.....she now seems to be back on track. So, a few additional thoughts:

- The repeatability of the improvements we have seen may depend on the other supplements she is taking in combination. They seem to work together, or affect different systems in complimentary ways. I now have her on just 2g/day of thiamine, and a strong b-complex (solgar 100) to assist after reading more reports from Dr Lonsdale etc. In addition to the 900mg/day of ubiquinol, reishi, lions mane, and now 200mg x 3/day of ambroxol. However, to re-iterate what I said above, I had her on these supplements prior to ambroxol, and we didnt see anything like these effects. So, well, they seem to work together, at least for her! Ambroxol dose, I kept her on the same while going through the above complications, not wanting to complicate further, just moved from 180 to 200mg yesterday, will look to increase properly again in the coming weeks.

- Previously when writing above, I had her on up to 7g/day thiamine, as from the Q&A with Dr Costantini he mentioned that HDT works also with MSA, but that higher doses are required (4-6g/day being his stated upper dose for Parkinsons). And while I only very gently took my Mum up to this dose, unfortunately that also meant I didnt realise it was causing other unpleasant side effects, and I now believe her insomnia was caused/worsened by too much thiamine. Although this is something she suffered from before I started her on HDT, there seems to be a correct dose that assists sleep, but above which it worsens a range of symptoms in unpredictable ways. Right now she's sleeping well, her condition is improving, she is become more sociable again, etc etc. So, fingers crossed, but looking good....!!!

Been a crazy couple of weeks, and I have only myself to blame - turns out you should never hard stop powerful medicines, and thiamine in these dose ranges really is medicine. But finally now starting to breathe a sigh of relief!

58 Replies

Awesome! Thanks for sharing! Keep us posted!

Quite amazing and encouraging. Hope the improvements will continue and we'll hear more good news!

Robb,

Your mom is very fortunate to have you as her advocate! These results must be very uplifting for both of you!

I hope your PS-128 test goes as well or better than your Ambroxol test! I look forward to your updates. These kind of results for MSA seem incredible! Thank you for this update!

Art

thanks for sharing. Hope the improvements continue. Keep us posted

Positive results, well done for trying to help your mother with this condition, please keep us updated and I wish her well with her recovery. Well done for trying other alternatives which seem to help many sufferers.

Wonderful very inspiring

Hi Robb48,

I understand perfectly when you say writing about this brings tears.

That was how I felt about PS 128- how a simple probiotic can bring about so much change.

1 Corinthians 1:27 KJV

[27] But God hath chosen the foolish things of the world to confound the wise; and God hath chosen the weak things of the world to confound the things which are mighty;

So happy for you that ambroxol is the light that shines so bright for you. Another member also shared about the great improvements experienced with ambroxol. Hope that PS 128 can bring about even more normal functions.

Your mother is so blessed to have a son who is so engaged in promoting her well being.

Wishing you continued improvements

Robb48 profile image
Robb48 in reply to Millbrook

Great quote, thanks! I'm so grateful for you and all the other people on this forum who are contributing, none of this would be possible without an engaged and driven community. I've always had a lot of faith in crowdsourced info, happy it is paying off! Myself, I cannot imagine doing anything other than throwing my all into trying to help her, there is no alternative.

Wow, that is wonderful and gives many hope. May I ask what brand of ambroxol you're using?

Robb48 profile image
Robb48 in reply to Marie1895

Sure, bought 1kg from Bionique Pharma in India (via Indiamart, cheapest supplier I could find, US$450 inc postage), putting tablets together on a weekly basis for her.

rebtar profile image
rebtar in reply to Robb48

I'm wondering if you researched the supplier and found them to be reputable and reliable as far as ingredients and contamination in their product?

Robb48 profile image
Robb48 in reply to rebtar

I did not, because I am not sure how I would go about doing so. They could provide certificated of purity, lab analysis, which I believe they shipped with the product, but how would I verify? No idea. Just had to go for it. If you can find better, please share!

