Enzyme Replacement Therapy (ERT)
Purpose: To compensate for deficient or malfunctioning enzymes in patients with inherited metabolic disorders.
Examples
Gaucher Disease: Caused by a deficiency of glucocerebrosidase. ERT with recombinant glucocerebrosidase (imiglucerase) reduces the accumulation of glucocerebroside in tissues.
Fabry Disease: Deficiency of α-galactosidase A leads to lipid accumulation. Administration of recombinant α-galactosidase A alleviates symptoms.
Pompe Disease: Lack of acid α-glucosidase causes glycogen buildup. ERT with alglucosidase alfa helps reduce glycogen storage in muscles. (Enzymes and Isoenzymes)
Thrombolytic Therapy
Purpose: To dissolve blood clots in conditions like myocardial infarction, pulmonary embolism, and ischemic stroke.
Enzymes Used:
Streptokinase: Activates plasminogen to plasmin, which breaks down fibrin clots.
Urokinase: Directly converts plasminogen to plasmin.
Tissue Plasminogen Activator (tPA): Recombinant enzyme that specifically targets fibrin-bound plasminogen, minimizing systemic effects.
Cancer Therapy
Asparaginase: Used in treating acute lymphoblastic leukemia (ALL). It depletes asparagine, an amino acid essential for the survival of certain leukemia cells, leading to cell death.
Digestive Aids
Pancreatic Enzymes: Such as pancrelipase, a mixture of lipase, amylase, and protease, used in patients with pancreatic insufficiency to aid digestion.
Lactase Supplements: Help individuals with lactose intolerance digest lactose in dairy products.
Anti-Inflammatory Agents
Serratiopeptidase and Bromelain: Enzymes used to reduce inflammation and edema in various conditions.
Wound Debridement
Collagenase and Papain: Enzymes applied topically to remove dead tissue and promote healing in chronic wounds and burns.
Treatment of Gout
Uricase (Rasburicase): Converts uric acid to allantoin, which is more soluble and easily excreted, reducing uric acid levels in patients with gout or tumor lysis syndrome.
Enzyme Therapy for Genetic Disorders
Adenosine Deaminase (ADA) Deficiency: Treated with PEGylated ADA (pegademase bovine) to restore immune function in patients with severe combined immunodeficiency (SCID).
