Tuesday, October 1, 2019

Gene Therapy Fundraiser

Nate seemed particularly happy on our walk to school the other day. I asked him how awesome he thought his life was on a scale of 1-10. His response? “Twenty. Wait, no. A trillion!!” This surprised me 0%. He loves his life, and we love our life with him in it. We want his life to be as long and fantastic as possible.

As I mentioned in our last post, a possible treatment for SMARD is in the pipeline at Nationwide Children's Hospital, and we are big fans of this news. This type of gene therapy has already been developed for other diseases and had amazing results (check out some of these articles!). Now Nationwide is ready to create a SMARD-specific version of this therapy. The therapy aims to replace the faulty gene with a healthy copy by delivering a one-time dose of a "virus" that carries the healthy gene. Gene therapy like this has the potential to stop the progression of the disease and even to help kids reclaim some of the strength that they've lost. Nate's decline has been so gradual that it has been almost imperceptible even to us until we look back at pictures from years ago and notice him doing things that he'd never be able to do now. Even stopping further degeneration would be life-changing for him. Children who are still very young with limited disease progression stand to gain even more from this treatment.

The nonprofit Smash SMARD has been working closely with researchers at Nationwide and have raised over $1.3 million in the last few months which will be used to fund this research. They need an additional $600,000 to get them to clinical trials next year for 10-12 kids. 

So, to recap...this gene therapy already exists, it has worked in other diseases similar to SMARD, it has worked in SMARD mouse models, Nationwide is interested and excited about moving forward early next year with developing the treatment specifically for SMARD, $1.3 million dollars have already been raised (mostly through Facebook!!), and clinical trials could happen as early as next summer. A lot of exciting stuff. But it’s all contingent on raising the rest of the money.
Over half of the money raised so far has been from Facebook donations, and most of those donations are small amounts from people just chipping in whatever they can. So, in honor of my and Scott’s October birthdays, we’ve set up a Facebook fundraiser. It's so easy to donate on Facebook - just visit this page and click the blue "Donate" button at the top. We invite you to just do whatever you can, no judgement. If you don’t feel comfortable donating on Facebook, let me know and I can give you an address to send a check.
Of course we can’t really predict what kind of impact this treatment would have on Nate, but all signs point to it being a low-risk, high-rewards option for him. Until now we’ve never had any real hope for a treatment and this project is worth seeing through to the end. I can’t stand the idea of money being the reason it might not happen. We are so grateful for every single donation! 

Donate here!

You can learn more about the therapy and the work Smash SMARD is doing at smashsmard.org. Smash SMARD is a registered 501(c)3 organization and all donations are tax deductible to help find a cure for SMARD.