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Enzymes in Medicine and Enzymopathies

For medical students2 min readUpdated 2026-10-10

Enzymotherapy refers to the use of specific enzymes as adjunctive therapeutic agents for treating various diseases. In contrast, enzymopathies are pathological conditions that develop due to impaired normal functioning of intracellular enzymes.

EnzymotherapyThe use of enzyme preparations to treat a wide range of pathologies.
HeredityPrimary enzymopathies are always associated with genetic defects.
Blood TumorsAsparaginase deprives leukemic cells of a necessary amino acid.
SurgeryProteases effectively debride purulent wounds of dead cells.

Main Directions of Enzymotherapy

Enzyme preparations are actively introduced into clinical practice as adjunctive therapy. Depending on their substrate specificity, they are prescribed for inflammation, thrombosis, viral infections, and oncology.

1. Preparations with Proteolytic Activity This group includes trypsin and chymotrypsin. They are used exclusively topically. The primary task of such enzymes is the cleavage of proteins in necrotic tissues, making them indispensable for debriding purulent wounds. In addition, proteases help remove blood clots and effectively liquefy excessively viscous secretions that accumulate during respiratory tract inflammation.

2. Application of Nucleases The enzymes ribonuclease and deoxyribonuclease are used in medicine as specific antiviral agents. Their direct indication is the treatment of viral eye conditions, such as adenoviral conjunctivitis and herpetic keratitis.

3. Fibrinolytic Agents To combat thrombosis, agents capable of dissolving pre-existing blood clots are used; this process is called thrombolysis. Clinicians have access to drugs such as fibrinolysin, streptokinase (also known as streptolyase), streptodekase, and urokinase. The main indications for their administration are life-threatening thromboses and thromboembolisms.

Specific Enzymes: Hyaluronidase and Asparaginase

In addition to proteases and fibrinolytics, targeted enzymes are used in therapy.

What are Enzymopathies?

While enzymotherapy is treatment using enzymes, enzymopathies are diseases whose primary cause is a disruption in the cell's own enzymes.

Globally, all enzymopathies are divided into two main categories:

  1. Primary. These are hereditary pathologies in which the enzyme defect is genetically encoded.
  2. Secondary. These enzymopathies are acquired. They accompany most known diseases and essentially present as proteinopathies (pathologies of protein structures).

Mnemonic

To remember the indications for nucleases, associate the root "nucleo-" (nucleus, viral DNA/RNA) with their antiviral action in eye infections (keratitis, conjunctivitis).

Frequently asked questions

Which specific diseases belong to primary hereditary enzymopathies?

Primary hereditary enzymopathies include disorders classified by the type of metabolic defect:

  • Amino acid metabolism disorders — phenylketonuria, albinism, alkaptonuria, tyrosinosis.
  • Carbohydrate metabolism disorders — galactosemia, hereditary fructose intolerance, glycogen storage diseases.
  • Lipid metabolism disorders — lipidoses.
  • Nucleic acid base metabolism disorders — gout, Lesch-Nyhan syndrome.
  • Connective tissue metabolism disorders — mucopolysaccharidoses, chondrodystrophy.
  • Gastrointestinal enzyme defects — cystic fibrosis, celiac disease, lactase deficiency.

Other hereditary enzymopathies include acute intermittent porphyria and desmosis.

What enzyme preparations are used for replacement therapy in gastrointestinal diseases?

Gastrointestinal replacement therapy utilizes preparations that compensate for insufficient digestive secretions:

  • Pepsin — used for gastric therapy in gastritis with reduced secretory function.
  • Festal, enzistal, mezim forte, and pancreatin — used to compensate for exocrine pancreatic insufficiency.
  • Microspheric pancreatic enzymes — modern preparations used to relieve maldigestion syndrome in exocrine insufficiency.
What are the methods and approaches for treating primary enzymopathies?

Based on the source material, approaches to treating primary hereditary enzymopathies (diseases linked to enzyme gene defects) include:

  • Symptomatic treatment — alleviating symptoms.
  • Pathogenetic treatment — diet therapy for phenylketonuria, replacement therapy, toxin removal.
  • Enzyme replacement therapy — using enzymes when deficient in the body.
  • Etiological treatment — gene therapy, viewed as a promising future direction.
How do proteolytic enzymes work in surgery?

Trypsin and chymotrypsin are used topically. They catalyze protein breakdown in dead cells, thereby debriding purulent wounds and dissolving blood clots.

What drugs belong to fibrinolytics and why are they needed?

They include fibrinolysin, streptokinase, streptodekase, and urokinase. They are essential for breaking down blood clots (thrombolysis) in thrombosis and thromboembolism.

How and why is hyaluronidase used?

Hyaluronidase (Lidase) is administered intramuscularly or subcutaneously. It breaks down hyaluronic acid, promoting the resolution of post-burn scars and postoperative adhesions.

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