That’s all folks!

So, as you will all have heard, the BYM338 trial for sIBM has been suspended.  This bombshell first appeared on Facebook and the American Myositis Forum (TMA) on Tuesday evening.  I had a phone call from one of the trial doctors at Queen Square on Wednesday morning to confirm that with immediate effect there will be no more infusions, although we are expected to keep our next appointment which will become an ‘end of trial’ visit with fasting blood tests and the full gamut of strength and walking tests.  He asked me not to make any public announcements until he’d had chance to speak to each of his trial participants personally, which I though was fair enough, but by the afternoon it was fairly common knowledge throughout the myositis community via postings on Facebook and the discussion forums. 

We knew a couple of months ago that the primary goal of increasing our 6 minute walking distance hadn’t been met, and I’ve said a lot about that already so I won’t go over it again.  There was still the hope that the secondary goals would prove to be successful, improvement in the pinch, grip and quadriceps strength tests, but I’m told they showed no improvement either in any of the treatment groups.  Interestingly, the questionnaires we fill in about our ability to do our daily living activities DID show improvements in the groups that were having some dose of the drug, but no improvements in the placebo group.  So those people on the drug felt that they were doing better, but these improvements were not measurable by any of the tests that have been devised, and are therefore considered irrelevant.

So, since there was nothing that Novartis could show to the FDA in support of an application to license this drug for use in sIBM, they will not be taking this any further.  To be fair, they must be pretty upset about this turn of events, because they have sunk literally £millions in it – the science behind it looked as if it would work, but as I have said before, there is a fundamental flaw in the calculations.  We have a disease that destroys our muscles.  You can probably build new muscle cells with this drug but unless you stop the disease from doing its thing, it will just destroy the new muscle cells too.  Perhaps if they’d blasted us with high doses, they could have out-paced the disease process, but they chose not to do that.  There was a slight suggestion that Novartis may devise a new trial using bigger doses, but we all know how long it takes these things to get off the ground so for now – game over.

So how do we feel about the trial being stopped?  Well, it’s understandable that Novartis can’t keep throwing money at a lost cause.  I’m just angry at the way it’s been done.  It really illustrates how little the drug companies think of us guinea pigs that they gave us no notice, no chance to talk about how we thought we were getting on, no consideration that we might not be getting significantly stronger but some of us weren’t deteriorating as fast as might be expected, no NOTHING – just the bleak notification that we’re not getting any more infusions.  After all the days we’ve given you, all the ridiculously early morning journeys, all the needles stuck in us and all the effort we put into the various tests, thanks a lot Novartis.  Yeah, I know – we all volunteered for it, nobody made us do it.  I’m incredibly angry that 25% of the trial participants only ever got the placebo, yet carried on jumping through every hoop that Novartis presented us with because we all thought the drug was working for some of us and we’d all get the best dose in the end.  I feel we’ve all been cheated.

That’s the bigger picture.  Personally I’m scared.  Regardless of what their results say, I know the drug has been good for me.  I know that before the trial, I struggled to type and was down to using just one finger as the others weren’t strong enough.  I know that when I sat on my stairlift I didn’t bother putting the footplate down because I couldn’t lift my feet onto it, and now I can.  I couldn’t pull plugs out of wall sockets, or ring pulls off drink cans, and I hadn’t been able to open bottles of medicine with child-proof (Steph-proof) caps for about 10 years.  All easy now.  I can breathe when I’m lying down flat, and I don’t have to constantly clear my throat.  My head doesn’t flop back when I look up, and I can shrug my shoulders again – not sure what benefit that is, but there it is anyway.  And the drug doesn’t work?  Yeah right.  Without the drug, I know all the gains I have made will gradually slip away, and the relentless downward slide will begin again.  Not a nice prospect.  I know some will say, quite rightly, that there are new drug trials in the pipeline and more treatments in development.  I know that’s true, but I’m not ready to jump on any new bandwagons just yet.

The only advantages I can see to not having the infusions any more are (a) that I won’t have to get out of bed at 5 am any more to start the horrendous journey into Central London, and (b) the diarrhoea might clear up. 

So Monday is my next scheduled appointment.  They would like me to arrive fasting, ready for blood tests, strength tests, walking tests, ECG, physical exam etc.  They say it will be a long day.  They have no idea how much the day takes out of people with IBM, but we’ve done it on numerous occasions because it was required of us in order to get the drug.  Now we’re not going to get any more drug because they’ve decided it doesn’t work.  So why do they need this extra set of data from us?  What difference will it make?  None to me.  Am I going?  Am I boll*cks.