In Vancouver, the Rare Trait Hope Society is a non-profit making a big difference for people and families dealing with rare genetic conditions. They’re all about offering support, raising awareness, and building a community for those facing these unique health challenges. The society is a valuable resource for people in Vancouver dealing with rare genetic traits. We sat down with Executive Director Barbara Insley to discuss their work more closely.
Describe your charity in a few sentences
Rare Trait Hope Society was established to fund research and human clinical trials to cure childhood rare diseases. Our current project is to fund a gene replacement therapy to cure Aspartylglucosaminuria (AGU), a fatal neurodegenerative disease that impacts children.
What problem does it aim to solve?
In Canada, there are no mechanisms currently in place to fund clinical trials for most rare diseases. We support parent groups who are funding ultra-rare disease research to address this gap in funding.
When did you start it?
Rare Trait Hope Society was founded in 2020.
What made you get involved?
My daughter was diagnosed with a rare disease in 2019 called AGU, that impacts only a few hundred children around the world and currently has no approved treatment or cure. It is my mission to change the outcome of her disease.
What was the situation like when you started?
By 2020, a global group of parents of children with the disease AGU had successfully funded and completed a proof of concept for a gene therapy-based treatment that showed success in animals – curing them and even reversing the disease. That work, and a clinical trial protocol, were reviewed by the FDA and all FDA comments were successfully addressed. However multiple steps still needed to be completed in order to start the children’s clinical trial. That’s when the Rare Trait Hope Society stepped in to help.
How has it changed since?
Since Rare Trait Hope joined the race for a cure, we’ve raised more than US$500,000 towards the clinical trial. The medicine necessary to save the children has been manufactured, and partially paid for. The final gene therapy safety study is underway. Once completed, we can submit our application for final FDA approval. The produced medicine will then be administered to children in a simple, one-dose, 1-hour injection.
The total cost to produce and safety test a new drug is about US$5.8 million. Our Rare Trait Hope global families raised the first US$4 million through corporate donations and discounts, as well as grassroots fundraising efforts. As it stands now, only US$1.8 million still needs to be raised. So we’re more than 2/3rds of the way there. In the meantime, the kids have to wait. And the clock is ticking.
What more needs to be done?
Everything we need to succeed is in place, except for the remaining funds. Although the life-saving drug has been manufactured, we need to complete payments on it, in order to use it in the clinical trial.
How can our readers help?
If you share our vision that every child with a rare disease can be treated, and you want to accelerate our funding to support this clinical trial, then we would be so grateful for donations that will help us complete the payments for our medicine! You can help us raise awareness by following us on social media. You can also introduce us to others who share our passion for curing rare diseases and may be able to help us. or anyone interested in participating can subscribe to our mailing list at our website, or contact us here.
Do you have any events coming up?
Our 50/50 started on April 15th! Please check our website to find out more about how you can help, and you can purchase tickets here.
Where can we follow you?
Facebook | Instagram | Linkedin | Website
PAY IT FORWARD: What is an awesome local charity that you love?
I am a big fan of Cause We Care, which supports single mothers and their children in need.
