Harry Sammut’s family is facing a crippling bill to get him to the US for treatment unavailable in Australia for his rare disease – as the CEO says, children die here regularly ‘because of paperwork’

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

Sydney boy Harry SammutAt the age of 10 he 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 in the Westmead Children’s Hospital receiving chemotherapy.

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

The condition, which affects about 40 children in Australia every year, is usually discovered in children under the age of five. However, Harry was diagnosed with the disease just before his ninth birthday.

Harry’s mother Julia told Daily Mail Australia 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 was showing promising signs of curing the cancer.

“We literally go overseas to pick up the drugs because they are not available in Australia,” she said.

Harry beat the cancer in November, but doctors told the family there is a 50 percent chance the cancer will return.

The young boy underwent several rounds of treatment, including two bone marrow transplants and immunotherapy, in his successful battle against the disease.

Determined to help Harry get rid of the cancer completely, Julia began doing her own research in hopes of getting her son further treatment.

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

Harry was admitted to the trial, but Julia said the family has gone into debt to cover the costs of his treatment.

“This trial was valued at $130,000 for us over two years,” she said.

‘We could end up in further debt if Harry relapses and I can’t go back to work.

“We don’t spend our money wandering around… we just get by and do what we need to do to save Harry.”

The US Food and Drug Administration approved DFMO in December last year after clinical trials showed a 50 percent reduction in the risk of relapse in patients at high risk for neuroblastoma.

Harry flew to the US with his mother two months ago and will make five separate trips over two years to receive treatment and doses of the medication.

“It’s frustrating that we have to take our child abroad and that you face so many health risks with a child who has a weakened immune system,” Julia said.

“If the FDA has approved this drug, there are certainly studies [that] We could start in Australia so they can consider it for all children, even if it is a rare form of cancer.”

The mother of 10-year-old 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 to receive ongoing treatment and doses of medication in an attempt to be cancer-free

Clinical trials for DFMO have yet to be approved in Australia, even though trials for adult patients started in July 2023.

At a Senate inquiry hearing in Sydney last month, Neuroblastoma Australia CEO Lucy Jones slammed Australia’s lengthy drug approval process.

She told the Inquiry into the Diagnosis and Treatment of Rare Cancers that delays in getting approval for life-saving drugs must be reduced.

“I hate to say this, but it is true that children routinely die in this country because of paperwork,” Ms Jones said.

‘Medicines dispensed in hospitals require MSAC (Medical Services Advisory Committee) approval, which can take 18 months.

“It doesn’t stop there, however, because the pharmaceutical company must then go through a lengthy negotiation process to agree on a reimbursement method at the state level.”

Daily Mail Australia has contacted the 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 for drug safety to be assessed based on studies conducted by pharmaceutical companies before a prescription drug is approved.