Biotherapeutics: Specialized medicine for life-threatening, chronic diseases

MANILA, Philippines - Biotherapeutics are specialized medicine that have a successful record in treating many life-threatening and chronic diseases.

Unlike conventional medicine that are made up of various chemical compounds, biotherapeutics utilize living cells — either bacteria or more complex mammalian cells — as “mini-factories” to manufacture the appropriate proteins.

These proteins, in turn, either initiate or promote a vital physiological process or block or inhibit a particular step of a disease process. This is why biotherapeutics are often called the “smart bombs” of medicine.

Also called biological medicine, biotherapeutics are used to treat, among others, anemia (erythropoietin; methoxy polyethylene glycol-epoetin beta), diabetes (insulin), blood clots that can cause heart attack and stroke (alteplase, tenecteplase), age-related macular degeneration (ranibizumab), lung infections (dornase alfa), rheumatoid arthritis (rituximab, tocilizumab), multiple sclerosis and lupus (ocrelizumab), chronic hepatitis B and C (pegylated interferon alpha), asthma (omalizumab), and various forms of cancers (trastuzumab, bevacizumab, rituximab, pertuzumab).

Advanced expertise, technology required

Because the proteins used in biotherapeutics are complex molecules that require advanced expertise and technology to synthesize, developing and manufacturing biotherapeutics require substantial investments in research, personnel, and equipment.

To ensure the production of a safe, active and consistent biotherapeutic product, biotechnology companies monitor, control and document all aspects of the complicated process — from the raw materials and environment to the utilities, equipment, and procedures.

The first step in manufacturing a protein or antibody is to genetically engineer a cell so that it produces the desired product. Genetic engineering involves the introduction of the genetic information, DNA, which provides the cell with the instructions it needs to produce the protein or antibody.

Once a cell has been engineered to express the product of interest, it is used to establish a “cell line,” i.e. thousands of copies of this original cell. This cell line is then frozen and stored for use in the manufacturing process.

Standardization is key

Unlike conventional drugs, biotherapeutics are too complex to be characterized by chemical and physical methods. According to the World Health Organization (WHO), production and quality control methods used in the production of biotherapeutics must be highly standardized to account for the inherent variability in raw materials, the potential for contamination with extraneous agents, and the relative instability of many active ingredients.

“Ensuring the quality, consistency, and efficacy of (biotherapeutics) has become an increasingly difficult challenge for manufacturers and regulatory authorities alike,” WHO said.

Working with the scientists, medical experts, regulatory authorities, manufacturers and expert laboratories worldwide, WHO has established International Biological Reference Preparations necessary to standardize biological materials as well as developed guidelines and recommendations on the production and control of biological products and technologies.

These norms and standards, based on scientific consensus achieved through international consultations, assist WHO member-states in ensuring the quality and safety of biological medicine and related in vitro biological diagnostic tests worldwide.