British journal of pharmacology
AUTHORS
- PMID: 25572005[PubMed].
ABSTRACT
Traditional pharmacology is defined as the science that deals with drugs and their actions. While small molecule drugs have clear advantages, there are many cases where they have proved to be ineffective, prone to unacceptable side effects, or where due to particular disease etiology small molecules cannot possibly be effective. A dominant feature of the small molecule drugs is their single-mindedness: they provide either continuous inhibition or continuous activation of the target. Because of that, these drugs tend to engage compensatory mechanisms leading to drug tolerance, drug resistance, or, in some cases, sensitization, and consequent loss of therapeutic efficacy over time and/or unwanted side effects. Here we discuss new and emerging therapeutic tools and approaches that have potential for treating the majority of disorders for which small molecules are either failing or cannot be developed. These new tools include biologics, such as recombinant hormones and antibodies, as well as approaches involving gene transfer (gene therapy and genome editing) and introduction of specially designed self-replicating cells. It is clear that no single method is going to be a “silver bullet”, but collectively these novel approaches hold promise for curing practically every disorder.
Traditional pharmacology is defined as the science that deals with drugs and their actions. While small molecule drugs have clear advantages, there are many cases where they have proved to be ineffective, prone to unacceptable side effects, or where due to particular disease etiology small molecules cannot possibly be effective. A dominant feature of the small molecule drugs is their single-mindedness: they provide either continuous inhibition or continuous activation of the target. Because of that, these drugs tend to engage compensatory mechanisms leading to drug tolerance, drug resistance, or, in some cases, sensitization, and consequent loss of therapeutic efficacy over time and/or unwanted side effects. Here we discuss new and emerging therapeutic tools and approaches that have potential for treating the majority of disorders for which small molecules are either failing or cannot be developed. These new tools include biologics, such as recombinant hormones and antibodies, as well as approaches involving gene transfer (gene therapy and genome editing) and introduction of specially designed self-replicating cells. It is clear that no single method is going to be a “silver bullet”, but collectively these novel approaches hold promise for curing practically every disorder.