While most thyroid cancers progress slowly and can be curable when detected early, anaplastic thyroid carcinoma (ATC) poses a significant challenge. This rare and highly aggressive tumour is associated with a poor prognosis, making effective treatment crucial. However, recent findings from a clinical trial conducted at the University of Texas MD Anderson Cancer Center provide new hope for patients suffering from a specific subtype of ATC.

The clinical trial explored a combination of cancer immunotherapy and targeted treatments aimed at a particular genetic mutation present in some ATC tumour cells. This innovative approach appears to improve survival rates among patients with this aggressive form of cancer. “Patients with anaplastic thyroid carcinoma need treatments that work fast, and we saw promising results with this combination treatment approach,” stated Dr Maria Cabanillas, the lead investigator and a professor specializing in endocrine neoplasia and hormonal disorders.

The findings were published on October 24 in the journal JAMA Oncology. According to the researchers, ATC tumours can exhibit significant genetic variation between patients, with each subtype harbouring distinct driver mutations that affect tumour behaviour and progression. Approximately 40 per cent of ATC tumours have mutations in the BRAF gene, which significantly influences the cancer's prognosis and characteristics. The trial specifically focused on 42 patients with BRAF-mutated ATC.

Eighteen of these patients received a treatment regimen consisting of three drugs: Atezolizumab (Tecentriq), a monoclonal antibody immunotherapy, combined with vemurafenib and cobimetinib, which are targeted therapies for the BRAF mutation. The results were promising, with a median overall survival of just over 43 months for patients in this group. About half of these patients responded favourably to the treatment.

In contrast, a second group of 21 ATC patients with different mutations—specifically RAS (NRAS, KRAS, or HRAS) or NF1/2 mutations—received a combination of atezolizumab and cobimetinib. The median survival for this group was significantly shorter, at just 8.7 months, with only 14 per cent of patients responding positively to the therapy.

Additionally, three patients whose tumours did not exhibit the mutations seen in the previous groups received atezolizumab plus bevacizumab (Avastin). Their median survival was approximately 6 months, with only one-third responding to the treatment.

These findings underscore the importance of identifying specific ATC mutations, as they can guide treatment decisions and potentially enhance survival rates. “The takeaway from this study is that immunotherapy really does add benefit for patients,” Dr Cabanillas emphasized. However, she acknowledged the need for further research to develop effective therapies for patients with non-BRAF mutations. “There are no approved and effective therapies for ATC with non-BRAF mutations, and we continue to focus our research in that area,” she noted. “Our goal is to optimize outcomes for our patients, enabling them to live longer, better lives. This study offers hope for patients with ATC.”

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