ISSN: 2044-9038 (Print) 2044-9046 (Electronic)

Clinical Practice

Drug Toxicology Research

Standard pharmaceutical toxicology programs run through a series of studies with increasing length, initially checking out a maximum tolerated dose then supporting clinical trials of accelerating length until ultimately a study is run which will support lifetime administration in humans. In the majority of cases for human clinical trials supporting gene therapy, the therapy is given just one occasion , and in many cases, the therapy is meant to be expressed in perpetuity—a single clinical dose may represent “lifetime administration.” Therefore, the preclinical safety program for a gene therapy usually consists of 1 mid- to long-term study, sometimes with interim timepoints to help evaluate the temporal nature of potential effects. The evaluation of drug safety and toxicology has benefited in recent years from biomarker applications, particularly within the area of biomarker discovery. As the number of drugs and drug targets grows, there is a commensurate need for readily measurable markers that can provide an early indicator of undesirable side effects. Indeed, the battery of established markers that can currently be measured in preclinical toxicology studies and in the clinic can all be considered broadly as biomarkers of toxicity; however, there remains a requirement for extra biomarkers which will serveas indicators of potential toxicity at the earliest possible time, and during a manner that's specific to a given target, pathway, or drug chemotype.    

High Impact List of Articles

Relevant Topics in Clinical