Drug discovery in the 21st century  pg. 2

Today the properties of known medicinal plants have been largely realized. We rarely have the luxury of embarking on a drug discovery program with this level of confidence in our ultimate success. Instead, we begin with knowledge of a biological system and identification of a potential drug target found among the many potential targets known from our new understanding of the human genome.

Instead of knowing that a drug interacting with this target will have clinical efficacy, we make a hypothesis based on our still limited and imperfect understanding of complex biological systems.

Typically, there are no existing drugs that interact with that target, forcing a search for a novel compound that has the desired effect and then engineering the properties required for a useful drug. This process is expensive and inherently high risk—we may reach the end of a project that cost hundreds of millions of dollars only to find that our original hypothesis was incorrect and the drug has no clinical efficacy or has unforeseen toxicity.

Translation of the extraordinary progress of recent years into fundamental advances in human health and patient care is a major challenge facing today’s biomedical research community. The complexity of this task requires the combined efforts of outstanding scientists, engineers and clinicians with strong expertise in a broad range of disciplines.

Traditionally, the NIH and academic institutions have supported basic biomedical research, while industry has supported commercial development of medicines and medical products. While scientists in academic and other basic science settings have made significant progress in furthering our understanding in biology, chemistry and related disciplines, they often fail to make the critical link that allows this information to be useful in an industry setting.

Likewise, fiscal pressures that govern research efforts in industry make it increasingly difficult for companies to invest significant resources in exploratory projects and basic research that capitalize on translating the most exciting discoveries of basic science in marketable products.

The most innovative and high-impact advances in therapeutics will likely come from aggressive efforts to provide a bridge that allows translation of advances in basic science to novel therapeutic agents. While this translation is clearly the mission of pharmaceutical and biotech companies, is it critical that scientists at NIH-funded institutions focus increasing attention on their role in contributing to the therapeutic discovery process.

In addition to the advances in basic biology, we have realized tremendous advances in combinatorial chemistry, development of large libraries of small molecules, and other approaches to high-throughput synthetic chemistry. These libraries are now widely available to the research community, and new high-throughput screening technologies have been developed that allow more widespread mining of the libraries.

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