The mother of a boy with a rare cancer has revealed that her family will spend more than $130,000 so that her son can receive life-saving treatment in the United States because a drug used to help fight the disease is not available in Australia.
sydney boy Harry Sammutaged 10, began complaining of stomach pains and, after a routine check-up and blood tests by her local GP, she was diagnosed with stage four neuroblastoma two years ago and spent 18 months at Westmead Children’s Hospital receiving chemotherapy.
Neuroblastoma is a form of cancer that grows in nerve cells and often develops a tumor in the adrenal glands, just above the kidneys.
The condition, which affects around 40 children each year in Australia, is usually detected in children under five years of age. Harry, however, was diagnosed with the disease shortly before his ninth birthday.
Harry’s mother, Julia, told Daily Mail Australia that she was forced to travel to the United States with her son just before Christmas last year to take part in a clinical trial of a drug that showed promising signs of curing cancer.
Harry Sammut (pictured) has been forced to receive life-saving treatment in the US using a drug effective against the recurrence of a rare cancer that has not yet been approved in Australia.
“We are literally going overseas to pick up the medication because it’s not available in Australia,” he said.
Harry beat cancer in November, however doctors have told the family there is a 50 per cent chance it will return.
The young man underwent several rounds of treatment, including two bone marrow transplants and immunotherapy, in his successful fight against the disease.
Determined to help Harry become completely cancer-free, Julia began doing her own research in the hopes that her son would receive more treatment.
He found a drug called DFMO (difluoromethylornithine) that researchers say is an effective form of treatment for children with recurrent neuroblastoma when combined with chemotherapy.
Harry was accepted into the trial, however Julia said the family had gone into debt to cover the costs of his treatment.
“This test cost us $130,000 over a two-year period,” he said.
‘We could go further into debt if Harry relapses and I can’t go back to work.
“We’re not spending our money, wandering around… we’re just getting by and doing what we need to do to save Harry.”
The U.S. Food and Drug Administration approved DFMO in December last year after clinical trials found that the risk of relapse among high-risk neuroblastoma patients was reduced by 50 percent.
Harry flew to the US with his mother two months ago and is scheduled to make five separate trips over a two-year period to receive treatment and doses of the medication.
“It’s frustrating having to take our child abroad and there are so many health risks with an immunocompromised child,” Julia said.
“If the FDA has approved this drug, there will surely be trials that could start in Australia to consider it for all children, even though it is a rare cancer.”
The 10-year-old’s mother, Julia (pictured right), said the cost of Harry’s treatment will cost $130,000.
Harry (pictured with his mother, Julia) will visit the US five more times for ongoing treatment and doses of medication in a bid to be cancer-free.
Clinical trials of DFMO have not yet been approved in Australia, although trials in adult patients began in July 2023.
At a Senate inquiry hearing in Sydney last month, Neuroblastoma Australia chief executive Lucy Jones criticized Australia’s lengthy drug approval process.
He told the research investigating the diagnosis and treatment of rare cancers that delays in getting life-saving drugs approved need to be reduced.
“I’m horrified to say this, but it’s true, children routinely die in this country because of paperwork,” Ms Jones said.
‘For medicines supplied in hospital settings, MSAC (Medical Services Advisory Committee) approval is required, which can take 18 months.
“However, it does not stop there, as the pharmaceutical company must go through a long negotiation process to reach an agreement on a reimbursement method at the state level.”
Daily Mail Australia has contacted MSAC for comment.
The Therapeutic Goods Administration (TGA), which regulates prescription drugs, vaccines and medical devices in Australia, It can take up to 11 months to review drug safety based on trials conducted by pharmaceutical companies before a prescription drug is approved.