MPSIIFund News

I still have a smile on my face from Saturday. How can I not? The number of people who supported us is unbelievable. I don’t even know where to begin (if you want to start with the sap, read the bottom first).
How about food and drink donors. Blue Goose Cattle Company donated 80 something pounds of organic ground beef (I don’t even want to know how much that would cost to buy). Happy Planet donated 370 juices (Trey & Ave get 1 Happy Planet juice each after Trey’s infusion on Monday’s, which costs over $5, so I can only imagine how much that would have cost to buy… and their juice is so tasty and healthy). Molson donated 12 dozen beer (we all know how much that would cost to buy :)). Que Pasa Mexican Foods donated fresh salsa and organic chips. And Save on Foods donated everything else. Produce, taco shells, pop, water, beans, cutlery, cups, napkins… name it. They make it so easy for us and everyone I have worked with from Save On is so friendly and helpful!
Now onto raffle and auction donors. We gave away dozens of of raffle items and there were 72 auction items, all of which were donated. I just do not have room to thank everyone who donated items for the raffle and auction, so if you were one of the amazing people who donated, please know we are so grateful. It was such a cool auction, I wish I’d had the time to bid on more stuff!
Entertainment… BOBS & LOLO!!!!!!!!!! If you haven’t heard their music, go to: www.bobsandlolo.com. They are awesome and wow did everyone love their performance. One of our friends, Asher, is such a die hard Bobs & Lolo fan, I can’t help but laugh when I see him making sure everyone is following along with the words & actions & paying attention. They were definitely a highlight of our event and I am so grateful they were able to perform.
The massive bouncy castle was a big hit for kids too (I had some adults request to go in, but there just wasn’t enough room!). I knew the kids would like it, but I’ve never seen so many kids go so crazy for something. Crafts and face painting (thank you Avrell & Shannon!!) were other things the kids spent a lot of time at.
There are just so many people to thank. Our volunteers were amazing. My mom and sister stayed up into the wee hours of the morning every night in the week leading up to T4T, my dad, my aunt, my mom, my grandma, and my mom’s friends picked up all the food, drinks and rentals, family and friends were at the school late Friday night and early Saturday morning setting up, and SOOOOOO many people helped during the event. Thank you!
And to all the people who came to the event, wow. It was amazing. We filled up the school gym and then some. You helped us raise $18,000. More than last year. You touched our hearts. When Ry and I got home that night, one of the things that came up was the disappointment from people who mean a lot to us and did not come. During that conversation, Ry said: “They don’t know what this means to us. They haven’t been through what we have.”
You really don’t know what you coming to our event means to us and unless you’ve been where we are, you probably never will. However, I will try to express our feelings in words: When Trey was first diagnosed, and for the first year, we were pretty much always crying and always overwhelmed. There wasn’t a minute that went by that MPS didn’t cross our minds.
Then Trey started ERT, we held our first T4T fundraiser, and we were told Trey’s brain would likely be okay. Things were starting to look awesome.
Then Trey had hernia surgery and a month later the hernia popped back out. Then he got another, different, hernia. Then Trey’s ear tubes fell out and he needed new tubes. In the span of 3 monthes, Trey had 3 surgeries. We were in a bit of a rough patch.
But since Trey’s ear tube surgery on October 1, 2007, Trey’s health has been awesome. No surgeries… nothing medical (other than infusions), really. Sadie was born, we were planning our next great fundraiser, and again, all was good.
About a week ago I saw a cardiology report of Trey’s for the first time, done back in January. I really don’t know what it means, and I am waiting for his doctor to interpret the results for me, but there are a lot of words on the page. And that scares me. And although I don’t know if anything is actually wrong, it is a reminder that Trey has a progressive disease. And that we need to find a cure. And that although Trey was diagnosed over 2 years ago, this isn’t over for us and never will be.
This is our life. I realize and some part of me understands that for others, life goes on without MPS. But ours doesn’t. I guess that is what is isolating for me. We don’t go away for more than 7 days at a time because we don’t want Trey to miss an infusion. I spend half the year working part to full time, organizing a fundraiser to find a cure for my son’s disease. BC Children’s is almost a second home. MPS is a central part of our lives.
So all of you coming to Trey’s fundraiser… it showed us we’re not alone. We have people around us who recognize this is not over for us. Many of you changed your plans, or took float planes to be on time, or drove from different parts of the country to be there. It showed us you care. Knowing that Trey is going to be okay and knowing that we have people around us who will stay with us and join us on this journey, that is what makes this journey okay. If given the choice, I never would have picked this path, but Trey and Ryan and Avery and Sadie and all of you who support and love us, make this a pretty darned awesome road to be on. Thank you so very much. We love you.

