Melanoma spread to the brain "now appears to be curable"
18 February 2025
Newly released long-term data from a groundbreaking clinical trial for advanced melanoma patients whose disease had spread to their brain has proven that long-term disease control has been achieved for over 50% of patients given combination immunotherapy as first line treatment.
The impressive 7-year follow up results from the ABC clinical trial were published today in the high impact journal Lancet Oncology.
Brain metastases are present in 30-40% of patients at diagnosis with Stage 4 (advanced) melanoma. In the past, these patients only survived for around 16 weeks.
The ABC trial was the first to demonstrate that these advanced melanoma patients could be successfully treated with combination immunotherapy using nivolumab and ipilimumab. So impressive were early survival rates from the trial, released in 2018, clinical practice changed virtually overnight.
The 7-year follow-up results released today show overall survival of 48% of patients on combination immunotherapy, with the survival rate increasing to 51% in patients given the treatment upfront.
The randomized, phase 2 study was conducted at four sites in Australia – Melanoma Institute Australia, Princess Alexandra Hospital, Royal Adelaide Hospital and Peter MacCallum Cancer Centre.
"Patients in this long-term study have been followed for seven years now, and the results are fundamentally changing how we think about melanoma that has spread to the brain,’ said Professor Grant McArthur AO who heads Peter Mac’s Molecular Oncology Laboratory and is co-senior author on the study.
"A situation that was considered terminal and with very limited treatment options now appears to be curable, based on these new data.
"More broadly, this continues the trend we’ve seen over recent decades as new immunotherapy-based approaches emerge and change advanced melanoma into not only a manageable disease but a curable disease for some patients,’ he said.
Between 2014 and 2017, 79 patients were enrolled in the ABC trial, with 36 given combination checkpoint inhibitor immunotherapy (anti-PD-1 plus anti-CTLA-4) and 43 given single agent immunotherapy (nivolumab).
Initial results released in 2018 showed a 46% response rate for those patients given the combination immunotherapy, versus just 20% response rate for those on single agent immunotherapy.
The 7-year results, released today, show progression free survival rate was 42% with ipilimumab plus nivolumab, compared to 15% with nivolumab alone. Overall survival was 48% and 26% respectively.
In patients treated upfront, or as first line treatment, 7-year progression free survival rate was 47% with the combination immunotherapy and 14% with the single agent, with overall survival 51% and 29% respectively.
This is the longest follow-up ever for melanoma patients with active brain metastases. Results show that, if treated with this combination immunotherapy upfront, these patients can have similar outcomes to those melanoma patients without active brain disease.
“This proves we have achieved long-term disease control in this group of advanced melanoma patients,” said Professor Georgina Long AO, Medical Director of Melanoma Institute Australia and lead author of the study.
“We are now confident these patients are cured, a term not used lightly in cancer. This combination immunotherapy should now become standard of care for melanoma patients with brain metastasis,” she said.
Australia has the highest melanoma rates in the world with one person diagnosed with the disease every 30 minutes and one person dying every 6 hours.
“We have previously shown that with immunotherapy, metastatic melanoma is no longer a death sentence,” said Associate Professor Alex Menzies from Melanoma Institute Australia and co-senior author on the study.
“This is now the case even for patients with brain metastases, who used to have the worst of the worst prognosis. Results here will not only transform melanoma care but also lead to improvements across the cancer spectrum,” he said.
"These phenomenal results show the critical importance of investing in research and clinical trials to push science and medicine forward and move us closer to our goal of reaching zero deaths from melanoma," Prof Long added.
“We are now expanding on these findings and are conducting an Australia-wide trial – the ABC-X trial - exploring the role of upfront stereotactic radiosurgery with combination immunotherapy for melanoma patients with brain metastases.”
Read the study - titled “Ipilimumab plus nivolumab versus nivolumab alone in patients with melanoma brain metastases (ABC): 7-year follow-up of a multicentre, open-label, randomised, phase 2 study” - in full here.
For more information, please contact:
- Jennifer Durante - Melanoma Institute Australia - 0412 798 990 -
This email address is being protected from spambots. You need JavaScript enabled to view it. - Danny Rose - Peter MacCallum Cancer Centre - 0417 123 048 -
This email address is being protected from spambots. You need JavaScript enabled to view it.