Date: 2014-12-04
Type of information: Publication of results in a medical journal
phase: preclinical
Announcement: publication of results in the journal Cell Stem Cell
Company: Geron (USA - CA)
Product: imetelstat
Action
mechanism: Imetelstat (GRN163L) is a potent and specific inhibitor of telomerase that is administered by intravenous infusion. This first-in-class compound, discovered by Geron and to be licensed to and jointly developed with Janssen Biotech, Inc., pending the effectiveness of the collaboration agreement upon the expiration of the applicable waiting periods under the Hart-Scott-Rodino Act, is a specially designed and modified short oligonucleotide, which targets and binds directly with high affinity to the active site of telomerase. Preliminary data suggest disease-modifying activity by imetelstat is by affecting the malignant clone associated with hematologic malignancies. Imetelstat has not been approved for marketing by any regulatory authority.
Disease: acute myelogenous leukemia
Therapeutic area: Cancer - Oncology
Country:
Trial details:
Latest
news: * On December 4, 2014, Geron Corporation announced the publication of preclinical data demonstrating activity of the company\'s telomerase inhibitor drug, imetelstat, on leukemic stem cells from acute myelogenous leukemia (AML). In addition, the preclinical study showed the role of telomerase in AML disease initiation and progression. In the study, mice were transplanted with AML grafts from normal and telomerase deficient donor mice. Mice that received telomerase deficient grafts were shown to have reduced LSC functions and experienced cell cycle arrest and programmed cell death. This resulted in prolonged survival of telomerase deficient AML mice as compared to normal AML mice, providing proof-of-principle of the role of telomerase in maintaining LSCs. To test telomerase as a pharmacological target for AML treatment, xenograft mice were engrafted with primary AML patient samples and treated with or without imetelstat. Imetelstat treatment resulted in inhibition of disease progression and prolonged survival in these mice. In addition, imetelstat was shown to inhibit disease relapse after doxorubicin chemotherapy in the AML xenografts. These data suggest that imetelstat has the potential for disease-modifying activity in AML by targeting LSCs. The study was co-authored by Dr. Steven Lane and colleagues at QIMR Berghofer Medical Research Institute in Australia in collaboration with Geron scientists and published online on December 4, 2014 in the journal Cell Stem Cell. In addition, companion preclinical research on imetelstat in animal models of AML was selected for presentation as a poster at the upcoming 56th American Society of Hematology Annual Meeting (Abstract #2322, Inhibition of Telomerase with Imetelstat is Detrimental to Leukemia Stem Cells in Acute Myeloid Leukemia).