Trey’s story was featured on Shaw’s ‘The Express’ in the week leading up to the 2nd Annual Tacos for Trey.
[flv width=”320″ height=”240″]http://treypurcellcom.nationprotect.net/video/theExpressApril29_2008.flv[/flv]

The North Shore News wrote a great article about Trey and the situation in Canada with rare drugs for rare diseases. Click here to read the article.
We hope to see you this Saturday, May 3rd at Lynn Valley Elementary School from 3-6pm for “Tacos for Trey” as it’s going to be tons of fun!!

It is very timely that this Friday we (Canada) will be celebrating our first ever International Rare Disease Day (more about it below). This past weekend, Kirsten Harkins and Simon Ibell (the ED and a director of the MPS Society) met in Ontario with a number of families affected by MPS II. They met to discuss Ontario’s decision by Health Minister George Smitherman not to fund Elaprase and to discuss where they could go from here. I spoke with Simon last night and he said it was a very emotional meeting. These families are trying everything they can to receive funding for this drug, deemed safe and effective by Health Canada and many other countries around the world, but Smitherman continues to deny Elaprase to people with Hunter Syndrome. Simon, who was a part of the Elaprase trial, is in his early 30’s, and who has an attenuated form of Hunter Syndrome, has been receiving funding for Elaprase from Shire HGT, who developed the drug. The Ontario government was supposed to start funding Simon’s infusions this past December, but has refused. I cannot believe and am brought to tears by the disregard that George Smitherman (and others who were a part of this decision) has for human life.
Simon Ibell, and his mum Marie, are the main reason that Ryan and I survived Trey’s diagnosis with hope. When Trey was diagnosed, I felt like I was drowning. Simon is without doubt, one of the kindest and most thoughtful people I have ever met. When I met him, I thought to myself that if Trey turns out to be anything like Simon, I couldn’t be happier. And Marie got me through the diagnosis as a mum. She has been told 6 times that Simon had less than a year to live. She taught me to believe in Trey instead of listening to people’s words and opinions of all my son would not do. But George Smitherman apparently does not think Simon’s life is worth living. If Simon isn’t worth helping out, I don’t know who is.
DO YOU KNOW WHAT SIMON SENT TREY YESTERDAY? A PHOENIX SUNS JERSEY AUTOGRAPHED BY STEVE NASH (Steve and Simon are good friends). Trey loves jersey’s-basketball, football, hockey, if it’s a sports jersey, he’ll wear it!! Some days he’ll wear one on top of the other because he loves them so much. And our whole family is into sports (it’s a toss up who was more excited by the jersey, Ryan or Trey), and Steve is from Victoria which is just a hop, skip and a jump from here… a very cool gift. Thanks Simon!!
Here’s the info on Rare Disease Day: On Friday February 29, 2008 is the 1st International Rare Disease Day. It is a day about raising awareness of rare diseases and of people with rare diseases. In conjunction with International Rare Disease Day, is a “Day of Action” on Thursday February 28th. Patients, family members, and friends are traveling to Ottawa to present a proposal for a “Chance for Life” fund. This fund will support treatment for Canadians with severe and life-threatening rare disorders. And last but not least, look for CORD’s newspaper insert in the National Post on February 29th. Even if you just talk about how a rare disease has affected you or someone you know to someone who has never heard of MPS II (or any other rare disease), that can make a difference. That’s all for now!

