Tekmira Pharmaceuticals Corporation (TSX:TKM), a leader in RNA interference (RNAi) therapeutics, announced today that, together with collaborators at the National Cancer Institute (NCI), it has published promising preclinical data in the prominent journal Cancer Research (Lee et al, Definition of Ubiquitination Modulator COP1 as a Novel Therapeutic Target in Human Hepatocellular Carcinoma.
Cancer Res; 70(21); 8264-9). The data stem from the use of small interfering RNA (siRNA) enabled by Tekmira's lipid nanoparticle (LNP) technology in preclinical models of human hepatocellular carcinoma (HCC) or liver cancer.
Scientists at the NCI identified a novel oncology target called COP1 that is over-expressed in human liver cancers and can accurately predict patient survival. Tekmira and scientists at the NCI designed specific siRNA that, when delivered systemically with Tekmira's LNP technology (COP1-LNP), silence the COP1 gene. In preclinical studies, COP1-LNP demonstrated a greater than 12-fold decrease in liver cancer cell growth in tumor bearing mice. This tumor growth inhibition exceeds the National Cancer Institutes criteria for promising therapeutic compounds.
Dr. Mark J. Murray, Tekmira's President and CEO, said, "This work is an illustration of one approach Tekmira is taking to identify targets of therapeutic interest which could lead to the development of product candidates. We are very encouraged by the results of this study and look forward to advancing this work with our collaborators at the NCI. By combining the NCI's expertise in identifying novel cancer targets and Tekmira's leadership in siRNA molecule design and delivery, we intend to build on what has been a very successful collaboration over the past several years."
COP1 has been identified as being over-expressed in human HCC and a predictor of patient survival. The silencing of COP1 via RNAi affects a number of genes in a cancer cell including restoring the expression of p53, a key tumor suppressor protein. The result is an inhibition of cancer cell proliferation and programmed cell death.
Primary liver cancer or HCC is associated with one of the poorest survival rates in oncology with only 30-40% of patients being eligible for curative treatment due to late diagnosis, underlying liver disease and lack of effective treatment options. HCC is the third most lethal cancer with an estimated 600,000 deaths annually.