Luna Kemmery is a pint-sized fighter, displaying bravery beyond her years.
The Brisbane toddler has endured five rounds of chemotherapy, surgeries, stem cell therapy, a bone marrow transplant, radiation and immunotherapy in her battle against a rare form of childhood cancer.
“She’s your regular two-year-old — happy, sassy, bubbly, very, very active,” mum Samantha Byrne said.
“Given everything, we thought she would have slowed down a little bit — but, if anything, it’s ramped her right up.”
Luna was diagnosed with stage 4 neuroblastoma in July last year.
It is a cancer which most commonly and cruelly affects children, with the average age at the time of diagnosis of just two years old.
“From as young as 11-days old, we were in and out of hospital for some breathing issues,” Ms Byrne said.
“Now we can see … it was something a bit more sinister.”
Luna’s future rests on access to a drug treatment available in the United States, but not currently used in Australia.
Known as DFMO, the Therapeutic Goods Administration (TGA) is considering its approval in Australia.
But, there are fears even a streamlined application process could take too long, and parents of kids like Luna are pleading for federal government support to help cover the exorbitant costs of getting the drug overseas.
“It’s as simple as a couple of tablets a day for a two-year period,” Ms Byrne said.
“It does come at a hefty cost though — it’s $500,000 for the course of treatment, which is around $21,000 a month.”
The miracle drug helping kids avoid the return of cruel cancer
The outlook for children diagnosed with neuroblastoma is devastating. About half do not survive.
Even after successful initial treatment, the relapse rate is shockingly high at about 60 per cent — and survival rates from there plummet to just 5 per cent.
DFMO was trialled in the US for four years, and was found to reduce the risk of relapse by 50 per cent, helping about three-quarters of children with the neuroblastoma diagnosis survive the deadly disease.
Chief executive of Neuroblastoma Australia, Lucy Jones said it is a difficult wait for the TGA to consider use of the drug in Australia.
The TGA has given the drug “orphan” status, which allows a fast tracked approval process of up to 12 months for a drug which could fill a significant gap in available medical treatment.
“Families desperately need this drug now, their children cannot wait, neuroblastoma does not wait,” Ms Jones said.
“We really need to get the approval for that drug as soon as possible, or if not we need the government to find an interim solution to provide access to this drug to these vulnerable children immediately.
“We don’t really want paperwork to get in the way of saving young children’s lives.”
About 50 Australian children are diagnosed with neuroblastoma every year.
For Ms Jones, this campaign is personal. She established Neuroblastoma Australia after losing her daughter Sienna, aged just two-and-a-half.
“We are asking the federal government to fund access to this drug in this country, to avoid children having to go to America,” she said.
“Right now families are having to fundraise hundreds of thousands of dollars to take their vulnerable children overseas to get that drug.”
Pleas for government action
In April, Neuroblastoma Australia and the Australian and New Zealand Children’s Haematology and Oncology Group asked the Health Minister Mark Butler to commit to $15 million in funding over two years.
That would cover treatment during the period prior to formal TGA approval in Australia.
A spokesperson for the minister said he “remains concerned about the situation faced by children with neuroblastoma in Australia”.
“The priority is for children to be able to access safe treatment, either overseas or in an Australian hospital,” the spokesperson said.
“This includes looking at support for families for treatment overseas through the Medical Treatment Overseas Program which supports families at no cost to access treatments they can’t currently access in Australia.
“The minister and his staff have discussed accelerating approvals and access to treatment for neuroblastoma with both health authorities and with drug manufacturers.”
Mr Butler’s counterpart, Shadow minister Anne Ruston said the benefits of DFMO were clear for all to see.
“It is our hope that the government will support these families with accessing this lifesaving medication until DFMO is finally able to be listed on the PBS,” she said.
In Senate estimates in early June, officials from the federal health department said public hospitals could choose to provide access to the drug if they chose to.
“Public hospitals are not restricted in terms of the medicines that they can fund in the way that the Commonwealth is through the Pharmaceutical Benefits Scheme and the legislative requirements we have around that,” Health department deputy secretary Penny Shakespeare said.
Ms Jones said hospitals do not have the budget to cover the cost fo the drug up-front.
“It doesn’t really help the matter by suggesting something which is totally improbable and impossible,” she said.
Luna’s parents hope an announcement could be imminent.
“I just really hoping that Mark Butler will be able to help us in our play to get the treatment to Australia. so we don’t have to uproot and split our family to travel overseas to get the medication that she needs to survive and thrive,” Ms Byrne said.