May 24, 2017

Spinal Muscular Atrophy Speech – 24 May 2017

I rise to bring to the attention of this chamber an important development, a new treatment, for people who are experiencing spinal muscular atrophy. The drug Spinraza has been developed and successfully trialled and commercialised in the USA for patients with spinal muscular atrophy. This drug was approved by the FDA in December 2016 and is being recommended by the European Medicines Agency for approval within the European Union. The drug is the first of its kind and the first potential genetic treatment option for sufferers of this often fatal disease.

Spinal muscular atrophy is the No. 1 genetic killer of infants under the age of three. It is the childhood version of motor neurone disease and leaves young children who are diagnosed with this disease unable to do simple everyday tasks, such as playing with toys and cutting fruit to eat.

Spinraza works by injecting the protein, which sufferers of spinal muscular atrophy are deficient in, straight into the spine through a lumbar puncture. It is an extremely invasive procedure; however, the families of infants suffering from spinal muscular atrophy are desperate for a treatment option. In most cases, infants who are diagnosed with spinal muscular atrophy have a short life expectancy, as there is currently no other effective treatment options available. This is why Spinraza is such a beacon of hope for children who suffer from spinal muscular atrophy.

The results, according to what I have been advised, speak for themselves. There is a young person, Aviana McElwee, who was born in Darwin in 2016. She was perfectly happy and healthy; however, three weeks after her birth, her movement started to deteriorate. She was diagnosed with SMA just one day after celebrating her three-month birthday. Luckily for her parents, they were invited to participate in a trial of this drug at a Sydney hospital. The effects of the treatment started to show within six to eight weeks after the procedure. Now, Aviana’s physical development has improved so much that she is sitting up unsupported and playing with her toys unassisted.

However, I would not have heard about Aviana’s story if it was not for the work of one amazing individual. I first met Julie Cini eight years ago when she told me the story of her loss and hope. Julie lost two children to spinal muscular atrophy. She also lost her partner in a tragic car accident during this time. Whilst most people would be unable to cope with such tragedy and loss, Julie became inspired to help other families so that there would be, hopefully, one day when people would not have to suffer as she did.

Through her tireless work with her charity, Spinal Muscular Atrophy Australia, she has been lobbying for the Spinraza drug to become available on the Pharmaceutical Benefits Scheme. This drug gives hope for those families who have no other options for treatment. Julie has asked that I bring this matter to the attention of this House and hopes that this drug will proceed through the testing process of the TGA and the Pharmaceutical Benefits Advisory Committee.


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