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Early Trial Results Targeting Cancer Mutations Promising

Lungs illustration

(National Cancer Institute)

20 August 2015. A clinical trial testing the efficacy of a cancer drug to treat multiple types of cancer based on a common genomic mutation, showed a melanoma drug could treat at least a few other cancers. A team from the U.S. and Europe, led by researchers from Memorial Sloan Kettering Cancer Center in New York, published its results today in the New England Journal of Medicine (paid subscription required).

The clinical trial, funded by the pharmaceutical company Roche and its biotechnology subsidiary Genentech, investigated effects of the drug vemurafenib, marketed as Zelboraf by Genentech, approved to treat melanoma resulting from mutations in the BRAF gene. The researchers, led by Memorial Sloan Kettering chief physician José Baselga, tested vemurafenib with patients having other types of solid tumor cancer that result from a specific mutation in the BRAF gene, BRAF V600.

The intermediate-stage trial, still underway and recruiting participants, aims to enroll 160 patients with a range of solid tumor cancers, — including  lung, colorectal, and ovarian cancer — and multiple myeloma, looking primarily for tumor response, or shrinkage in tumors.  The researchers reported on a part of the study testing vemurafenib with 122 individuals having solid tumor cancers enrolled at 23 sites worldwide.

The early findings show vemurafenib had mixed results with the solid tumor cancer participants. More than 4 in 10 individuals (42%) with non-small cell lung cancer responded to vemurafenib treatments, with median progression-free survival of 7.3 months. About the same percentage (43%) of participants with the rare cancers Langerhans cell histiocytosis and related Erdheim-Chester disease also responded to vemurafenib, with none of the individuals having these types of cancer reporting disease progression.

For other types of cancer treated with vemurafenib, the numbers of participants in the trial were too small to report statistics, but the team says anecdotal evidence supports responses with individuals having anaplastic thyroid cancer, salivary-duct cancer, ovarian cancer, clear-cell sarcoma (soft-tissue cancer), cholangiocarcinoma (bile duct cancer), and pleomorphic xanthoastrocytoma (a rare brain cancer). Anecdotal reports of responses were also reported for colorectal cancer, when vemurafenib was combined with cetuximab, marketed as Erbitux by Eli Lilly and Co.

“This kind of study is a beneficial way to do rare tumor research,” says first author David Hyman in an institute statement, “because it allows us to open the study to patients with diseases that are completely underrepresented in clinical trials in general, such as anaplastic thyroid cancer and glioblastoma.” Hyman is the acting director of developmental therapeutics at Memorial Sloan Kettering.

This clinical trial, says the institute, is expected to be the first of a number of studies addressing mutations associated with cancer, resulting from the wealth of genomic data accumulated in recent years, a strategy known as precision medicine. “Precision medicine has come to the forefront as the future of cancer treatment,” says Baselga. “This is the first example of what it looks like, and proof of how it works. This study demonstrates we can design trials based on genomics as opposed to site of origin of the cancer.”

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