’Life-changing’ drug for kids with dwarfism divides community

This toddler will get daily injections until he’s 18 as part of a controversial drug trial - and not everyone is happy about it.

Daisy Broadway knows there will always be people against what her and her partner Justin Ebbels are doing for their son Casper.

The Melbourne bub was enrolled in a controversial drug trial when he was months old and will receive daily injections until he’s 18 and old enough to decide for himself if he wants to continue treatment.

The world-first treatment, led by the Murdoch Children’s Research Institute, is allowing those with achondroplasia to grow at the same rate as their peers and possibly avoid surgery.

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Daisy Broadway says they’re doing what they think is best for Casper’s health.

Achondroplasia, the most common form of dwarfism, is caused by a gene mutation affecting about one in 25,000 babies and until recently there was no treatment.

Dr Ravi Savarirayan, who leads the study 18-month-old Casper is taking part in, has found vosoritide can increase the annual growth rate in children with achondroplasia.

“It is controversial and it’s a tricky one and I sort of feel sometimes I have to filter who I talk to about it,” Ms Broadway said.

“There’s some people who are vehemently against it and that’s totally fine. Their attitude is they’re trying to fix something that doesn’t need fixing and undoing a lot of work that’s been done in short statured pride.

“I think they’re very valid points, however at the end of the day we made a decision we believe is in our son’s best interests and I hope people respect that.”

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Casper will receive the drug every day until he’s at least 18.

Daisy and partner Justin didn’t know Casper had achondroplasia until a couple of weeks after he was born.

Ms Broadway said it was very important to emphasise they were not trying to “change” their son or weren’t proud of him.

“It was very hard to come to terms with obviously, but for us it’s more than aesthetics and cosmetics, it’s reducing the risk of Casper needing medical intervention as he grows,” she said.

Children with the condition have impaired bone growth in the limbs, spine, and base of the skull. They may also have curvature of the spinal cord, bowed legs and spinal cord compression, with about half needing corrective surgery.

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MCRI clinical geneticist Professor Ravi Savarirayan has spent almost 10 years working to counter inhibited growth and other complications of the condition.

Dr Savarirayan explains on Dateline on SBS tonight that Casper’s parents are on the “front lines”, taking the risk to put their child through a trial with unknown side effects.

“If it works as hoped, vosoritide could help children with achondroplasia grow taller and avoid surgery,” he said in an article for the institute.

“This could be life-changing for many children and their families.”

Ms Broadway said after Casper turned 18 he could decide whether he wanted to continue treatment because participants could take the drug until they reached their final adult height, which could be early to mid-20s.

“Hopefully it will improve the quality of life for kids like Casper and other kids and hopefully he will be able to avoid some of the nasty health complications that go along with his condition,” she said.

You can watch Dateline’s episode Born Small tonight at 9.30pm on SBS