Biotechnology drug


Current application of biotechnology already impacts our day-to-day lives. More than 100 years ago, leeches were commonly used to treat illness by so-called Bloodletting i.e. doctors believed that by using leeches to suck the blood out of a patient, diseases were removed from the body. Nowadays, medical biotechnology is a very exciting field of medical science because of sophisticated advancement in this field are occurring due to day-to-day discovery. Even the leech is also getting importance, not for blood-letting but the enzyme found in its saliva that can dissolve blood clots and possibly bound to treat stroke and heart attack.
After the discovery of the structure of DNA by James Watson & Francis Crick, molecular biology has advanced at the astonishing pace providing molecular techniques that give scientists and doctors very powerful tools for combating human diseases.
Recombinant DNA technology procedures allow molecular biologist to splice a DNA fragment from one organism into DNA from another organism and to clone (i.e. make many identical copies of) the new recombinant DNA molecule and applied in advance research fields. This r-DNA technology used to produce a number of antibiotics, hormones and other medically important agents such as clotting factor and human insulin (Hamelin) and diagnose, treat a number of human genetic diseases.
The discovery of Single Nucleotide Polymorphism (SNPs) is partially responsible for a newly emerging field called pharmacology, genomics, customized medicine. It involves designing the most effective drug therapy and treatment strategies based on the specific genetic profile of a patient.
Because of genetic polymorphism, the individuals can react differently to the same drugs which can have varying degrees of effectiveness and side-effects.
Current biotechnological processes essentially involve five different groups of organisms: bacteria (e.g. Escherichia coli, Pseudomonas spp. Serratia mascescens, Erwenia herbícola, Lactococcus lactis and Bacillus sub­tilis), fungi (e.g. Saccharomyces cerevisiae, Pichia and Hansenula, Trichoderma and Aspergilli), plants (e.g. to­bacco plant, rape and transgenic potatoes (Tourte, 1998)), insects (e.g. Spodoptra frugiperda) and few mammalians.
The application of different techniques allows chan­ges to be made in microorganisms, in order to highlight a particular feature or increase their production and ultima­tely the production of new products. For this, conventional genetic techniques such as mutagenesis, fermentation, sexual and Para sexual processes or modern techniques such as recombinant DNA techniques or the hybridoma technique, can be used (Ferreira, Sousa, 1998).
Examples of drugs obtained by biotechnology
Examples of drugs obtained by biotechnology processes
Biopharmaceutical forms are potent, reactive, unsta­ble and very expensive. They have several advantages such as the provision of effective treatments for chronic and uncommon diseases. Recombinant drugs (Factor VIII for hemophilia), offer sa­fer and reduced side effects, improve on existing therapies and can be produced on a large scale by biotechnological processes.
Some new drugs are now in daily use for the tre­atment of chronic and rare diseases, for which there was hitherto no therapeutic or conventional therapies were ineffective.
In the future, biopharmaceuticals may be used against the AIDS virus, different types of cancer, asthma, Parkinson’s and Alzheimer’s disease. There are different groups of biopharmaceuticals, including antibiotics, blood factors, hormones, growth factors, cytokines, en­zymes, vaccines and monoclonal antibodies.
Antibiotics are the largest group in terms of econo­mic importance among the products obtained by fermen­tation. Some examples of antibiotics whose synthesis involved microorganisms include penicillin produced from Penicillium notatum; cephalosporins (usually semi-synthetic process) from the genus Streptomyces; chloram­phenicol from Streptomyces venezuelae; streptomycin from Streptomyces griseus; cycloserine from Streptomyces orchidaceus; clindamycin from Streptomyces lincolnensis; vancomycin isolated from cultures of Streptomyces orien­talis (Nocardia Orientalis); teicoplanin from Actinmopla­nes teichomyceticus and mupirocin from Pseudomonas fluoresces (Osswald, Guimarães, 2001).
