VCU Massey Cancer Center to Partner with Israeli Biotech Firm on $1M Pancreatic Cancer Clinical Research Study

By Andrea Butler and Donna Edmonds

The Virginia Commonwealth University Massey Cancer Center will open a Phase I pancreatic cancer study later this year in conjunction with leading researchers from Israel, marking the first time cancer researchers at VCU have partnered with their counterparts in Israel.

The study is supported by a $950,000 grant from the U.S.-Israel Bi-National Industrial Research and Development Foundation (BIRDF). Leaders from the Virginia Israel Bioscience Commercialization Center (VIBCC) helped to foster Israeli interest in research at VCU.

The clinical trial, designed by Massey principal investigator Ray Lee, M.D., Ph.D., involves a novel targeted therapeutic agent developed by BioCancell of Jerusalem and will offer new hope for one of the most difficult-to-treat cancers.

"This collaboration is a tangible outcome of the initiative launched by the Virginia BioTechnology Research Park more than a year ago to recruit Israeli life science companies with innovative products and technologies to Virginia and the Research Park," said Robert T. Skunda, President and CEO of the research park.

"The world, indeed, is getting flatter," said Donna Edmonds, executive director of the Virginia Israel Bioscience Commercialization Center. "By importing and exporting high-quality medical research and building collaborative clinical studies such as this, we can work toward saving more lives of people with cancer in Virginia and across the globe."

Gordon D. Ginder, M.D., director of the VCU Massey Cancer Center, said, "Having an organization dedicated to promoting Virginia's academic, clinical and biotechnological expertise is a boon to the Commonwealth. As federal funding for cancer research in the United States has shrunken in the face of expanding scientific opportunities for better treatment and prevention, it's vital that we explore international collaborations and funding sources, too. We are grateful to the VIBCC for serving as a beacon to guide international funding and research opportunities to us."

About Pancreatic Cancer
About 30,000 people die from pancreatic cancer each year. It has a high mortality rate and ranks as the fourth leading cause of cancer-related death as its symptoms often do not present until the disease is in a late stage. Fewer than 5 percent of patients survive for more than five years, giving pancreatic cancer the lowest survival rate of any common cancer type.

Currently the best treatment option for pancreatic cancer is through high-risk surgical removal of part of the pancreas. However, if the cancer affects surrounding vessels and lymph nodes, surgery often is not an option. Chemotherapy and radiation provide alternative treatment options, however, the median survival rate on those protocols is just one year.

About the Clinical Trial
This study involves a novel, gene-based therapy for pancreatic cancer developed by BioCancell. Its new therapeutic agent links a DNA fragment that is responsible for tumor-specific expression of a gene, with a suicidal toxin gene. When the agent is injected directly into the tumor, the DNA fragment will induce the expression of the suicidal toxin in the cancer cells but not in normal tissue. The researchers expect to avoid damaging normal tissue while killing tumor cells. 

Candidates for the study are adults with locally advanced pancreatic cancer who are not candidates for immediate surgical resection. Researchers will inject the new agent into cancer directly through a laparoscopic procedure.

Using this approach in pancreatic cancer, they hope that tumors could be down-staged from unresectable status, or inoperable, to resectable, giving patients a better chance of remission.

This agent has been tested and shown to be effective in animal models and anecdotally in two metastatic cancer patients in Israel. The liver lesions of those two patients showed success in shrinkage. 

Beginning in the summer of 2008, Massey expects to begin enrolling patients. Lee is the principal investigator, and Brian Kaplan, M.D., a surgical oncologist at Massey, will be the co-principal investigator performing the laparoscopic surgery. Abraham Czerniak, M.D., Ph.D., of Sheba Medical Center, Ramat Gan, Israel, will be responsible for the Israeli arm of the study, which expects to enroll one-third of the total patient base.

About BioCancell
BioCancell Therapeutics Inc. is a biopharmaceuticals corporation specializing in the development of patient-oriented, targeted therapy for the treatment of numerous types of cancer. The Company's proprietary technology constitutes a novel paradigm for the targeted destruction of cancer cells, with no effect on normal surrounding tissue and no observed side effects, allowing for long-term, safe treatment and prevention of cancer.

BioCancell was co-founded in 2004 by Avraham Hochberg, Professor of Molecular Biology at the Hebrew University of Jerusalem, based on technology developed by him over the past 15 years.

In 2006, BioCancell successfully completed a private round of funding and an initial public offering totaling $8.5 million. Its securities are traded on the Tel Aviv Stock Exchange, with the major stockholders being Clal Biotechnology Industries (a member of the IDB group of companies), and Prof. Hochberg. For more information, please visit www.biocancell.com.

BioCancell's Technology – Patient-Oriented, Targeted Therapy
BioCancell's technology is both personal and targeted. The approach is based on the identification of particular genes that are highly expressed only in tumors ("Target Genes"). The regulatory sequences of these Target Genes are used to drive the expression of a toxin gene exclusively within tumor cells, enabling targeted tumor-cell destruction, leaving normal cells intact. In effect, the plasmid acts as "smart bombs," activated only inside their targets thus destroying only the cancerous cells, while leaving healthy cells intact.

The patient's eligibility for the treatment is determined by analyzing the patient's tumor for the expression of the specific Target Genes. The diagnosis of the expression of the Target Genes are, therefore, a prerequisite for treatment and is made possible through the Company's proprietary diagnostic technology that enables detection of even a single malignant cell. Only those patients with high expression levels of the Target Genes in their tumor are eligible for treatment with high confidence of success. The Company has designated two genes as Target Genes – H19 and IGF2.

H19 Gene
Discovered by Professor Avraham Hochberg in humans, H19 is an oncofetal gene that encodes RNA (with no protein product) that is expressed at high levels in over 30 types of human cancer tissues, while existing at a nearly undetectable level in the surrounding normal tissues, thus making it an optimal weapon in the fight against cancer.

The gene is expressed abundantly in the human placenta and in several embryonic tissues, but is repressed post-natally and only re-expressed with the appearance of cancer, within cancer cells. Studies show that H19 fulfills an important role in the process of tumorigenesis, and it is thought that the gene enables tumor cells to survive and proliferate under stress conditions.

About Virginia Biotechnology Research Park
The Virginia BioTechnology Research Park is currently home to a unique mix of more than 55 public and private bioscience companies, research institutes affiliated with the VCU Medical Center and major state and national medical laboratories. These companies are housed in nine buildings totaling more than 1.1 million square feet of space, representing an employee base that exceeds 2,000. The VBDC is the Park's business assistance program located in its 27,000 square-foot incubator. Since opening in 1995, 63 companies have started in the Park's incubator facility; 31 of which have graduated into larger spaces in the Park or greater Richmond community. The Park's Commercialization Center has been created to take incubator graduates through commercialization to M&A or IPO, giving the Park's tenants full-scale business assistance. To date, the Park has graduated three companies that are now publicly traded.

About Virginia Israel Bioscience Commercialization Center
In addition to the Park's nationally recognized incubator program, the Virginia Biosciences Development Center, the newly formed VIBCC provides substantial support, experience and expertise in bringing "graduates" of Israeli incubator programs to successfully commercialize their products, making them more likely candidates for additional funding sources, business alliances and exit options. Specifically, the VIBCC assists companies in developing tailored commercialization strategies, including regulatory and reimbursement support, obtaining market validation, supporting product launch through national clinical leadership networks and assisting in the ongoing funding process, as well as formation of strategic partnerships with industry leaders.