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MPSIIFund News

Trucking Along- An Update on Trey!

It’s been ages since I last updated on Trey. Fortunately, this is because there’s little to update, at least in the world of Trey combined with MPS II. I read back through my blogs- the last Trey update I gave was in April 2018!

Trey has been receiving IV Elaprase since February 2006 and IT Elaprase since October 2011. We have been waiting for two years to receive news about the approval of IT Elaprase, but there are no answers yet. This is a very loaded question/topic/conversation. While we wait, Trey and our family are good. Trey is receiving drug and has a great quality of life. While we wait, other families who are waiting for drug are not doing so well. Some boys have passed, others have progressed to a stage of disease where the drug is unlikely to help. I remember being on the waiting end of things before IV Elaprase was approved. Trey was diagnosed with MPS II in February 2006 and the drug wasn’t approved by the FDA until July 2006. That was only a 5-month wait and it was hell. Every day you wait while you know irreparable damage is being done to your child; a baby you birthed and love and have huge hopes and dreams for is getting brain damaged and losing hearing and experiencing progressive heart disease and joint contractures while you gaze into their beautiful eyes. I used to stare at Trey, after diagnosis and pre-IV Elaprase, and wonder exactly what damage was being done RIGHT NOW. Every day you are angry. Every day you are sad. Every day you are scared. Every day you are in trauma. Every day is HARD.

Once we have an answer about the IT trial, things for us will either be good for us, and things will truck along like they have been ever since Trey got IT drug, or they will be very very bad. It’s like this: IT drug= trucking along. No IT drug=progression and death. IT drug= being different from others in that Trey has special needs, but being stable so that we know what to expect and can get on with living our lives with Trey as a part of our family, instead of a child who requires so much physical care that the other members of the family get neglected (including ourselves), and so much emotional work to figure out how to be remotely sane while watching your child lose skills and abilities, it often leads to exhaustion and insanity. When I first heard we would receive news “soon” about IT approval- two years ago- I operated with some degree of panic. I feel the panic rising while I write this. However, I have gotten used to waiting, so when it’s not actively on my mind, I’m able to get busy living.

In April 2018 I wrote about Trey’s obstructive sleep apnea (OSA) and limp. After trying for about 8-months to get Trey to wear a C-Pap machine progressively longer each night, I gave up. If at some point I get the energy back up to try again, I will. It’s the only treatment for OSA, so I felt like a horrible mother for giving up, but I just couldn’t do it anymore. I tried so hard, I was so exhausted, I was doing it on my own 7-nights/week, and I just didn’t see how Trey could get from where he was (tolerating it while lying in bed for up to 20-minutes in the dark, followed by a reward of watching movies while wearing it for progressively less time each night) to where he needed to be (sleeping with it all night- he didn’t even sleep with it once). It didn’t help that research shows boys with developmental disabilities are the hardest population to get to wear a C-Pap machine. Unfortunately, OSA can cause heart issues if it’s bad enough (it’s not in Trey yet) and daytime exhaustion, which would impact Trey’s quality of life, which both suck. L

I also wrote in April 2018 about Trey’s limp. It’s still a mystery. Trey’s had multiple x-rays, MRI’s, orthopaedic appointments, hip specialist appointments, all to no avail. He still limps periodically, and for that reason we don’t really take him to basketball anymore. Aside from basketball, he still gets to do everything he likes to do. Speaking of which, he’s in all sorts of activities. At school, Trey lifts weights, is in swimming lessons, does recycling and shopping for the school and is doing work experience at Maplewood Farm. After school Trey has his weekly infusion, dinner club where a group of teens go shopping for, prepare, eat, work on social skills, and clean up from a meal, with support. He also goes to forest club where they learn about the forest and survival skills! On Saturday’s Trey goes to Teen Club where he hangs out with friends from different high schools and they do all sorts of activities and go on all sorts of trips. Trey has a busy life and he LOVES it!

That’s about it in Trey’s world! Except that… HE TURNS 16 IN MARCH 2020. I can’t quite believe this yet.

