New Hope Against a Rare, Aggressive Form of Thyroid Cancer With An Extremely Poor Prognosis

Thyroid cancer is among the rare cancers that do not trigger or show any symptoms in the early stages. Experts say if caught early, this slow-moving disease is curable. However, some patients can present with what is known as an anaplastic thyroid carcinoma or ATC - a rare and very aggressive tumour with a very poor prognosis.

And now, a clinical trial offers new hope to patients with a certain subtype of this tumour.

Doctors from the University of Texas MD Anderson Cancer Center in Houston have reported that combining cancer immunotherapy with another treatment -- targeted to a particular genetic mutation found on some ATC tumor cells -- appears to extend patient survival. “Patients with anaplastic thyroid carcinoma need treatments that work fast, and we saw promising results with this combination treatment approach," said Dr. Maria Cabanillas, lead investigator and professor of endocrine neoplasia and hormonal disorders at the University of Texas MD Anderson Cancer Center in Houston.

The study, published in the journal JAMA Oncology, said ATC tumors can differ genetically from patient to patient, and "each subtype has distinct driver mutations that can influence tumour behaviour and progression." About 40 per cent of ATC tumors have mutations in the BRAF gene that help drive the cancers' behaviour and prognosis. The new trial focused on 42 patients battling a BRAF-mutated ATC. Eighteen of the patients received three drugs – Atezolizumab or Tecentriq - a monoclonal antibody immunotherapy drug, plus a combination of vemurafenib and cobimetinib - two drugs targeted to the BRAF mutation.

The median overall survival of patients in that group was just over 43 months, with about half of the patients responding favourably to the regimen, the Houston team said.

A second group of ATC patients included 21 people battling tumors with a mutation known as RAS (NRAS, KRAS, or HRAS), or patients with NF1/2 mutations. This group got a combination of atezolizumab plus cobimetinib, but overall median survival was much shorter, just 8.7 months. Only 14% of patients responded to that therapy, the team said.

Finally, three other ATC patients with none of the tumor cell mutations detected in the prior two groups received atezolizumab plus bevacizumab. Their median over survival was just over 6 months, with only a third of patients responding.

The new trial shows how important pinpointing specific ATC mutations can be in determining a treatment course that might extend survival. "The takeaway from this study is that immunotherapy does add benefit for patients," Cabanillas said in a university news release. However, she added that more research is needed into devising effective treatments for patients with ATC whose tumors carry 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 said. “We are working to optimize outcomes for our patients. We want them to live longer and better lives, and this study offers hope for patients with ATC.”

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