Enzyme therapy

Also known as: Enzyme replacement therapy

Anatomy or system affected: All

Definition: The use of enzymes as drugs to treat specific medical problems

Indications and Procedures

Enzymes are large, complex protein molecules that catalyze chemical reactions in living organisms. The phrase enzyme therapy sometimes refers to enzyme preparations given as dietary supplements, often as digestive aids. Such treatment is of questionable value because enzymes, like all proteins, are degraded in the stomach. Legitimate enzyme therapy is an innovative procedure based on emerging technology. Enzymes used to treat various medical conditions are delivered intravenously.

Enzyme therapy is used to dissolve clots in stroke and cardiac patients. Enzymes such as streptokinase, plasmin, and human tissue plasminogen activator (TPA) are able to dissolve clots when injected into the bloodstream.

Some types of adult leukemia can be treated by injection of the enzyme asparaginase, which destroys asparagine. Tumors in these patients require asparagine, and the asparaginase removes it from the blood, thus inhibiting the ability of these tumors to grow.

Enzyme therapy can also be used to treat certain inherited diseases. One of these is Fabry’s disease. Patients with this disease are deficient in an enzyme called alpha-galactosidase A. Without this enzyme, harmful levels of a substance called ceremide trehexoside accumulate in the heart, brain, and kidneys. If left untreated, patients usually die in their forties or fifties after a lifetime of pain. On the other hand, patients injected with alpha-galactosidase A once every two weeks lead nearly normal lives.

Gaucher’s disease is another severe inherited disease characterized by a deficiency in an enzyme that normally prevents the buildup of a chemical to injurious levels. It can be treated by injections of the enzyme glucocerebrosidase.

Uses and Complications

The use of enzymes to treat clotting problems and genetic diseases started in the early 1990s; thus, the long-term effects are unknown. Enzyme therapy does not cure genetic diseases, so the therapy must be lifelong.

Some people are encouraged to swallow enzyme preparations to aid digestion. For example, the enzyme papain is promoted as a digestive aid. Although papain is very effective at breaking down proteins in the laboratory or as a meat tenderizer in the kitchen, it does not function well in the stomach. Papain is most effective at a nearly neutral pH of 6.2, whereas the stomach is highly acidic with a pH of about 2.0. On the other hand, papain is active in the more neutral esophagus. Although normally little or no food is found in the esophagus, papain has the potential to damage the esophageal lining, especially in people with esophageal disorders.

Enzymes have seen a recent resurgence in the promising new ways they are being employed. They have been shown to be effective against a wide range of pathologies, including fibrosis conditions, cancers, and Alzheimer's Disease. Enzymes can have many advantages, particularly in that their use generally does not come with negative side effects. There are, nonetheless, several challenges that the employment of enzymes entails. These include a relatively short useful life, an inability to target their use, high cost, and, very importantly, a risk of hyper-immune response. This is when a person’s immune system overreacts to the presence of harmful intrusions such as viruses and bacteria.

Bibliography

Cichoke, Anthony J. The Complete Book of Enzyme Therapy. Garden City Park, N.Y.: Avery, 1999.

De la Fuente, Miguel, et al. "Enzyme Therapy: Current Challenges and Future Perspectives." International Journal of Molecular Sciences, 22 Sept. 2021, www.mdpi.com/1422-0067/22/17/9181. Accessed 12 Aug. 2023.

Devlin, Thomas M., ed. Textbook of Biochemistry: With Clinical Correlations. 7th ed. Hoboken, N.J.: Wiley-Liss, 2010.

Hennigan, Jennifer and Michael Lynch. "The Past, Present, and Future of Enzyme-Based Therapies." Drug Discovery Today, Jan. 2022, pubmed.ncbi.nlm.nih.gov/34537332. Accessed 12 Aug. 2023.

Padrosa, David, et al. "Back to the Future: Taking Enzymes to the Next Level of Sustainability." The Biochemist, 28 Jun. 2022, portlandpress.com/biochemist/article/44/3/19/231491/Back-to-the-future-taking-enzymes-to-the-next. Accessed 12 Aug. 2023.

Rimoin, David L., et al., eds. Emery and Rimoin’s Principles and Practice of Medical Genetics. 5th ed. Philadelphia: Churchill Livingstone/Elsevier, 2007.

Saptashwa, Datta. et al. "Enzyme Therapy: a Forerunner in Catalyzing a Healthy Society?." Expert Opinion on Biological Therapy, 27 Jun 2020, www.tandfonline.com/doi/abs/10.1080/14712598.2020.1787980?journalCode=iebt20. Accessed 12 Aug. 2023.

Scriver, Charles R., et al., eds. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York: McGraw-Hill, 2001.