Aussie bub helps cure brain killer
A WORLD-FIRST cure for a rare and fatal brain disease is now saving the lives of newborns around the world thanks in part to Australian doctors.
The brain of “Baby Z” started to disintegrate soon after her 2008 birth, with an enzyme deficiency causing a toxic buildup of sulphite in her body.
No child had ever survived the metabolic disorder Molybdenum cofactor deficiency (MoCD), which affects one in about 150,000 babies and can cause a painful death in the first few months of life.
But Monash Health neonatologist Alex Veldman and the parents, who cannot be named, found a compound was being tested on animals in Germany.
After gaining approval from the Melbourne hospital’s ethics committee and the Family Court for its use in humans, Baby Z received the drug cPMP in her first month. Her sulphite levels normalised and she is now aged seven; however, she suffers severe disabilities.
A study published today in The Lancet shows a daily lifelong infusion is curative in the type A variant of MoCD.
Monash University researcher Flora Wong said while Baby Z was suffering ongoing health issues, she helped save other children. “I think of her as the heroine,” she said.