The genetic component of variations in human responses to pharmacological agents is called pharmacogenetics while the molecular basis for these variations are most often identified as pharmacogenomics. Pharmacogenomics as a field of scientific endeavor is so new that in the scientific literature the two terms are often used interchangeably. In fact, the search for new drugs at the molecular level start with the identification of variations in DNA sequences whose products produce alterations in the amino acid structure of the active (...) portion of a protein which are then identified with the clinical disorder in question. This process is just the reverse of standard pharmaceutical approaches which begin with exploration of the physiological and potentially the biochemical basis of the specific disorder under study. The completion of the first draft of the human genome in the first months of the new millenium has increased expectations that molecular genetic approaches to drug disovery and development will significantly shorten the time-frame of new drug testing, significantly reduce the level of adverse events and anable the design of drugs to treat those with unique disorders.Based on such rosy expectations and the potential economic opportunities afforded by those who obtain such patents, there has been a rush of patent applications both in the United States and in Europe by private industry and university laboratories conducting genomic research to secure the rights to specific DNA sequences and federal agencies attempting to place the control of such information in the public domaine. This paper will document and discuss from a historic prospective the public policy and bioethical issues associated with the availability of patents for drug development. (shrink)

The genetic component of variations in human responses to pharmacological agents is called pharmacogenetics while the molecular basis for these variations are most often identified as pharmacogenomics. Pharmacogenomics as a field of scientific endeavor is so new that in the scientific literature the two terms are often used interchangeably. In fact, the search for new drugs at the molecular level start with the identification of variations in DNA sequences whose products produce alterations in the amino acid structure of the active (...) portion of a protein which are then identified with the clinical disorder in question. This process is just the reverse of standard pharmaceutical approaches which begin with exploration of the physiological and potentially the biochemical basis of the specific disorder under study. The completion of the first draft of the human genome in the first months of the new millenium has increased expectations that molecular genetic approaches to drug disovery and development will significantly shorten the time-frame of new drug testing, significantly reduce the level of adverse events and anable the design of drugs to treat those with unique disorders.Based on such rosy expectations and the potential economic opportunities afforded by those who obtain such patents, there has been a rush of patent applications both in the United States and in Europe by private industry and university laboratories conducting genomic research to secure the rights to specific DNA sequences and federal agencies attempting to place the control of such information in the public domaine. This paper will document and discuss from a historic prospective the public policy and bioethical issues associated with the availability of patents for drug development. (shrink)

The rapid sequencing of entire genomes based in large measure on a DNA cloning procedure, the polymerase chain reaction (PCR), has opened new frontiers in the discovery process for novel therapeutic agents. DNA cloning is a basic tool in genomics and it has been used for over a decade. Drug discovery is currently focused on the identification of gene databases, gene arrays and protein arrays aimed at therapeutic modulation of disease-related genes—which require procedures that may involve cloning techniques. Currently, cloning (...) for pharmacologic products is most applicable in the preclinical area of drug discovery. For example, the identification of specific receptor subtypes occurred via cloning whereas traditional pharmacologic approaches that use agonists and antagonists may have failed to separate binding affinities. Successful therapeutic agents where cloning played vital role resulted in the development of synthetic insulin, growth hormone, erythropoiten and other tissue factors in blood products, Vaccines may be another successful application derived from cloning. Another area where cloning can make a significant impact upon human therapeutics is that this technique may provide a new source of cells for tissue engineering and transplantation. Stem cells, and in particular, embryonic stem cells can differentiate into other cell types such as hepatocytes and neurons to provide normal products for hepatic and neurological diseases. However, the use of cloning embryonic stem cells introduces important ethical, medical and legal issues which require that leaders in government, academic, and religious groups be fully educated in these applications. A close working relationship among these diverse groups is needed to respond wisely to the introduction of new therapeutic opportunities while at the same time providing protection for the individual and the public at large from unethical practises. (shrink)