Lovely to read your story Robb. I had a break with 600 mg Ambroxol to include magnesium and B12 into my drug regime. Soon as I started back on the Ambroxol two days ago, I’m more able to push myself up and out of bed. So yes positive for result there. Hope your mum will continue to improve. X

Robb48 profile image
Robb48 in reply to Trudy2020

Awesome, congrats! And really interesting you are seeing short term relief with ambroxol as well, rather than the months/years of slow burn I had expected.

Amazing. One of the best posts I ever read on this forum. Thank you so much for sharing this.

That is awesome!

Amazing to read and thanks for taking the time to make such a detailed post. I hope this upward swing continues for your mother and the whole family.

Hi, curious about how you came upon ambroxol as a therapy for MSA. I write often on this forum about symptom overlap in neurodegenerative disorders, which I have concluded are forms of brain injury. I first thought I had MSA because of the early onset of autonomic dysfunction, turns out I have an atypical form of ALS. I'm intrigued about the therapeutic potential of ambroxol for motor neuron disease. Thanks for sharing your testimony.

SE

frontiersin.org/articles/10...

Robb48 profile image
Robb48 in reply to SilentEchoes

Sure, I came across it here, remember reading a post from someone saying that it was the only thing that helped their partner (Parkinsons), so I researched more and decided it was worth a shot. The original study indicated that ambroxol may work by clearing out the misfolded alpha-synuclein, which is of course a common factor in different forms of Parkinsonisms, so it was reasonable to consider it might have an effect on MSA also. Even more so given this indicates it may be working on the cause, rather just reducing symptoms. Hugely speculative of course, the original study was more to assess safety, but the fact that they saw a real decrease in Parkinsons related symptoms - not just slowing the rate of decline - is still an excellent result. And, well, the results of inaction are well documented, so what is there to lose except money! I am not sure I agree that these conditions are a form of brain injury though, or at least not exclusively that, given the studies on gut connection, effects on mitochondria, and more recent studies exploring the link to SIRT systems. Too many unknowns though, too many interlocked systems spiralling into complexity. But research seems to be moving forward at a good pace now, and who knows ambroxol and PS128 seems to have incredible effects on some people, if followed up this should expand our understanding.

JayPwP profile image
JayPwP in reply to Robb48

DHPSR was our resident Ambroxol expert and promoter, but the account seems to have been closed

Millbrook profile image
Millbrook in reply to JayPwP

Sorry to hear that. She was the one I was referring to but I could not remember her name.

Have you heard about the Phenylbutyrate TUDCA study? It is done by two Brown grad students and Dr. Rudolph Tanze of Harvard. In human trial, it slowed motor decline by 25%. Phenylbutyrate was trialed in Colorado in approx 2012 for PD and it looked good. Likely didn’t continue because without it being combined with something it can not have IP rights. TUDCA is currently being trialed for PD. Anyways, you might want to look in to it?

Robb48 profile image
Robb48 in reply to matcharoe

Thanks, will check it out!

Thanks much Robb48 for taking the time out to write in such detail about Amboxol

I’ve been diagnosed with MSA and will definitely check it out.

What dosing frequency do you give your mum for the 180mg - is it split up or a single dose daily?

Please do keep us posted - you’ve been very kind sharing

Very best regards

JayPwP profile image
JayPwP in reply to binnyrox

jamanetwork.com/journals/ja...

The 186-day exposure period comprised 28 days of dose escalation, with each dose administered 3 times per day as follows: 60 mg (days 1-7), 120 mg (days 8-14), 180 mg (days 15-21), and 300 mg (days 22-28). This exposure period was followed by 158 days of administration of ambroxol at 1.26 g per day (420 mg 3 times per day). Patients were issued ambroxol therapy in 2 batches, 1 at baseline and 1 at 93 days.

binnyrox profile image
binnyrox in reply to JayPwP

Really appreciate the note It’s very helpful and I’ll try it out.