I know the holidays can be emotionally loaded for many of us. For those of us who have kids with progressive and rare diseases, the holidays can become even more loaded. My hope this holiday is that you can find at least a few moments of peace and beauty; where you can feel the brilliance of fresh air or good food or good company (even if there are people missing) or a sound or sight or memory that lights you up. Moments… I now aim for great moments… great days or weeks or years set the bar too high I now believe. Great moments seem achievable. 😉

Love Deb

PS. The two pictures are of me and my boys, Trey and Avery, and our five kids (in age order): Trey, Avery, Sadie, Raleigh and Cora.

2nd Annual Construction Cares a Wrap!

That’s a wrap! Our second Construction Cares Event was a HUGE success thanks to all of you wonderful people who attended our event and showed their support, as well as all of you who donated from afar. Construction really does care!! We raised $41,700 for the MPS II Research Fund at The Isaac Foundation this year and we couldn’t be happier with how this whole day went!
BIG thank you to all the volunteers, sponsors, donors, partners and vendors who have made our second annual Construction Cares event the great success it was!

If you’d still like to donate, you can do so at: You can read more about the research we’ve funded here:

AND, in more great news, there will be a 3rd Construction Cares in 2020, so if you’d like to be involved in any kind of volunteer, donor, partner or sponsorship role, you can let me know or keep checking in on Trey’s website: or the MPS II Research Fund on Facebook!

We truly cannot thank you enough.

The Purcell’s

Construction Cares 1 month away!

Save the date! 09-06-2019

We hope you can make it out to Construction Cares on Sunday, June 9. It’s just over a month away! It’s going to be a super fun event for a great cause and we’d love to see you there.

If you can’t make the event, but would like to donate to fund our MPS II research, you can do so here. We are grateful for every donation and make sure to do right by all of our donors.

For more information about Construction Cares, click here.

Construction Cares approaching!

Save the date! 06-09-2019

Construction Cares is a fun-filled indoor fair for adults and children of all ages on Sunday, June 9th from 11am to 4pm at The Pipe Shop in North Vancouver.
115 Victory Ship Way, North Vancouver, BC V7L 0B2

Event is rain or shine and offers bike valet by donation; wide variety of games, activities, interactive displays, live auctions; food vendors; emcee Kevin Evans and Tall Music DJ on site; and more!

Admission is free and activity tickets can be purchased to participate in your favourite games and activities including a kid’s construction & decorating station; Top Shot Hockey game; face painting; LifeSpace Gardens Veggie Mobile, GardenWorks gardening station; balloon twisting; Axewood Axe Throwing; caricature artist; Beere Brewing beer tasting; roaming characters including Elsa, Olaf and Spiderman; a visit from the Vancouver Canadians’ Bob Brown Bear, free colouring and block building station for kids, SPCA table; live auctions and 50-50 draws hourly at 12pm, 1pm, 2pm, 3pm and 4pm. 

Net proceeds benefit the MPS II Research Fund at The Isaac Foundation (Canada).

For volunteer information, please contact Louise LaRochelle at 
For sponsorship or live auction donation information please contact Presenting Sponsor Joe Geluch at or Pamela Buck, Event Organizer at 



$150,000 Awarded to Dr. Brian Bigger for MPS II Gene Therapy Research!

Our MPS II Research Fund and the entire Isaac Foundation is always on the lookout for hopeful research and researchers in MPS II. Dr. Brian Bigger’s work is just that. We are THRILLED to announce that we just granted $150,000 to Bigger’s Lab in the UK and we are hopeful that this will lead to anther gene therapy option for boys and men with MPS II. Please read below for details of Biggers’ work:

“The Bigger lab have developed several gene therapies for neurodegenerative lysosomal storage diseases including most recently Mucopolysaccharidosis (MPS) II (Hunter), MPSIIIA, MPSIIIB and MPSIIIC. Most patients have no treatments, and where treatments do exist, they are ineffectual in the brain.  Delivery of replacement enzyme via haematopoietic stem cell gene therapy (HSCGT) using a lentiviral vector can effectively target the brain, correcting disease.

