By Cam Lucadou-Wells
A Berwick woman has travelled to the halls of Federal Parliament in the latest step to make a life-saving drug accessible to children afflicted by a rare, cruel disease.
Julie Cini met with Health Minister Greg Hunt on 28 March, arguing for the drug Nusinersan (Spinraza) to be fast-tracked onto the PBS list for children suffering Spinal Muscular Atrophy by June.
She is hopeful of favourable news when Pharmaceutical Benefits Advisory Committee announces new PBS listings on 20 April.
“Every time I go to bed I will have to talk with the man upstairs.”
Without the PBS, the drug is prohibitively expensive – about $750,000 for a 12-month course of six injections, she says.
Up to 25 families have been given access to the drug – under a compassionate scheme offered by its pharmaceutical manufacturer.
More than 200 families still await access.
Without the drug, the illness destroys motor neurons and muscles – the means for children to crawl, walk, feed, sit, cough, swallow and breathe.
Dubbed the childhood version of Motor Neuron Disease, it is the No.1 genetic killer of infants aged under 3.
The drug Nusinersen has “phenomenal” properties, halting the disease’s degenerative march and increasing patients’ motor-function.
“Normally type-1 SMA children have only 12 months of life,” Ms Cini says.
“Children on this treatment are now seeing their second birthday and they’re growing stronger.”
Timely access is the difference between living and dying, walking and not walking, she says.
Ms Cini knows too well the cruelty of the illness, losing two baby daughters Montanna and Zarlee to type-1 SMA.
She founded and heads the Hallam-based charity Spinal Muscular Atrophy Australia, and has fought for the PBS listing for the past two years.
She said she was “blown away” by the support of a 11,201-signature petition, which was tabled in Parliament by Holt MP Anthony Byrne on 28 March.
Mr Byrne said he was honoured to present the petition, watched on by families with children with SMA.
“Every day that passes without this drug means that parents have to watch their children’s motor skills deteriorate, unable to do simple tasks…
“These families live in hope that one day soon they too will be able to access this vital drug.”