Thanks much

Robb48 profile image
Robb48 in reply to binnyrox

Oops sorry, oversight on my part not to be clear on that - I will edit the original post. She is on 180mg three times per day - with breakfast, lunch and dinner. So a total of 540mg. Decided with food was best due to it being easily absorbed, having a short half life and potentially causing stomach upset.

binnyrox profile image
binnyrox in reply to Robb48

Thanks much

binnyrox profile image
binnyrox in reply to Robb48

Hi again Robb48

Many thanks to you I’ve started on Mucosolvan - brand name for ambroxyl where I live

I happen to belong to the MSA Coalition group as well but find this group has been the most helpful

Just wondering if you would like to post there as well regarding ambroxol

Your sharing has made a world of difference for me - at least something to try when there doesn’t seem to be any thing else

Perhaps it might help someone else similarly over at the MSA group

Just a thought

Again immense thanks Robb48

Very best

BR

Robb48 profile image
Robb48 in reply to binnyrox

Hi, I wont share for now, sorry to say but in the last few days she's just crashed down again - but then I ran out of thiamine and some of the other things I was giving her before, and hoped they were unnecessary now, perhaps I was wrong. So we're just trying to get her back on an even keel again. Fingers crossed. Good luck!

binnyrox profile image
binnyrox in reply to Robb48

Thanks for the note

Robb48 profile image
Robb48 in reply to binnyrox

Hi, just to let you know, I've edited an addendum onto the original post, you may wish to read :). I think I can tentatively say back on track now, hopefully no more issues in the future!!

binnyrox profile image
binnyrox in reply to Robb48

Hi Robb48

Thank you so much for taking the time out for the note

I was thinking about your mom and as a fellow MSA patient - you may find some of the following information useful. Pardon the lengthy post but I am writing from personal experience

1. Stressors

With MSA, any stressor brings about a setback. If its small - eg rushing for an appointment, then I may take a few hours / half a day to recover. If its bigger, - eg over exercise at therapy - it may take a day or more. The threshold is rather small so my neurologist recommends doing all activities in what he calls snacks

So there are exercise snacks, speech therapy snacks etc and its done for about 10mins and I rest for an hour till the next session throughout the day. I have been told not to underestimate the value of physical exercise and it has helped very significantly with reducing disease progression

2. Physical Therapy

I am not sure if your mother is mobile. A very important exercise explained to me was a Sit to Stand. It helps keep the lower body strength. Also, having been bedbound due to aspiration pneumonia, I am slowly starting to walk again. I found sitting on a chair with ankle weights and marching on the spot helped with the cadence and the sense of planting weight on my feet. Stretching (including fine motor for the hands) is paramount to prevent irreversible contracture . For cardio, I use a recumbent bike HIIT to get more out of it as I can't exercise for long. If she is mobile then you may be curious about this done at Innsbruck Medical University, a leading centre for MSA research

multiplesystematrophy.org/w...

3. Dysphagia

One of the most problematic area for an MSA patient is swallowing. This can lead to pneumonia. Does she have a speech therapist? If she is still having foods orally, then you might like to view the link below.. From a videoflouroscopy done - I have been prescribed the Masako and Effortful Swallow The best practice for swallowing is simply to swallow the secretions that may collect in the mouth. If I am overwhelmed by it, I rinse and dry it out so that I can restart with less difficulty,

The chest specialist recommends rinsing the mouth many times a day to minimize bacteria pooling and going down the airway accidently. If anytime she sounds gurly, there is probably secretions sitting on the vocal cords that can accidently go down the airway so try clearing it often

southtees.nhs.uk/content/up....

4. Chest Infections

I was recently introduced to 'Cough Assist' and it was a game changer for me. It works much better than a suction machine and less invasive. It also strengthens the lungs in the process.

I use a spirometer and the following exercises have also helped

mskcc.org/cancer-care/patie...