We recently developed a stem cell gene therapy that can target the brain in Hunter disease, via a lentiviral vector expressing a blood brain barrier crossing peptide coupled to the IDS gene (LV-IDS-APOEII). We have successfully performed a proof-of-concept study in MPSII mice demonstrating correction of disease (Gleitz 2018 Embo Mol Med) and superiority of the LV-IDS-APOEII vector over the normal enzyme.

We obtained funding for an InnovateUK Manchester Advanced Therapies Centre Hub (iMATCH), a consortium of academic and industry partners with a co-ordinated strategy to scale up gene therapies for patients in Manchester. Our remit on iMATCH is to develop scaled up GMP transduction of haematopoietic stem cells, but vector production was excluded from the call.

The grant from the Isaac foundation will allow us to purchase GLP grade IDS-APOEII (Hunter disease genome) plasmid from plasmid factory to enable production of a large-scale GMP-like lentiviral vector for Hunter disease, which would both validate our large-scale transduction procedure in CD34+ cells for iMATCH and provide data for a potential filing for phase I/II clinical trial.”

 If you have questions, please reach don’t hesitate to contact us!

Construction Cares was a HUGE Success!

Construction Cares, an indoor fair for adults & children of all ages with net proceeds going to the MPS II Research Fund, happened this past weekend and it was incredible! These events never fail to touch our hearts and souls at the deepest level.
When Joe Geluch, President of Naikoon Contracting (the company that built our house), approached me last summer after getting to know our family through the build of our house, to put on a fundraiser for our MPS II Research Fund, I thought it was a kind gesture, but never did I have any idea that it would be brought to fruition. Fruition was Sunday, April 22, 2018.
$24,000 was raised for our Fund. For research. For our boys with MPS II. I don’t give as many updates on Trey as I did when he was first diagnosed, and although Trey is mostly stable due to the IV & IT treatments he receives, our life is still far from typical. It’s hard to describe, to a large group of people, what our life is like unless you see us regularly. What do you focus on? What and how much do you say? Trey’s progression due to the build up of GAGs as a result of MPS II is minimal to none after he began receiving Intrathecal ERT over seven years ago, but he still has MPS.
This past year, two issues have arisen for Trey that at times have gotten me pretty down:
One, we found out Trey has Obstructive Sleep Apnea (OSA). There’s no surgical treatment, no cure and since November 2017, we’ve been working with his Behaviour Analyst to get him to wear a C-Pap machine at night. Due to his OSA, Trey is tired most days and now due to our routine trying to get him to wear a C-Pap machine at night, misses most of his night time activities like floor hockey, bowling and softball, which he loves. If he doesn’t learn to tolerate the C-Pap machine, that could lead to heart issues due to lack of oxygen when he obstructs.
The second, which has a longer history than a year but we thought was resolved, is a severe limp with no known cause, other than MPS (he has hip dysplasia as a result of MPS). It was awful to the point of Trey not being able to walk for weeks at a time last summer, but somehow resolved on its own. Then a week ago, it came back with full-strength after the one activity he still participates in because it’s early enough to get him home for his C-Pap routine, soccer. He could barely walk (I contemplated a wheelchair because I don’t think he could use crutches) from Monday night until Thursday morning and then was fine. Sunday night after Construction Cares, the limp was back. Avery and Sadie helped carry him back to the couch when he’d try to get up. It is so hard to see. We now might have to take him out of soccer. His life is already limited by MPS cognitively, with multiple trips to hospitals here and in North Carolina, and with weekly infusions, it gets me down to see his quality of life (which I think is pretty good, he might just be the happiest member of our family) further impacted by MPS.
I say all this with the hopes of you understanding just how much it means to our family that you showed up yesterday, in whatever form that was. MPS can be sad and hard and lonely and it is you who lifts us up when we are down. Even to those of you who couldn’t make it but sent a text, that matters. You remembering matters. You asking how we’re doing despite us being twelve years into this journey, matters. To all who came and donated and volunteered and sponsored and attended as guests, you honestly fill our hearts. You make it so we can pick ourselves up and say “We’ve got this. We can do it. We are surrounded.” Ryan and I left Construction Cares last night knowing we’d never be able to fully express how thankful we are. We didn’t get to thank each of you and even if we did, it wouldn’t be enough. I hope my words can in some small way help you know what it means to us that you came.
Thank you to everyone from Naikoon who volunteered. It was SO cool and overwhelming to walk into The Pipe Shop and see all these people we know, all these people who began conversations with us years ago about building a sustainable and durable house for our family, to the people who actually built our house and know the guts of our house better than we do, to the people who emailed and organized and showed up for the build of our house when we had questions, and to the people from Naikoon we didn’t know until this fundraising began! We are so touched by how involved you all were. It took a team. Thank you.
And an extra special thanks goes out to Madeleine Funk, Tyler Waddell, Pamela Buck and Joe Geluch. Gosh, when I think about how stressed and anxious and insanely driven I was putting on other events, and then I think of what you all did for me, for us yesterday, I am forever grateful. Joe, we will never EVER forget what you have done for us.
We will share more pictures from the event in the coming weeks, and here is a link to a CBC video, our clip starts at 10:34:
With love,
The Purcell’s

