Advances in Gene Therapy

Gene therapy is bringing renewed hope to patients with diseases like cancer, neurodegenerative conditions, and other ailments. It’s advancing quite nicely since the passage of the 21st Century Cures Act with both the Food and Drug Administration (FDA) and National Institutes of Health (NIH) making significant progress in the science of gene therapy. 

So how is gene therapy evolving the world of healthcare? Let’s explore the science.

What is Gene Therapy?

Gene therapy is the introduction, removal, or change in someone’s genetic code with the objective of treating or curing a disease. It’s still considered experimental. Researchers are testing multiple ways to execute gene therapy, including:

• Replacing a mutated gene that causes a disease with a healthy one
• Inactivating a mutated gene that isn’t functioning properly
• Introducing a new gene into the body to help fight an ailment
• The majority of gene therapy work relates to diseases that have no current cure.

The Gene Therapy Process

So, how exactly does gene therapy work? The gene therapy process focuses on introducing new genetic material into cells to compensate for abnormal genes or to cultivate a beneficial protein. Mutated genes can cause a necessary protein not to be available, so the gene therapy process is used to introduce a normal copy so that the function of the protein can be restored. 

The process of injecting the gene into the cell is done so through the use of vector, which is a carrier. It’s similar to how a virus infiltrates a cell, except in this process, the new element isn’t going to make someone sick.
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The vector is either injected or given through an IV to a specific tissue in the body. This approach is still being refined and can be challenging. Scientists are attempting to find better ways to deliver genes and better target the cells.

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Types of Gene Therapy in Development

​There are many different trials happening right now in gene therapy, many of which are showing progress. Here are some examples:

Sickle Cell Trial

​A stem cell gene therapy trial has been underway for those that suffer from sickle cell disease. Sickle cell is an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” in situations of low oxygen. It was even featured on 60 Minutes with the NIH director Francis Collins, MD, Ph.D., stating, “This looks like a cure.”

Muscular Dystrophy

​Gene therapy company and biotech firm Exonics Therapeutics is currently using gene therapy to treat a canine version of muscular dystrophy. This disease can be devastating to children and is caused by a mutation of the dystrophin protein gene on the X chromosome, making it another disease that is attractive for gene therapy. Thus far, their work has shown great promise in the ability of the dogs to move more freely and without restriction and pain.

Cancer

​Two gene therapy treatments were approved by the FDA in 2017--Kymriah and Yescarta. These treatments use a patient’s own immune cells to fight rare types of cancer. These are considered to be “living drugs” made by extracting T cells from patients then genetically engineering them to destroy cancer cells. Kymriah works on bone marrow cancers, and Yescarta treats a type of lymphoma. Both treatments have allowed patients to enjoy complete remission.

Hemophilia

​Gene therapy company BioMarin has created a gene therapy for replacing the faulty gene that causes hemophilia, a disorder where the blood doesn’t clot properly. The company has published clinical trials that indicate that with the gene therapy, they had much improvement in clotting. Other patients with hemophilia B, have shown complete recovery after only one treatment.

Vision Restoration

​Spark Therapeutics introduced a gene therapy treatment, Luxturna, which was designed to correct retinal mutations and restore sight. The company uses adeno-associated viral (AAV) vectors to target cells. This revolutionary treatment has already enabled many patients to get their vision back and works primarily for those with inherited retinal diseases.

Gene Therapy: A New Medical Frontier

Though much of the science is still in its infancy and there are substantial roadblocks to future development, the potential of gene therapy treatments is potentially infinite, and will only grow more viable. New gene therapy companies and treatments are emerging every day. Some would say it’s the future of medicine. In the next year, experts believe there will be new therapies for spinal muscular atrophy, hemophilia A, and AADC (Aromatic L-amino acid decarboxylase) deficiencies. 

The above examples show that a unique and novel sector of the healthcare market is open, and it’s one that needs services and solutions, which is where you can see opportunity. It’s an excellent idea to keep abreast of the advances in gene therapy for anyone in the healthcare industry. And you can do that by subscribing to our blog below, where we cover healthcare trends, challenges, and more.

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About the Author

Carevoyance contributor Beth Osborne is a professional writer and content marketer with multiple years of experience in healthcare IT marketing. Learn more about her by visiting her website.