5. Urinary Track Infections

I was having constant UTI until an SPC was done. I have not had any infections after

6. Nutrition

With dyphagia , I was dangerously undernourished and finally had a PEG done. I am now back at a healthy weight and take a combination of home blended food (3x) and nutritional feeds (2x) daily

7. Medications

I am on Madopar and Comtan. The Comtan makes the Madopar work longer and is very effective. For orthostatic hypotention, I take a combination of Midodrine and Florinef/Potassium

A tip you may find helpful - consistently, when my bp is low and its not time for the meds, I take a bolus of very cold water (250ml or more) and it brings my BP up 20 points for about 60-90mins

I am now on the Ambroxyl that your so kindly shared. Its my third week dosage and I feel its been helpful. Easier to do exercises and helps with the walking

Like your mom I'm on many supplements including the Red Helmut and PS128. I believe it's a confluence of many factors that I am seeing a remarkable improvement for a disease that has a very dismal prognosis

Again, many thanks for your posts Robb48, it has been very helpful and please do ping if you have any other queries. I'd be glad to share if I know

PS A patient on the MSA forum mentioned she put her mother on a Gluten free diet and it helped immensely. I have been doing that as well

Robb48 profile image
Robb48 in reply to binnyrox

Hi! Sorry for the delay in getting back to you, I appreciate your writing this all out for me though! Extremely kind of you, I'll have to save it for reference. And fantastic that you are seeing such improvements! Re ambroxol, my Mum is back to where she was now before our recent hiccup, which is SUCH a relief. But it is curious talking to her about it, she's not sure if she feels like she has improved, even though it is night and day for myself and my father. I wonder if this is something you have seen? Of course it's hard for her to compare how she feels, to how she felt a couple of months ago, but still. It reminds me of Dr Costantini actually, taking photos of peoples faces before and after thiamine injections to prove how different they looked. Anyway, quickly re the other points you mentioned, yes we have noticed my Mum coughing sometimes while eating, swallowing issues, but it's not so severe yet, thankfully. Still, I had not considered the pneumonia angle, definitely one to bear in mind. She is mobile, my Dad goes out on walks with her typically twice per day. Often short but it's something. With stressors, my Dad had a theory that if she went on walks that were too long, more than she could manage, that it set her back, but I'm not sure. One to consider though!

Another therapeutic angle I've been looking into again in the last few days is telomere based therapies. Listening to interviews with a Dr Michael Fossel, the whole telomere science is fascinating, but not one I've been up to date on for a while. Dr Fossel was the editor of a high ranked anti-aging journal, and the last interviews I can find with him are from a year ago, when he was developing a gene therapy to controllably switch on telomerase and use this (broad range technology) to target Alzheimer's specifically. He was hopeful that they could cure it, but only the data will say for sure. His most optimistic guess then was that they would move into human trials in 2 years, and mentioned that other neurodegenerative conditions would be good initial targets. So I'm hopeful this is something we may see on the horizon soon-ish.... I'd like to get my Mum on a product called TA-65, a telemoerase activator, it's not massively effective but it's all thats commercially available, unfortunately it's also monstrously expensive :). Still, I think I have a bit more kudos with my parents now so maybe I'll cajole them into trying it in a bit! Holding off on PS128 for now, we werent sure if it was making her feel sick and they're off on a cruise in a couple of days (!!!). Have you noticed anything from it? After they get back we'll try it again. I have other angles to consider long term, I think the sertuins approaches are promising, resveratrol perhaps, unfortunately another pricey one. Well, going to take it a bit more gently now! Ambroxol has given us breathing room :). Hope you are well!

binnyrox profile image
binnyrox in reply to Robb48

Good to hear from you, apologies for the late reply. Based on what you have mentioned, I am further along the disease than your mum

I thought about it and felt the most useful information I could share with you would be what I would have done differently if I could turn back the clock from when I was first diagnosed

1. Know that MSA can deteriorate very rapidly. For example, there was a period of 2 months when I was being treated with TCM -accupuncture and accupressure. Because of time constraints, I was passive and did not manage to exercise and continue with speech/swallowing practice. When I finally realized it, I had lost my ability to cough, hawk, do strong swallows. Functionally, I went downhill - warp speed - unsteady gait, hand tremors with tasks etc. I know if I had continued being active, the decline would have been very much slower

So practicing doing all important functional activities and exercises daily is very important.

