Savannah Hamilton
Professor Philip Randall
STS 1010
19 November 2018
Scientific Advancement: Gene Therapy 2.0
According to James Wolfe in Genetic Testing and Gene Therapy, “genetic therapy, also called gene transfer therapy, is the introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease” (96). Gene therapy is the altering of the genes in a body’s cells to treat or stop a disease (Mayo Clinic). This is not a new scientific technology, it has been experimented with for many years, and has helped scientists solve fundamental issues that were hindering cures for hereditary diseases. “Gene therapy 2.0” is a new advancement, scientists are now trying to use it to cure diseases other than ones that are hereditary; such as, cancer and heart disease. Gene therapy is a scientific advancement that will help improve the health of people; we will see this in its history, how it has changed lives so far, and how it will impact society in the future.
Background
It is necessary to examine the history of gene therapy to understand how it has reached a new period of advancement. Gene therapy is not a new concept, scientists began discussing its possibility in the early 1960s. “By the late 1960s early 1970s, gene therapy became the subject of an increasing number of articles and meetings” (Wolff and Lederberg 471). Gene therapy was not made possible at this time, but it was becoming a more public and researched idea. Although it was made public, and scientists and others in the medical field knew it was possible and could benefit medical applications, it still received criticism. Due to the criticism “it suddenly became clear that human gene therapy would be more complex technically and ethically than had been envisioned” (Friedmann 95).
It was in the late 1990s that gene therapy began to rapidly develop. At this time, numerous successful treatments in small and large animal models of human diseases were reported (Herzog 2). On September 4, 1990, a four-year girl became the first human gene therapy patient. “She had adenosine deaminase deficiency, a genetic disease which leaves her defenseless against infections” (“Gene Therapy- A Revolution in Progress”). She had treatments for a period of two years, and the treatment was successful; she now leads a normal life. This success led researchers to use the same technique in newborn babies with adenosine deaminase deficiency. After a baby had a single therapy, there was a steady increase in their immune cells. This did not lead to gene therapy being perfected, although it has found a cure or a way to manage some illnesses; such as, several inherited immune deficiencies and other illnesses that will be further discussed. The development of gene therapy has been a long and complex process, that has many more obstacles, but it has already made a difference in the lives of people. With the many new advancements and new trials, gene therapy has many scientific obstacles before it can become a practical form of therapy (“Gene Therapy- A Revolution in Progress”).
How Gene Therapy Has Changed Lives So Far
Gene therapy has been researched and has had many clinical trials to perfect the technique since the 1990s, however very few patients have received effective gene therapy treatments (“Gene Therapy Successes”). Even though this has been the case, doctors and researchers still believe gene therapy is a promising future medical application. It has not been perfected, but it has had successes that have affected the lives of people. One of the successes of gene therapy is that it has effectively treated several inherited immune deficiency disorders. As discussed in the previous paragraph, the first gene therapy treatment was given to a child with an immune deficiency known as adenosine deaminase deficiency, and her treatments were successful. This led doctors to start using this therapy on other people with this deficiency. The therapy for adenosine deaminase deficiency is done with multiple trials where the patient’s blood stem cells are removed, treated with a vector that delivers a functional adenosine deaminase gene, and is then returned into the patient. “For the majority of patients in these trials, immune function improved to the point that they no longer needed injections of adenosine deaminase enzyme”, and none of the patients that have gone through gene therapy for this disease have developed leukemia.
Gene therapy has also helped the lives of people with another immune deficiency called severe combined immune deficiency, or SCID. Severe combined immune deficiency was also one of the first genetic disorders that had success with treatment using gene therapy; further proving that this approach was possible. However, the first clinical trials for SCID had obstacles that scientists needed to overcome. The major obstacle was that the viral vector used to fix the mutated genes caused leukemia in some patients. Because of this, researchers now have begun trials with new vectors that are safer and less likely to cause cancer. Although the gene therapy for SCID was not one hundred percent successful, it still helped patients treat their disease, proving that gene therapy needs to be further researched, so in the future it can allow the people suffering from this disease lead a normal life. “Gene therapy has made important medical advances in less than two decades. Within this short time span, it has moved from the conceptual stage to technology development and laboratory research to clinical translational trials for a variety of deadly diseases” (“Gene Therapy for Diseases”). Gene therapy, so far, has done great things in the lives of people, and with its new advancements, it will do even more in the future.
