Course: SAT 170 Introductory Biotechnology

Unit 11 Learning Module

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- Unit 11
- Table of Contents (Selected)
- Unit 11 Key Terms (Selected)

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Biopharmaceutics and Pharmacogenomics

This figure diagrams the overall process that is used to tailor medical treatments to a particular individual based on their genetic make-up.
Biopharmaceutics and pharmacogenomics are fields that are often intertwined so we will discuss them together here. Biopharmaceutics is a field which studies medical drugs that are made using biotechnology; such drugs are known as biopharmaceuticals. Pharmacogenomics is a field of study which focuses on designing effective drug therapy for an individual based on that individual’s genetic make-up. The idea being that different people will react to and metabolize drugs differently, this can influence the efficacy and toxicity of these drugs in that individual. With pharmacogenomics, treatments can be designed to be more specific and effective for a particular individual.

Protein therapy is an example of biopharmaceutics which often relies on genetically modified organisms (GMO’s). Recall from unit 7 that protein therapy focuses on replacing a missing or non-functional gene, researchers are replacing a missing or non-functional protein. Protein therapy has been practiced for decades but the techniques have changed significantly with the introduction of genetic engineering. At one time, the proteins used for protein therapy were sourced from healthy organisms that naturally produced that particular protein. These proteins can be isolated from the tissues of these organisms once they die or in some cases can be isolated from their bodily fluid such as their urine. Once the protein has been extracted and purified from these sources it can be used for protein therapy. The major downfall to these isolation and purification methods is the limited amount of proteins you can collect from these samples for therapy as well as the time and cost associated with these collection methods. Additionally, since the source of these proteins is often non-human (e.g. the isolation of insulin from the pancreases of pigs for the treatment of diabetes), there is some potential for unwanted reactions and reduced effectiveness. Modern protein therapy, which relies on genetic engineering, is done following these basic steps:  isolating a gene from a healthy individual, inserting that gene into an organism (or host cell) which will express that gene, collecting and purifying the protein product, and finally inserting the protein product into an individual with the disease. The host cells which are used as protein factories vary from bacteria, to fungi, to plants and animals. This is an example of how transgenic plants and animals can provide valuable therapeutic agents as was mentioned in our last unit. The uses of insulin for the treatment of diabetes as well as human growth hormone for the treatment of dwarfism are examples of successful applications of such forms of protein therapy.

The process which forms hybridomas from the fusion of antibody producing cells with myeloma cells that can then be used for the mass production of a particular antibody.

Immunotherapy represents a diverse set of biotechnology techniques which are employed in the field of medicine. One such example if the use of antisera. Antiserum is derived from the blood of an animal that has been exposed to some form of antigen. An antigen is a substance which triggers an animal to produce antibodies. Examples of antigens include toxins and cell surface markers present in bacteria.  Thus, a sample of antiserum will contain antibodies that are ready to attack the antigen to which that animal has been exposed. Antibodies that are able to attack one antigen very specifically are referred to as monoclonal antibodies (Mabs). Because of their therapeutic potential, a lot of interest and research has been devoted to designing ways that these Mabs can be made using cell cultures rather than animals. The process most commonly used involves the formation of hybridomas. A hybridoma results from the fusion of an antibody producing immune cell, with a cancer cell known as a myeloma. The immune cell will provide the traits needed to make the antibody, while the cancer cell allows the cells to divide indefinitely creating an effective antibody producing culture. Other forms of immunotherapy include the use of vaccines such as those which we introduced in unit 1. A vaccine is used to stimulate the immune response. One form of vaccine is to inject a patient with an attenuated (weakened) form of the pathogen. This will trigger the immune response without causing the disease. Vaccines can also be genetically engineered by creating genetically modified organisms that will carry a gene for a particular antigen. The antigen can then be isolated from that GMO and used as a vaccine to trigger the immune response.