First of its kind medicine harnesses
DNA profile to target cancer
14 September 2020 – The first 'pan-tumour' medicine to harness DNA profiling to target and treat a specific form of cancer in the youngest of children, as well as adults, has been registered for use in Australia.
VITRAKVI® (larotrectinib) can be used to treat more than 15 types of cancer with a distinct genetic alteration known as NTRK (neurotrophic tyrosine receptor kinase) gene fusion, and where the cancer has advanced or spread and cannot be controlled by other therapy.
Until now there has been no proven targeted treatment for this unique form of cancer that can occur in any part of the body – from the skin or bones to the breast or kidney – irrespective of age.
NTRK fusion is a gene abnormality that occurs for no apparent reason and acts as an 'ignition switch' for tumour growth. VITRAKVI works by blocking the protein that is produced following the fusion process, thereby halting tumour development.
Professor David Thomas, Head of the Genomic Cancer Medicine Laboratory at the Garvan Institute of Medical Research and Director of The Kinghorn Cancer Centre described the Therapeutic Goods Administration approval of VITRAKVI as a "major milestone".
"Cancer is a disease of the DNA. We know that genetic information holds the key to treating it most effectively," he said.
"We estimate that 'gene fusions' are responsible for one-in-five cancers. While NTRK gene fusion is just one piece of a bigger puzzle, it is exciting to be able to test for and treat cancers caused by this specific gene abnormality."
To date, nine Australian cancer patients have been treated with VITRAKVI on compassionate, last resort grounds after tests confirmed the presence of NTRK gene fusion.
Associate Professor Jordan Hansford, Paediatric Oncologist at The Royal Children's Hospital Melbourne, said that VITRAKVI allows "treatment to be matched to the genetic blueprint of the cancer irrespective of tumour location or the age of the patient".
"Treating childhood cancer is enormously challenging. It is often a balancing act, using aggressive chemotherapy and radiotherapy while working to minimise the potential for long-term damage or developmental side-effects," he said.
"Genetic testing of young patients to find out what is driving their particular cancer helps doctors select treatment approaches that target the root of the disease."
The Kids' Cancer Project Chief Executive Owen Finegan said VITRAKVI offers new hope to the parents of children with some of the hardest to treat cancers.
"We are thrilled that scientific discoveries are leading to more treatments for our most vulnerable children," he said.
"Every day, three Australian families discover their child has cancer. But even more devastating is that three kids are dying each week from this indiscriminate disease, making it the number one killer by disease in Australian children," he said.
"Understanding the genetic makeup of cancer and being able to target the treatment of it not only means more children will survive, but those that do, will thrive."
Cancers with NTRK gene fusions can occur anywhere in the body, in both children and adults, but are most common in salivary gland and secretary breast cancer, as well as several paediatric cancers.
Bayer Australia confirmed it has made a submission for a PBS subsidy and is working to secure an MBS item number so patients can readily access the necessary testing to identify cancer with this specific genetic blueprint.
"Bayer shares the cancer community's excitement about the registration of this truly novel therapy and is working towards securing access to effective testing and treatment for patients at the earliest opportunity," said Bayer Pharmaceuticals ANZ General Manager, Ashraf Al-Ouf.
"TGA registration of VITRAKVI is an exciting advance in precision therapy as it will enable patients with around 15 different types of cancers to receive the same treatment. Rare Cancers Australia is hopeful that the reimbursement applications will navigate the system quickly so that patients will soon have access to publicly funded NTRK screening and, for eligible patients, treatment with VITRAKVI on the PBS," said Richard Vines, Chief Executive, Rare Cancers Australia.
