Colorectal cancer kills more than 49,000 Americans each year and is the second-leading cause of cancer deaths in the United States. Dr. Scott Waldman, professor and chair of Sidney Kimmel Medical College’s Department of Pharmacology & Experimental Therapeutics at Thomas Jefferson University, and Dr. Adam Snook, Instructor in that department, with a team from Jefferson’s Departments of Microbiology and Immunology, Dermatology and Cutaneous Biology, and Medical Oncology, have pioneered an innovative vaccine to stop the spread (metastases) of cancers originating in the gastrointestinal tract, including cancers of the colon, rectum, pancreas, stomach, and esophagus.
In the early 1990’s, Dr. Waldman first identified Guanylate Cyclase C (GCC), an enzyme shown to be highly accurate for detecting the spread and predicting recurrence of colorectal cancer. For one in two people with colon cancer, rogue tumor cells escape the colon. In those cases, colon cancer returns within two to five years and when it does, it’s no longer confined to the colon (metastatic), making treatment more challenging and the prognosis often bleaker. The vaccine, which targets GCC, instructs the immune system to spot metastatic tumor cells and destroy them. With this breakthrough vaccine, metastases outside the colon may be prevented. This is good news for patients because containing the cancer leads to better outcomes.
“When we diagnose a patient with colon cancer, removing the primary tumor is not always the entire story,” says Dr. Waldman. “Indeed, at the time of surgery, tumor cells can already be on their deadly mission attacking other areas of the body. Our preliminary findings regarding the vaccine mean we have the potential to limit the aggressive nature of this disease and prevent metastases.”
As early as 1994, Targeted Diagnostics & Therapeutics, Inc., a biotechnology company headquartered in Exton, PA, obtained the worldwide exclusive rights to the technology from Thomas Jefferson University. Since then, it has been supporting the work in Dr. Waldman’s laboratory. After 15 years of research, and numerous pre-clinical lab and animal studies to evaluate biological activity and safety, Dr. Waldman filed an Investigational New Drug Application (IND) which was approved by the FDA in 2013. A phase I clinical trial was successfully completed over a two-year period that concluded in 2015. This phase I trial tested the safety and tolerability of the vaccine in stage I and II colon cancer patients. Results revealed that the vaccine was safe and well-tolerated, and elicited an immune response in some patients.
Viral Gene Inc., a company established to conduct and obtain funding for the vaccine’s phase II trial in the U.S., recently received a large investment from Alpha Holdings, Inc., formerly Alphachips Corp., a Korean-based company. The investment of $10 billion Korean Won ($8.7 million) is dedicated to funding the phase II trial, directed by Drs. Waldman and Snook, and bringing the vaccine to market. The phase II trial is anticipated to commence in 2017, pending FDA approval, and will include patients with the targeted cancers, to assess the vaccine’s effectiveness, and to commercialize the vaccine. Trial completion should take about two years. Also, both companies plan to conduct a Korean trial for the Asian market under the direction of Drs. Waldman and Snook.
Viral Gene plans to apply to the FDA for Orphan Drug Status for this vaccine, through its Office of Orphan Products Development (OOPD). OOPD’s mission is to advance the evaluation and development of drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases (conditions which affect fewer than 200,000 people) or conditions which affect more than 200,000 people but are not expected to recover the costs of developing and marketing a drug. If orphan drug status is approved and the phase II trial demonstrates efficacy, the vaccine will then be fast-tracked directly to market.
Filed Under: Drug Discovery