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Pre-Clinical Data Support Cancer Stem Cell Drug

Beakers and test tubes (Horia Varlan/Flickr)Geron Corporation in Menlo Park, California said today two studies with preclinical data demonstrate that the company’s telomerase inhibitor drug imetelstat (GRN163L) targets cancer stem cells from multiple myeloma, pancreatic, and breast cancers. Imetelstat is currently in Phase 2 clinical trials.

The company says imetelstat targets cancer stem cells, found in many types of cancer, and are often resistant to chemotherapy and conventional anti-cancer agents. They are rare populations of malignant cells with the capacity for endless self-renewal and believed to be responsible for tumor growth, recurrence and metastasis.

The first study indicates an inhibitory effect of imetelstat on multiple myeloma cancer stem cells in vitro and in animal models of the human disease. The research was published in the journal PLoS ONE.

Treatment with imetelstat for two weeks in vivo in a xenograft (cross-species graft) model of established human multiple myeloma resulted in survival benefits compared to controls: eight of eight animals alive in the imetelstat treated group, versus none of the eight were alive in the control group at day 110. Also, cancer stem cells isolated from human multiple myeloma cell lines and treated with imetelstat in vitro showed reduced proliferation with five-fold fewer colonies after treatment for three weeks and 100-fold fewer colonies after five weeks compared to controls.

The second study demonstrates an inhibitory effect of imetelstat on cancer stem cells from breast and pancreatic tumor cell lines in vitro and in animal models of the human disease. The data were published in the journal Cancer Research (paid subscription required).

Treatment in vitro of pancreatic and breast cancer cell lines with imetelstat reduced the proportion of cancer stem cells from the bulk tumor populations by 1.6 to 12 fold in all cell lines tested. In addition, imetelstat inhibited the growth of bulk breast cancer cells, resulting in cell death after three weeks of treatment.

Related: Clinical Trial of Stem Cell Therapy Underway

Photo: Horia Varlan/Flickr

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