SILVER SPRING, Md.—PharmaCyte Biotech Inc., a clinical stage biotechnology company focused on developing targeted treatments for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, announced April 20 that its international “world-class team of scientists, physicians and academics” that constitute its Diabetes Consortium is now complete with the addition of the final two members, Dr. Thomas Stratmann and Dr. Axel Kornerup Hansen.
Stratmann is an associate professor in the Department of Physiology and Immunology at the University of Barcelona in Spain, bringing to the consortium extensive experience in using a diabetes mouse model of type 1 diabetes in which the disease develops rapidly. This in contrast to other mouse models where the disease only develops after several weeks. Through the use of this model—as the Diabetes Consortium progresses in its animal preclinical studies with Cell-in-a-Box-encapsulated Melligen cells—it is fully expected that the overall development timeline for PharmaCyte Biotech’s diabetes treatment will be shortened significantly.
Hansen is a professor in the Department of Veterinary Disease Biology at the University of Copenhagen in Denmark. He will perform specially designed preclinical studies to determine the minimum dose of encapsulated Melligen cells that will normalize blood glucose levels in diabetic rats as well as pigs, which are an important model for mimicking the dosing that will be required for human patients.
PharmaCyte Biotech has the exclusive worldwide rights to use the Melligen cells to treat diabetes. Melligen cells are genetically engineered from human liver cells and have been shown to secrete insulin in response to the concentrations of glucose (blood sugar) in their environment. When Melligen cells were transplanted into diabetic mice whose immune systems were essentially not functioning, the blood glucose levels of the mice became normal. This observation illustrates that Melligen cells could perhaps even reverse diabetes.
The initial formation of the Diabetes Consortium was announced in November 2014, and part of that announcement was to note that, in addition to key personnel from PharmaCyte Biotech, the consortium would consist of principals from Austrianova—Dr. Walter H. Gunzburg, the chief technical officer of Austrianova and PharmaCyte Biotech’s chief scientific officer, and Dr. Brian Salmons, the CEO of Austrianova (Gunzburg and Salmons co-developed the Cell-in-a-Box technology)—and Prof. Ann Simpson of the University of Technology Sydney (UTS) who, along with her professional colleagues at UTS, developed the Melligen cells.
All members of the Consortium are committed to developing a treatment for insulin-dependent diabetes using PharmaCyte Biotech’s Cell-in-a-Box cellulose-based live-cell encapsulation technology combined with human non-pancreatic, insulin-producing cells (specifically, at this point, the Melligen cells).
“We are very pleased that our international Diabetes Consortium is now complete after only a few months of development,” said PharmaCyte Biotech’s CEO, Kenneth L. Waggoner. “We feel that the scientists, physicians and academics that make up our consortium are of the highest caliber and have already proven that they can work in concert as the development of our diabetes treatment progresses. We firmly believe that, although the members within the consortium are diverse in terms of their geographic location and expertise, their talents and knowledge are most complementary. Because of this, PharmaCyte Biotech’s treatment for insulin-dependent diabetes could be in clinical testing much sooner than would otherwise be the case.”
The members of the Diabetes Consortium and their expected contributions are:
- Dr. Eva-Maria Brandtner, head of the Bioencapsulation Unit at the Vorarlberg Institute for Vascular Investigation and Treatment will handle program development for the consortium. Brandtner will coordinate all of the Diabetes Consortium’s research activities and also have a scientific advisory role. She previously served as chief scientist at Austrianova, where she conducted studies related to the encapsulation of Melligen cells and the early testing of their use in PharmaCyte Biotech’s encapsulation technology as a treatment for diabetes.
- Dr. Matthias Löhr, professor of gastroenterology and hepatology at the Karolinska Institute in Stockholm, Sweden, will serve “a pivotal scientific advisory role” for the Diabetes Consortium. Löhr is exceedingly familiar with the Cell-in-a-Box technology, having served as principal investigator for the clinical trials in pancreatic cancer that employed the technology together with the cancer chemotherapy drug ifosfamide. In addition to pancreatic cancer, Löhr has a specialty in diabetes and its treatment.
- In addition to Günzburg and Salmons, Dr. John Dangerfield of Austrianova will play a significant scientific advisory role. Dangerfield, the chief operations officer of Austrianova, is intimately involved in dealing with the Cell-in-a-Box encapsulation process on a daily basis. All three will be responsible for the preparation of cells and their encapsulation as required for preclinical studies and ultimately be involved in human clinical trials as the Diabetes Consortium develops its diabetes treatment.
