Since the early 1970s, scientists have had a theory of how to manipulate genetic structures, but they unfortunately did not have the proper equipment to do so. As technology evolved over the course of the next ten years, scientists were finally able to craft a procedure that would substantiate their theory. They coined this procedure as “gene therapy.” The essence of gene therapy is to install a “microscopic factory” into one’s body that supplies the body systems with missing enzymes or proteins. Like a vaccine, the “factories” are made from a mild form of the disease/virus and specifically engineered DNA. These “factories” can be inserted into the body in numerous ways such as inhalation, injection, or by altering previously removed cells from the patient and reestablishing those cells in the body (Kirsner). Although we now have the power of gene therapy in our hands, there are many concerns and controversies regarding the risks and the benefits, the weightage of past failures against successes, and whether or not there is a market and a promising future for this method of treatment.

A number of companies all over the world have instituted programs in support of the research for gene therapy. Genzyme is one of these companies. They are working on a gene therapy treatment targeting Parkinson’s disease, a disorder of the central nervous system. According to the Parkinson’s Disease Foundation, “between seven and ten million people worldwide” suffer from this disease. Genzyme has already been able to gather enough evidence to conclude that the effects of the treatment will last up to five years (Kirsner). A Dutch company called UniQure is making serious advances with their gene therapy treatments, specifically Glybera, as well. Glybera treats patients with “a rare metabolic disorder that causes inflammation of the pancreas.” The medication takes action by breaking fats down, in turn reducing inflammation of the pancreas (Kirsner). The development of these new treatments calls for some form of inspection, regulation, and distribution, which The Food and Drug Administration (FDA) is in charge of in the U.S. The FDA performs several tests and completes thorough examinations of each treatment prior to putting their stamp of approval on it (Lattime). 

One of the first successes with gene therapy was with a young boy in Germany. He was born with a life-threatening disease called “junctional epidermolysis bullosa.” His mutation of the disease made his skin “blister and tear,” making him prone to various types of bacterial infections. Doctors first experimented with antibiotics, bandages, special nutritional measures, and even a skin transplant from the boy’s father–none of which worked; the disease had progressed too far. After concluding that gene therapy was the only option, doctors first extracted a sample of the boy’s skin and sent it to Dr. DeLuca’s team of Italian doctors in Modena. They manipulated the genes from the patch of skin by inserting the normal form of the mutated gene. Using the engineered cells, they cultivated large skin grafts for the boy. The grafts were rushed back to Germany where they were transplanted onto his body (Grady). As he was recovering, doctors marveled over the results of the procedure. “It was spectacular… we removed the gauze and saw the epidermis underneath. We got the feeling that the body of the kid was recovering, was responding,” Dr. DeLuca beamed, ecstatic about the outcome of the procedure. Thorough analysis of the skin showed that the new genetic material had not spread to any unaffected areas. It also showed that the procedure needs “careful cell-culturing” for a higher success rate (Grady).

Gene therapy has also shown high success rates in patients who have a rare hereditary brain disease called adrenoleukodystrophy (ALD). The brains of ALD patients are very fragile and there is no way to determine how fast the patient will deteriorate to “the point of no return.” For this reason, gene therapy must be provided to the patient as soon as possible (Kolata). The first step is to extract bone marrow stem cells hosting the ALD gene. Upon extraction, doctors need to insert a well-functioning gene in the cells and then reintroduce them into the patient. They gradually move up into the brain where they are naturally converted into glial cells, cells that are necessary for the functions of the brain. The new glial cells take over, “stopping the brain deterioration that would otherwise occur” (Kolata). A study was conducted using seventeen ALD patients, all boys aged 4-13. They were treated with gene therapy and of those seventeen patients, fifteen were “functioning normally without obvious symptoms.” (Kolata) 

