Dwarfism drug offers ‘nearly normal’ growth
Controversial treatment could allow children with achondroplasia to grow at almost the normal rate
People with Dwarfism are divided over the prospect of a drug that could fight the condition by helping children with the genetic abnormality grow at almost the normal rate. A US Biotech firm has submitted the results from trials of vosoritide to US and European regulators, making its release possible within months. Children who took the drug grew an additional 0.6 inches on average in one year, with minimal side effects. But some activists said the new drug was “divisive”.
PEOPLE with dwarfism are divided over the prospect of a drug that could fight the condition by helping children with the genetic abnormality grow at almost the normal rate. An American Biotech firm, Bio-marin, has submitted the results from trials of vosoritide to US and European regulators, making its release possible within months.
Dwarfism is defined as an adult height of less than 4ft 10in. It affects around one in 25,000 infants.
Vosoritide was shown in tests to increase the growth rate in children with the most common form of dwarfism, achondroplasia, according to a study in The Lancet. Trials were carried out in seven countries – Australia, Germany, Japan, Spain, Turkey, the USA and the UK. Scientists recruited 121 children to participate, with half receiving the drug and half receiving a placebo. Children who took the drug grew an additional 0.6 inches on average in one year, with minimal side effects.
If taken over many years, scientists reported, vosoritide could produce a significant increase in adult height – although the study was limited to a year and does not look at other health problems associated with dwarfism. Vosoritide utilises a synthetic form of a protein that humans produce naturally. It targets the overactive signal that prevents bone growth.
The issue of drugs to combat dwarfism is controversial in the United States. Mark Povinelli, president of the Little People of America, told the New York Times that vosoritide “is one of the most divisive things that we’ve come across in our 63-year existence.
“We want to show that you can have a completely fulfilling life without having to worry about growth velocity.”
More than 80 per cent of those with achondroplasia are born to parents of average stature, and a child with the condition has a 50 per cent chance of passing it on to their own children.
Melissa Mills, of Jacksonville, Florida, said her four-year-old daughter Eden would use vosoritide if it was approved by the Food and Drug Administration. “The world wasn’t built for my child, so if there is something I can do to help her navigate the world a little bit better and on her own, I want to do it,” she said.
The scientist who led the trial, Dr Ravi Savarirayan of the Murdoch Children’s Research Institute in Melbourne, said the drug could be life-changing. “We’ve got 12- and 13-year-old girls who now, for the first time, can do their own feminine hygiene,” he said.
Megan Schimmel, who, like her husband Jeremy and two-year-old daughter Lily Grace, has dwarfism, said she was uninterested in the drug.
“I can do everything that someone a foot taller can do, with minor accommodations,” she said. She felt that vosoritide sent a message that those with achondroplasia “are broken”.