Construction Cares is Coming Up!!

Naikoon Contracting Ltd. has stepped up to support our MPS II Research Fund in the most incredible way. Last year, the owner of the the company that built our house, Joe Geluch, approached me to put on an event for our fund after learning about Trey through building our house. The event has come to fruition!! On Sunday, April 22, 2018, Naikoon Contracting Ltd. is presenting Construction Cares, an exciting indoor fair running from 11am to 5pm at The Pipe Shop in North Vancouver, with proceeds going to our MPS II Research Fund! This indoor fair is for adults and children of all ages, with games and activities hosted by local businesses. Bike Valet and admission are by donation, and activity tickets will be on sale for $2 each.

Activities include: a Construction Station sponsored by Naikoon Contracting Ltd.; Decorating Station sponsored by Pro Works Painting; Gardening Station sponsored by LifeSpace Gardens & GardenWorks; and Face Painting sponsored by KYBE Electrical Contracting.

There are super-fun games too, all with a prize for the highest score of the day, including a Spider Boxer game sponsored by Griffins Boxing; Electronic High Striker Game & Kiddie High Striker Game side by side (so adults and children can play together); and an Xtreme Skateboard game.

Also look for our bike valet sponsored by The North Shore News; music from Tall Music DJ Services; hourly 50-50 draws from our emcee Kevin Evans; and some amazing prize baskets put together by local businesses that are being raffled for $2 a ticket.

Everyone welcome, rain or shine, with net proceeds from this event benefitting Hunter Syndrome/MPSII research.

Spoke with Dr. Muenzer Regarding IT Trial Set-back

I spoke with Dr. Muenzer today regarding the IT Idursufase trial not meeting it’s endpoints. If you have any questions regarding the future of the trial, please feel free to reach out to myself or Dr. Muenzer. If you don’t have Dr. Muenzer’s contact information, I can provide you with it (he gave me permission). For any family in the trial or for concerned extended family or community members, he would like to provide as much information as he can in order to relieve anxiety, so please don’t hesitate in reaching out. The trial did not meet its endpoints, but this does NOT mean the drug does not work and it does NOT mean the trial is shut down. Dr. Muenzer knows the drug works. He sees our kids and he knows where they would be without the drug.
People have pointed out to me in the past day that gene therapy is coming up if this trial does get shut down. One: this trial is not getting shut down unless it’s over my jailed or dead body. Two: Gene therapy is hopeful and likely, but I will not rest my child’s life on hope and possibility. Nobody knows this better than families who have kids with MPS II Trey’s age and older. When Trey was diagnosed at the age of two in 2006, IT trial research was already going on. I spoke with Dr. Muenzer about it every few months. Every spring we were told human clinical trials would begin in the fall. Every fall we were told it would begin in the winter. Every winter we were told it would begin in the spring. For three years. During that time, most of Trey’s peers fell outside of the IT trial criteria and therefore did not qualify.
So yes, gene therapy will hopefully come to fruition, but I will not let a drug that is holding my child stable and saving his life, go, with the hope for gene therapy. I don’t know gene therapy will come, and if it does come, I don’t know if Trey will qualify for the trial, and I don’t know if it will come in time to save Trey.
Trey has IT therapy. I am not giving that up until or unless there is another treatment that is available and works.
With love,


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