Wednesday, June 22, 2016

Becoming a Smurf for Science

Because of the work I do, I often find myself meeting with and talking to people who are involved in clinical research. It has made me an advocate for research, and I feel that people everywhere are missing opportunities because they simply don't know about what's out there.

Almost as soon as Alex was diagnosed, I began to trawl and every other web site, book, or pamphlet that might yield new information, insight, or hope. In the summer of 2013, there really wasn't anything out there - but we did try both Namenda and Rivastigmin. There was a slim chance that some portion of his symptoms might have an Alzheimer's component to them, and since there was nothing else, it was worth a try.

Then, a new drug appeared ready for Phase 3 research trial -  a tau disrupter known as leuco-methylthioninium. At first, it seemed like we would not be eligible because of age restrictions - but by spring of 2014, the criteria were changed, and we were able to be screened.

I would LIKE to say that this development was on the radar of Alex's doctors here in Minneapolis - but it really wasn't. We signed up for TRIAL MATCH the week after we were diagnosed, and they did not rush to contact us about this. It was our own effort and curiosity (and some of my "insider" knowledge) that got us on the docket early. Maybe it all would have happened at the same time anyway - I am just not a person who is very good at waiting, so I  went after it with a lot of energy, and a sense that time was of the essence.

Alex and I both felt  very strongly (and still do ) that we wanted to do everything we could to benefit others - and yes, of course, we were hoping that it would make a difference for us. But we were very aware that this would be a double-blind study - meaning that half of the participants would get the placebo, and half would get the real thing, and we would not know which arm we were assigned to.

This is a drug with really only two side effects. The first doesn't sound like much - the need to urinate more often and a stronger more urgent need to urinate. Okay - not easy, especially since Alex's dementia has made surgical intervention for enlarged prostate too risky.

But here's the second side effect - methylinium is an ACTIVE ingredient of this compound, and it is a vivid blue dye. Everything coming out of Alex would be a BRIGHT blue green which permanently dyes all kind of surfaces. Very special.

People asked me, "What about the placebo?" Well, if we were on the placebo, the fake compound would contain enough blue dye to have the same effect so that no one would be able to tell who was in the control group.  

As we got through our learning period being on the drug, we were finding splashes of blue EVERYWHERE in the bathroom. I joked with Alex that when he went to public restrooms, he should just tell people he was from Venus. But it was Laura who really nailed it when she said, "It looks like a Smurf exploded in our bathroom," and that describes it better than any other image I could give you.

When we visited people, I DREADED his using the bathroom - I was horrified when I saw white rugs or old (and therefore much more likely to stain) toilets in people's homes. I swear to God, this stuff will permanently dye some toilet seats in about 2 nanoseconds. 

As far as bedding and underwear goes - well, let's just say we keep trying our best. Supposedly, this stuff will lift with ascorbic acid. Yeah - kind of. Sometimes. In my darker moments, I would think, "If we are going through all of this and I find out we're on the placebo, someone will pay!"

But look - when you are faced with hopeless situation, you just learn to get creative. We got a fancy Japanese toilet seat that has a rinse and dry feature, we put protective shields on the bathroom walls, we found ways to BOOST the ascorbic acid, and we learned to be very open with people about this so that they would understand.  And we learned to accept the fact that by this point in time Alex quite literally has blue balls.

It is still a very exciting adventure. We know that we are lucky to get to play any kind of role in this. The research team that we work with is simply phenomenal - some of the kindest, most capable, most supportive and most responsive people I have ever encountered in a clinical setting. They are full of sound advice, they are sincere advocates, and when things get rocky, they understand and listen and offer whatever comfort they can.

Sometimes  out of nowhere, Alex will say, "When are we going back to Mayo?" and I know that's it partly because he likes seeing everyone there , and partly beause he hopes they're going to "fix" the blue thing.

Here's what I could never have been prepared for.

Not knowing whether you are on the "real thing" or not, and not knowing whether it is working or not can make you a bit insane. Any good day, I would be CERTAIN we were on the drug, and that it was working. Any bad day, I was sure we weren't. I would watch Alex like a hawk for any sign of deterioration or improvement, and when he would laugh at a joke or volunteer some information, or connect some dots, I was SURE the cure was working, and I would make sure that life woudl be full of actitives that would almost certainly re-build Alex's brain. (Sounds stupid, I know, but hope is a powerful drug, too).

At the end of the year, we were offered an extension - meaning that we could stay on until they decided whether or not to make the drug available. We are now on the real thing for sure, and we still do not know whether we were during the first year. At first, I thought that would trouble me. It doesn't anymore. It's just part of the deal and part of what we signed up for.

Recently, it is clear that Alex is really losing ground.I am absolutely unable to gauge whether the drug has helped Alex or not. I have no way of knowing how Alex would be if we hadn't done all of this - and no one call tell us that. But if this drug does benefit some people, then I will be thrilled that we played a part in it  and I have started to think of every single blue spot as a little victory for all of us.

This fall, the drug sponsor will publish their findings, at least that's what we think. If it fails (and it well might) I can only imagine how difficult that will be for everyone who has played a part in this.

When I see things on the web that say things like, 'Doctors don't want you to know that this one simple thing will cure dementia," I could scream. What I see when I go to Mayo is a team of people who have watched drug after drug fail, and had to be there for all kinds of people like us who showed up with hope and enthusiasm and came up empty. I can only imagine the rejoicing they will do when this disease gets cured - and there is no doubt in my mind that the day is coming, even if it might be too late for us.

Knowing what I know now, would I do it all over again? Unequivocally "Yes!"  This is one of the only ways I can think of to try ot make some good come from this - and if ANY of you are offered a chance to be a guinea pig, I will tell you that you have nothing to lose. Go for it!  If you need advice on removing blue stains, just ask!


  1. Dawn, if I had been drinking tea (which is my usual early morning FB catch-up ritual), I would have spewed it all over my keyboard when I read the ultimate TMI line. Snort. I'm posting you second-to-last para on my FB screed/uh, wall/ because I, too, want those who believe the stupid conspiracies aka snake oil salesman bullshit "news" to THINK about what they are saying to those of us who actually have to face the reality of dementias and looking for treatments that work. OK, now I have to stop and make a cup of tea to calm myself down.

    1. You crack me up! I am glad you are part of the "A" team.

  2. Hello Dawn! Thank you for your courage in writing about your lives with this devil of a disease. Few people know or understand FTD unless you have/had a loved one with it. (My Dad did.)
    Thanks for putting yourself out there and writing. It is a great public service. I hope you don't mind, I mentioned you and this blog on the Facebook page for the book I wrote with a dear friend who died of ALS.
    Take care! Warm thoughts are with you from people you don't even know!

    1. Thanks so much for your supportive words. Since I just about murdered Alex last night, your timing is perfect! I am always happy to have the posts shared - my original intention was just to help people understand that we're going through - but I am starting to see how marginalized this illness is, and if we can help raise awareness, maybe the awful early stages will be diagnosed and understood more quickly and managed better.
      And I would like to say that you began your stories about Bruce when Alex's diagnosis was fresh. I LOVED listening to them - the things the two of you talked about were SO in line with my thoughts and feelings. It was inspiring and comforting, and I was cheering the two of you on every single time you broadcasted a new story.
      I am so sorry your family had to go through this, and I will certainly check out your facebook page.