Gene therapy is an experimental technique in medical science that uses genes to either prevent or treat disease. The goal with this approach is to create a future world where doctors could treat specific disorders by inserting genes into a patient’s cells instead of using surgery, drugs, or other interventions to improve health.
There are several different approaches to gene therapy being researched today. One method involves the replacement of a mutated gene with a healthy copy of it. There is the possibility that a mutated gene could be inactivated so that its improper functionality stops. Doctors could even introduce new genetic material into a patient’s body to help them fight a disease.
The advantages and disadvantages of gene therapy show that this promising treatment option is potentially useful for some inherited disorders, cancers, and viral infections. It is also a technique that comes with a more significant set of risks than conventional approaches. Researchers are still studying the approach to determine if it is safe and effective for regular use by targeting diseases that have no other cures.
List of the Advantages of Gene Therapy
1. It offers the possibility of a positive medical outcome.
About 3% of the births in the United States involve a condition which is potentially treatable by using gene therapy techniques. Many of the children born in this demographic die soon after birth because of the devastating effects of their condition. Birth defects are also potentially preventable with this option, which impacts about 20% of families each year. Instead of paying for hospice care or being forced to say goodbye immediately, doctors and scientists are giving parents more hope for a brighter future because of the availability of this technology.
2. We can treat disease or illness in meaningful ways.
About 1 in 10 people in the United States have a condition or disease classified as being rare. That means there are over 33 million Americans who could benefit from gene therapy. These interventions could reduce or eliminate the pain and suffering that occurs when a genetic abnormality is present in a person’s body Almost 4 out of every 5 diseases that adversely impact human health have a genetic foundation to them as well. When doctors can replace or deactivate faulty cells that led to detrimental conditions, then the individuals and their families can find the relief they require to live a more fulfilling life.
3. It could improve a person’s life in other ways.
The focus of gene therapy research is to create new medicines and treatment approaches that can alleviate suffering. Whether it is a curative solution or not is less important than the positive changes that occur for people who need this help. When we discover beneficial processes through this work, then we might be able to apply it to farming, environmental causes, pet health, and food transportation to ensure that we all have access to the best possible life. Simple changes could create profound results in almost every industry.
4. We already know that it can be a successful treatment option.
There is always a measure of unknowns that patients experience in the medical world. Individual bodies react differently to the same treatment option. What we know about gene therapy is that the clinical trials of this intervention have shown some success when treating some specific diseases. If you are dealing with hemophilia, leukemia, blindness that’s caused by retinitis pigmentosis, or a severe combined immune deficiency issue, then you might be a viable candidate for this therapy option.
5. There are out-of-body treatment options available.
If your doctor recommends out-of-body gene therapy, then researchers or technicians will take bone marrow or blood to begin separating the immature cells away from the sample. Then they’ll add the necessary gene to them before injecting the cells back into the bloodstream. Once returned to the body, the immature cells go to the bone marrow, start maturing, and eventually multiplying to replace all of the defective cells. This technique looks to be especially helpful for people who suffer from sickle-cell disease or something similar.
6. Gene therapy could be useful in the treatment of multiple diseases.
Although there are not any gene therapy products that are currently available in the United States healthcare market, there are clinical studies that suggest this approach could be useful in the treatment of a variety of conditions. Recent studies suggest that a rare form of blindness called Leber congenital amaurosis could be treated with it, and there is also data to suggest that Parkinson’s disease responds well to the therapy.
As we get to know more about the overall capabilities of what gene therapy can do, there could be hundreds of different diseases that are treatable with this approach.
7. The medical impact of gene therapy can create permanent results.
Once the faulty genetics are replaced by the correct genes, this therapeutic approach earns the advantage of being a long-lasting, sometimes permanent result. Although this advantage doesn’t apply in every situation, many patients can reverse their bothersome symptoms in a short amount of time in either the in-the-body or out-of-the-body approach. There is even the possibility that some of the changes could be heritable to the next generation, which would reduce their risk of suffering from a similar future.
8. New treatment options are coming out all of the time.
In May 2019, the Food and Drug Administration approved the gene therapy medication called Zolgensma. This product is approved to treat spinal muscular atrophy in children who are under the age of 2. Several additional products, including Luxturna, Novartis, and Yescarta, have also come to the market since 2017. These items create new options for treating lymphoma, leukemia, and some genetic-related vision loss issues.
9. It can work in combination with cell therapy techniques.
Gene therapy involves the transfer of genetic materials. Cell therapy is the process of transferring cells with relevant function into the patient when that genetic material is missing in the first place. Some protocols utilize both, which can give patients a powerful way to recover from their medical condition. Stem cells can be isolated from a patient, and then genetically modified in tissue culture to start the expression of a new gene. Once the treatment expands to suitable numbers, then the patient receives the therapy as part of their overall care.
List of the Disadvantages of Gene Therapy
1. There could be unwanted immune system reactions.
The body’s immune system might see the various viruses that we use to replace unwanted genes as invaders that must be extinguished before they cause harm. When the white blood cells attack the newly introduced genetic material, it is not unusual for a patient to experience health issues like inflammation, dizziness, and headaches. In severe reactions, it is even possible for the immune response to target the body’s organs and cause them to fail.
That’s why treatment options include an immunosuppressant, but this medication can make someone more susceptible to infections and illness.
2. Current gene therapy methods can sometimes target the wrong cells.
Viruses have the capability of affecting more than one type of cell in the human body during a gene therapy treatment. It is entirely possible that the altered viruses that deliver the information to the cells could infect additional ones – not just the ones that contain the mutated or missing genes. When this disadvantage occurs, then the healthy cells could receive damage that may have unpredictable results. There is the possibility that the outcome could involve disease development, illness sensitivity, or even cancer.
