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Dr. Andrew Campbell:
Well, what I noticed, even as early as medical school and as I continue to train, there's a lack of research in sickle cell disease compared to a lot of other chronic conditions. It affects child health and adult health. And what I always noticed was that sickle cell patients had diverse what we call phenotypes. That means how their disease presents, how their disease, for example, some patients may have few pain crises per year, other patients may have 30 to 40 pain crises per year.
Other complications such as pneumonia, is what we call acute chest syndrome, may happen in the subset of patients much more often than the other. So we knew there are lots of differences. Yet, you would hear it at the same amount of sickle cell percent in your blood, we have a very poor understanding why the symptoms differ so much. So for me, that was my drive.
The second is lack of therapies for sickle disease, and curative therapies, for example. Or even disease-modifying treatments. Since sickle cell disease has been around, since 1910, which means it's been around for over 110 years, up until two years ago, there was only one FDA approved drug for sickle cell disease. So now there have been actually three additional new ones, which I'm very happy about. However, even with that, we still have a long way to go to improve the life of sickle cell disease patients.
The third reason is a lot of patients, they do well their first maybe 10 years of life, it's that second and third decade of life, when we start seeing more and more symptoms in patients. They have more pain, they may have more complications. And even though we have these disease-modifying treatments, some patients to continue to break through those disease-modifying treatments such as hydroxyurea, and we just have a poor understanding of how we could come up with new therapies, and how we can really design studies that allow us to place patients in one bucket, when you're more at risk with developing complications in XYZ versus others.
So that really got me into sickle cell disease research, trying to understand. So that then led me to do more international research, because we know that sickle cell disease is an international disease, right? It affects millions of patients worldwide. And secondly, patients in India, patients in Ghana, patients in the United States, again, inherit the same sickle cell hemoglobin, but they may have different clinical, we call expressions or phenotypes, clinical symptoms. And some of that might be environment, some might be genetic, but there are so many unanswered questions to explain those differences.
