Fros, the good news is that this is science, which can be proven. In fact, Cell in a Box(R) technology has already been proven. This is evident by the number of peer reviewed studies reporting on the results of studies using Cell in a Box(R) technology. Peer reviewed, btw, means that other scientists verify the results. They are not misleading but simply reporting findings, related specifically to this technology. Nothing generic in these reports.
Q: You’ve had several major peer-reviewed papers? What is the significance of these studies? Can you explain why these are key to PharmaCyte’s medical story?
KENNETH: There are three papers that have a direct effect on what we are doing at PharmaCyte Biotech. The first two have to do with our pancreatic cancer treatment and the third concerns our work with diabetes.
The first paper reports on the results of the Phase 1/2 clinical trial of our treatment in patients with advanced, inoperable pancreatic cancer. In this trial, 14 elderly and very sick patients were treated at a single study site in Germany with the combination of Cell-in-a-Box® plus low dose ifosfamide. All patients were treated with a single implantation of 300 Cell-in-a-Box® capsules with each capsule containing about 10,000 cells that are capable of converting the ifosfamide into its cancer-killing form. The capsules were implanted, using interventional radiography, with a catheter that was threaded through an artery in the leg to the pancreas where they were deposited as close to the cancerous tumor as possible. Then ifosfamide was administered intravenously at one-third of its “normal” dose. Only two treatments of ifosfamide were given about three weeks apart. This was a single-arm study; no comparator arm was used, but the results were compared to historical data for gemcitabine – the only drug approved at that time for the treatment of advanced, inoperable pancreatic cancer.
The results from this clinical trial showed that the Cell-in-a-Box® plus low dose ifosfamide combination increased the average lifespan of patients from about 5.7 months for gemcitabine to about 11 months with the combination and doubled the percentage of one-year survivors from 18% to 36%. Importantly, while the use of gemcitabine was associated with serious side effects, virtually no side effects were seen with the Cell-in-a-Box® plus ifosfamide treatment. The “quality of life” of the patients was vastly improved because of treatment. In addition, some beneficial effect was seen on metastatic lesions (from the pancreatic cancer) in the liver as a result of treatment with the Cell-in-a-Box® plus low dose ifosfamide combination. Thus, the major conclusion from this trial was that the combination of Cell-in-a-Box® plus low dose ifosfamide was a safe and effective treatment for patients with advanced, inoperable pancreatic cancer.
The second paper reports the results of a single-arm Phase 2 clinical trial in which 13 patients with advanced, inoperable pancreatic cancer were treated at 4 study sites in Europe. The only difference between this Phase 2 clinical trial and the Phase 1/2 clinical trial was that the dose of ifosfamide used was doubled to two-thirds of the normally used dose of this drug. This was done in an attempt to increase the anticancer effectiveness of the ifosfamide. The main conclusion taken from this trial was that the combination of Cell-in-a-Box® plus one-third the normal dose of ifosfamide was the appropriate treatment to use in all future clinical trials for a safe and effective antitumor treatment of advanced, inoperable pancreatic cancer.
The third peer reviewed paper titled, “Reversal of diabetes following transplantation of an insulin-secreting human liver cell line: Melligen cells” was published in the journal Molecular Therapy – Methods & Clinical Development. The article describes the development and preclinical testing of the Melligen cells developed by Prof. Simpson and her colleagues at UTS. Most importantly, however, the authors note that, for the Melligen cells to be effective in treating Type 1 diabetes in humans where the insulin-producing ß cells of the pancreas have been destroyed, it will be necessary to protect those cells from rejection by the body’s immune system after they have been introduced into the body. The article points out that one way to protect the Melligen cells would be to encapsulate the cells in protective “cocoons” prior to being placed into a diabetic patient. If this is done, the authors believe that encapsulated Melligen cells may offer a cure for Type 1 diabetes.
PharmaCyte Biotech’s live cell encapsulation technology, Cell-in-a-Box®, appears to be the ideal encapsulation technology for this purpose. In fact, Melligen cells have already been successfully encapsulated using the Cell-in-a-Box® process and experiments are already underway to ensure that encapsulation does not detract from the beneficial properties of the Melligen cells in any way.
