Here's a link to more info about Efgartigimod:
https://myastheniagravisnews.com/2018/06/07/myasthenia-gravis-pivotal-phase-3-trial-argenx-efgartigimod-on-track/

It is in trials and I believe is given as an infusion. I am glad that they are also including seronegative MG patients in the trials as there seem to be a lot of us.

I thought it is a orally taking drug. If it is a cure that will be a great news! Anyone want to participate in this trial?

I tried to understand what the drug does. I found this line "efgartigimod blocks antibody recycling through FcRn binding and leads to fast depletion of the autoimmune disease-causing IgG autoantibodies"

If I understand that, it gets rid of our immune system created bad antibodies -- sort of a direct attack on what is our problem.

The phase 2 study results "While safety and tolerability of the treatment were the study’s primary objective, researchers also evaluated ARGX-113’s effectiveness. Using four tools to measure the treatment’s impact on disease severity, it became clear that ARGX-113 improved scores on all four compared to placebo. Specifically, 75% of patients treated with the investigational therapy had clinically and statistically significant improvement through at least six weeks compared to 25% of patients on placebo."
Improvements already were seen after one week of the four weekly infusions. The treatment also lowered the levels of antibodies, a measure that correlated with patients’ disease severity.

An interesting note on the last sentence above -- level of antibodies correlated with patient diseases severity -- that is something that most of the time we hear is not the case.

It also says the drug was given by infusion, but they were also testing a "subcutaneous" version.

News to me . Bring up this new drug with my doctor and I'll likely wind up in a straight-jacket!!

An interesting note on the last sentence above -- level of antibodies correlated with patient diseases severity -- that is something that most of the time we hear is not the case.

Agreed, there follows a snip from a conversation with my Neurologist the other week.

"Anti-acetylcholine receptor antibodies are odd. Some patients have multiple symptoms and only very low levels of antibodies whereas others even with high levels are controlled on pyridostigmine alone".

Makes me wonder what else is in play and perhaps offers an explanation why so many of us have different symptoms and indeed severity. Interesting that they are arguably going for the result rather than the cause albeit I do appreciate that not in all people with MG can it be attributed to the antibodies.

My antibody levels at diagnosis were “abnormally high”. (400 ish). My antibody levels one or two years post thymectomy - 700 ish. Three or four years later - 1240! I have been symptom free and medication free since three weeks after my surgery. Ive had scans to see if anything else, somewhere else, is producing thymic tissue, so far nothing has been found. For me, the antibody level does not correlate to the severity of the disease.

I too have been told that there is no correlation between anti - body levels. I have difficulty getting my head around such a statement, because this disregards that we are all different i.e weight, metabolism etc. Do you know if anybody has had plasmapheresis treatment after thymectomy ? If the source of the antibodies is the thymus , surely cleaning the blood will eliminate or radically reduce the antibodies. After all you would not change the oil filter on a car without changing the oil.

I would be very interested in joining trials for this drug. I love in the UK.Anybody know how I could volunteer for such a trial

Why not ask Donald while he is here? Alternatively, try this route

http://www.argenx.com/en-GB/content/contact/41/

Andrew: This might not be the answer you expect but, as far as I am aware, no one, certainly not the scientists, are absolutely sure of the mechanisms associated with MG. I like your analogy but, the oil, in this case, is simply the medium that carries the antibodies through the system? Something or someone else is adding additives to the blood? We can have IVIg and that works much as you describe, but only for a month or so, before, things get back to 'normal'.

At this point, I am unsure what happens in the case where the patient has had their Thymus removed? If they were young at the time they do seem to be cured of MG. For us older ones there's no point in removal, it has done its deed. Not everyone has MG, of course, it's a rare disease. I too wonder where the source of our antibodies rests, crack that and I suspect a cure won't be too far away.

Another question. Any ideas at what point our blood started to carry the antibodies - have they always been there, just biding their time?

Perhaps it is also true that irrespective of the level of those antibodies in our blood our muscle receptors are not a constant - other events impact on them?

It would be useful if there was a direct correlation between the level of antibodies and the severity of our MG as it would provide a measure of the efficacy of the drugs we take and in so doing allow far better control of the disease. Sadly, not the case?

There are multiple antibodies involved in MG. For a summary of the types so you can read your test results better try this link.
https://www.questdiagnostics.com/testcenter/testguide.action?dc=WP_MyastheniaGravis

My own initial blood test results:

Blood Test Name, Date, My test, Normal value

AChR Binding Ab(S) May 22, 2012 5.80 nmol/L <=0.02
Positive for MG
AChR Modulating Ab (S) May 22, 2012 100 % 0-20%
Positive for MG
This serological profile suggests thymoma.

With positive ACHR tests the following were done too

AChR Ganglionic Neuronal Ab, S May 22, 2012 0.00 nmol/L <=0.02 Negative
to rule out dysautonomia-- is an umbrella term used to describe any malfunction of the autonomic nervous system. -- something other than MG

CRMP-5-IgG Western Blot, S May 22, 2012 Neg. Negative
Not sure but maybe for thymus cancer (or other cancers).

Striated Muscle Ab May 22, 2012 Positive 1:120 <1:60
As a serological aid in the diagnosis of thymoma, especially in patients with onset of myasthenia gravis (MG) younger than 45 years. As a screening test for MG in older patients, especially when tests for muscle acetylcholine receptor (AChR) antibodies are negative


GAD65 Ab Assay, S May 22, 2012 0.07 nmol/L <= 0.02 Positive
Diabetes susceptibility and other autoimmune likely--helps doctor decide if prednisone might push me into type 2 diabetes -- and so my neuro was worried about that. Also suggests predisposition to related autoimmune thyrogastric disorders
.
This serological profile suggests thymoma.

I didn't have thymoma. I do have immune system thryroid problem.

I have not had a retest of my blood since 2012. I am in remission and think it would be interesting to see what the test says now, but my neuro says I would have to pay for it -- so we wait until remission goes away -- maybe never!

Russ, interesting and in far more detail than I recall we get from the NHS. There again with it all being so new to me, I doubt, even had I asked, it would have meant much to me. I had half my thyroid removed a few years before being diagnosed with MG. It was benign but was assured that the two things had nothing in common. In those early MG days, I had terrible trouble with mucus, at the time they said, nothing to do with MG and was even sent to the ENT department for tests, we now know it is very much a side effect, certainly of Mestinon, or even a marker that things are not going so well.

Nice, for us, not to have to pay for the blood tests - are they very expensive? I also gather that there is a shortage of IVIg and certainly here in the UK strictly limited in its use compared to a few years ago.