enzymtherapy

Enzymotherapy, or enzymotherapy, refers to the therapeutic use of enzymes or enzyme systems to treat disease. It encompasses enzyme replacement therapies for inherited deficiencies, digestive enzyme supplements for pancreatic or gut disorders, thrombolytic enzymes to dissolve clots, and oncologic approaches that use enzymes to activate prodrugs or to deplete tumor substrates. The term covers both approved clinical treatments and experimental strategies.

Enzyme replacement therapy (ERT) provides functional enzyme to patients with metabolic disorders caused by deficient or defective enzymes. Approved ERTs include enzymes such as imiglucerase, agalsidase beta, alglucosidase alfa, and taliglucerase alfa, used for various lysosomal storage diseases. Digestive enzyme products, such as pancrelipase, aid digestion in pancreatic insufficiency. Thrombolytic enzymes, including streptokinase and tissue plasminogen activator (tPA), dissolve clots in acute cardiovascular or pulmonary events.

In cancer therapy, enzymes are used in targeted strategies like antibody-directed enzyme prodrug therapy (ADEPT) and gene-directed enzyme prodrug therapy (GDEPT) to convert inactive prodrugs at tumor sites, increasing local drug activity while limiting systemic exposure. L-asparaginase is a traditional enzyme used to deplete asparagine in acute lymphoblastic leukemia. Enzyme therapy also includes enzyme-based wound debridement, such as collagenase formulations.

Delivery and safety vary by application. Most systemic enzyme therapies are given intravenously or subcutaneously; pancreatic enzymes are taken orally. Immunogenicity, allergic reactions, and dose-related risks are considerations in clinical use. Regulation and availability differ by country, with several therapies requiring specialist oversight.

Research continues in improving targeting, reducing immunogenicity, and expanding the range of enzymes used in therapy, including newer prodrug and gene-delivery approaches.