Targeted Cancer Therapeutics

The superior efficacy of targeted therapy for cancer (the ability to treat the tumor based upon its molecular biology) has moved chemotherapy to a different paradigm. Chemotherapy for cancer was synonymous with adverse reactions, nausea, vomiting, hair loss and a host of other illnesses because these conventional drugs kill the good cells as well as the tumor cells.  By knowing the molecular biology of the tumor and targeting the therapy directly to the different molecular nature of the tumor, a drug can be effective against only the tumor and not the healthy cells.  One such example of molecular diagnostics and targeted therapy for breast cancer is the test Her2Nu and the targeted therapy Herceptin.  This drug has revolutionized the treatment of breast cancer and has been very effective, but only in the subset of women with tumors tested to have the molecular biology susceptible to this treatment.  InterGenetics has identified a particular gene that is now in preclinical studies as a tumor suppressor and has been linked to a number of different types of cancers, such as lung, brain, pancreas and breast.  This biological therapy is being advanced in animal models and when fully developed its intended use is to target pancreatic cancer and end-stage lung disease.

Research has demonstrated that cancer is not just one particular type of disease but rather several hundred different types of diseases that are usually named only by the organ or tissue it initially grows in (i.e. breast cancer, colon cancer, prostate cancer).  Because cancer can develop through multiple different molecular mechanisms, treating all cancers with broad cell killing therapy is not as effective as targeting the specific cause of a particular cancer.  Equally important are the unwanted side effects that are produced by non-targeted cancer therapy because in order for them to be effective they work by killing the healthy cells as well as cancer cells.   “Targeted Cancer Therapy” refers to, first, the identification of the difference in the mis-directed machinery of the cancer cell compared to the healthy normal cell, and then targeting and treating the cancer based upon that particular difference.  Some very effective targeted therapeutics are on the market for treating some forms of cancer, and they are making a significant difference in the treatment and morbidity and mortality of breast cancer patients.

InterGenetics has developed a class of novel cancer therapeutics in this emerging area of targeted cancer therapy.  The core technology is based upon the discovery that injection of the patented RNA molecules causes complete remission and cure of both primary and metastatic tumors in a rat breast cancer model.  The response rate for the therapy is high with ~75% of treated animals being cured.  Most importantly, the majority of the cured animals (48/50) were resistant to tumor growth when re-challenged by transfer of fresh tumor.  Additionally, no adverse side effects were evident in the cured animals.  This novel RNA therapeutic stimulates anti-tumor immunity and has the potential, either directly or indirectly, to activate the body’s immune system to fight cancer.  We have characterized this novel RNA as a tumor suppressor with a role in controlling cell proliferation.  This work had shown that introduction of the gene product in deficient cancer cell lines originating from a range of different cell types caused a reversion to normal growth.  Our research demonstrates that this novel RNA exhibits a duality with the potential to function both as a biological therapeutic (stimulating cancer immunity) and a molecular targeted therapeutic (replacement in defective cells) in cancer. InterGenetics is targeting an indication for use in pancreatic cancer, end-stage lung cancer and other cancers that are inoperable, or have no other effective therapies available to the patient.