Where does medicine come from?

The challenge facing medicinal science is to fill the demand for new drugs. Despite technological advances, there are fewer new medicines approved every year. The task of getting a drug to your local pharmacy is complex, arduous, and frequently expensive. Therefore, pharmaceutical companies have established elaborate business models to research, develop, and produce molecules to treat diseases

Phase One: Drug Discovery

Drug discovery relies on the collaborative efforts of researchers including chemists, biologists, and physicians

• Chemists build molecules that may eventually become drugs while biologists investigate the relevant molecules that cause diseases
• They select drug targets in the form of large biomolecules (such as proteins) or cancer cells, then search for molecules that will disrupt the protein or kill the malignant cell

Phase 2- Clinical Trials

These are the stage of drug development where a drug’s effect on humans is tested.

• In clinical trials, the drug is primarily assessed for how effectively it treats the disease it is designed to target and whether it is safe for use by patients.

Challenges Going Forward

As new technologies in genetics and disease identification become available, our understanding of the molecular basis for diseases will improve

• Unfortunately, the rate at which new diseases are emerging appears to be outrunning the pharmaceutical industry’s ability to find remedies
• Drugs can become ineffective through the evolution of resistance in certain diseases-causing bacteria, viruses, parasites, and cancers

Drug Discovery: Natural Products

Historically, medicines were administered in the form of herbal concoctions, and many traditional medicines continue to be taken this way.

• As science advanced, chemists were able to extract the active ingredients from natural sources to make more potent medicines such as aspirin from the bark of the willow tree.

References:

Corey, E. J., Czakó, B., & Kürti, L. (2001) Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings. Chem. Del. Rev. 46:3-26.

• Goodman, J., & Walsh, V. (2008) The Story of Taxol: Nature and Politics in the Pursuit of an Anti-Cancer Drug. In Drug Discovery and Development, Volume 2: Drug Development. Ed. Chorghade, M. S. Hoboken: John Wiley & Sons, Inc., 2007
• Clinical Trials
• Lipinski, C. A., Lombardo, F., Dominy, B.W., & Feeney, P.J. (2011) Federal Research Center Will Help Develop Medicines. The New York Times, January 2011

Drug Discovery: High-Throughput Screening

Robots use robots to mix miniscule amounts of potential drugs with tiny samples of the drug target.

• Through the screening process, a molecule may show potential as a drug by binding to the target biomolecule (i.e., the key for the lock).

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