The FDA has issued approval for biopharmaceutical company Verastem to begin
a Phase 2 clinical study of a new drug for the treatment of malignant
VS-6063 is an orally available, small molecule inhibitor of a crucial signaling
pathway inside stem cells called the Focal Adhesion Kinase (FAK) pathway.
FAK is vital for tumor development and is critical for the survival of
cancer stem cells. VS-6063 was well-tolerated in a Phase 1 study and demonstrated
signs of clinical activity in advanced solid tumors.
Dr. Dean Fennell, Chair of Thoracic Medical Oncology at the University
of Leicester, incoming President of the International Mesothelioma Interest
Group (iMig) and a member of the Verastem Mesothelioma Steering Committee,
presented promising data at a
briefing session on VS-6063 at the annualAmerican Society of Clinical Oncology meeting
in Chicago, IL, in early June of this year.
Studies by Verastem and others have shown that
chemotherapy agents most commonly used in the treatment of mesothelioma do not kill
cancer stem cells in mesothelioma, but actually increase growth of cancer
stem cells. This includes the only FDA approved chemotherapy drug pemetrexed
(Alimta), along with other commonly used agents cisplatin, vinorelbine
and gemcitabine. VS-6063 has the ability to kill these cancer stem cells.
Studies also show that mesothelioma patients who lack the tumor suppressor
gene Merlin have increased sensitivity to FAK inhibition, which would
greatly enhance the effectiveness of a drug like VS-6063. Currently a
biomarker test is being developed in conjunction with LabCorp to identify
mesothelioma patients low in Merlin, this accounts for approximately 40-50%
of mesothelioma patients
“These early results suggest that a targeted therapy, particularly
when used in combination with a specific biomarker, has the potential
to significantly improve treatment of this aggressive and deadly disease,”
said Dr. Fennell, “There is a large unmet medical need in mesothelioma
and an opportunity to develop targeted agents to bring new hope to patients
struggling with their disease.”
VS-6063 is also being studied in a Phase 1/1b trial in combination with
paclitaxel for the treatment of ovarian cancer.