Elaprase

Elaprase is a recombinant DNA-derived form of the human enzyme iduronate-2-sulfatase. It is used as an enzyme replacement therapy for patients with Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II). Elaprase is produced by Shire plc and was approved by the Food and Drug Administration (FDA) in 2006.

Medical Uses

Elaprase is indicated for the treatment of Hunter syndrome, a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This enzyme deficiency leads to the accumulation of glycosaminoglycans (GAGs) in various tissues and organs, causing a wide range of symptoms including developmental delays, organomegaly, and skeletal abnormalities.

Mechanism of Action

Elaprase works by replacing the deficient or absent iduronate-2-sulfatase enzyme in patients with Hunter syndrome. This enzyme is essential for the breakdown of GAGs, specifically dermatan sulfate and heparan sulfate. By providing the functional enzyme, Elaprase helps reduce the accumulation of GAGs, thereby alleviating some of the symptoms associated with the disorder.

Administration

Elaprase is administered via intravenous infusion. The recommended dosage is 0.5 mg/kg of body weight, given once weekly. The infusion typically lasts for 3 to 4 hours and should be administered under the supervision of a healthcare professional.

Side Effects

Common side effects of Elaprase include headache, fever, rash, and infusion-related reactions such as flushing, chills, and hypotension. Severe allergic reactions, including anaphylaxis, have also been reported. Patients should be monitored for signs of hypersensitivity during and after the infusion.

Regulatory Status

Elaprase received orphan drug designation from the FDA and the European Medicines Agency (EMA) due to the rarity of Hunter syndrome. It was approved by the FDA in July 2006 and by the EMA in January 2007.

See Also

• Hunter syndrome
• Mucopolysaccharidosis
• Enzyme replacement therapy
• Shire plc
• Recombinant DNA

Related Pages

• Hunter syndrome
• Mucopolysaccharidosis II
• Enzyme replacement therapy
• Shire plc
• Recombinant DNA

Engineered Monoclonal Antibodies

Diagram of engineered monoclonal antibodies

Engineered monoclonal antibodies are a class of biological therapies that are designed to target specific antigens on the surface of cells. These antibodies are produced using recombinant DNA technologies and are used in the treatment of various diseases, including cancer, autoimmune disorders, and infectious diseases.

Structure and Function

Monoclonal antibodies are composed of two identical heavy chains and two identical light chains, forming a Y-shaped molecule. The tips of the "Y" contain the antigen-binding sites, which are highly specific to the target antigen. This specificity allows monoclonal antibodies to bind to their target with high affinity, blocking or modulating the function of the antigen.

Types of Engineered Monoclonal Antibodies

There are several types of engineered monoclonal antibodies, each designed for specific therapeutic purposes:

• Chimeric antibodies: These antibodies are composed of murine (mouse) variable regions and human constant regions. They are less immunogenic than fully murine antibodies.

• Humanized antibodies: These antibodies are mostly human, with only the antigen-binding sites derived from murine sources. This reduces the risk of immune reactions.

• Fully human antibodies: These are entirely human in origin, produced using transgenic mice or phage display technologies.

• Bispecific antibodies: These antibodies are engineered to bind two different antigens simultaneously, offering unique therapeutic mechanisms.

Applications in Medicine

Engineered monoclonal antibodies have revolutionized the treatment of many diseases:

• Cancer therapy: Monoclonal antibodies can target specific tumor antigens, leading to direct tumor cell killing or recruitment of immune cells to attack the tumor.

• Autoimmune diseases: By targeting specific components of the immune system, monoclonal antibodies can reduce inflammation and tissue damage in diseases such as rheumatoid arthritis and multiple sclerosis.

• Infectious diseases: Monoclonal antibodies can neutralize pathogens or their toxins, providing passive immunity or enhancing the host's immune response.

Production

The production of engineered monoclonal antibodies involves several steps:

1. Antigen identification: The target antigen is identified and characterized. 2. Hybridoma technology: B cells from immunized animals are fused with myeloma cells to create hybridomas that produce the desired antibody. 3. Recombinant DNA technology: Genes encoding the antibody are cloned and expressed in suitable host cells, such as Chinese hamster ovary cells. 4. Purification and formulation: The antibodies are purified and formulated for clinical use.

Challenges and Future Directions

While engineered monoclonal antibodies have shown great promise, there are challenges such as high production costs, potential for immune reactions, and the development of resistance. Ongoing research aims to improve antibody design, reduce immunogenicity, and enhance therapeutic efficacy.

Related Pages

• Monoclonal antibody therapy
• Biopharmaceutical
• Immunotherapy
• Hybridoma technology

This article is a stub related to hospitals. You can help WikiMD by expanding it!