The Burden of
Proof
Kids helping kids helping science— that’s the promise of pediatric clinical trials by Elizabeth Cooney
Four doctors file into Becca’s hospital room on their daily rounds, thir-
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teen days after the teenager received a stem cell transplant. They rub their hands with sanitizing gel, then proceed to listen to her chest. The stethoscope they use stays in her room; when not in use, it hangs from the IV pole. An array of hair bows pinned next to cheerleading pictures on one wall leads a doctor to venture that Becca’s room could win a best-decorated contest. The 15-year-old says she always wears a bow in her hair. Not losing her hair to chemotherapy is perhaps the most tangible sign of the gentler approach doctors are taking to treat her rare disease. Dyskeratosis congenita is an inherited disorder in which telomeres—the protective caps on the ends of chromosomes— are not properly maintained, a situation that can lead to changes in the controls on cell division. Becca’s rapidly dividing cells burn out quickly, leading to bone marrow failure. Because her cells do not repair damage as well as they should, she is more susceptible to cancer and organ failure. The chemotherapy or radiation typically used to prepare a body for a bone marrow transplant is extraordinarily harsh, and Becca’s condition makes her even more vulnerable to the aggressive nature of these conventional tools. To help her body accept the donated stem cells, doctors instead gave Becca immunosuppressive drugs only. In other studies, this approach has shown that
the more robust cells will outcompete the leftover damaged cells and will survive. One of Becca’s doctors wears two hats: clinician and researcher. He oversees her care—and the research study’s protocol. Becca is one of five participants in the pilot study. Her parents wouldn’t have it any other way. Their unease with having their daughter be one of only a handful of patients in the world to receive this alternative treatment was calmed when they learned that their daughter’s doctor is an expert in her disease. Today, the disease neither of them had heard of until Becca’s diagnosis four months ago has become their own object of study. They know her dates and diagnoses; they understand there is a 10 percent risk that her body won’t accept the transplanted cells or be able to produce its own. They check her online patient portal daily to see what her blood counts are, willing her absolute neutrophil count above 500, the threshold of transplant success.
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