Gene therapy is a medically-based practice that uses normalized genetics to replace genes which are either not present or abnormal for some individuals. Doctors would take the specific gene sequences that need adjustment, and then insert them into the cellular information of the patient in various ways. Most forms of gene therapy are still in the clinical research stage, but there have been stories of encouraging results.
Several inherited immune deficiencies are being treated successfully right now with gene therapy. When the blood stem cells are removed from patients, retroviruses then deliver working copies of the defective genes to the body.
For the gene therapy options which have been approved for use, there are many success stories to consider. Sebastian Misztal is one such story. He was a patient in a hemophilia gene therapy trial in 2011. After receiving the therapy, Misztal no longer experiences episodes of spontaneous bleeding.
Roughly 70% of the currently active gene therapy clinical trials are based in the United States. Europe approved their first treatment in this area in 2012. These are the pros and cons of this scientific approach to consider.
List of the Pros of Gene Therapy
1. Gene therapy provides hope for those who may not have had any in the past.
About 3% of American children are born with a genetic condition which requires gene therapy as a way to treat the issue. At this time, the diseases and disorders which are present in this population will take the life of the child before there is an opportunity to correct the condition. Birth defects are the leading cause of newborn death in the United States, with as many as 1 in 5 children suffering from them. Advances in gene therapy could help to correct these issues instead of forcing parents into a heartbreaking scenario.
2. Gene therapy could change the perspectives that people have about disease.
Roughly 10% of all Americans are affected by a rare disease or condition on any given day. Approximately 33 million people are suffering from a disability that is directly attributed to their genetic profile. The promise of gene therapy is that it can reduce or eliminate the pain and discomfort that these abnormalities cause. 80% of the diseases that we know impact human health in negative ways have a genetic foundation. If we can replace the cells or chromosomes that are at-fault, then it becomes possible to offer relief.
3. Gene therapy could offer the potential of new discoveries.
Our world is a better place when there is an emphasis on diversity. When we have effective gene therapy treatments that can save lives or prolong them, then we are adding strength to our existence. There will be more opportunities to research, new ideas that could lead to critical discoveries, and relationships that can lead to future generations that experience these benefits as well. There will always be a segment of society that looks at gene therapy as a way to “play God.” The reality of this medical treatment is that it can help people continue to live a life that they love.
4. Gene therapy could be used in different ways to improve life.
Right now, the focus of gene therapy research is to provide solutions for people who are suffering from specific illnesses or diseases. When we begin to experience successes in this field, then the information we learn can apply to other treatment areas as well. Gene therapy could be useful in the treatment of infertility issues. The processes involved may help people struggling with vision or hearing issues. Even if the only thing that we can do with this science is to relieve chronic pain, that would be tremendously beneficial for the futures of many people.
5. Gene therapy does not just apply to human treatment options.
When we discuss the pros and cons of gene therapy, it is essential to remember that the benefits we can experience as humans apply to other forms of life as well. The technologies we create from this research could help us to grow crops that adapt more effectively to changing climate conditions. We could use this information to correct the various genetic conditions that we know about in the animal kingdom. This data could help us to grow healthier foods, increase the shelf life of harvests, or produce more items in our overall yields.
6. Gene therapy allows us to use technology to improve the quality of life for people.
Many of our medical discoveries rely on technological processes that we apply to natural items. Even some of the most critical advancements of our era, such as the development of a polio vaccine by Dr. Jonas Salk (and the work of many others) relied on the use of inactivated virus materials to create the first usable product. Gene therapy would become one of the first treatment options for doctors that was purely technological. That means our opportunity to develop new resources from it are virtually unlimited.
7. Gene therapy allows us to treat the “untreatable” diseases.
Gene therapy is potential miracle worker when we start to look at its full potential for humanity. It offers us the opportunity to eliminate, and then prevent hereditary diseases like hemophilia and cystic fibrosis. The technologies behind this treatment option could provide us with a possible cure for heart disease. Potential medical options include cancer and AIDS cures. Even if there is a fair amount of risk involved when treating these health issues, there are a lot of patients who don’t have much to lose. Gene therapy opens a door that we once thought was permanently locked.
