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Science Overview

Biospecimens Are Critical to Cancer Research

Biomedical cancer research relies upon the use of human biological specimens ("biospecimens") to help clarify the nature of health and disease. In recent years, the use of well-characterized human cancer biospecimens has significantly accelerated cancer research as shown in the following two cases:

Case 1: HerceptinĀ®

The development of trastuzumab (Herceptin) for the treatment of breast cancer illustrates the importance of access to well-characterized and preserved biospecimens. Human epidermal growth factor receptor 2 (EGRF-2) normally controls aspects of cell growth and division, but it was shown in tumor samples from the National Cancer Institute ("NCI") to be amplified in 20% - 30% of breast cancer cases. The development of an antibody (Herceptin) to this receptor as a therapeutic agent might not have succeeded if it had been tested on the general breast cancer patient population. The biospecimens, however, pointed researchers toward a targeted, highly effective therapy for a sub-population of breast cancer patients.

Case 2: GleevecĀ®

Imatinib mesylate (Gleevec) was originally developed for the treatment of chronic myeloid leukemia by targeting the BCR-ABL protein. After conducting molecular profiling studies on biospecimens collected from different tumor types, scientists discovered that a mutant form of KIT, a protein related to BCR-ABL, is responsible for the progression of a rare but deadly type of cancer, gastrointestinal stromal tumors ("GIST"). This led to the hypothesis that Gleevec could be used to treat GIST, and subsequent clinical trials confirmed the effectiveness of this new indication for Gleevec


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