Meet David, Your Summer Scamper Patient Hero
“Being in the hospital isn’t scary to me. Having a new problem isn’t scary to me anymore because it’s happened so many times. I don’t know, my blood just does not like me very much, I guess.”
At age 15, David had been living in and out of Lucile Packard Children’s Hospital Stanford so much that he could explain his sickle cell disease, an inherited red blood disorder, better than anyone.
“Your red cells are supposed to be round and have oxygen in them. Mine are half-moon-shaped, sickle-shaped, which is why it’s called sickle cell, so I don’t get the same amount of oxygen,” David said in the 2019 Netflix documentary “Human Nature” by Adam Bolt.
“It’s often called the first molecular disease. It’s caused by a singular change in the DNA sequence,” explained Matt Porteus, MD, PhD, Sutardja Chuk Professor of Definitive and Curative Medicine at Stanford University. “It’s the letter ‘A’ changed to the letter ‘T.’ It becomes very stiff, and it can’t squeeze through and you’re not able to get red blood cells to the tissues where they can deliver oxygen. When you block the ability of oxygen to get to those tissues, those tissues won’t work well, and they’ll get damaged.”
Currently, the life expectancy for a patient with sickle cell disease is just 42-47 years. That’s 42-47 years full of dialysis, severe pain episodes, and risk of strokes and organ failure. And yet, these risks have not stopped David from being a kid. He can’t be held back from what he loves most: playing basketball.
“It’s like pulsating,” David said about the pain. “I can have a little pain crisis where it really doesn’t count, and then I could have something really bad. But I’m not just going to not play basketball. You can’t just not play basketball.”
But now there’s hope for more kids like David. Dr. Porteus is using CRISPR, a revolutionary gene editing tool, to develop a cure for diseases like sickle cell disease.
“We just didn’t have the tools, to make that single letter change in a precise fashion—especially one letter. Before CRISPR, we were getting 1-2% correction. We’re now up to 50-80% of the cells,” Dr. Porteus said excitedly. “This could really work. This could really cure a patient. We have the expertise to do that all here. We have the infrastructure with the 5th floor of Lucile Packard Children’s Hospital to treat these patients.”
Recently, David surprised Dr. Porteus with a video call to catch up and thank him for his work to find a cure.
“The hospital has been a second home to me, and it feels really weird to say but some of my favorite people I met through the hospital or work there,” David said. “And I don’t even have to know them, I can speak for all of the other people with sickle cell, and basically every disease. Thank you, Dr. Porteus.”
“I really appreciate that,” replied Dr. Porteus. “But I’ll also point out that it’s really a large group of us who are all part of that. Things like this don’t take one person. It takes a whole team of people, and you have a whole team of people behind you, and you’ll always have a whole team of people behind you.”
Thank you for being a part of David’s team. David continues to receive treatment at our hospital (although now that he’s 18 years old, he’ll be graduating to the Stanford Hospital soon!) and is proud to Scamper in support of other patients, his care team, and innovative researchers like Dr. Porteus. We may be apart, but as David says, “you can’t just not Scamper.”