2. You mentioned your mum coughs when eating. That's a first sign of dysphagia. The cough indicates some thing has gone down the wrong way. Her swallowing may have gotten weaker. I didn't realize it and continued eating and have ended up in the hospital for aspiration pneumonia twice. That is not desirable as I became deconditioned (again rapidly) in bed and it takes a long time to try to get back to where I was (and still trying). I would have seen a good speech therapist earlier. A videofloroscopy is usually done to determine the problem and appropriate exercises prescribed. Most MSA patients end up with chest infections so minimizing it is crucial

3. I pushed myself so hard trying to get better. And I'd be so tired out - couldn't do anything for the rest of the day. With MSA, its a little tricky, try doing things up to a level when its just right. My benchmark is that I should be able to rest for an hour and be ready to start another activity. If I can't do that - then I've overworked the system

4. Because MSA is neurodegenerative, , I find the concept of neuroplasticity very helpful and optimistic. I read Norman Doidge's The Brain that Changes Itself and The Brain's Way of Healing. You may be interested. It really keeps me from despair

5. I've also found what really works for me with regards to aspects of MSA medication, managing orthostatic hypotention, nutrition etc. If you do cross that bridge, just ping and I'd be glad to share what has worked for me. I won't bore you with that now

6. The MSA Coalition is peerless with regards to assistance and information. I have written to a few of the professors and doctors associated with the organization with regards to issues and they have reverted promptly and have been incredibly helpful multiplesystematrophy.org/

The world forefront chap would be Professor Gregor Wenning. There are experts in various areas,- check them out in the about us

Well that is all I can think of. You have been so kind with the Ambroxyl lead, I am now on 300mg thrice daily. Your mother is very lucky with you helping her out

Take care and best wishes for your mum

BR

Robb48 profile image
Robb48 in reply to binnyrox

Hi, apologies again for the late reply! And again, thanks for the info. Yes, we have seen periods where my Mums condition has rapidly deteriorated, which confused us, so it is useful to know this can happen. And also thanks for the other details, I wont reply in detail now but it is useful! Another one you, if you are interesting in cutting edge research. I was unsure for a long time what the bodies normal mechanism is for clearing the misfolded synculein, discovered the other day that there are two pathways for this, and one is autophagy. Autophagy appears to be a somewhat broad heading, but is useful, because it is recognised to be impaired in all neurological conditions, and can be targeted. I went looking for trials on spermidine, a compound found in many foods but sold in the form of wheatgerm concentrate, it boosts autophagy, although unfortunately it is another extremely expensive supplement and quite new to the market. However, I found one trial for dementia in which it actually improved the condition of test subjects - didnt just slow their rate of decline, it actually improved their dementia score. Which is highly interesting, although they "administered" it in the study by feeding the individuals bread rolls! Also trials on older adults at risk of Alzheimers, with subjective cognitive decline, etc, which all came up positive, and used actual extract pills I think. There are other trials with animal models on synuclein that look good, but very basic animal model (fruit flies and flat worms if I remember right). So this looks hopeful to me, as it is addressing what may be a key pathway in the disease condition. One to consider perhaps.

Hey Robb, glad that your mom is seeing improvements. I have ambroxol in the closet still waiting to try. I've been on ps128 for about 3months and at first the improvement did not show, however the last month , my symptoms (tremors, fatigue, anxiety, insomnia) have diminished significantly. I've also cut sugar and carbs off, I truly believe the combination is the cause of the improvements. I was reluctant at first to attribute ps128, it may be the mind or body adjusting to the PD , meds after experimenting for 3 yrs, but after 2 months of steady improvements I'm beginning to have hope that I can survive long enough to see my 3 and 8yo grow up. Keep fighting for your mom and good luck.

