Myasthenia Gravis Support Group

Myasthenia gravis (MG) is a neuromuscular disease leading to fluctuating muscle weakness and fatiguability. The hallmark of myasthenia gravis is muscle weakness that increases during periods of activity and improves after periods of rest. Although myasthenia gravis may affect any voluntary muscle, muscles that control eye and eyelid movement, facial expression, and...

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Treatment for severe refractory disease

angepugin
angepugin
Hi all. I'm new to this site but have had severe MG for 10 years. It has turned my whole life upside down! My MG is generalised and has affected my respiratory muscles especially severely so that I am in chrnic respiratory failure. I use a BIPAP ventilator at night and on bad days aswell. I am taking mestinon 4hourly, azathioprine, prednisone and methotrexate and rely on 4 plasma exchanges evry 6-8 weeks to keep me alive. I have also tried a whole host of other immune suppressants including rituximab,with little success. I have had to stop working as a paediatric physiotherapist 5 years ago and spent 7 months of last year in hospital.I was told in March by 2 of my specialists that there was nothing more they could do for me. It's been a very traumatic time and I feel completely alone! I'm from SA. There is quite a large clinic with about 500 MG sufferers that I attend but I have never met many patients and those that I have met seem so much milder. My specialist tells me there are no other patients in SA with similar disease. Is there anyone out there who is going through a similar thing? Has anyone thought of or heard of anyone else having that "rebooting the immune system treatment"done at John's Hopkins? I am determined not to let this disease beat me! Any advice would be greatly appreciated!
deleted_user
deleted_user
I am so sorry to hear you have been battling such severe disease course. Mine started with my breathing and swallowing and it took 6 months to get a diagnosis or appropriate treatment. I have been on supplemental oxygen, take mestinon, imuran, prednisone and have Ivig every 2 weeks. I too have found that many of the people in my local support group have a milder disease course. I am not sure what to tell you on the rebooting of the immune system but have heard some good reports of chemo drugs such as cytoxan. There is also a drug trial going on right now for something that starts with an e. I will try to find the info for you.
jen
Sarah100
Sarah100
I read somewhere that UCLA was doing the rebooting. You may want your docs to contact the neurology department there.
angepugin
angepugin
Hi Jen. Thanks for your response, it's great to have contact at last with people like you! I have in the past been on moderate doses of IV Cytoxan but that had little effect. I've noticed that the extremely high doses used in that particular approach actually wipe out the entire immune system and allow it to redevelop without the myasthenic antibodies. It sounds pretty risky especially since I have had tuberculosis twice previously and there would be a risk of the TB re-emerging in the resistant form when my immune system is gone. The few patients who have had the treatment and been written up, seem to have benefitted enormously from it though. Would be great for us to actually speak to someone who has had it hey. Have you taken part in any of the trials? Would you think of trying them? Really hope you are having a stronger patch and breathing easy! Big hug!
Ange
deleted_user
deleted_user
Thanks Ange,
I actually would be game for a trial. Anything that might make life a little more symptom free but, I unfortunately have too low of an antibody level to qualify for any of the trials I have seen. My official diagnosis is severe generalized sero negative MG but I do have a few tests where the antibodies were present.

Jen
angepugin
angepugin
I'm exactly the same. I've had one antibody study come back low positive but all others negative so am not actually sure if those weak positives make us sero-negative or mean that we are actually sero-positive? Don't suppose it makes much difference in the long run hey. I have taken part in a few of the studies here with some interesting results but nothing that would really lead to a different treatment approach yet. One in particular gave a possible cause for my disease severity and maybe that will eventualy lead us to a successful treatment who knows? It showed that I had a particularly low complement inhibiting factor which makes my tissue more vunerable to damage when my complement factor is activated by my antibody production. The fact that I require regular plasmaphoresis seems to exclude me like you from most of the studies which test new treatments but you never know what could happen in the future hey. O by the way I think that drug trial you spoke about with an e is for something called etanercept and I would have been particularly interested to take part i n it since they have had such fantastic animal results. If I'm correct it is a complement inhibiting drug and perhaps you should look into that. From what I remember the sero-negative disease was't an exclusion criterion.