Introduction in Medical Chemistry

May 14, 2009 – 1:51 pm

Designing drug molecules to alleviate human disease and suffering is a daunting yet exhilarating task. How does one do it? How does a researcher sit down, paper in hand (or, better yet, a blank computer screen), and start the process of creating a molecule as a potential drug with which to treat human disease? What are the thought processes?

What are the steps? How does one select a target around which to design a drug molecule? When a researcher does design a molecule, how does she or he know if it has what it takes to be a drug?

These are important questions. The previous century ended with an explosion of activity in gene-related studies and stem cell research; the new one is emerging as the “Century of Biomedical Research.”We have now witnessed the global spectre of SARS (Severe Acute Respiratory Syndrome) and avian flu, which has emphasized the looming importance of infectious disease to global health.

Concerns about the capacity of “Mad Cow” disease to infect humans have focused attention on the safety of our food supply. AIDS and obesity-related disorders have not gone away, but rather are increasing in incidence and prevalence. Long-recognized diseases, such as stroke and Alzheimer’s dementia, are becoming more common as a greater proportion of the human population reaches old age.

Not surprisingly, the need for drug discovery to address these important diseases is increasingly being recognized as a societal priority. Not only is drug discovery important to the medical health of humankind, it is also an important component of our economic health. New chemical entities (NCEs) as therapeutics for human disease may become the “oil and gas” of the 21st century. As the world’s population increases and health problems expand accordingly, the need to discover new therapeutics will become even more pressing. In this effect, the design of drug molecules arguably offers some of the greatest hopes for success.


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