Rational drug design
Rational drug design is a focused strategy for drug development that is grounded on a solid foundation in the basic sciences, driven by the rigor of laboratory research and proven clinically.
Drug discovery technology
At Novartis Oncology, our targeted research strategy leverages biomarkers and targeted drug development focused on individual patients. Our targets are selected based on extensive research to understand the molecular structure of drug receptors, the specific biochemical characteristics of intracellular proteins, the unique products of aberrant genes and the cellular processes underlying the etiology of cancer.
State-of-the-art technology is used to identify the best compounds to hit specific molecular targets accurately and effectively. When a natural compound is not readily found, synthetic molecules that mimic the natural ligands of receptors can be engineered from the understanding of the underlying mechanisms gleaned from previous research.
Working from the roadmap of life—the human genome—researchers have gained an understanding of the complete genetic makeup of human cells, which has provided a new resource for current drug design. The challenge of functional genomics is to understand the function of each gene and its interaction with each of the nearly 30,000 others in the genome.
Tools are now available to facilitate microexperiments that can answer complex questions about each gene in the genome simultaneously. For example, a single experiment can identify the genes that are expressed at high levels in lung tumors, but not in healthy lung tissues.
This type of research can be used to identify molecular targets and compounds, to genetically profile tumors or patients, and in the future, to guide and customize therapeutic choices.
Novartis Oncology uses the lessons learned in the laboratory to deliver clinical benefits. Additionally, observations made in clinical settings help to refocus or innovate basic research hypotheses. Active basic and clinical research programs ensure that optimal cancer treatments will be developed as rapidly as possible.
"As we continue to research the details behind small molecules, we can attack genetic abnormalities more efficiently and aggressively."—William R. Sellers, MD
Vice President and Global Head of NIBR Oncology Research