Resisting Pediatric Research and Missing Chances for Cures

Is it riskier to give children experimental treatments, or not to?
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Romeo Ranoco/Reuters

In Colorado, a scientist places a needle in a developing embryo. He removes the cells and melts them to isolate pure DNA. Then he experiments on that DNA. This is the earliest research we can perform on a human. They select a child free of the perils of breast cancer and then implant that child into her mother. After the medical meddling, she becomes pregnant. 

A thousand miles away, in rural Mississippi, an HIV positive mother gives birth. This mother couldn’t be more different from the one in Colorado. She’s received none of the benefits of prenatal care, none of the drugs that might possibly spare her newborn from a lifetime of HIV. Desperate, her doctors do something they don’t usually do: they give the baby an aggressive mix of drugs

Both of these children, born from adversity, represent a revolution in science. Because of the experimentation performed in their earliest hours, these two newborns are protected from two deadly diseases: breast cancer and HIV. Today they are healthy three-year-olds, not unlike my own daughter.

We have a thorny history with pediatric research. We either do too much or too little. The challenge of conducting research in children is manifest. They are a vulnerable population, one whose bodies are in constant flux, and who cannot suitably consent for the procedures they undergo. Despite these limitations, withholding research in children yields an equal disservice. In the 1950's, the drug thalidomide caused tens of thousands of deaths and deformities in children because its influence on the developing fetus wasn’t evaluated.

On the other side of the spectrum, the genetic roots of some adult diseases, such as cardiomyopathies and colorectal cancer, were elucidated only after studies in children. Because of the challenges of pediatric research, it’s rare that a disease is solved in children before adults. For breast cancer and HIV, advances in pediatrics are as unexpected as they are unprecedented.

The child in Colorado was screened as an embryo for a gene called BRCA. This gene carries an increased risk of breast cancer. By weeding out the embryos with the gene, this little girl is spared the fears of her mother, whose genetic code includes BRCA. This kind of analysis, called pre-implantation genetic testing, is only one facet of the growing field of embryo research. The Food and Drug Administration is also considering approving the creation of three-parent embryos. This technique could spare babies from mitochondrial diseases. In this rare group of diseases, the cell cannot keep up with energy demand, resulting in blackouts of body function. The diseases are difficult both to diagnose and treat. For women with a drained cell battery, mixing in the mitochondrial DNA from another woman could save lives. But there’s no denying this powerful research is risky and controversial.

In Mississippi, doctors hoped that their daring treatment might prevent the mother’s HIV from infecting her baby girl. Instead, they watched as the virus gained a foothold in the infant’s body. On her medical records they marked her HIV status as positive. From that day forward doctors assumed she’d have to take medication to keep the virus in check. But then something odd happened. At a check-up her mother admitted she hadn’t given the baby her medication in five months. She started missing doctor appointments. Unexpectedly, her HIV didn’t come back. Puzzled, her doctors ordered tests. Where did the virus go? They could only find remnants of HIV, like wounded soldiers unable to fight, hiding in the girl’s cells. This baby is now a healthy three-year-old. Last week, we learned she’s not alone. A second baby, treated even sooner, within four hours of birth, is similarly cleared of the virus. This research is part of a trend in pediatric HIV; one that many believe will result in children being the first population cured of the disease.

When I look at my three-year-old daughter I can’t imagine her participating in a research study. I’m a scientist and yet the idea of her being given an aggressive drug regimen to cure her of HIV, or having her embryo poked with a needle and then analyzed, is terrifying. But the only thing more terrifying is the thought of her sick and dying.

Children need our protection. First we have to figure out what to protect them from. Do we shield them from highly experimental research? Or do we pursue cures at all costs? Our answer has to be as nuanced as the science itself. In our debate over research in children, these two, healthy toddler girls serve as positive examples. They represent the best science has to offer. Research based on these two cases has the potential to reach beyond pediatrics. Their experiences will inform new therapies to treat disease in adults. There may be nothing more frightening than a research study performed on our children, unless it’s a baby with an incurable illness.

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Nathalia Holt is a microbiologist and author of Cured: How the Berlin Patients Defeated HIV and Forever Changed Medical Science and Rise of the Rocket Girls: The Women Who Propelled Us From Missiles to the Moon to Mars.

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