Damian Garde in STAT Newss:
Scientists have come up with a drug, injected once a day, that appears to make children’s bones grow. To many, it’s a wondrous invention that could improve the lives of thousands of people with dwarfism. To others, it’s a profit-driven solution in search of a problem, one that could unravel decades of hard-won respect for an entire community. In the middle are families, doctors, and a pharmaceutical company, all dealing with a philosophically fraught question: Is it ethical to make a little person taller? The most common cause of dwarfism is known as achondroplasia. People with the condition, caused by a rare genetic mutation, have shorter limbs and shorter stature than those without it, and they deal with a lifetime of skeletal issues that often require a battery of corrective surgeries.
For years a U.S. company called BioMarin Pharmaceutical (BMRN) has been developing a drug that targets the genetic roots of achondroplasia, a mutation that stops cartilage from turning into bone. The goal, according to the company, is to prevent the medical complications associated with achondroplasia, which include sleep apnea, hearing loss, and spinal problems. But proving the drug’s long-term skeletal benefits would require a decades-long study, which is an expensive and, to BioMarin, impractical proposition. Instead, the company is measuring the the most immediate byproduct of bone growth: height. And that’s where the philosophical schism begins. For thousands of little people, the short stature that comes with achondroplasia is not a disability in need of treatment but a difference to be celebrated. Organizations like Little People of America have spent years dispelling stereotypes, advocating for fairness, and pointing out that having achondroplasia doesn’t preclude anyone from a fulfilling life. Dwarf pride means flourishing because of one’s body, not in spite of it.
Through that lens, the drug looks to some like a threat of erasure, a so-called cure for people who are not sick. Furthermore, about 80% of babies with achondroplasia are born to parents of average height. That means, if the drug goes on to win Food and Drug Administration approval, the decision of whether to give it to a child would often be made by people with no exposure to little people culture, who know nothing of dwarf pride. “People feel like this is an effort to eliminate the dwarfism community,” said Becky Curran Kekula, a disability advocate and motivational speaker who was born with achondroplasia.