While many trials have had successful outcomes, not all have had the same results. Recently, eighteen-year-old Jesse Gelsinger lost his life due to complications in his experimental gene therapy treatment. Many years ago, nine children with an immunodeficiency disease were also treated with gene therapy. Of those nine children, four developed leukemia, a type of blood cancer (Kolata). The outcome was disappointing in a few other trials as well. “... dose escalation of an adenovirus has resulted in the death of a participant and another with an underlying genetic disorder” (Lattime). Similarly, “...after retroviral gene therapy for SCID, four participants developed a viral insertion-related leukemia” (Lattime). There are a few other concerns about gene therapy that doctors and scientists are working hard to eliminate. The vectors, or “factories,” may “alter genomic DNA in a manner that can manifest much later,” making the unpredictability of the risks a deterrent for those looking into gene therapy. (Lattime)

Some people believe that the practice of gene therapy will completely alter one’s character, creating an entirely different person. Opponents of gene therapy argue that each individual is created with a purpose, and to change that purpose would be considered morally unethical (Ferguson). In some cases of Down Syndrome, patients display a lack of fear towards strangers. Clare Ferguson, mother to Russell Ferguson, a young boy who has Down Syndrome, voices her stance on whether gene therapy is an acceptable method of treatment or not. She believes that “... removing all characteristics belonging to Down Syndrome but not belonging to Russell is impossible” as he is not just “a sum of his parts” but a whole individual. 

Despite the opposition, gene therapy is gaining popularity at a swift rate. A meeting of investors was held at the Royal Society in London during which Alan Smith, former officer of Genzyme, said, “... the consensus was that gene therapy… is now a promising place to deploy capital” (Kirsner). The gene therapy study conducted on the seventeen ALD patients had a massive impact on the economy. That small pilot study was successful enough “to inspire the funding of a company, Bluebird Bio, which sponsored the bigger study in hopes of marketing gene therapy for ALD” (Kolata). As of date, Bluebird Bio’s financial position is soaring. In December 2016, their stock on NASDAQ was trading at around $50. Exactly one year later, their stock is trading at $171 with a market capital of about $7.695 billion.  People are curious to know what these advances mean for the future of medicine and the economy.

As with everything, gene therapy has two sides to it too. There are opposers while there are also supporters. There have been successes while there have also been failures. There are risks while there are also benefits. Given the success to failure ratio, is it worth taking the risk or is it better to play it safe? Is there a promising future for this method of treatment or will it fade away with passing time? To opt or not to opt? That is the real question.

References:

-Ferguson, Clare. “A Mother Tells Why She Would Not Want Her Child Changed By Gene Therapy.” Genetic Engineering, edited by Sylvia Engdahl, Greenhaven Press, 2006.

Contemporary Issues Companion, Opposing Viewpoints in Context,

link.galegroup.com/apps/doc/EJ3010423214/OVIC?u=conc63834&xid=4d1575c.

Accessed 26 Nov. 2017. Originally published as “A Mother’s Reflections on Perfection,” Voices Across Boundaries, Winter.

-Grady, Denise. “Gene Therapy Creates New Skin to Save a Dying Syrian Boy.” 

New York Times, 9 Nov. 2017, p. A6(L). Opposing Viewpoints in Context, 

link.galegroup.com/apps/doc/A513934554/OVIC?u=conc63834&xid=6d8d0e4d. 

Accessed 16 Nov. 2017.

-Kirsner, Scott. “Gene Therapies Have The Potential to Improve Human Health.” Human

Genetics, edited by Louise I. Gerdes, Greenhaven Press, 2014. Opposing Viewpoints. Opposing Viewpoints in Context, link.galegroup.com/apps/doc/EJ3010916206/OVIC?u=conc63834&xid=01980f17. Accessed 15 Nov. 2017. Originally published as “Gene Therapy Shows New Signs of Promise,” Boston Globe, 2 June 2013.

-Kolata, Gina. "Study Shows Success of Gene Therapy in Treatment of a Rare Brain Disease." New York Times, 6 Oct. 2017, p. A22(L). Opposing Viewpoints in Context, link.galegroup.com/apps/doc/A508258793/OVIC?u=conc63834&xid=b347ad0d.

Accessed 21 Nov. 2017.

-Lattime, Edmund C., and Stanton L. Gerson. Gene Therapy of Cancer: Translational Approaches from Preclinical Studies to Clinical Implementation. Elsevier/AP, Academic Press Is an Imprint of Elsevier, 2014.