Born in Baghdad in 2002 without access to advanced medical care, Maryam Idan stood little chance of receiving anything more than the most basic treatment for her sickle cell disease (SCD). By the time Maryam was a baby, her parents, Basim and Janin Al-Zoubaidi, had already lost three of their children to the blood disorder, so they knew the condition’s telltale signs. “When she started showing the same symptoms as her siblings, we immediately thought the worst,” says her father. “We knew there was treatment in America, but we had no idea if we would have the means to get there.”
An inherited disease, SCD causes a child’s red blood cells to malform and become stiff, taking on the shape of a sickle, like the letter C. These abnormally shaped blood cells can stick together and interrupt healthy blood flow throughout the body. This can cause a host of problems that can affect nearly every major organ, but it affects every patient differently. “Some kids don’t have pain or complications until they’re teens, while others suffer strokes and extreme pain and need blood transfusions when they are very young,” says Leslie Lehmann MD, director of the Stem Cell Transplant Program at Dana-Farber/Children’s Hospital Cancer Center (DFCHCC).
Like Maryam, most kids with SCD experience breathlessness, delayed growth, extreme fatigue and pain in their bones from the time they’re young. Kids with SCD also have weaker immune systems, making them susceptible to infections, and they’re at a greater risk for strokes, blindness and fatal iron-overload—another dangerous blood complication. In developed nations, where symptoms can be treated with medication, patients often still only survive until their late 40s. For patients like Maryam in developing countries, even the most basic treatment for these symptoms isn’t usually available, and death at a young age is very common.
The only cure for SCD is a stem cell transplant when the patient is young, similar to a treatment that someone with cancer would undergo. It involves the complete destruction of the immune system (as well as the sickle cells) through chemotherapy, then rebuilding the person’s immune system using healthy stem cells from a matched donor. …