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What Is Cystic Fibrosis?
A young child struggles to keep down the food her mother so carefully prepared, and the clean air just fills her lungs with sandpaper. She can't go outside to play like a normal child; her disease makes her especially susceptible to infections that only worsen her condition. She is seven years old and she knows that most kids with her disease will only live until ten years old. The year is 1962 and this young child has to carry the burden of knowing her parents will be devastated when she passes. Jump forward a few decades to 2002: an uncle in his twenties comes to visit his older sister and his young niece. He can feel his body weakening as the wear and tear of the years is only heightened by the fact that he struggles to breathe normally. He walks out to the patio where his family awaits him for brunch. As he makes his way over, he trips over a crack in the sidewalk. He falls, breaking his fragile hip. Within the next twenty four hours he is gone; his weakened body could not take the surgery to heal him. His poor family is devastated, now left with dozens of medical bills that have piled up over the years and the weight of having to plan a funeral. These conditions described are not some hypothetical tragedy for an overhyped young adult novel. These conditions describe the ruthless disease known as Cystic Fibrosis.
Cystic Fibrosis (CF) is a fatal disease that affects thousands of people all over the world. Cystic Fibrosis is a genetic disease; if genes spoke a language, what would trigger Cystic Fibrosis would be a misspelled word that changes the whole meaning of a sentence. Meaning, the coding of the genes get out of order (either with an addition of one or more of the tiny genetic building blocks, nitrogenous bases, or when one or more nitrogenous bases are missing in a sequence), and code for something they were not intended to code for. When the genetic mutation that creates Cystic Fibrosis does occur, it primarily affects the respiratory system and the digestive system. The mutation initiates the overproduction of abnormally thick mucus in the lungs, which then clogs major organ systems such as the lungs, pancreas, and intestines. Furthermore, the buildup of mucus makes it incredibly difficult to breath and, because it blocks the enzymes that aid the digestive system, makes it challenging to digest food as well ("Cystic Fibrosis - How Is Cystic Fibrosis Treated?"). Because this is a genetic disease, it makes it incredibly difficult to treat, let alone find a cure for. In recent times, it has been proposed that genetic editing and gene therapy may be the solution. However, it has been met with incredible opposition as bioethics laws have not outlined clearly the limitations that doctors and researchers should take when altering genes. Because of this there are many misconceptions about what it means to edit genes. Such misconceptions include the production of designer babies, or that it alters the divine plan that many people believe in. To benefit those afflicted with genetic diseases such as Cystic Fibrosis, it is thereby imperative that these misconceptions are dispelled in favor of providing patients with the best treatment to lengthen their life and give them the best quality of life possible. Furthermore, these treatments are still in the experimental stages, making them neither proven to work nor risk free. However, risks are normal for any kind of drug or treatment that is still being experimented with. Additionally, the progress has been clearly shown throughout history, and researchers are very close to finding the key that could fix the defective genes of Cystic Fibrosis patients.
One of the most common misconceptions about gene editing is the fear surrounding the production of what is colloquially known as "designer babies". Designer babies is a term used for when gene editing, usually in the embryonic stages of development, is used to change physical features of the fetus for social reasons rather than medical reasons (Sample). For instance, this includes targeting the gene that would allow the unborn child to grow taller so that they can play basketball in high school. In doing so, it would be difficult to limit what is ethical and what is not when editing genes for social reasons. This, however, differs incredibly from the use of gene editing for medical reasons. Editing genes for medical reasons would instead target the mutated genes that cause genetic diseases and change the order in which they occur to force the genes to function normally. For example, with Cystic Fibrosis, this would target the defective gene that codes for the overproduction of thickened mucus and alter the gene to code for a normal production of healthy mucus found in the bodies of healthy humans. Peter Glazer, Ph.D., M.D. describes this process in his interview with The National Heart, Lung, and Blood Institute, explaining that "The majority of people with cystic fibrosis will have the genetic mutation called Delta F508. If you put in the donor DNA that has the healthy sequence, it replaces the segment of the person's chromosome that has the mutant sequence, the one that causes the disease." This clearly does not describe anything that fits the designer baby stereotype; it purely alters the genes that control the disease and does nothing to change the physical appearance of the patient. Therefore, if this misconception were dispelled and backed by bioethical laws, it would make the general public more accepting of genetic editing as a form of treatment for Cystic Fibrosis. Furthermore, limiting gene editing to use for medical reasons only would completely disregard the possibilities for designer babies.
