Monday, July 27, 2009

bloody friggin' hope

I am, as it happens, a great fan and a great skeptic where clinical trials are concerned.

I want clinical trials to happen, and I want lots and lots of people to sign up for them, so that we have mounds of data – swamps of the stuff, stuffed with mainstream, riddled with outliers galore - all the data we'd need to warn doctors of the ROUSes that may lurk, should a drug or treatment come to market.

Swamps, morasses, dancing mountain ranges of data. Not that I have any intention of contributing to it, myself. Or jumping on the results when they show up as headlines. Back when gene therapy was the Great New Hope, we were often asked, wouldn't it be wonderful, if they come up with a way to cure hemophilia?

Sure, I’d say. About oh, twenty or so years later. Once other people have field-tested it. A lot.

Because bodies, as we know well, are weird. And the dancing mountain ranges may not contain that wierdness, o Horatio. As much as we may know about them, bodies can nonetheless be unpredictable, unreliable, or just plain quirky. Imperfect bodies are especially good at this, and when the Imperfects quirk, their doctors have to admit just how much medicine relies on strong, date-fed guesswork, or on the lessons of experience – which are not always backed up by understanding. This works, although we don’t really know why, we are told, and I respect that. I often don't know why, either.*

But a trial and quirk and ‘don’t really know why?’ Too much unpredictability in one room.

Count us out.

Except sometimes, count us in.

The Eldest’s allergy whizzbangers are conducting some of the trials on these new, daring ideas about tolerizing children with allergies. And tomorrow, we’ll join the ranks of hopeful families, to see if the Eldest might be able to be tolerized to dairy. A bit. Not cured, mind you – just more tolerant. Immunologically open-minded, you might say.

I'm sitting on my inner cynic here, but I think it's worth it. Unlike those gene therapy trials, this is a risk we can measure:

  • the risk of exposing him to an allergen, and having his offended immune system crank the allergy up a few notches. Stasis vs manufactured change? Or, stasis despite our attempts to create change?
  • The risk of a reaction – and he will have one, given the structure of the trial. He knows this, but chose to sign up regardless. It's okay to be nervous, I told him tonight. He nodded, looking down. Breathed for a moment. And then looked up. I know. I'm glad he knows, but there's a frozen knot in my stomach. Because my job is to protect him, and maybe at this moment, protecting him means also putting him in harm's way. It's true on the playground, when he climbs to the top of the rope webbing. But is it true here? I don't know. So, risk of a reaction, risk of responsibility, risk of - well,
  • The risk of the psychological impact of that reaction, because he’s not had a reaction in long enough that this will shake him.
  • The risk of hope.
I wrote about hope and this clinic shortly before our first visit, and they keep yanking that chain. On our most recent visit, we were flabbergasted to see that the boys' RAST tests had dropped. All of them. This has not happened, ever. The boys are famous for their RASTS, for the thin ar up there with their scores, and here they were, having dropped from holyfreakin'molyI'veneverseenanythinglikethat, to ohwowbutthat'sreallyhigh. Which, you have to admit, is one hell of a drop. So, if these allergy wizards can help us swing that, what else might they be able to do?**

So, the trial. Where they will feed the Eldest dairy until he reacts. If he reacts too early in the trial, then we're out. But if he makes it far enough before reacting, he'll eat measured amounts of dairy - only - daily, for a measured amount of time. The reaction is a given, because he's still allergic to the stuff. Think about it: he's allergic. We're feeding it to him anyway. And the good news would be getting to feed it to him daily. Maybe, possibly, perhaps this might help his body adjust to the allergen over time. Maybe.***

How the hell could we want to do this?

Dairy is a big pain in our Imperfect ass: it lacks the PR of peanuts, and frankly, it’s an easy protein that folks find hard to replace in school lunches. And birthday celebrations. Skip the eggs? No problem. But the pizza-n-icecream? Well, you’ve got me there. A little more PR, and folks would understand that dairy really can be scary, just like the Peanut ‘o’ Doom, but hey, if we can’t fix the PR, could we maybe tinker with the kid?

Ask the question, of course, and you then have to wait for the reply. Stomach clenched, waiting for the inevitable reaction and please don’t let it be too bad because I’m why he’s here, and damnit, cold fist in the stomach, clogging harsh lump in the throat, swallowing past it and – oh, fuck me– hoping.

*see here, especially the last two 'grafs for more on this.

**at the initial visit to this clinic, both boys were diagnosed with environmental allergies. Exposure to an allergen can raise the immune system from Defcon 3 to 4, 5, or 17. And drag the lab results upwards, as the immune defenses crank into gear. If the kids are exposed to their allergen, say, like a dust mite's poop, for oh, 8-10 hours at a time, that'll wreak merry havoc with their immune systems. Get rid of the allergen, and the body will settle back to normal. Or whatever it thinks normal oughter be. Personally, I'm not knocking it.

*** Tolerizing is a protocol known pretty well to folks in the bleeding disorder community, especially those in the lightning-struck, punch-drunk inhibitor circles. Those lucky folks are people who typically don't make any of a certain clotting protein naturally. As a result, their bodies are making antibodies to their clotting meds, in an honest but oooh, cripes attempt to protect them against a foreign protein. The standard protocol is to

  1. hope that the poor guy's antibody, or inhibitor levels are low enough so that you can
  2. do 12-18 months of daily infusions of massive, expensive amounts of clotting protein. While
  3. hoping that the guy doesn't bleed much, because the clotting protein is going to get infused into him, but not work, thanks to the antibodies. Which means that you'll need lots of infusions of another, even more expensive clotting med - oh, every 2-3 hours while a bleed is going. And that stuff won't work as well, because it's so short-lived. Ow. Oh, and did I mention that
  4. success does not mean that the antibody is gone. As the Eldest proved. He got mostly over his inhibitor, after 18 months of some pretty intense needle-work. Mostly. But, keeping an eye on this post's original topic, we learned then that we're very willing to measure success by whether something gets easier to live with. Or, just plain better.


magid said...

Oh, wow.

I hope it goes well (*insert avoidance of evil eye behavior here*). That would be great to have dairy back.

And kudos to Eldest, whatever the outcome, for being willing to do this.

Rachel said...

good luck!

Auntie A said...

Well that explains the lack of sleep. Hope all goes well and I echo magid re: the kudos.