What are the benefits of the drug development process?

The theory behind much of today's drug discovery, known as rational drug design, is that if a target is known for a particular disease, different molecules that will interact with this target, altering the course of the disease can then be found. The process of finding a new drug against a chosen target for a particular disease usually involves high-throughput screening (HTS), involving large libraries of chemicals that are tested for their ability to modify the target HTS, also demonstrates how selective the compounds are for the chosen target. The goal of this process is to find a molecule that will interfere with only the chosen target, but not other, related targets. For this reason, other screening runs will be made to see whether the "hits" against the chosen target will interfere with other related targets. This is the process of cross-screening. Cross-screening is important because the more unrelated targets a compound hits, the more likely that off-target toxicity will occur with that compound once it reaches the clinic, thus increasing side effects and drug interactions.

Recent evidence supporting the theory of rational drug design is the discovery and design of imatinib, an anti-cancer drug. This drug specifically targets a fusion protein that is only present in the Philadelphia Chromosome, an abnormal minute chromosome found in white blood cells, in certain related leukemias. It is different from previous cancer drugs because it only targets cancer cells, rather than all rapidly diving cells in the body.

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