Today Trey has been on ERT for one year. We had a small party at the MDU yesterday for his 53rd infusion with cake, presents and many many thanks to all of our family, nurses, doctors and other staff who have been here with us through this journey.
What does one year mean? It’s really more of a feeling. A great feeling of relief and accomplishment. We did it!! Trey is still only one of 4 kids in Canada who have been approved for treatment. The other 3 boys are all brothers in one family in Alberta. The feeling is knowing what would have happened to Trey’s body in the past year had he not been on ERT. It’s a relief knowing that I’m finished fighting for him to be on treatment (at least for now) as I know many other parents who are still fighting as hard as they can for their kids to get Elaprase. It’s knowing that he breathes and sleeps easily at night, and he can run, play and dress himself like any other kid his age.
When I think about where we were at one year ago: we had no idea what ERT would bring and if Trey might have a serious allergic reaction to the drug, and we were still not sure if Trey’s brain was affected by the disease.
This year, like I mentioned before, life is surprisingly like others around us. Besides our weekly jaunt to the hospital, which we treat like a job, our life is like our friends who have kids. After being through what we have in the past 2 years, I’ll admit, it’s strange. I even get to forgetting sometimes that Trey has anything at all. I’m glad for that for so many reasons, mostly because it means I treat Trey like any other kid. I know other people probably see Trey as different, but I don’t, and I think that’s important for our family so that Trey grows up thinking and believing he is capable of doing anything he puts his mind to.
It’s also coming up on Trey’s 2 year diagnosis anniversary which is Valentine’s Day. No matter how far we’ve come since then and how awesome Trey’s doing, remembering that time brings tears to my eyes and I get this sinking feeling in my chest and heart.
In the past weeks I have tried to think about what we can do as a family on that day, and we will do something for and with the kids, but I don’t want to celebrate with Ryan. It doesn’t feel right. All I can think about when I think of Valentine’s Day is sitting at Gotham restaurant with Ryan and 3 month old Avery in my lap, trying to enjoy dinner at a posh restaurant we’d made reservations at, when the only thing running through my head were the doctor’s words from earlier that day: “I think he might have an enlarged liver”, “possibility of a storage disorder”… Valentines Day has been ruined for me. I think I’ll talk to Ry about changing Valentines Day for us.
But then I get back to what’s important and how far we’ve come since then. It is a strange experience having been at a point where I honestly believed my life was over (and I can still feel those feelings) and then coming to the place where I’m at now. We’ve gotten back into the groove of living life again. I am so grateful for that.
And last but not least, a bit of “Tacos” talk. We checked out the elementary school last week where the fundraiser is being held this year. We’d love to have it at our house again, but there were so many people who came last year, if even the same numbers came this year and it rained, we’d be up a creek without a paddle. So we’re going to do it at a brand new and awesome elementary school.
It’s a perfect location for our fundraiser. And now because we’ve got more space, we are going to have 2 bouncy castles, a space for hockey and crafts for kids, as well as the pinata, raffle, auction, food and drinks we had like last year… and that’s just what we’ve got planned right now. Who knows who else will stop by or what might be brought… I don’t know if it’s possible, but the fundraiser will quite possibly be bigger and better than last year. I am really excited and I hope you are too!!

Trey began ERT one year ago today. What a fantastic year it has been. He has not missed one infusion, which is unbelievable. He is growing like a weed and continues to be very tall for his age, his speech is improving quickly and his development is doing great, his range of motion and joints are where they should be at for a kid his age, his xray and othopedics appointment two weeks ago week showed us that his bones are growing and doing well (Trey had and EKG and echocardiogram yesterday at the hospital, but we do not yet have results)… our life is strangely and wonderfully “normal.”

In disappointing, but expected news, the CDR (aka. Common Drug Review) did NOT recommend Elaprase for funding. As defined by the Canadian Agency for Drugs and Technologies in Health, on January 4, 2008: “The Common Drug Review (CDR) conducts objective, rigorous reviews of the clinical and cost effectiveness of drugs, and provides formulary listing recommendations to the publicly funded drug plans in Canada (except Québec).” Next week, The Canadian MPS Society will be issuing a press release responding to the CDR’s decision. This does not mean that funding cannot be secured for people with MPS II, it just means that funding will continue to be a struggle and will continue to be decided on a case by case basis.
The CDR has incorrectly concluded that the effectiveness of Elaprase has not been established. In contrast to the CDR’s decision, Health Canada gave Elaprase a Priority Review based on their conclusions of efficacy and the life threatening nature of Hunter syndrome.
Coincidentally, global treatment recommendations for Elaprase have just been published.The CDR needs to make changes to the way it deals with rare disease drugs. It is clearly not able to properly value and assess rare disease therapies and has just created another barrier to patients with rare diseases trying to access approved and effective drugs in Canada. Canadian patients with rare diseases are at a serious disadvantage, and as far as I’m concerned, this is a human rights violation.