How Gene Therapy Will Impact Society in the Future
Gene therapy hit a stopping point in the early 2000s, it was not until 2017 that it began having major breakthroughs, therefore it is considered a new emerging scientific advancement. It is using similar techniques to the past gene therapy, but now scientists are researching ways to use it on more complex diseases and disorders. Gene therapy has had a bumpy past, and continues to experience obstacles, but once it is perfected it will have an important impact on society. One way gene therapy could impact society is by finding a cure for types of cancer. “Cancer is one of the leading causes of death in the world with the prevalence of more than ten million mortalities annually” (Zaimy et al. 233). Currently, the treatments for cancer are surgical intervention, radiation, and chemotherapy; these treatments are not guaranteed to cure the patients suffering from cancer, and some of the treatments often kill off healthy cells which can result in toxicity (Zaimy et al. 233). This is a reason why researchers are looking to gene therapy to finally find a way to cure it. There are promising gene therapy treatments for cancer in progress now, and some have made it to the process of clinical trials. “Clinical trials are research studies performed in people that are aimed at evaluating a medical, surgical, or behavioral intervention” (“What are Clinical Trials”). One clinical trial for cancer gene therapy was tested on patients with leukemia. They used a gene therapy technique where the patient’s immune cells are removed, treated with a virus that has been genetically altered to recognize the proteins that are in the surface of cancer cells, then the immune cells are returned to the patient. Fifty-nine patients were used for this clinical trial, and twenty-six of them experienced complete remission. This is just one example and one method of gene therapy that has shown promising results that could, in the future, lead to a cure for cancer.
Another way gene therapy can benefit our society is by finding a cure for sickle cell anemia. “Sickle cell disease is among the most prevalent inherited monogenic disorders. Approximately 90,000 people in the United States have sickle cell disease, and worldwide 275,000 infants are born with the disease annually” (Ribeil et al. 848-855). People with this disease have a life expectancy of about 42-47 years old, and during their lives they experience many challenges; including, sever pain episodes, possible stroke, and organ damage. The first human clinical trial for sickle cell anemia was done on a thirteen-year-old boy who had had the disease his entire life. Doctors transplanted stem cells with a sickle cell disease fighting gene into his body and observed the side effects for approximately fifteen months. After the trial, “…he had no sickle cell disease related clinical events or hospitalizations…” (Ribeil et al. 848-855). He was then taken off all his medications that were involved with his disease, and reported full participation in academic and physical activities. He was cured of sickle cell anemia. This success will help guide researchers design methods for future clinical trials for sickle cell anemia genetic therapy. The curing of that boy is further proof that gene therapy is the future of medicine in society.
Conclusion
In conclusion, gene therapy needs to be further researched and developed. This could help find a cure for many life-threatening diseases that take away loved ones too soon. Cancer and sickle cell anemia are only two examples of diseases that show progress with gene therapy; others include, Parkinson’s disease, hereditary blindness, and hemophilia. Imagine a world where getting diagnosed with these diseases was not a death sentence or life altering. Gene therapy would involve s long process of treatments, but you would be guaranteed to live. Our society should encourage further development of gene therapy for all the people diagnosed with incurable diseases annually.
Works Cited
“Gene Therapy- A Revolution in Progress: Human Genetics and Medical Research.” National Institute of Health, U.S. Department of Health and Human Services, history.nih.gov/exhibits/genetics/sect4.htm.
“Gene Therapy for Diseases.” Gene Therapy for Diseases| ASGCT- American Society of Gene and Cell Therapy, American Society of Gene and Cell Therapy, 2017, annualmeeting.asgct.org/about-gene-therapy/diseases.php.
“Gene Therapy Successes.” Nutrition and the Epigenome, learn.genetics.utah.edu/content/genetherapy/success/.
Herzog, Roland W et al. “Two Decades of Clinical Gene Therapy— Success is Finally Mounting.” Discovery Medicine, vol. 9, 45 (2010): 105-111.
Mayo Clinic. “Gene Therapy.” Mayo Clinic, Mayo Foundation for Medical Education and Research, 29 Dec. 2017, www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619.
Ribeil, Jean-Antoine, et al. “Gene Therapy in a Patient with Sickle Cell Disease.” New England Journal of Medicine, vol. 376, no. 9, 2017, pp. 848-855.
“What are Clinical Trials and Studies?” National Institute on Aging, U.S. Department of Health and Human Services, www.nia.gov/health/what-are-clinical-trials-and-studies.
Wolfe, James. Genetic Testing and Gene Therapy. Edited by Christine Poolos. Britannica Educational Publishing, 2015, http://ebookcentral.proquest.com/lib/clemson/detail.action?docID=2101657.
Wolff, Jon A., and Joshua Lederberg. “An Early History of Gene Transfer and Therapy.” Human Gene Therapy, vol. 5, no. 4, 1994, pp. 469-480.
Zaimy, M. A., et al. “New Methods in the Diagnostics of Cancer and Gene Therapy of Cancer Based on Nanoparticles.” Cancer Gene Therapy, vol. 24, no. 6, 2017, pp 233-243.