,I went to London and met Doctor Robin Lachmann. He is going to start me on the drug soon then I will need to go bk to London after being on it for 4 weeks so they can tweak the dosage. He views this more of a temporary solution though and I think has concerns over just how well it could work long term. He says at 39 being fit and healthy and basically symptom free with a loes score of just 5 he feels I am prime candidate for bone marrow transplant. To say I am worried about this is an understatement. What are peoples thoughts on this and the transplant in general? I've been speaking to someone who had it done successfully and is now cured and doesn't need to worry about brain side of things. But he took a year to get over having it done and it was 7 years ago and his life is still changed from it with certain dietary requirements. My sick pay at work doesn't cover pre existing medical conditions so wouldn't cover me for this so I'm worried about things financially also. Any advice or guidance will be appreciated. I am hoping to meet with transplant team soon to get full fact's and discuss before any decisions thanks
Original post:
Hi! 39 years old now and still symptom free without walking issues although I do get heavy feeling legs and my muscles feel strange in them in the mornings. I have been having mris every couple of years since 2005 and they've never picked up anything. On my last MRI there is still no involvement on the spine but they found a cerebral change in the area they would expect ALD to be. I have seen the scan and it is a tiny affected area, they showed me other scans that look wildy different from other ALD patients. I had a follow up contrast dye MRI and they had a quick look at it on Friday but couldn't see anything additional to worry about. I've taken Lorenzos oil for years but they don't know if it's helped or not. My doctor at addenbrookes has asked for compassionete access to leriglitazone and the company has said yes I believe, I just need to travel to London for a chat and assessment and to see what they will require from me. The data looks good for halting cerebral change. Has anyone else been on the drug at all and can provide feedback? I know the trial they previously did was looking to try and help people who had ALD on their spine and it failed as a trial for this but they discovered that ir stopped brain changes. I will stop taking Lorenzos oil and hopefully start on this drug soon all being well and not running into any roadblocks but anything anyone else can add on things would be greatly appreciated. Many thanks. Justin
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Thanks for your post. I have AMN (with walking/bladder issues but without brain impact so far) and I've been on the MIN-102 (leriglitazone) trial since 2018. While I knew there are discussions I didn't know they'd got to the point where they are prescribing it for patients! I've seen the research/publicity papers about the trial and they appear to indicate that there isn't a great deal of evidence of improvement of peripheral symptoms but there is a hope it could stop the development of cranial ALD. So I guess that's why they are giving it to you.
As for me it hasn't made much change. I take 12ml orally per day. It does give me water retention (oedema) which is a known side effect. I am 10+kg heavier now than before the trial but otherwise no other issues.
Thanks for your reply that's really helpful! They asked for compassionate access to the drug and it looks like it will be agreed with the providers. I've heard about water retention and putting on weight, I could probably do with gaining some weight anyway as I am quite small. I will update once I start on the medication. Thanks again for your reply
Things have changed already. They have said although my loes score has changed they have re scored all my MRIs now and the first one I had 18 years ago was a score of 3.5 and my latest one was a score of 6. There was also no sign of active disease on the latest scan which means I don't meet criteria for bone marrow transplant. Plan is to go on the medication and then scan every 6 months to keep a check that there is no sign of active disease, if this ever shows up they said would scan after another 3 months and check again and if still active then I would need to make a decision whether I want the transplant or not. Feel a lot better about things now but still very worried about future. I hope I can get some sleep tonight it's not been a good time.
Maybe i'm not understanding but you're generally symptom free and you want to start a drug that isn't even proven to help or thinking about undergoing a last resort bone marrow transplant?? If I were you I'd find a new doctor. Focus on staying active and eating well. Many on here would kill to be in your situation. This disease generally is slow progressing.
I'm just going on advice of doctors with regards to the medicine as plenty of people are taking it and it seems to halt any further progression with the disease on the brain, I was against the transplant personally but the doctor in London made it seem like it would be the best choice. I know it's generally slow progressing but also some people can start progressing quicker and if it's suddenly active and there's inflammation on the brain they make it seem like I would start seeing really bad symptoms quite quickly if I declined the transplant. I definitely need to eat a bit better and be more active for sure.
The disease usually progresses to cerebral in patients who've dealt with spinal involvement for decades. And it's still only a percentage of them. But that's the situation for all of us. But it seems like you've been spared even the basic effects of AMN. Be happy. If the drug is safe like they're saying it can't hurt, but I don't know if it's the answer yet. Staying active everyday, eating properly, getting proper amount of rest, keeping low stress really makes a difference in my opinion.
Thanks. The drug seems to have only minor side effects so I can tolerate it well I see no reason why not to try it like they are saying. After all, I've taken Lorenzos oil for nearly 20 years and now they tell me it has done absolutely nothing for me and has no effect whatsoever 🤦🏼♂️
Exactly, it was nearly 20 years ago I started it. Which is a lot longer than almost ten years. They didn't really know then if it would help but I was then just kept on it because I was already taking it. Which is about as stupid as it sounds 🤣
I think this sounds like a prudent plan—the Leriglitazone may impede the growth/progress of the lesion and buy you extra time to determine if a BMT is the right course of action for you. I’m hopeful this is the fix (and enjoy being done with LO—that restrictive diet is no fun!)
I’d like to add that if you’ve dealt with headaches for a long time, supposedly that can cause lesions on your brain which are harmless. I have a few lesions now. I’m 32 and was diagnosed when I was 19. I have had lesions for at least 10 years with no side effects from them. I mainly have neurological symptoms. No Addisons. No active brain involvement even with the lesions.
Thanks for your reply man! I've had the headaches for as long as I can remember, some now are due to screen time at work but I always wondered if some were linked to the illness. So, no side effects but you mention you have neurological symptoms can I ask what they are? Sorry to be so blunt.
In terms of myself, I started on leriglitazone, but it gave me stomach spasms (not a known side effect they say) this led to dirrarhea for nearly a week so I stopped it. I then re started it again but took mebeverine one hour before the leriglitazone, this stopped spasms and diarrhea but my toilet habits were not exactly normal. After being on it two weeks I started experiencing bladder problems too especially through the night (not a known side effect either they say) in the end it got a bit much and I've stopped again possibly for good. Think I have some fluid retention too around ankles and my eyes started looking puffy I think all linked to leriglitazone. Bit gutted I can't seem to stomach the stuff as not much else out there for me at same time my condition hasn't really changed in nearly 20 years so maybe I'm just fine as I am, I just worry as I hit 40 next year things could start happening. I sometimes struggle with my memory and wouldn't want this to get even worse
Memory is a symptom IMO. I don’t remember details from a few days ago. I’m almost to a point of not being able to walk. I have neuropathic pain. I now have a colostomy due to the battle with constipation. I have no control of my bladder. Headaches constantly. I don’t have Addisons or brain involvement other than the lesions that they think are from the headaches.
Thanks for reply sorry to hear of what you have to go through. I still don't understand this disease at all if I'm honest and really have no idea what the future could hold. I've had some real problems with constipation over the years.
I had to choose a colostomy for a better quality of life. I spent a third of my time trying to fight constipation. After 12 years of it I just had enough.
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