Not long ago, I found myself speaking to a program administrator at the FDA. It was a classic elevator pitch, I told him all about the breakthroughs in gene therapy for leukodystrophy, specifically, MLD. My voice cracked with emotion recounting how I had recently told one father that while gene therapy had shown promise for other children, it would not be available to his daughter.
I pleaded with the FDA program officer, "What would you have me do?"
The man leaned over and whispered: "Let me be frank, money gets things done."
For over two years, I called reporters, sold cupcakes, wrote letters and spoke to researchers, business leaders and politicians. It has been humbling and frustrating to see a treatment that could help children not be available to families in the US.
Since the piece on CBS, so many people have written to blame the dysfunction of the FDA or point fingers at pharmaceuticals. The reasons for the delay are not so simple, it seems to me there are no villains in this story, just victims.
But, just as I wondered if all this work was going to make any impact, I received a one- line email this morning: "Don't give up."
Since yesterday's airing, we have raised $5000, with nearly $10,000 raised since we launched our kickstarter grant project in July, that's pretty good, but we have a long way to go.
Let's be clear, this money is not going to salaries or office spaces , we want this money to launch US leukodystrophy gene therapy trials. So, please take a moment and share the piece with friends and family. We are not asking any one person to give more than they can afford. But, small donations can add up. It will be years before gene therapy gets to the US, so for a small gift, we will be working every day to make miracles possible sooner.
- Maria Kefalas, co-founder, The Calliope Joy Foundation and Cal's mom