Wonder drug helping children with dwarfism grow faster with no negative effects

A wonder drug is giving babies with dwarfism a new lease on life, helping children grow at a rate almost on par with their peers who don’t have the rare bone condition.

Tori English is part of a trial of a new drug that is seeing children with dwarfism increase their rate of growth. Picture: Simon Dallinger

Babies with the most common form of dwarfism now have the chance to be treated with a wonder drug that is booting the growth of older children with the bone growth disorder.

A first-in-human Melbourne-led trial has seen young people with achondroplasia grow 50 per cent faster each year — from 4cm to 6cm — through daily injection over almost four years.

This is almost on par with peers who don’t have the rare genetic condition, and there were no negative effects on body proportion or bone composition.

Murdoch Children’s Research Institute lead researcher, Professor Ravi Savarirayan said while the aim of the world-first international study was to relieve children of the medical complications from being short statured such as sleep problems, bowed legs and compression of the spinal cord, he was hopeful it would also increase their final adult height.

“Children’s legs are getting straighter, so they may not need surgery. For some of the girls, it’s the first time they are able to toilet themselves without needing help,” Prof Savarirayan said.

“They are able to run faster, get less tired, reach things and be independent.

Tori English, 12, is running cross country and competing in athletics since joining the trial. Picture: Simon Dallinger

“We’re at a tipping point right now. It’s going to change practice forever.”

Results from the phase II trial of Vosoritide, involving 35 young people aged five to 14 years, are published today in the prestigious New England Journal of Medicine.

Most of these children will receive the drug until they stop growing, around age 18 to 20 years.

It works by acting on the genetic mutation impairing growth of bones in the limbs and spine.

Following these results, the two larger trials — one involving kids aged five to 18, and another pitting the drug against placebo in newborns to age five — are already recruiting participants.

“All the standard answers I gave parents to their questions of how tall their child will be, the incidence of dying in the first five years and the chance of operations — it’s changing everything I tell them, because we are redefining it all,” he said.

“It won’t eradicate the condition, but I’m hoping it will eradicate the medical complications.”

Professor Ravi Savarirayan with patient, Tori English, who took part in the trial. Picture: Mark Stewart

MORE: COULD CANNABIS HELP TREAT PTSD?

VICTORIA’S SHOCKING RATE OF BIRTH HEARTBREAK

In Prof Savarirayan’s office, there are photos of Tori English scaling a rock climbing wall on school camp — the first time this has been possible.

The 12-year-old from Wodonga is one of the 80 per cent of people with achondroplasia who have parents of average height, with the condition caused by a spontaneous genetic mutation.

Tori has received the drug for the past four years and is now able to be more active, she can brush her own hair and stand in the shallows of the swimming pool for the first time.

“It’s amazing what an extra 2cm a year can actually do,” said her mum Anthea.

“If these babies get that growth over 16 years, they’re going to be a foot taller at the end of it and perhaps drive cars without pedal extenders, reach bank machines and shop counters.

“I think it’s going to change the face of achondroplasia.”