Christine White

_“When my two daughters were diagnosed with Gaucher disease in 1991, I felt terrified and alone. I’d never heard of Gaucher. There was no information, no internet to speak of. I had to borrow medical textbooks and the main thing I learned from them was how severe and life-shortening and incurable this condition is. I had two little girls, Meghan and Kate, both under the age of two, and I thought they were going to die. That was highly motivating. That was my “why.”

I started seeking out any patient, any parent, any family I could find who had any experience at all with this disease. I was desperate to find someone who could show me a path forward. Finally, I made contact with a few patients in Toronto who were meeting and sharing information, and it was there that I learned about a new Gaucher treatment that was available in other countries, but not here in Canada. And there were other promising treatments coming down the research pipeline, without any assurance that those who needed them would get access to them. These drugs were among the most expensive in the world at the time, and it was unbelievably hard to know that there was a medicine that might help my daughters, but that it was out of reach.

We started working like crazy to bring these treatments to Canada, lobbying the government, doing anything we could think of. We organized and formed the National Gaucher Foundation of Canada. It was a long and difficult road, but our efforts finally saw some patients in Canada being treated with these new therapies. But, with Gaucher, a treatment is never a cure.

The first Canadian patient we got on treatment was a friend of mine named Bill, who was 32 years old. Bill has been a ray of light for me. To see that he’d survived so long inspired a cautious hope that my girls might have a chance. But, over all those years of his life, Gaucher disease had been wreaking irreversible havoc on Bill’s organs, and the treatment came too late to save his life.

The earlier someone is able to get on the treatment, the more the inevitable progression of this disease can be slowed. My girls were fortunate that, in the years that followed, they were both able to get on treatment while still very young. It was life-changing for them. But I still had a lot of close friends in the community who weren’t so fortunate and who were fighting for their lives.

I continued to volunteer with the foundation until 2009, when Mark Freedman, our founder and first president, grew too sick and ultimately died of complications from Gaucher. Before he passed, Mark asked me to take over his role. Having worked side by side with him all those years, and knowing how much work was still to be done, I knew I needed to step up.

I started doing this difficult work for Meghan and Kate, and then I kept at it for the incredible people I’d met in the Gaucher community and for the memory of those like Bill and Mark who we’d lost along the way. It’s a constant battle, and it’s emotionally devastating. I could share story after story of people I’ve known who have suffered, who have died unnecessarily, who have lost their children. It’s heartbreaking. But every one of those stories is a new “why” that keeps me fighting.

I’m 67 years old and I’ve been in these trenches for half my life. I’m not going to lie, I’m tired. But it’s impossible to stop now, especially when the landscape has changed so much and, for the first time, there are possible curative Gaucher treatments on the research horizon. We’ve had a lot of victories, and there’s still a lot more that’s worth fighting for.”

{{CTA}}