- In addition to Simpson, Dr. Brenton Hamdorf of UTS in Australia, along with their scientific colleagues at UTS, will be “essential to the success of the efforts of the Diabetes Consortium.” Simpson is a professor of biochemistry at UTS, and has spent a significant portion of her professional career in developing and characterizing Melligen cells. Hamdorf serves as the business development contact at UTS and partner for the Diabetes Consortium. Dr. Simpson and her fellow scientists at UTS will be responsible for performing laboratory and animal studies on the Melligen cells both before and after encapsulation that will be necessary for defining the parameters under which the Melligen Cells produce insulin.
- Dr. Constantine Konstantoulas and Mag Helga Petznek of VetMed will be responsible for conducting animal studies with encapsulated Melligen cells that will test the potential of these cells to produce tumors, define the biocompatibility of the encapsulated Melligen Cells and test the ability of the encapsulated cells to produce insulin in a mouse model in which type 1 diabetes has been induced by the introduction of a virus. Konstantoulas will coordinate the day-to-day activities of these tests, and Günzburg will oversee all of the studies done at VetMed. The studies at this institution are already underway.
- Stratmann at the University of Barcelona in Spain, has (as noted earlier) extensive experience in using a diabetes mouse model of Type 1 diabetes in which the disease develops rapidly; in addition, Stratmann has two other mouse models of type 1 diabetes in his laboratory. Through the use of these other animal models, and in combination with other test systems, the Diabetes Consortium “should be able to obtain irrefutable data as to the effectiveness of PharmaCyte Biotech’s diabetes treatmen,” according to PharmaCyte.
- Hansen at the University of Copenhagen in Denmark will perform preclinical studies to determine the minimum dose of encapsulated Melligen cells that will normalize blood glucose levels in diabetic rats. In addition, Hansen will perform the same tests on pigs that have been made diabetic by injecting them with a drug known as streptozotocin. This drug destroys the ability of the pancreas to produce insulin and thus results in diabetes.
- Dr. Andreas Blutke, Dr. Rüdiger Wanke and Dr. Eckhard Wolf, of the Laboratory of Functional Genome Analysis, Gene Center, at the Ludwig-Maximillians University in Munich, Germany, have developed unique transgenic mouse and pig models in which the mice and pigs exhibit diabetes from birth without the use of diabetes-inducing drugs. When these models are ready, they will be used by the Diabetes Consortium to test the effectives of PharmaCyte Biotech’s diabetes treatment and to study the effectiveness of the treatment in minimizing the kidney and pancreas alterations that occur as diabetes progresses.
- Waggoner and PharmaCytes’ Dr. Gerald W. Crabtree, the chief operating officer, will be responsible for providing overall coordination and management of the Diabetes Consortium as well as funding for the activities within the Consortium. PharmaCyte Biotech will also provide scientific direction and support when it is required.
In other recent news of the Diabetes Consortium, PharmaCyte noted in February of this year that consortium partner UTS had published a review article on cell and gene therapies for the treatment of diabetes. The publication, titled “The use of β-cell transcription factors in engineering artificial β cells from non-pancreatic tissue,” which was authored by Simpson and her colleagues at UTS, appeared in the scientific journal Gene Therapy.
The article reports that cell and gene therapies have shown promise as a potential cure for type 1 diabetes through the genetic engineering of non-pancreatic cells that make them capable of regulating blood glucose levels by producing insulin on demand. Such genetic modification and augmentation of non-pancreatic cells has the ultimate goal of producing glucose-responsive “artificial” β insulin-producing cells that mimic the function of pancreatic β cells in non-diabetic individuals.
PharmaCyte Biotech is a clinical-stage biotechnology company focused on developing and preparing to commercialize treatments for cancer and diabetes based upon the proprietary cellulose-based live cell encapsulation technology known as Cell-in-a-Box. This unique and patented technology will be used as a platform upon which treatments for several types of cancer, including advanced, inoperable pancreatic cancer, and diabetes are being built. PharmaCyte Biotech’s treatment for pancreatic cancer involves low doses of the well-known anticancer prodrug ifosfamide, together with encapsulated live cells, which convert ifosfamide into its active or “cancer-killing” form. These capsules are placed as close to the cancerous tumor as possible to enable the delivery of the highest levels of the cancer-killing drug at the source of the cancer. This “targeted chemotherapy” has proven remarkably effective and safe in past clinical trials, the company says.
PharmaCyte Biotech is also working towards improving the quality of life for patients with advanced pancreatic cancer and on treatments for other types of solid cancerous tumors. In addition, PharmaCyte Biotech is developing treatments for cancer based upon chemical constituents of the Cannabis plant, known as cannabinoids. In doing so, PharmaCyte Biotech is examining ways to exploit the benefits of Cell-in-a-Box technology in optimizing the anticancer effectiveness of cannabinoids, while minimizing or outright eliminating the debilitating side effects usually associated with cancer treatments.