ideas almost certainly exist and need to be vetted in order to
effect real change in this complex problem.
The research that underpinned advances in genomics and
powered the molecular biology revolution has demonstrated
what is possible when a country agrees to invest in science,
technology, and innovation. However, this visionary strategy
also underscores the need to develop the infrastructure
necessary to support molecularly based medicine: a robust
network of high-quality, clinically annotated tissue samples,
or biospecimens, collected using global standards in privacy
protection and archiving; and platforms in genomics and
proteomics with the accompanying informatics for data
analysis and communication between research laboratories.
The effectiveness of both molecular classification of tumors
and targeted drug development, for the benefit of patients
everywhere depends on the availability of biospecimens that
are properly consented, collected and annotated.
Figure 14: Development of Imatinib (Gleevec). Improvements in microscopy and chromosomal characterization led to the groundbreaking discovery that
patients with CML had a specific chromosomal rearrangement, now known as the Philadelphia chromosome (pink dots, A) 26. This rearrangement produces
a fusion of two chromosomes that creates a novel protein kinase, called BCR-Abl (green ribbon, B) that is responsible for the immune cells’ uncontrolled
growth. Technologies for chemical-based screens, structural biology, and libraries of inhibitor compounds made it possible for public and private research
collaborations to design and test chemicals (blue structure, B) that could block the activation of BCR-Abl (red ribbon, B) 27. Thus, imatinib (blue structure,
B) became the first rationally designed oncology drug; see Imatinib Sidebar, p. 52.