Innovative gene therapy shows promise in fighting disease

Friday, Sept. 22, 2017 9:07 PM

The history of modern medicine has been dominated by three great leaps forward in the understanding of human health and disease. These are sanitation, anesthesia and antibiotics.

While the roots of sanitation date back over 1,000 years, it was the work of Dr. John Snow in the mid 1800s, linking the spread of cholera to contaminated water, that truly advanced the understanding of a connection between sanitation and human health.

Similarly, the practice of anesthesia transformed the practice of surgery.

Today, we live in the antibiotic era of medicine, ushered in by the discovery of antimicrobial medications during the first half of the 20th century.

These three advances represent the great paradigms of modern medicine – each transforming medical practice in a new and fundamental way. For the past two to three decades, we have been on the verge of the next great paradigm shift in modern medicine: gene therapy. It now appears that the dawn of gene therapy is upon us.

Last month, the U.S. Food and Drug Administration approved a first-of-its-kind treatment that alters a patient’s own DNA to fight leukemia. The new treatment turns a person’s own immune cells into cancer-fighting cells and is used to treat an aggressive form of childhood leukemia that is resistant to more common types of cancer therapy.

Each cell in the body is made up of components determined by our DNA, which is a kind of road map to its structure and function. Genes are the basic units of DNA that encode this structure and function. Disease often results from defects in the DNA, either from a single malfunctioning gene or a combination.

According to the National Institutes of Health, gene therapy is an experimental technique that uses genes to treat or prevent disease. Researchers are testing several approaches to gene therapy, including,

Replacing a mutated gene that causes disease with a healthy copy of the gene.Inactivating, or “knocking out,” a mutated gene that is functioning improperly.Introducing a new gene into the body to help fight a disease.Gene therapy poses many challenges, which have made progress in this technique slow. Technically, it has been difficult to engineer delivery of genetic material into DNA in a way that permits function of the new gene. Also, practical and ethical concerns persist regarding the safety and appropriateness of altering the human genome.

Currently, gene therapy research is focused on the treatment of diseases for which other effective therapies do not exist. The FDA notes that more than 550 types of experimental gene therapy are being studied, mostly targeting cancer.

In a news release last month, FDA Commissioner Scott Gotlieb said, “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.

“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

Dr. Matthew A. Clark is a board-certified physician in internal medicine and pediatrics practicing at the Ute Mountain Ute Health Center in Towaoc.