In my last entry I spoke about being attracted to people who are real. Well, here’s an example. This excerpt is taken from a friend of mine’s Caringbridge website. Her son has MPS III and he was diagnosed in August:
“It’s a sad day…too much on my mind. Tyler takes the front seat but more baggage is piling in than I can handle right now. Physically I am tired. I am hurting. I am sick of the kicks, the scratches, the bites. I am sore from the hair pulling, head butting and endless pinching. I am stiff from ducking flying objects like sippy cups and lunch bowls. I am cramped from living on my hands and knees scraping up wet rice from the floor and picking out crushed cheerios from the carpet. I have been searching for an outlet…preferably a safe one. Even time away seems to go too swiftly and I find myself back here again being disappointed all over. I fantasize about running away. Just me. I imagine myself in another city, another state where no one knows me. I hide there and try to forget my past and reinvent my future. In my fantasy I am happy and I don’t look back. I can picture myself smiling, laughing and running through my days. I am free of the weight of the baggage that slows me down here. This is my escape. I am sad to me right now.”
It is this kind of honesty that makes it okay for people, me included, to be less than perfect. Life has ups and downs, and it is impossible to recognize the beauty in life without having experienced its despair and sorrow. Thank you, Donna Kay, for sharing yourself and for making it okay to be human.

I copied the below out of the Winter issue of the Canadian MPS Society’s newsletter, The Connection:
Support Patients with Rare Disorders in their Fight for Life-Saving Therapy
“Canada is the only developed country without an “Orphan Drug Policy” so Canadians with rare disorders are denied access to therapies available in other countries. There are up to 6,000 rare disorders affecting nearly 10% of the Canadian population. Thanks to advances in science, some disorders can now be treated so patients live longer and have improved quality of life.
We need your help to get governments to put in place an “Orphan Drug Policy.” Your support is urgently needed to assure that Canadians with serious and life-threatening rare disorders get the same chance for life as those with more common disorders. A letter of support is available on the website of the Canadian Organization for Rare Disorders (www.raredisorders.ca).”
I sent in a letter myself, and it can take as little as a few minutes. All you have to do is click on the link at the top of the home page, fill in your name and address and the site will tell you who your local MP is. It then gives you a generic letter that you can submit as is, or you can edit it as you wish. It is actions like this that make change, so if you can take 2 minutes to send in a letter that will save lives, possibly Trey’s, we would appreciate it.
How can an ODP help Trey? If this new Intrathecal Therapy for MPS II gets approved for use, and Trey needs it for spinal cord compression he may one day experience, or if another treatment becomes available, an ODP means that we will not have to fight tooth and nail to get the treatment for Trey. We will not have to wait months, while Trey’s disease progresses, to see if the treatment will be funded. We will not have to fight for new treatments like we had to fight for Elaprase.

In the past weeks, we have had a bit of a rough go with adjusting to life with Sadie. Ry went back to working long film hours, often at night, 2 weeks after Sadie was born. Soon after, everyone got sick. The boys were up at night having trouble breathing from being congested, Avery is up at least once a night with nightmares, and since Sadie’s birth, he’s decided that he wants bottles of milk in the night (he’s never had a bottle of anything in the night). Add this to Sadie being up 3-4 times/night for an hour and a half (she’s a puker and burper and has trouble settling at night), and Ry and I were getting less than 5 hours of broken up sleep /night, often less than that. It is the first time I have experienced sleep deprivation like that and it was tough.
With this experience and the experience of Trey’s diagnosis and the journey this has brought, I am beginning to find that I am having a harder and harder time with our culture’s “everything is great” attitude. I am finding even more than before, that I am attracted to people who will admit when things suck. They will talk about the highs and the moments of beauty, wonder and pleasure, but they will also talk about the lows and not hide the lows like they are a sign of weakness or shame.
And on a different note… this past year… This time last year we were still considering transplant and Trey had not yet began ERT. 2007 has been an awesome year. Trey began ERT, we began an MPS II Fund through the MPS Society, we got pregnant, we found out we were having a girl, an echogenic bowel on my 19 week ultrasound was later shown to be wrong, we raised over $30,000 at our first “Tacos for Trey” fundraiser, Trey’s development tests in May and November showed us that Trey’s CNS is doing well and has a 99% chance of staying that way (for those who know this disease, that is monumentally huge and I don’t know if there is anything else in the world I will ever be more grateful for), and we experienced the birth of our beautiful baby girl on October 30. What a year to be thankful for. Definitely one of the best years of my life. Happy Holidays!! We wish you much love and all the best for 2008.