Recombinant blood products and therapeutic enzymes

CH12 RECOMBINANT BLOOD PRODUCTS AND THERAPEUTIC ENZYMES... 

Recent reviews Hst more than 400 biotechnology-based pharmaceutical formulations either registered in clinical trials or undergoing review by the regulatory agencies for the treatment of nearly 150 diseases including cancer, infectious diseases, autoimmune diseases, and AIDS/HIV (1,2). Biotechnology-based pharmaceuticals already on the markets include recombinant blood factors, recombinant hormones, cytokines, vaccines, monoclonal antibody-based products, and therapeutic enzymes. 

Proteins intended for therapeutic use, including C5rtokines (e.g., interferons), enzymes (e.g., thrombol5rtics), and other novel proteins, except for those that are specifically assigned to CBER (e.g., vaccines and blood products). This category includes therapeutic proteins derived from plants, animals, or microorganisms, and recombinant versions of these products. 

For this purpose, the SOD enz5mies (natural, recombinant, and modified) have been used in prechnical animal studies and chni-cal trials 3a,b) and have been shown to possess efficacy in the disease states associated with the overproduction of 02. SODs have also proven usefiil as a probe for detection of this radical ). An example is Orgotein (bovine Cu/Zn SOD) which showed promising results as a human therapeutic 12). However, there are major drawbacks associated with the use of the enzymes as potential medicaments immunological problems, lack of oral activity, short half-fives in vivo, costs of production, solution instability, reduced efficacy due to the large size, and inability to access the target tissues (e.g., native enzymes do not penetrate the blood brain barrier) (36). 

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