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Targeted Therapy Extends Survival in BRAF-Mutated Anaplastic Thyroid Cancer

• A clinical trial demonstrates that combining immunotherapy with targeted therapy improves survival for patients with BRAF-mutated anaplastic thyroid carcinoma (ATC). • Patients with BRAF-mutated ATC treated with atezolizumab, vemurafenib, and cobimetinib showed a median overall survival of over 43 months. • The study highlights the importance of identifying specific genetic mutations in ATC tumors to guide effective treatment strategies. • Further research is needed to develop effective therapies for ATC patients with non-BRAF mutations, as current options are limited.

Anaplastic thyroid carcinoma (ATC) is a rare and aggressive form of thyroid cancer with a historically poor prognosis. A recent clinical trial conducted at the University of Texas MD Anderson Cancer Center offers new hope for patients with a specific subtype of this disease. The study, published in JAMA Oncology, reveals that combining immunotherapy with targeted therapies significantly extends survival in patients with BRAF-mutated ATC.
The trial focused on 42 patients with ATC, with a subset of 18 patients harboring BRAF mutations. These patients received a combination of atezolizumab (Tecentriq), a monoclonal antibody immunotherapy drug, along with vemurafenib and cobimetinib, two drugs that target the BRAF mutation. The median overall survival in this group was just over 43 months, with approximately half of the patients exhibiting a favorable response to the treatment regimen.
In contrast, a second group of 21 ATC patients with RAS (NRAS, KRAS, or HRAS) or NF1/2 mutations were treated with atezolizumab and cobimetinib. This group experienced a significantly shorter median overall survival of 8.7 months, with only 14% responding to the therapy. A third group of three patients without the mutations received atezolizumab plus bevacizumab (Avastin) and had a median overall survival of just over 6 months, with a third of patients responding.

Implications for ATC Treatment

The findings underscore the importance of genetic profiling in ATC to identify specific mutations that can inform treatment decisions. Dr. Maria Cabanillas, professor at the University of Texas' MD Anderson Cancer Center in Houston and lead investigator, emphasized the need for fast-acting treatments for ATC, noting the promising results observed with the combination therapy. "Patients with anaplastic thyroid carcinoma need treatments that work fast, and we saw promising results with this combination treatment approach," said Cabanillas.

Unmet Needs in Non-BRAF Mutated ATC

While the study provides a significant advancement for patients with BRAF-mutated ATC, it also highlights the urgent need for effective therapies for those 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," Cabanillas stated. The research team is dedicated to optimizing outcomes for all ATC patients, aiming for longer and better lives through continued research efforts.
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Reference News

[1]
New Hope Against a Rare, Aggressive Form of Thyroid Cancer
drugs.com · Apr 17, 2025

A clinical trial combining immunotherapy with targeted treatment for BRAF-mutated anaplastic thyroid carcinoma (ATC) ext...

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