Neuroblastoma treatment option expands with IFINWIL®'s inclusion on Australia's Pharmaceutical Benefits Scheme
In a significant development for families affected by high-risk neuroblastoma in Australia, a world-first clinical trial of copper chelation therapy combined with chemo-immunotherapy is underway. This innovative approach aims to overcome immune suppression by the tumor and enhance existing therapies, potentially offering a promising advancement in treating this aggressive childhood cancer.
Neuroblastoma, a cancer that originates in the body's nerve cells (neuroblasts), typically presents as a primary tumour in the adrenal glands. Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high-risk neuroblastoma (HRNB), a rare but aggressive form of cancer predominantly affecting children and most commonly presenting in the first 5 years of life.
The therapy uses trientine tetrahydrochloride to chelate copper, combined with drugs dinutuximab-beta, irinotecan, and temozolomide. Copper chelation is expected to safely enhance treatment effectiveness because copper modulation can reprogram immune cells in the tumor environment to fight cancer more effectively, potentially improving outcomes of therapies such as CAR-T cell therapy.
The ongoing clinical trial, primarily assessing safety and tolerability, is anticipated to recruit 10 patients starting in 2025 and lasting about five years. If safety is established, the treatment's effectiveness will be rigorously tested in subsequent larger international clinical trials (BEACON2 study).
Regarding risks, copper chelation medication (trientine) is widely used in children with few side effects, so the trial aims to confirm its safety in combination with chemo-immunotherapy. The PBS listing of eflornithine, a new treatment for high-risk neuroblastoma, is another recent development in Australia.
Eflornithine, marketed under the brand name IWILFIN, offers a new approach in the limited therapeutic pathways for high-risk neuroblastoma. The PBS listing comes 3 months after its registration by the Therapeutic Goods Administration (TGA). Parents should speak to their doctor for guidance on the best treatment approach for their child regarding eflornithine.
The IFINWIL Consumer Medicines Information (CMI) provides safety information on risks, side effects, and precautions associated with eflornithine, including the risk of anaemia, low neutrophils, low platelets, increased liver enzymes, and hearing loss or problems balancing.
Lucy Jones, CEO of Neuroblastoma Australia, has welcomed the PBS listing of IFINWIL, stating, "This is a landmark moment for families affected by high-risk neuroblastoma in Australia." Gus Rudolph, General Manager of Norgine's Australia division, expressed gratitude towards the Australian Government for providing access to eflornithine.
Jones also expressed gratitude towards the Federal Government for their support in ensuring early access to this treatment. The PBS will make eflornithine available to all Australian families, marking a significant step forward in the fight against high-risk neuroblastoma.
As research continues into immune modulation and personalized medicine strategies, the hope is that these advancements will lead to improved survival and quality of life for children with neuroblastoma.
Science has shown promise in a novel treatment approach for high-risk neuroblastoma, with a clinical trial underway that combines copper chelation therapy and chemo-immunotherapy. This medical-condition, known as neuroblastoma, primarily affects children and is often aggressive, requiring effective treatments like the new medication eflornithine.
The ongoing trial aims to safely enhance treatment effectiveness using trientine tetrahydrochloride, which can reprogram immune cells in the tumor environment to fight cancer, potentially improving health-and-wellness outcomes for children with high-risk neuroblastoma.
