“People like me are endangered and now they want to make me extinct.”
A drug to improve the quality of life of children with achondroplasia, the most common form of dwarfism, is coming under fire for reinforcing stigma.
Early last month, Australian researchers published an article in The Lancet claiming that an experimental drug, vosoritide, appears to return growth rates to normal.
Professor Ravi Savarirayan, of Murdoch Children's Research Institute, in Melbourne, says that: “This drug is like releasing the handbrake on a car, it lets you get up to full speed instead of having to drive with the brakes on.”
Achondroplasia is a genetic bone disorder affecting 250,000 people worldwide, or about one in every 25,000 children. It is caused by a mutation in the FGFR3 gene that impairs bone growth and means that children grow around 4 cm per year, instead of the usual 6 to 7 cm.
Current achondroplasia treatments, like surgery, only address the symptoms. Vosoritide is a precision therapy directly targeted at the molecular cause of the disease. But, according to a feature in The Guardian, some people with dwarfism question whether it is ethical.
Leah Smith, a spokeswoman for Little People of America (LPA), the largest organisation in the US for people with dwarfism, said [in 2015]: “People like me are endangered and now they want to make me extinct.” Recently, the actor Mark Povinelli, who is president of the LPA, told the New York Times that the drug “is one of the most divisive things that we’ve come across in our 63-year existence”.
There are, says Gillian Martin, a tutor and chair of the Restricted Growth Association, the British charity that supports people with dwarfism, “people who are genuinely afraid of where this could lead”. Because about 80% of children with achondroplasia are born to parents without it, some “adults with dwarfism in the community feel that average-height parents are being an advocate for a disability that doesn’t directly affect them. There is a fear – irrational in my view – that this research is leading the way to eradicate dwarfism.”
One issue is that parents have to consent to the treatment because the drug only works on children.
Michael Cook is editor of BioEdge
- How long can you put off seeing the doctor because of lockdowns? - December 3, 2021
- House of Lords debates assisted suicide—again - October 28, 2021
- Spanish government tries to restrict conscientious objection - October 28, 2021