List of the Cons of Gene Therapy
1. Gene therapy does not have a reliable delivery method.
Retrovirus delivery systems are the most common way for gene therapies to be delivered to patients. The problem with this option is that the enzyme used to encourage the transfer of genetic data can be eliminated by the immune system before it has the chance to work. There could be issues with cell division or replication that limit the effectiveness of the treatment.
When there is a noticeable change to the cell, the body might attack itself without the presence of an immunosuppressant. Until we can remove and replace genetic data with more reliability, the success stories for gene therapy will always be hit or miss.
2. Gene therapy is an expensive procedure.
There are several gene therapy options which are available right now, but they come at a steep price. If you use Luxturna to treat both eyes as a way to treat blindness, then the final cost could be more than $1 million. Even the “affordable” options in this field start at $200,000 per treatment. That’s why many patients weight for clinical trials to begin, and then apply for a spot in one to receive the help they need. Most healthcare insurance plans will not cover the cost of these procedures because of their uncertainty.
3. Gene therapy requires ongoing treatment s to be effective.
Many people have found that the benefits of their gene therapy treatments began to wear off as soon as they were no longer taking their medicine or visiting their doctor for treatments. It can be a lifelong course that someone must follow to reduce or eliminate the genetic issues that hold back their health. Unless you can keep taking the products which are often priced above $100,000 per treatment, then you will experience a reversal in your condition.
4. Gene therapy may not be able to adapt to a changing world.
It has taken less than a century for prescription-grade antibiotics to no longer be as effective for the treatment of bacterial infections as it once was. Antibiotic resistance can impact anyone at any age, and in any country. Sometimes it occurs naturally, but the most common reason for this issue is that antibiotic misuse has led to a growing number of infections, including pneumonia, tuberculosis, and salmonellosis being more challenging to treat because the medicine is not as effective against the bacteria.
This issue could occur with gene therapies too. We have already seen people begin to have their progress reverse itself when they stop following their treatment plan. Over the next couple of generations, the body could start resisting this option too.
5. Gene therapy might only delay the inevitable.
Jolee Mohr was lying in a Chicago hospital, her body swollen by internal bleeding and organ failure. The sight was so difficult that her husband decided not to bring their 5-year-old daughter into the room to say goodbye. Although there was no evidence to suggest a link, Jolee had taken an experimental treatment for rheumatoid arthritis. She was only 36 years old.
The National Institutes of Health approved the first human gene transfer study in 1989. Through 2006, there were 800 gene therapy studies that involved 5,000 patients. In those 17 years, the total number of approved therapies was zero. The only success story was a cure for the “bubble boy” disease that also caused leukemia thanks to the virus that delivered the treatment.
And Jolee wasn’t the only story. A teenager named Jesse Gelsinger also died because of treatments offered inn a clinical research study. We must remember that there are sad stories to tell in addition to the happy ones when evaluating this treatment option.
6. Gene therapy will shift society toward new polarization.
The United States is already highly polarized from a political perspective If gene therapies are approved for widespread use, then it may create another layer of separation from a medical perspective. Although most people can get behind the idea of creating a cure for cancer, birth defects, or chromosomal disorders, the processes used could also create designer genetics that promise a specific outcome. Should we pursue a scientific field that could help our children become smarter, faster, or better looking?
7. Gene therapy could change the way we think about competition.
Although the discussion of “designer babies” often involves looks, the science behind gene therapy could also encourage specific traits to develop in children. Parents with wealth could work with their doctors to support a healthier muscle mass, faster fat burning capabilities, or an adaptive body frame that allows for greater flexibility in sports. People could design an outcome where results could follow a curve where outcomes could be planned for years in advance. This process would result in another layer of socioeconomic separation that would likely lead to even more polarization.
The pros and cons of gene therapy still require a lot of soul searching, even though we are 30 years and counting into this field of research. We are beginning to see some successes, but it has also come at the expense of some high-profile failures. Only time will tell if we can put this information to good use for the betterment of humanity. Until then, we must continue searching to find more solutions to the significant health issues our race faces each day.
Natalie Regoli, Esq. is the author of this post and the editor-in-chief of our blog. She received her B.A. in Economics from the University of Washington and her Masters in Law from The University of Texas School of Law. In addition to being a seasoned writer, Natalie has almost two decades of experience as a lawyer and banker. She is a child of God, devoted wife, and mother of two boys. If you would like to reach out to contact Natalie, then go here to send her a message.