Technology Overview

A Patient-Oriented, Targeted Therapy Approach

The Patient-Oriented, Targeted Therapy approach is based on the identification of particular genes that are highly expressed only in tumors. The regulatory sequences of these genes are used to drive the expression of a toxin gene exclusively within tumor cells, enabling targeted tumor-cell destruction.

H19 – A clear indicator
The first gene to be used by BioCancell Therapeutics for this purpose is H19. This is the first oncofetal gene ever discovered that is present at high levels in human cancer tissues, while existing at a nearly undetectable level in the surrounding normal tissues. It is thought that the H19 gene enables tumor cells to survive under stress conditions, such as low serum and low oxygen levels. The H19 gene is present in 70-85% of bladder, ovarian, pancreatic, breast, colorectal and hepatocellular cancer patients, and in significant percentages in a large number of other cancer types.

Targeted Cancer Cell Destruction
The product developed using the H19 gene is a plasmid in which H19 regulatory sequences drive the expression of the diphtheria toxin A gene (DTA-H19). This occurs in tumor cells only, triggered by the presence of H19 transcription factors found only in the tumor cells. The selective initiation of toxin expression results in selective tumor cell destruction, enabling highly targeted cancer treatment.

For more details regarding ‘Patient-Oriented, Targeted Therapy’ see the following animation (push buttons 1 to 5):

The Patient-Oriented Approach – Screened for Treatment Success
Diagnosis of H19 gene expression is a prerequisite for treatment. Only those patients displaying the characteristic high level of H19 in tumor cells are eligible for treatment with confidence of success. This is what makes it a Patient-Oriented therapy.

The Platform - Theragnostics
 

Our Patient-Oriented, Targeted Therapy is a novel combination of 2 approaches:

Diagnostics, for the detection of a specific gene - H19 - with patient-screening to ensure success of treatment, and Therapeutics that selectively targets cancer cells (using the H19 gene). This places it within the emerging paradigm of Theragnostics.

The first type of cancer treated with the H19 plasmid BC-819 in a clinical trial was refractory superficial Transitional Cell Carcinoma (TCC) of the bladder. The technology may be used in treatment of additional types of cancers displaying a high expression level of the H19 gene in humans, such as ovarian, pancreatic, liver, lung and colon.

Diagnostic Tool
H19 is highly expressed in various types of cancer. The proprietary diagnostic technology is able to detect even a single malignant cell expressing the H19 gene, making it also a promising diagnostic tool.

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