Harry Sammut’s family face a crippling bill to get him to the US for treatment not available in Australia for his rare disease – as CEO says kids regularly die here ‘because of paperwork’

The mother of a young boy with a rare cancer has revealed her family are set to spend more than $130,000 so her son can receive life-saving treatment in America because a drug used to help fight the disease is not available in Australia. 

Sydney boy Harry Sammut, 10, started complaining of stomach pains and after a routine check up and blood tests by his local GP, he was diagnosed with stage four neuroblastoma two years ago and spent 18 months at the Children’s Hospital Westmead receiving chemotherapy.

Neuroblastoma is a form of cancer that grows in nerve cells and a tumour often develops in the adrenal glands just above the kidneys.

The condition, which affects around 40 children in Australia annually, is usually detected in kids under five years of age. Harry, however, was diagnosed with the disease just shy of his ninth birthday. 

Harry’s mother Julia told Daily Mail Australia that she was forced to travel to the US with her son just before Christmas last year to take part in a clinical trial for a drug that showed promising signs to cure the cancer. 

Harry Sammut (pictured) has been forced to get life-saving treatment in the US using a drug effective against re-emergence of a rare cancer that is yet to be approved in Australia

‘We’re literally going overseas to pick the medication up because it’s not available in Australia,’ she said. 

Harry beat the cancer in November however doctors told the family there is a 50 per cent chance it will return. 

The young boy went through several rounds of treatment including two bone marrow transplants and immunotherapy in his successful fight against the disease. 

Determined to help Harry be completely free of the cancer, Julia began doing her own research in the hope of getting her son further treatment. 

She came across a drug called DFMO (difluoromethylornithine) which researchers have found is an effective form of treatment for children with relapsed neuroblastoma when combined with chemotherapy. 

Harry was accepted into the trial however, Julia said the family has been plunged into debt to cover the costs of his treatment.

‘This trial was estimated at $130,000 for us over the two-year period,’ she said. 

‘We could go into debt even further if Harry relapses and I can’t get back to work.

‘We’re not spending our money, galivanting around…we’re just purely getting by and doing what we need to save Harry.’ 

The Food and Drug Administration in the US approved DFMO in December last year after clinical trials found the risk of relapse among patients with high risk neuroblastoma was reduced by 50 per cent. 

Harry flew to the US along with his mum two months ago and is set to make five separate trips over a two-year period to receive treatment and to be given doses of the medication.

‘It’s frustrating that we have to take our child overseas and there’s so many health risks you’re taking with a child that is immunocompromised,’ Julia said. 

‘If the FDA has approved this medication, surely there’s trials [that] could start in Australia for them to consider it for all children even though it’s a rare cancer.’

The 10-year-old's mother Julia (pictured right) said the cost of Harry's treatment is set to cost $130,000

The 10-year-old’s mother Julia (pictured right) said the cost of Harry’s treatment is set to cost $130,000

Harry (pictured with his mum, Julia) will visit the US five more times to receive ongoing treatment and be provided with doses of medication in a bid to be cancer free

Harry (pictured with his mum, Julia) will visit the US five more times to receive ongoing treatment and be provided with doses of medication in a bid to be cancer free

Clinical trials for DFMO are yet to be approved in Australia even though trials for adult patients got underway in July 2023.

At a Senate inquiry hearing in Sydney last month, Neuroblastoma Australia CEO Lucy Jones slammed the lengthy approval process for drugs in Australia. 

She told the inquiry investigating the diagnosis and treatment of rare cancers that delays in getting approval for life-saving medication needs to be reduced. 

‘It appalls me to say this, but it’s true, children routinely die in this country because of paperwork,’ Ms Jones said. 

‘For drugs provided in hospital settings, MSAC (Medical Services Advisory Committee) approval is required, which might take 18 months.

‘However, it doesn’t stop there, as the pharmaceutical company then needs to go through a lengthy negotiation process to agree on a method of reimbursement at the state level.’ 

Daily Mail Australia contacted the MSAC for comment. 

The Therapeutic Goods Administration (TGA) which regulates prescription medication, vaccines, and medical devices in Australia, can take up to 11 months to review the safety of drugs based on the trials conducted by pharmaceutical companies before a prescription medication is approved.

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