Sickle Cell Disease is an inherited condition that, if left untreated, kills 80 percent of children born with it before they reach their fifth birthday. In sub-Saharan Africa, where 75 percent of cases occur, every year about 300,000 infants are born with the disease.
Taking advantage of decades of experience that comes from battling Sickle Cell Disease among its own population of African Americans, the United States is partnering with countries in sub-Saharan Africa that have the largest number of people suffering from the disease. “There are a couple of approaches we can use,” said Brett Giroir, a pediatric doctor and Admiral in the United States Public Health Service who currently serves as the assistant secretary for health at the U.S. Department of Health and Human Services.
“We could mitigate the effects of the disease. We could treat people like we treat all other conditions. And we could do that very simply if we give penicillin and vaccines. We know we can get children who die before age five up into their 20s with nothing else and probably into their 30s or 40s. If we [treat with] hydroxyurea…there's no reason that people can't live to their 40s and 50s.”
Now we can also take advantage of treatments that deal with a genetic defect in the blood. That’s important, because a human being replaces his or her red blood cells every 90 days.
“You could correct the gene defect in the bone marrow and all of a sudden people with sickle cell will start pumping out normal cells. That is not science fiction; that's being done in clinical trials right here in the United States. It has a potential for being scalable to hundreds of thousands or millions of people.”
Admiral Giroir believes that within ten years, we could see relatively inexpensive, accessible treatment that would enable people with Sickle Cell Disease everywhere to lead normal lives. “There has never been a time in history that there's been so much activity and excitement about helping people with sickle cell disease,” he said.
“There's never been this opportunity and we want to seize it. And the time is really right. Just think about that. Nine million children by 2050 that we can save have them have happy, safe, productive lives for their countries, for them to raise families on their own.”