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.