Robb48 profile image
Robb48 in reply to Fighttolive

Interesting! Most the other reports were from people seeing results for PS128 in hours to days, good to know benefits can still happen on a longer timescale. Good luck to you too! Keep fighting :).

"I'm a little hesitant to post this, as it is still VERY early days, but if there is any chance that what I am seeing is caused by ambroxol, and if it might be replicated by other people, then I think I have a moral duty to put it out and give people the option to test for themselves, or their loved ones."

I think you have no proof that ambroxol does anything positive, and no "moral duty" to suggest to people that it might (nor can you say it has no negative effects).

"However, the improvements have been (mostly) steady and consistent, and I'm struggling to think of anything else that could cause it."

No need to struggle any longer, it is most likely a placebo effect, which is strong in people with Parkinson's, and can actually work quite well, until it doesn't

But don't feel bad, this is what this site is mostly all about - people suggesting that they have seen an improvement, and attribute it to some supplement, or some apparatus, and the rest of the folks going and buying it, and then trying it on themselves...

Robb48 profile image
Robb48 in reply to Levod

Nope, not placebo. I'm not the one taking it, and I didnt expect these improvements. They are, as I said, quite striking and obvious. That said, I was quite explicit in saying multiple times that I had no "proof", just putting it out as it is. People can make up their own minds.

Levod profile image
Levod in reply to Robb48

I think you don't understand the placebo effect, for if you did you wouldn't insist there is no placebo effect in you grandmother's case...

Robb48 profile image
Robb48 in reply to Levod

I think you have not read my post, because the person in question in my mother. And again you make presumptions about me and my situation. I understand placebo very well, and recognise the scale of effect it can have, but in this case nobody expected effects, yet they have been strong and sustained. Make up your own mind, decide I am an n=1 case you cannot verify, fine, I have no problem with that, in fact I would encourage it, but to TELL me it is probably just placebo, and that I shouldnt feel bad - oh thank you, how magnanimous of you! - when you know next to nothing about the situation? You think I have not considered that, with something so serious? Please. I could go on, with specific metrics and behaviours, but I think there is no point. I will not continue this conversation.

Levod profile image
Levod in reply to Robb48

I did read your post and understood that the patient was your mother.

"in this case nobody expected effects"

So she took some medicine with no expectations or hope whatsoever of a positive effect? I am not sure how you could say this.

"I will not continue this conversation."

OK. Good luck and peace.

Robb48 profile image
Robb48 in reply to Levod

OK, I will answer the question. No, none of us expected any results - in the short term. I have thrown a LOT of stuff at her to try, beyond the things discussed on this board, and hoped and expected some of them to have results, such as HDT. None of them did, at least nothing significant, and yes I was looking and hoping constantly to see improvements in her. And telling her what I hoped to see. If placebo was active, it would have been much more active with previous supplements etc I tried her on. Conversely here, I considered it enormously speculative, and told both my parents that if there were going to be effects, we would probably see them 3-6 months in, perhaps longer. So when we saw improvements in the first week, and my Dad asked me if I thought it could be the new supplement, I said no, that it was very unlikely. Even when it looked more probable, I was constantly cautioning myself against misplaced hope. I'm beyond that now. She is, without a doubt, improving, and I cannot see any other explanation. So, yes, I can reasonably rule out placebo as a cause.

.....and you said grandmother.

Levod profile image
Levod in reply to Robb48

"and you said grandmother."

You're correct. I mispoke. But it is a "red herring", for nothing I have asserted in my original response, has anything to do with the identity of the patient.

Now I am done...