Out-of-body gene therapy for severe combined immunodeficiency, which is the boy-in-a-bubble syndrome, have already experienced this disadvantage. Five of the 30 children who receive gene therapy for their condition went on to develop leukemia. One of them was unable to beat the resulting disease.
3. The delivery viruses might recover their ability to create disease.
There are specific viruses used to create results during gene therapies. The carrier, which is referred to as a vector, receives genetic engineering so that it can perform its job. They can be given intravenously or injected into the specific body tissues that require the extra help. It is possible for the initial infection to develop once it is introduced in the body, which could create problems for the information transfer. Although scientists wouldn’t use life-threatening viruses for this process, there is a risk of becoming sick from other issues other than the genetic concerns that doctors are trying to treat.
Researchers currently use adeno-associated viruses to deliver genetic information. Although it is not known to cause any disease in humans, it is related to those that can cause colds.
4. Gene therapies could cause a potential tumor.
If the new genetic information from a virus gets inserted in the wrong spot in your body, then there is a risk that the treatment process could lead to tumor formation. There is a risk of malignancy with this disadvantage as well. This disadvantage is present even when vectors aren’t being used to deliver information to the cells. Scientists can use stem cells for gene therapy, or the fatty particles called liposomes.
5. There is no guarantee that the gene therapy will work.
Most gene therapy options are still in the developmental or experimental stage. A number of the clinical studies that are available were based on animal research instead of human subjects. There have already been numerous failures of promising medicines because the results that animals experienced didn’t transfer to humans during the clinical portion of the approval process.
When gene therapy occurs, anything can happen. It is entirely possible that the treatment could fail. Some patients might see a limitation in their abilities to conduct the activities of daily living. It could even make their health worse.
6. Some patients might experience incompatibility issues.
There are a lot of similarities in our DNA that make genetic therapy a possibility. Each person also has about 300,000 unique points in their cellular profile that make then an entirely unique individual. Even when a gene therapy treatment session provides positive results for others, there is no guarantee that each person receiving this option will get to experience a similar outcome. Doctors could inject a virus with genetic material and there might be nothing that happens as a result.
7. Humans could develop a resistance to the gene therapy.
The benefits of gene therapy might be short-lived. We have already seen how antibiotic resistance changes our approach to medicine. This treatment option might be effective right now because it is so new. As more people discover its benefits and receive the IV or shots that deliver updated genes, the human body could develop resistance to the information transfer. Some patients might not experience a positive outcome even if their immune system does not attack the new genes. This disadvantage means that we might be able to treat today’s disorders effectively, but the next generation might be unable to experience the same benefit.
8. The cost of gene therapy is prohibitive to a number of families.
The fees for gene therapy can easily exceed $1 million in the United States. Even though competition can limit these costs over time, the impact of this expense means that some individuals may not consider this treatment option even though it is the best solution for their needs. If the price does not decline, then it will create a new layer of economic segregation that pits the wealthy against everyone else. Patent laws in the U.S. reduce the immediate availability of generics as well, which means it is up to each person to manage their costs if this option is necessary.
9. Ethical concerns about gene therapy exist.
Most people would say that the introduction of gene therapy provides several benefits that are worth taking under consideration. There are also some who have ethical concerns about using this technology to fix illness or disease. By changing the nature of a person’s genetic profile, even if it is for the better, then it works to eliminate the natural variations that occur within the human race.
Is it right for people to “play God?” Could this work begin to reduce the available gene pool to the point where genetic bottlenecks occur? We may not have answers to some of these questions until we learn more about the long-term impacts of gene therapy.
10. It typically works best in single-gene mutations.
There are some diseases that have a hereditary predisposition to them. Alzheimer’s, Parkinson’s, and some cancers are examples of this trait. Their cause might be due to several mutations or variations that occur on several different genes. Some people might be influenced by environmental causes as well. At this point in time, the treatment options for these complex situations is not as refined as it is for those who have a single gene defect. Some diseases, such as cystic fibrosis, might impact different areas of the body simultaneously as well. That would mean it would become necessary to deliver the treatment into all tissue types to begin the work of recovering.
11. There are funding challenges to consider in this category.
Moving gene therapy interventions into the clinical trial stage is the most daunting fiscal challenge these new approaches face. The additional funding needed to produce clinical-grade reagents, formal toxicology studies, document preparation, and additional manufacturing needs can limit the impact of this approach before it gets the chance to get started. Many of the most promising therapies are slowed by a lack of funding right now, which means the people who could be benefiting from this medical approach are forced to wait.
Verdict of the Advantages and Disadvantages of Gene Therapy
Before companies can market gene therapy products for use in humans, the item in question must go through testing to determine its safety and effectiveness. The Food and Drug Administration is the area of government responsible for this oversight. Then the results from each study are carefully examined to determine if the risks of the therapy are acceptable when compared to the potential rewards.
The advantages and disadvantages of gene therapy hold the promise to transform medicine as we know it. This option could create more choices for patients who currently live with challenging, or even incurable diseases. With scientists making numerous discoveries each year in this field, there may be an increase in the number of groundbreaking treatments that become available in the next few years.
If you think that your health and wellness could benefit from gene therapy, then speak to your doctor about the key points in this guide to determine if your treatment plan would improve with this option.
Keith Miller has over 25 years experience as a CEO and serial entrepreneur. As an entreprenuer, he has founded several multi-million dollar companies. As a writer, Keith's work has been mentioned in CIO Magazine, Workable, BizTech, and The Charlotte Observer. If you have any questions about the content of this blog post, then please send our content editing team a message here.