Blood Factors
Even with identical causes, two types of hemophilia can be distinguished, namely, hemophilia A (the deficient or abnormal element is Factor VIII or antihemophilic factor A) and hemophilia B (the deficient or abnormal element is Factor IX or antihemophilic Factor B)
TABLE – Some examples of diseases and respective biopharmaceuticals used in the treatment
Active substance
Hepatitis C
Interferon α
Multiple Sclerosis
Interferon β
Renal Cancer
Factor VIII and Factor IX
Human Insulin
In 1982, the first dosage form obtained through biotechnological processes was approved,  as recombinant human insulin for the treatment of patients with diabetes, using recombinant DNA techniques in the bacteria E. coli. Today, recombinant human insulin is available in different concentrations under different forms of therapeutic action (insulin lispro, insulin aspart, insulin glargine – respectively, very fast, fast, long-acting) and for different applications (intramuscular, subcutaneous, etc.).
The recombinant human growth hormone impro­ved the long-term treatment of children whose body was not producing enough growth hormone. Somatropin is a recombinant human growth hormone, marketed under different brand names such as Saizen®, Nutropin®, Huma­trope®, and Serostin®.
Growth Factors
Many Hematopoietic Growth Factors (HGFs) have been isolated, and the understanding of their clinical potential continues to grow. HGFs have had a significant impact on the prevention of infections associated with chemotherapy-induced neutropenia, chemotherapy-induced thrombocytopenia, and chemotherapy-induced anemia. Patients with HIV/AIDS can also be helped by the administration of recombinant HGFs.
Erythropoietin, a hormone produced by the kidneys, stimulates the bone marrow to produce red blood cells. The recombinant human erythropoietin (Procrit®, Epogen®, Eprex®, NeoRecormon®) may appear in different forms: alpha (produced in CHO), beta (produced in CHO) and gamma (produced in BHK). This recombinant growth fac­tor is used in the treatment of anemia associated with renal failure, HIV infections, surgery, etc. Erythropoietin alpha is targeted for the treatment of anemia due to chronic renal failure, HIV infection, and cancer.
Another example is Mircera® (beta methoxypolye­thyleneglycol-epoetin) used for the treatment of anemia associated with chronic renal failure. On the other hand, Palifermin (Kepivance®) is very similar to a natural growth factor that exists in the human body, known as keratinocyte growth factor (KGF). Kepivance® stimulates the growth of cells, helping to reduce the inci­dence, severity, and duration of oral mucositis in cancer patients subjected to intensive care.
Cytokines are molecules that activate the immune cells (e.g. lymphocytes and macrophages), regulate growth and differentiation of immune cells, also important mes­sengers in cells, influencing the response in inflammation, response immune, and tissue repair.
Example of a recombinant enzyme is a plas­minogen activator, known as alteplase (Activase®), used to dissolve blood clots formed in the circulatory system, which can cause heart attacks, pulmonary embolisms, and strokes. Elaprase® (idursulfase) is another enzyme produced by biotechnological processes used in the treatment of patients with Hunter syndrome (patients are not able to de­grade glycosaminoglycans, which gradually accumulates in cells, affecting most organs, causing difficulty breathing and walking). Another case of using biotechnology to produce drugs in the production of essential enzymes in patients with Gaucher syndrome type 1 and 3 (a disease charac­terized by the deficiency of the beta-glucosidase enzyme). This disease is usually characterized by a neurological disorder that includes mental degenera­tion and seizures. There are a few effective therapies for treatment including VPRIV® (velaglucerase alpha – a hu­man cell line derived enzyme replacement therapy – for the long-term treatment of type 1 Gaucher disease), the Protalix Biotherapeutics (taliglucerase alpha – a plant cell-expressed recombinant glucocerebrosidase enzyme), Cerezyme® (imiglucerase – produced by recombinant DNA technology using mammalian cell culture, CHO) and Za­vesca® (miglustat -reduces the harmful build up of fatty substances throughout the body by reducing the amount of glycosphingolipids produced by the body – used in patients who cannot be treated with enzyme replacement therapy).
A different enzyme produced using human cell lines is alfagalsidase (Replagal®). This enzyme is a copy of the human enzyme used in enzyme replacement therapy for Fabry’s disease (chronic and progressive genetic diseases caused by absence or deficiency of an enzyme called alpha-galactosidase A, responsible for the decomposition of lipids in the body, consequently the lipids accumulate in vital organs causing serious problems.
