Miracle cancer medicine curing children’s tumours ‘in days’
Eight year old Jaxon Hach had just months to live, but is walking and talking again after a "revolutionary" new medicine began to reverse his terminal cancer.
The Lego master was diagnosed and treated for a brain stem cancer at 18 months of age but his cancer returned shortly before Christmas last year and his prognosis was not good.
That was until genetic testing of his cancer revealed he carried an NTRK gene fusion that could be targeted by an experimental new treatment called VITRAKVI.
NTRK gene fusions produce abnormal proteins which may cause cancer cells to form and grow rapidly and are found in many different types of cancer.
The new treatment blocks these proteins and shrinks the tumours.
Jaxon's mum Lin Mean said when Jaxon's cancer returned last year he deteriorated quickly and needed a wheelchair.
"He wasn't able to eat again, his walking deteriorated and his speech wasn't great, we couldn't quite understand the words he was saying," she said.
After the treatment she could see the improvement on his scans.
"The last MRI scan he had, just visually I could see that it shrunk, we can actually understand him, like the words actually are words, they don't sound like mumble, he's taking a few steps," she said.
Cancers with NTRK gene fusions can occur anywhere in the body, in both children and adults, but are most common in salivary gland and secretory breast cancer, as well as several paediatric cancers.
The $21,000 a month treatment has just been approved for use in Australia in any cancer where a genetic test shows the patient carries an NTRK gene fusion. But there is no government subsidy available yet.
The genetic test needed to diagnose the cancer costs up to $2000 and experts say our drug subsidy system needed to be modernised to fund this type of precision cancer medicine.
Doctors involved in clinical trials of the drug told News Corp it was "revolutionary" and "spectacular".
"I know one case in Australia where a child under five years of age with a tumour called an infantile fibrosarcoma was in ICU and literally walked out of ICU, after a relatively short exposure to the drug," Garvan Institute researcher Professor David Thomas told News Corp.
"My memories of the chest X-ray which had half the chest filled with this tumour in the little baby and it literally disappeared in the space of a matter of days," he said.
"It can be really quite spectacular," he said.
Dr Jordan Hansford, the head of neuro-oncology at the Royal Children's Hospital in Melbourne, is treating Jaxon. He described the drug as "revolutionary".
In the case of Jaxon "certainly, it has changed his outlook from a matter of months to a much brighter outlook," he said.
If the drug could be used as a first line treatment in childhood cancers young children could avoid damaging radiation and chemotherapy that affects their IQ and cognitive abilities and their motor development, he said.
More than 25,000 patients die from cancer each year and funding the genetic test to identify all those fit enough for these new types of treatments would cost just $42 million a year, Dr Hansford said.
Adverse events from the medicine include fatigue, constipation, dizziness, anaemia, nausea and vomiting.
Bayer Australia has made a submission for a Pharmaceutical Benefit Scheme subsidy for the drug and is working to secure a Medicare rebate for the genetic test.
Originally published as Miracle cancer medicine curing children's tumours 'in days'