I have to “go there” a bit in order to write this blog. But I don’t want to and I’m not diving in. Human clinical trials for gene therapy in MPS II Hunter Syndrome are around the corner. We could begin next year. *IF* we raise $2.5 million dollars. Right. That.
Along with a few other MPS II parents, I spoke with our gene therapy researchers, Dr. Doug McCarty and Dr. Haiyan Fu, a couple weeks ago. Mice with MPS II start dying around 7-months of age. When Dr’s Fu & McCarty treated their mice that made it to 9-months old with double the dose of what they give their younger mice, these 9-month old mice came back. They normalized. I don’t know how many months old my Trey mouse would be, but can you imagine? I don’t even know what that would look like (to be clear, I don’t want Trey as anybody other than who he is, but avoiding early death, surgeries, hospital trips, and letting him be as independent as he can and wants to be would be my dream). Trey as a typical 13-YO kid? I won’t. I can’t. Not yet. It’s too scary. The letdown would be too monumental. Even writing this I’m choking back tears and emotions. Shove them back down. For now.
BUT, I do believe it is possible. WITH YOUR HELP. The thing is, with a rare disease, we NEED your help raising awareness. Nobody knows about MPS II Hunter Syndrome but us and in order to raise $2.5 Million dollars, we need people to know.
With gene therapy SO close, our MPS II community has banded together. We’ve organized teams. If each team raises $10,000, we’ll reach $1 Million. I think we can do better. Our runs raise $10,000 each year without a cure on the horizon. Tacos for Trey raised $20-40,000. Our gala raised $80,000. $10,000 is attainable. Let’s beat $10,000. $10,000 to see a Trey who has no physical or cognitive limitations?? I’d give a f@*k of a lot more than that.
Our MPS II community has also created a documentary series about MPS II Hunter Syndrome to raise awareness. The first of the episodes will be released on May 15- International MPS Awareness Day. Here’s the trailer.
So what can you do? 1. Watch the trailer. 2. Along with the trailer, share a link to our Scotiabank Run fundraising page:
https://www.gifttool.com/athon/MyFundraisingPage?ID=2012&AID=3532&PID=606221
Ask your community to donate and tell them why they should donate. Tell them how close we are to human clinical trials. Tell them how you know Trey and how MPS II Hunter Syndrome affects him. Tell them how MPS II Hunter Syndrome effects you.
THANK YOU. If you have questions, ask. I’m a mom on a mission to save her kid’s life. I’ll do anything. The fire-breathing dragon has been mostly sleeping for the past few years, but she’s being called back out… it is time.