Currently, vaccines are not only developed against infectious diseases but also against drug abuse (nicotine, cocaine) and against allergies, cancer and Alzheimer’s disease. Despite the success of conventional vaccines, there are still many infectious diseases and other chronic diseases against which no effective vaccine exists. In addition, the growing resistance to the existing arsenal of antibiotics increases the need to develop vaccines against common bacterial infections. It is expected that novel vaccines against several diseases will become available, and in these case, recombinant technologies hold great promise. Although conventionally produced vaccines are generally harmless, some of them may, rarely, contain infectious contaminants. Vaccines whose active ingre­dients are recombinant antigens do not carry this slight risk.
Vaccines produced by recombinant DNA techni­ques have been used to combat seasonal influenza virus (Fluarix®, Istivac®, Fluzone®, FluMist®, Agriflu® etc.) and hepatitis A and B. The first vaccine against hepatitis B was made from plasma derived from patients with chronic hepatitis B, and a recombinant vaccine whose sole active ingredient is a recombinant antigen has now replaced it (Steinberg, 1998b).
There are also other types of vaccines produced by genetic engineering, using the yeast Saccharomyces cerevisiae for the production of HBsAg or by entering the HBsAg gene in mammalian cells (Recombivax HB®, Engerix B®). The Ambirix® vaccine is another example of a bivalent vaccine used to protect against hepatitis A and hepatitis B (diseases affecting the liver) in children aged between 1 and 15 years old, who have no immunity to these diseases. This vaccine con­sists of inactivated hepatitis A virus (produced in human diploid cells, MRC-5) and surface antigen of hepatitis B (produced in Saccharomyces cerevisiae yeast cells by recombinant DNA technology). Another example of a vaccine used to protect against hepatitis A and B infection is Twinrix®, which contains inactivated hepatitis A virus and parts of the hepatitis B virus as active substances (surface antigens obtained by recombinant DNA technology).
On the other hand, the Myobloc® vaccine is a botu­linum toxin type B vaccine for the treatment of cervical dystonia, produced by fermentation using the bacterium Clostridium botulinum type B, Botulinum toxin type A (Botox®) is indicated for the treatment of cervical dystonia. Example of a vaccine produced by genetic engineering is Dukoral®, used in protection against cholera (an ex­tremely serious disease caused by V. cholerae, which is contracted from contaminated food or water and causes severe diarrhea). This vaccine contains small amounts of dead cholera bacteria and a part of the cholera toxin called “B subunit” (produced by recombinant DNA).
Malaria, cholera, herpes, lupus, rheumatoid arthritis, tuberculosis, HIV/AIDS, cancer and gastrointestinal dise­ases are diseases for which effective vaccines are expected to be developed. In 1998, U.S. researchers announced that they had genetically engineered potatoes to produce a “vaccine” against cholera (Arakawa et al., 1998). We can also highlight other vaccines that are on the horizon such as varicella, otitis and acute chronic infectious respiratory diseases such as pneumonia caused by Streptococcus pneumonia, Haemophilus influenza type B, parainfluen­za virus, rotavirus, Shigella, Vibrio cholerae and certain types of Escherichia coli.
Monoclonal antibodies
Monoclonal antibodies provide targeted immuno­suppression that, when used in conjunction with specific maintenance immunosuppression, may allow more speci­fic therapy and can be used not only for tumor therapy but also in other therapies or diagnoses. In recent years, this group of drugs has undergone more extensive research, and shown a very promising future, as evidenced by the number of drugs that are already on the market.
Modern medical biotechnology uses a wide range of methods to diagnose and treat diseases – from the biotechnological production of simple natural products to gene therapy. The most important group of biotechnological drugs by far, however, are the therapeutic proteins. Most therapeutic proteins are chemical messengers, enzymes or, especially in recent times, monoclonal antibodies. Some occur naturally in the body. For example, many long-established biotechnological products such as the hormones insulin and erythropoietin (EPO) are natural chemical messengers. Now, these molecules can be produced in genetically modified cells that carry the hereditary information for producing human protein.
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Written by: Ranjini Chakraborty.

Health and well-being

I am an MSc in Chemistry and working in Quality Assurance department of a Pharmaceutical product manufacturing company since last eight years. With this, I am learning and working on different issues related to health awareness, positive mental attitude, and prevention of microbiology contamination, creating awareness on the hazardous chemical used in the FMCG product, packaged food product, cosmetics, etc. This site is created to promote awareness on the said issues to keep people healthy and happy.

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