SilentEchoes profile image
SilentEchoes in reply to Levod

The placebo effect doesn't apply here, the mother doesn't appear to have the capacity to know or decide what meds she's given and is unbiased. Robb48 is the observer in this case and it is a legitimate report. Levod, no one is twisting your arm to take/try ambroxol. I am going to give it a go based upon my own research and combine it with NAC and appreciate that Robb48 wrote the post.

SE

Despe profile image
Despe in reply to SilentEchoes

NAC can be used in a nebulizer.

That’s wonderful Robb 48!!! I’m so happy for you and your mom. Definitely keep us posted but it sounds great. She is indeed very lucky to have you. A difficult part of these kinds of illnesses is doing everything on your own. I realize your mom wouldn’t be up for that anyway. But sometimes I wish I had someone who was frantically researching things for me and with me. The burden often falls on the patient. But of course this forum helps so much and everybody is so knowledgeable and nice about things. Anyway, thank you for the good news!

Robb48 profile image
Robb48 in reply to Godiv

Thanks, I am sure trying to do everything on your own must be incredibly difficult, well done for pushing forward anyway! So many other people here looking for solutions, it's really fantastic.

Godiv profile image
Godiv in reply to Robb48

Thank you! It gets a little difficult sometimes LOL. But yes the forum is great. It empowers all of us to search for solutions or some thing that will at least help.

Robb48, what are your mother's atypical symptoms?

SE

Robb48 profile image
Robb48 in reply to SilentEchoes

Sure. So first the persistent feeling of indescribable sickness or unwellness, generalised but also related to the gut, none of the specialists really know what that is about. Also orthopaedic hypotension is classic in MSA, which she seemingly didnt display at first, even when other symptoms were presenting fairly severely. With the first specialists she saw, he tested her exhaustively but it wasnt present, he gave a very tentative diagnosis of MSA at the time but was quite unsure. Later tests have shown a mild form of orthopaedic hypotension. Sleep, insomnia is less common I *think*, though it can be present, more regular is excessive sleep or sleepiness. But MSA presents as a spectrum, I emphasised atypical because I dont want to imply any certainties with regard to reproducibility.

Despe profile image
Despe in reply to Robb48

Our Vanderbilt MDS had explained to us the different symptoms for different neurodegenerative conditions. He told us that MSA never or very seldom presents itself with resting tremors, but patients experience muscles twitching.

SilentEchoes profile image
SilentEchoes in reply to Despe

For Robb48 too: the muscle twitching is called fasciculations. They are found in parkinsonisms (MSA and atypical ALS (see Table 1)). ncbi.nlm.nih.gov/pmc/articl...

So how do you tell them apart? Reflexes. They are normal in MSA and hyper in ALS and in MS. Fasciculations occur in MS too, which along with ALS, is a neuromuscular disorder (so is muscular dystrophy).

Dementia co-occurs in ALS about 50% of the time, FTD or frontotemporal dementia is part of the ALS spectrum. If dementia is present it is something other than MSA, such as PSP.

neurologyadvisor.com/topics...

The line is blurred between the neurodegenerative disorders.

SE

Robb48 profile image
Robb48 in reply to SilentEchoes

Thanks, yes with my Mum she gets leg twitches at night, but no tremors, and definitely no dementia thankfully. Understood about blurred lines, hard to say for sure sometimes.

This recent paper mentions ambroxyl and the associations between GBA mutations and MSA peripherally. Have you had your mom tested for other mutations? (There are over 300 of them). I hope she doesn't have it..it is supposed to be super rare. Since ambroxyl is working I wonder if she has N370S.

Robb48 profile image
Robb48 in reply to pmmargo

Hi, no I have not - genetics is not an area I've looked into in depth, only peripherally aware that genetic components are a bit controversial. 300 mutations seems to be a wide range of possible associated genes, rather than a direct genetic cause! I'll check out N370S though thanks.

Here's a source for ambroxol.

Retail box of Ambroxol 30mg tablet

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