BC-819 has been administered, under "compassionate use" provisions in Israel, to patients who had failed chemotherapy and were in the ultimate stages of cancer. In 2003, two patients with resistant bladder cancer were treated with BC-819. Prior to the BC-819 treatment, they underwent a transurethral resection, but the tumors returned. Both patients were treated by direct introduction of BC-819 into the bladder using a catheter. The BC-819 treatment resulted in a significant decrease of the superficial bladder tumor, no toxicity was demonstrated in healthy cells, no severe adverse side effects which can be related to the drug were diagnosed, and no plasmid was detected in the patients' blood.

Two additional patients with very large metastases in their livers showed shrinkage of these tumors following treatment with BC-819. No unwanted toxicity was demonstrated in healthy cells and no severe adverse side effects which can be related to the drug were diagnosed.

One patient suffering from ovarian cancercharacterized by intra-peritoneal distribution of metastases and ascites (liquid containing cancerous cells that builds up in the peritoneum as a result of the cancer), was treated with BC-819 after the failure of conventional chemotherapy. The results of the trial show that the drug caused no serious adverse events (SAE's) at any dosage, and that a decrease of 50% in the ovarian cancer marker protein CA-125 in the patient's blood was measured, as well as a significant decrease in the number of cancerous cells in the ascites, and reports by the patient and her doctors of a clinical improvement in her condition. Today, the patient's condition continues to be stable, with no new growths. Her quality of life was increased during the course of the treatment.

Clinical Trial History - Phase I/IIa Trial - Bladder Cancer
The trial was designed to determine the optimal dose and assess the safety and preliminary efficacy of BC-819 complemented with PEI (a transfection enhancer) given as intravesical infusions into the bladder of patients with superficial bladder cancer expressing the H19 gene, who had previously failed at least one standard treatment type.

• Safety - No Serious Adverse Events relating to the treatment were detected at any dose given.
• Optimal Dose - There was no dose-limiting toxicity at any dose given, so the optimal dose was set as the maximum dose administered.
• Primary Efficacy - In total, 72% of patients responded to treatment. The preliminary evaluation of the efficacy of BC-819 suggests that it has the ability to cause tumor ablation and regression at doses that are well tolerated.

Details of BioCancell's clinical trials are available at ClinicalTrials.gov (external link).