Therapeutic programs

Cancers

Pancreatic cancer, one of the most malignant neoplasms, is usually diagnosed at a relatively advanced stage; early detection is difficult because of the remote internal location. Furthermore, it is often difficult to resect curatively, which results in poor prognosis and the highest mortality rate among all gastrointestinal malignancies. At present, the only options for treating advanced pancreatic cancer are radiation and chemotherapy with anticancer drugs such as 5-FU and gemcitabine. Gemcitabine is considered the standard first-line therapy for patients with advanced pancreatic cancer. Treatment with gemcitabine produces clinical benefit and symptom improvement but little to no survival improvement.

InvivoGen Therapeutics is developing a program to enhance the efficacy of gemcitabine for the treament of pancreatic cancer. This program is based on increasing the conversion of gemcitabine to its toxic metabolites by the coexpression within the tumor of DCK and UMK, the rate-limiting enzymes involved in the metabolism of gemcitabine. Using a non viral gene therapy approach, we will deliver intratumorally a plasmid expressing the dck::umk fusion gene complexed to the synthetic polycationic PEI vector followed by the systemic administration of gemcitabine. We have already generated compelling pre-clinical data demonstrating the antitumor efficacy of the dck::umk/gemcitabine treatment. Our clinical development program is expected to start early 2008.


Thrombocytopenia is a common side effect of chemotherapy, responsible for increased risk of bleeding and delay of treatment schedules in cancer patients. Currently the only method to alleviate thrombocytopenia is platelet transfusion. Thrombopoietin (TPO) is the primary regulator of platelet production. Over the past 10 years, recombinant forms of TPO have shown various degrees of effectiveness in the treatment of thrombocytopenia associated with chemotherapy.

InvivoGen Therapeutics has initiated a program to increase the number of platelets in cancer patients with chemotherapy-induced thrombocytopenia. The goal of this program is to investigate the potential of using the hydrodynamic technology to deliver and express high and sustained levels of the TPO gene in the liver. The plasmid developed is totally CpG-free and contains the TPO gene under the control of a strong liver specific promoter. Preclinical studies are underway in the large animal model of the pig.