Biomarker Test Next Step for New Mesothelioma Drug


A company that makes stem cell-focused treatments for cancer has taken an important step closer to testing a promising new mesothelioma drug.

The biopharmaceutical company Verastem, Inc. specializes in agents that destroy cancer by killing cancer stem cells. One of its lead medicines is VS-6063, a focal adhesion kinase inhibitor scheduled to be the subject of a mesothelioma study later this year. In a Phase I safety study of 36 patients conducted by Pfizer (original developers of VS-6063), the drug was “well-tolerated” and showed enough efficacy to warrant further, larger studies.

A focal adhesion kinase (FAK) inhibitor is an agent that regulates the growth and spread of tumor cells by inhibited a crucial signaling pathway. In preclinical cancer models, when this pathway is disrupted, there is a noticeable reduction in cancer stem cells, primary tumor mass, and metastasis.  Verastem co-founder and chair of the Scientific Advisory Board, Robert Weinberg, Ph.D., said earlier this year, “Verastem has identified the FAK pathway as a critical regulator of the survival of cancer stem cells, which are an underlying cause of cancer recurrence and metastasis.”

Now, the new drug may be a step closer to reality for mesothelioma patients. Verastem has contracted with LabCorp, Inc. to validate biomarkers for VS-6063. It is estimated that about half of mesothelioma patients are lacking a particular tumor suppressor, a fact which could make them more significantly more sensitive to FAK inhibitors like VS-6063. It will be LabCorp’s job to develop a test for that biomarker, which will then help Verastem identify mesothelioma patients for the upcoming clinical trial of VS-6063. The company calls such a test “an integral component” of undergoing the FDA approval process. They have announced plans to run multiple clinical trials of VS-6063 in 2013, including what they call a “potentially pivotal study in mesothelioma”.

According to the Centers for Disease Control and Prevention (CDC), approximately 2,000 to 3,000 cases of malignant mesothelioma are diagnosed in the U.S. each year.  This cancer is caused by asbestos, a naturally occurring carcinogenic mineral that has been used in thousand of products and applications. Conventional cancer treatments have done little to improve the prognosis for mesothelioma patients, which is typically poor.


“Verastem Enters Biomarker Agreement with LabCorp for Cancer Stem Cell Agent Companion Diagnostic”, Press Release, January 9, 2013, Wall Street Journal Marketwatch.
“Verastem Acquires Clinical-Stage FAK Inhibitor from Pfizer”, July 11, 2012, Press Release distributed by Reuters.

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