Where We Are Going…

This is our next step.

The irony is that I have been waiting for this drug for seven years. Literally. I
found out about it when it was in early trials in Japan and I followed it
closely. Caitlin was 5 and had been fighting arthritis for two years. It was
known as MRA and it is a biologic that targets a completely different cytokine
than any of the existing biologic medications. It targets inter-leukin 6,
(IL-6) as opposed to TNF or IL-1. Scientists believe that different forms of
auto-immune disease probably have different cytokines in the lead. Children
with systemic JIA have often not responded well to TNF drugs. But in Japan,
they were doing trials with MRA and really sick systemic kids – and they were
getting good results. It truly seemed to me to be the best hope for remission
for Caitlin.

Seven years ago – it just about killed me to think there was a drug in existence that
could “cure” my daughter, and I could not get it for her. I was fairly obsessed.
I contacted the drug maker about petitioning for compassionate use. I learned
more than I ever wanted to know about clinical trials, FDA approval and drug
pipelines… as well as the truth about the business behind it all. Still – I
just wanted my daughter to be able to have a medicine that would control the

I contacted doctors around the world. I learned of a clinical trial taking place
in London, and seriously considered moving there if it meant Caitlin could have
been included. We have family there – and it would have meant taking a leave of
absence from my job, leaving my husband behind, and taking the kids across the
pond…. But that seemed reasonable if the result was remission. Ultimately, we
were too late and the trial was closed. I tried more docs, but it often felt
like I was beating my head against the wall.

I started following this drug in 2003. Over the years, I have been known to tell
other parents of systemic kids – “Don’t worry! MRA is coming!” Slowly, over
time, my frenzy died down into reluctant anticipation. Getting a drug through
trials is a long, expensive process. For very good reason – it is a slow

Again– the irony – when they finally started doing pediatric clinical trials for
this drug in the U.S. about 18 months ago, Caitlin was well. As in, she was in
remission already. Not sick enough to be included. But when the FDA approval
for this drug came through in January of this year, I was still very happy that
the long wait was over.

Our doctor recommended we try this over some of the other new medications, because
Caitlin seems to be having some systemic symptoms (some rashes) and some
typical systemic blood markers are high. Caitlin was horribly upset – because
the last infusion she remembers is IVIG – which took 10 hours and caused BAD
side effects. She cried as we tried to explain that this is only a 2-3 hour
infusion, without those side effects. Our doc was very hopeful that we will see
results fast, and might be able to “peel back” off this drug in less than a
year. Caitlin was not convinced and she is fairly inconsolable. I don’t think
anything will change that until we get the first infusion and she can know for
herself what it is like.

As long as we get insurance approval, two weeks from today, we’ll go in for the
first round. The doc will add a large dose of Solumedrol for the first infusion
(a steroid pulse to help knock back this flare). We’ll go back every two weeks
thereafter… until we can hopefully spread it out to once a month.

Seven years is a long time to pour hope into something. I was willing to chase this
chance around the globe – will it be worth it? I’m almost afraid to see it come
to fruition, but I really want to see Caitlin feel better.

Don’t let us down, Tocilizumab!

This entry was posted in Actemra - Tocilizumab, Medications. Bookmark the permalink.

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