Ambroxol trial

maryna

New member
Joined
Aug 17, 2024
Messages
6
Reason
Loved one DX
Diagnosis
06/2024
Country
UK
State
NY
City
Swansea
Hello,
I am a newbie here. My husband was diagnosed with ALS limb onset in June 2024. He had a rapid progression to the point that I thought he would die in August. Extreme SOB, respiratory rate went persistently to 40. Unable to move from a toilet to a wheelchair.

I have received Ibudilast from Japan and overnight he moved from 8 pillows and a wedge pillow to one pillow. I don't know whether it was a sudden overnight remission or it was a response to Ibudilast. The drug is a free radical scavenger, a small molecule penetrating BBB, works in a different pathway to Radicava ( Edavarone in the UK), it is licensed for ALS treatment in Japan and South Korea. I have read a lot about it and don't have any qualms for using it.

However, my question is whether have anyone of you tried Ambroxol? Again, it is a small molecule, derived from an Indian herb, penetrating BBB and is changing lipid metabolism in motor neurones. The question is out of curiosity. There is a trial conducting currently in Australia, the doses are high.
 
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Amboxol been used for decades outside the US OTC in syrups to break up phlegm. At marketed doses, major concerns are stomach damage (avoid if you have ulcers), liver and kidney damage if either is already a concern. It's also being repurposed/trialled in Parkinson's, Gaucher, etc.

With an RR that high, is he on BiPAP?

The current trial is a dose-finding study, so multiple doses will be used. If you're comfortable with that, it's worth exploring but the ibudilast (also in ALS trials) would probably disqualify him from any other interventional trials. Of course, there are ways to get the OTC dosage even while living in the US.

It is hard to believe that ibudilast would have that dramatic an effect overnight, but hope is a powerful medicine.
 
Thank you for your comment.
BiPAP is not his choice.
 
Hi Maryna, my husband was diagnosed last week. Do you mind keeping me posted on how the medication is working? We have our first meeting 10/16 with the clinic. I’m not sure if he should go on anything until we meet with them?? There seems to be so many things being tried I feel very overwhelmed but am willing to try anything, we have savings so we can pay for medications. Please how fo you know about all these medicines in other countries? If there is something in another country that works won’t they share that with the US? God I feel lost 😞
 
Hi JAM,
I would start with medications, which your husband’s doctor prescribed. I would also look for trials which are in your area.
Afterwards, I would look for Healey platform videos, ALS news today website, Canadian ALS association and Australian ALS websites. It will give you plenty of information what’s going on in ALS research area.
I completely understand you, have been myself there a few months ago. Though I am a doctor, albeit not a neurologist I was lost, desperate, feeling useless and powerless to help my husband. And still feel it.
You will also need adjustments for your house, depending on how fast ALS will progress. A stairlift, wheelchairs, crutches, handles, toilet and bathroom adjustments. It will take a lot of money, I spent £8000
in one month, some grants were given by our UK MND association, but it took time for them to move.
I would advise you to keep fighting for your husband.Yes, depression and desperation are always with you, but there are only hope and anger which helps you to go through it.
 
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Every situation is different, but in the US, ALS tends to outpace stairlifts and crutches. I think we more often do the rollator-to-wheelchair progression here and build out ground floors if needed for bathroom access. Still, for many PALS, bathroom use becomes impractical at some point so extensive renovations are not always worth it. It might come down to hovering in a Hoyer over the toilet, or a commode bucket if the Hoyer does not work in your bathroom space.

If someone is not going to be using BiPAP, their life expectancy is likely considerably reduced, so that might play into planning. Same for a feeding tube in most cases.

Building/sidewalk accessibility varies among countries and that affects the mobility devices and aids that are recommended.

Also, just a reminder about clinicaltrials.gov for finding studies worldwide.

As for drugs, not all are approved in all countries -- this relates to many things, including where the manufacturer wants to ship/market, how/in whom trials were done, and government budgets. Some are approved for use only in clinical trials until they are approved for general marketing.
 
Ty for all the information, I have a lot to do. Is your husband still getting better since starting the ibudalist? I am praying it’s working for him! You never know if a miracle drug is it until it is. 🙏
 
I apologize if this question has been asked/answered. We are in the US, is Ibudilast available in US? Does Dr. prescribe? I know we have to pay for it bc not covered by insurance but did you say it’s OTC? We are leaning heavily toward getting him started on that as soon as we are able. Ty.
 
It is not an fda approved drug. I believe some people source it from abroad which usually requires paperwork from the doctor to import it legally even if the source doesn’t require a prescription. There is a pathway to import medication when therapies for serious illnesses are inadequate. Be aware if you have any interest in trials this would be a disqualifier. You can’t take anything that is currently in trial elsewhere as it could skew results.

Ibudilast’s proposed mechanism is anti inflammatory. Even if it works as proposed not all ALS is inflammatory. This may factor into the varying results but be aware this is not yet proven
 
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I read this morning Mayo in MN will be conducting a trial with it. I guess we will discuss it when we finally meet with his team in a couple weeks- isn’t there a test they could do to determine if his/anyones aLS is inflammatory or no? Then the patient would know whether or not to even try Ibudilast. I’m sure as a newbie I am missing things 😕
 
There are some inflammatory markers. They apparently did not think it was worth stratifying people in the trial and I haven’t followed this closely to know if it was even considered. If they did limit the trial it would also limit availability if it was ever approved
 
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That makes sense. Thank you.
 
Jam, we found the ALS doctors we’ve encountered (at two different ALS clinics in Seattle) seem to be unwilling to try anything other than the medications that are approved for ALS. When we asked to try something experimental we were told that it wouldn’t hurt to try, but we should ask our Primary Care dr to prescribe it. Fortunately our PCP is awesome and has been willing to help read research and look at things for us. Not sure what clinic you’ll end up at and if it’s different but maybe make sure you have a supportive PCP on board to help, if needed.
 
Thank you Maria. It is so disheartening to get a devastating diagnosis and then not given any guidance or reach out/follow up from anyone. Granted maybe when we meet his clinic that will be helpful. I just feel like given all that these sweet souls are going through the doctors should help as much as possible not be reluctant. I hope your husband is having a good day:)
 
Jam, I agree. We wonder if the ALS clinic doctors that we’ve encountered are just focused on treating his symptoms caused by ALS (ordering a BiPap, medications for secretions, feeding tube, etc). And of course they prescribed the 3 R’s (we tried Relyvrio from the time of approval until the time it was disproven to do anything). But not much assistance with anything experimental we’ve wondered about …
 
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