Dr. Waldman, because it provides design parameters for creating immunotoxins with various agents. The immunotoxin Dr. Waldman used was an antibody against GUCY2C bound to the active portion of the ricin molecule with a chemical linker that degrades in acidic environments. However, other deadly toxins that could be kept inactive by the linker molecule could be coupled to the antibody, traveling throughout the body without causing damage until they are taken up by colon cancer metastases.
And the therapy wouldn't be limited to colon cancer. Gastric, esophageal and pancreatic cancers also express GUCY2C molecules, suggesting that the immunotoxin approach could be effective against the difficult-to-treat metastases in those diseases as well.

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In fact, the concept is currently being tested in a phase II clinical trial for gastric, esophageal and prostate cancers by Millennium Pharmaceuticals, a Takeda Oncology Company.
These studies were supported by grants from the National Institutes of Health (R01 CA75123, R01 CA95026, RC1 CA146033, R01 CA170533; and P30 CA56036), the Pennsylvania Department of Health (SAP #4100059197, SAP #4100051723), and Targeted Diagnostic and Therapeutics Inc. The Pennsylvania Department of Health specifically disclaims responsibility for any analyses, interpretations or conclusions.
Dr. Waldman was the inventor on patents related to this technology, which was licensed to Millennium Pharmaceuticals. The authors have no other conflicts of interest.
For more information, contact Edyta Zielinska, 215-955-5291, edyta.zielinska@jefferson.edu.
About Jefferson
Thomas Jefferson University (TJU), the largest freestanding academic medical center in Philadelphia, is nationally renowned for medical and health sciences education and innovative research. Thomas Jefferson University includes the Sidney Kimmel Medical College (SKMC), one of the largest private medical schools in the country and ranked among the nation's best medical schools by U.S. News & World Report, the Graduate School of Biomedical Sciences and the Jefferson Schools of Nursing, Pharmacy, Health Professions, and Population Health. Jefferson University Physicians is TJU's multi-specialty physician practice consisting of the full-time faculty of SKMC. Thomas Jefferson University partners with its clinical affiliate, Thomas Jefferson University Hospitals.
Article reference: G.P. Marszalowicz "GUCY2C lysosomotropic endocytosis delivers immunotoxin therapy to metastatic colorectal cancer," Oncotarget, 2014.
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