President, Grace Wilsey Foundation
Wilsey: Good morning, everyone. Thanks, David. The title of my 180 talk is “Rare Unlocks the Common.” Rare diseases, when added together, affect more people than AIDS and cancer combined. More people than AIDS and cancer combined. My daughter Grace is one of those people. She suffers from something called NGLY1 deficiency. From unknown 18 months ago, it has now been diagnosed in approximately 20 people globally with a few more out there.
I’ve spent most of my career as a tech entrepreneur. I’ve left that role. My new job now is hunting down and curing rare diseases that affect millions of people, especially children. With my 180 seconds I would like to change your mindset about rare diseases. Over the last five years, I’ve heard repeatedly that rare diseases like NGLY1 aren’t important. While people feel sorry for my daughter, they think I’m chasing windmills. They argue that it’s crass that we have assembled a world-class team of people to tackle a disease that affects so few. They tell me that our time would be better spent on cancer, Parkinson’s, Alzheimer’s, Ebola, essentially more important diseases. What these people don’t understand is that rare diseases are critical to unlocking big ticket diseases. Rare conditions are the long tail in medicine. Much like Amazon, YouTube, and iTunes, clinicians and basic researchers don’t yet know what will connect various pockets. How will associations form? Where will the breakout sensation come from? There’s much connectivity here and we know very little so far.
There’s a devastating neurological condition that exists today called Niemann-Pick C and it’s relevant because of Ebola. Patients that have Niemann-Pick C are immune to Ebola. I mean, that’s pretty amazing. It’s also remarkable and quite important. Why are they immune? We don’t exactly know why. There’s a few ideas, but we’re uncertain. It’s critical we figure it out quickly because it could lead to a treatment for Ebola and other medical breakthroughs.
That’s just one example today. There are others that I don’t have time to discuss in 180 seconds. But really quickly, are there any examples that exist throughout history of this long tail? Absolutely. Those include penicillin, Pap smears, X-rays, insulin, and the smallpox vaccine, essentially everything that we benefit from today. It doesn’t come as a surprise to any of us to be focused on the long tail. That’s a must for anyone in the tech world. It’s time the medical world grabs the tail of the common. It will lead to breakthrough discoveries. The associations are fundamental to us, our families, and our friends. Each rare disease is as important as each common disease. Please remember, rare unlocks the common. Thank you.