A spokesman for the makers of a promising new mesothelioma drug says the company plans to begin enrolling its first clinical trial participants this summer.
Dr. Joanna Horobin is Chief Medical Officer for Verastem, Inc., the developer of a drug that aims to treat malignant mesothelioma by targeting the stem cells that give rise to it. The company’s lead compound, an oral drug called VS-6063, inhibits a crucial signaling pathway inside stem cells called the Focal Adhesion Kinase (FAK) pathway. VS-6063 was approved by the FDA earlier this year as a ‘orphan drug’, a designation given to drugs designed to treat rare illnesses like mesothelioma.
“We are moving quickly to bring new treatment options to patients with mesothelioma,” Dr. Horobin said at a recent meeting of the American Society of Clinical Oncology in Chicago. “We have received allowance from the FDA to initiate a study of VS-6063 in mesothelioma and are in discussion with regulatory agencies worldwide to start the trial. We… expect to being enrolling patients into the trial this summer.”
According to Verastem data, the primary chemotherapy drugs for mesothelioma, including pemetrexed (Alimta), cisplatin, vinorelbine and gemcitabine are not effective at killing stem cells. In fact, although these agents can sometimes shrink mesothelioma tumors, they also increase the percentage of stem cells, leaving patients vulnerable to relapse. By killing the stem cells that lead to cancer recurrence and metastasis, it is hoped that VS-6063 may delivery better long-term results for mesothelioma.
Another important point of discussion at the ASCO meeting was the relationship between cancer and the tumor suppressor Merlin. As many as half of all mesothelioma patients lack the Merlin suppressor, making them particularly sensitive to the effects of an FAK inhibitor like VS-6063. The planned clinical trial of VS-6063 will include both standard and Merlin-low mesothelioma patients. LapCorp, Inc. has been working to develop a biomarker test to identify these patients.
Malignant mesothelioma is the rarest and most deadly of the illnesses associated with exposure to asbestos. The Centers for Disease Control and Prevention estimates that there are 2,500 new cases in the U.S. each year. Conventional cancer treatments have done little to improve the prognosis for mesothelioma patients, which is usually poor.
“Verastem Hosts Dr. Dean Fennell for a Mesothelioma Briefing at ASCO”, June 3, 2013, Business Wire.