The problem with fighting a rare disease is that it’s hard to gather support.
Not many people are directly affected by the disease, and those who are affected are “trying to grapple with the disease and just manage their life,” says Christine Waggoner, president and founder of Cure GM1. “To take on extra work is very hard.”
That’s why finding effective ways to extend their reach is critical for nonprofits working to cure rare diseases.
For Christine and Cure GM1, Charity Miles has become a new way to do that.
Cure GM1 funds research on GM1 gangliosidosis, a neurological disease that primarily impacts babies and children. Over 60% of those affected are infants, and most will not live beyond age two.
“It’s a tragic and utterly horrible disease,” says Christine.
Christine’s daughter Iris, who is now 14, was diagnosed with type 2 juvenile GM1 gangliosidosis in 2013. At the time, Christine and her family found no clinical trials available for Iris and few specialized resources.
“For ultra rare diseases, there’s not always a charity as a resource. We felt this was a big gap,” says Christine. “We had connected with other families affected by it, so we got together and decided to try to move the needle on getting treatment.”
In 2015, Christine founded Cure GM1, the only nonprofit focused entirely on GM1 gangliosidosis.
“We created the foundation in honor of Iris, and in honor of all those who suffer from GM1 gangliosidosis,” says Christine.
Since 2015, Cure GM1 has raised $4.1 million through grassroots fundraising. They’ve used the money to organize the patient community and provide seed grants for research projects.
“We aim to structure the environment so biotech companies and researchers will be interested in working with the disease and the community,” says Christine. “It’s extremely motivational for researchers to see and interact with the people that exist, as opposed to just reading about the disease in a paper.”
Their work led to big change. There were no clinical trials for GM1 gangliosidosis when Iris was diagnosed, but now there are four ongoing. Each trial offers a critical chance to patients.
Cure GM1 was also instrumental in getting GM1 gangliosidosis included in the largest newborn screening pilot study in the US. Only fourteen out of 7,000 rare diseases are included in the study. “It’s a huge privilege to be included, and so important,” says Christine. “Even if a treatment was approved tomorrow, people have to get diagnosed quickly for treatment to be efficacious.”
They’ve also funded gene therapy, mouse research, conferences, and support groups.
But a lot more is needed, says Christine, because “research is so expensive.”
That’s why Christine and her team were excited to add their “small, rare disease nonprofit” to Charity Miles. “Whatever tools are out there to help us reach more people and increase awareness, we embrace wholeheartedly,” Christine says.
Cure GM1 started on the platform in February for Rare Disease Month.
“It’s another way to organize events,” says Christine, adding that big events like marathons and races are hard for small nonprofits to organize without a tool like Charity Miles.
“People love the app,” she adds. Christine especially appreciates that the app is health-oriented. “A rare disease can be isolating, and mental health is very important. So I like that it gives people something to do to encourage wellbeing and mental health.”
Going forward, Christine hopes to use Charity Miles to increase awareness, find sponsors, and collect more money for life-saving research.
“What we ultimately want is for all the people impacted by this horrible disease to live long and fulfilling lives,” says Christine. “Every person deserves that.”
We’re honored to be a part of helping you reach that goal, Christine. #EveryMileMatters