Most of the recent progress and promise of cancer research is based on new understandings of cancer on a molecular and cellular level. What does this mean for treatment?
Robert Weinberg, PhD
Daniel K. Ludwig Professor for Cancer Research
Massachusetts Institute of Technology
C. Norman Coleman, MD
Director, Radiation Oncology Sciences Program
National Cancer Institute
The big hope for the future is that if we understand the molecular defects inside cancer cells, we can target them, and we can create drugs that specifically interfere with those defects, allowing us to kill cancer cells selectively while leaving normal cells untouched, thereby reducing undesirable side effects, and there have been several home runs that have been scored in recent years using this rational drug design. Gleevec is perhaps the best known of these.
Molecular therapeutics are therapies designed for a relatively specific molecular process. A perfect example is the drug Gleevec, which targets a mutation in chronic myelogenous leukemia and the mutation is the central feature of that disease, so without that mutation you wouldn't have the disease. If you can knock out the function of that mutation, the disease goes into remission. So molecular therapeutics are therapies designed to hit a specific molecular target.
What's interesting is a lot of the drugs that we use and that work, we really don't quite understand at the molecular level how they work. So as we understand even drugs like platinum and Taxol and so forth, a lot of them are molecularly targeted drugs when we understand their pathways.