Genetic Editing would indeed improve the quality of life for those suffering from Cystic Fibrosis; however some hesitations arise with the fear that by editing genes, doctors, researchers, and scientists are interfering with a divine plan for these patients. This is understandable in the sense that faith is very important to many people around the world, and it drives them to behave and follow how and what they think will please whatever divine being they believe them. When the term "genetic editing" is brought up, it is understandable that this comes across like researchers are trying to "play God" and alter the code that makes human beings what they are. This concern is similarly related to designer babies in that people fear the controlling of genes for social purposes. In the scenario, it often runs deeper as people fear that by changing genes, scientists can change the person's identity that their divine being had created for them. To ease this concern, common awareness should be spread that gene editing does not change personalities or physical appearances, but instead can be targeted to alter the gene that controls just the disease. Sample describes this by using the genetic editing being tested on HIV patients, stating "Scientists have collected immune cells from patients' blood and used gene editing to cut out the DNA that the cells need in order to make these surface proteins. Without the proteins, the HIV virus can no longer gain entry to the cells." This is a procedure that can be done outside of the body, which is similar to the procedures many scientists are working on to treat Cystic Fibrosis. This treatment only targets the genes that control the disease by essentially inactivating them so that these genes are no longer harmful to the health of the patient. Therefore, those concerned about tampering with a divine plan may be eased that genetic editing does not mean altering physical features or personalities, but allows the patient to live out their longest and best life without being hindered or distracted by a life threatening disease.
While scientists have developed highly technical procedures for gene editing, and have even engineered medication that works as "gene therapy", gene editing is not perfected and still requires more, sometimes risky, testing. However, if doctors and scientists are able to perfect this process, the benefits would far outweigh the costs. This can be supported by all of the progress already made for the treatment of Cystic Fibrosis. In 1962, the median age for CF patients was only ten years old; today the median age is between forty and fifty ("Cystic Fibrosis Research Milestones"). This is solely credited to the careful experimentation and medical advancements made in the medical community. One of the biggest advancements was the use of the drugs Ivacaftor and Lumacaftor in combination. These drugs work in tandem to reduce the functionality of the mutated genes of CF and to aid the immune system in fighting bacterial infections that can worsen the symptoms of CF. Ivacaftor in specific targets the protein that binds the defective genes and hinders its ability to bind and control the overproduction of mucus, which greatly improves the function of the lungs and reduces the clogging of other vital organs. Lumacaftor then works, in short, as a long term antibiotic to assist the immune system in fighting off bacterial infections (Boseley). These drugs are known as gene therapy drugs; meaning they are nonpermanent forms of gene editing that only work when continually treated. The use of these drugs only proves that these defective and mutated genes can be identified and can be targeted. This supports that gene editing, a more permanent solution, can be achieved. Additionally, while highly effective, these drugs can be costly, at about $136,300 per year (Buckland). A more permanent solution such as gene editing will be no doubt expensive, but rather than paying more than Arizona State University's tuition cost yearly, this expensive treatment can be a one-time payment. If this treatment furthermore becomes normalized, perfected, and less risky, it could also then be covered or partially covered by health insurance to aid patients.
Gene editing has come so far it would be ludicrous if progress were halted. It makes logical sense to persevere through more trials to find a cure for these suffering patients than to cease experimentation because it is slightly controversial. Additionally, there are different methods that yield different levels of risk, which should be considered in the development of these methods. For instance, the more well known method known as Crispr has its risks, such as "when Crispr gets where it is needed, the edits can differ from cell to cell, for example mending two copies of a mutated gene in one cell, but only one copy in another. For some genetic diseases this may not matter, but it may if a single mutated gene causes the disorder" (Sample). Yet when using newer technologies, such as the PNA method that Glazer describes:
"One of the big advantages of PNA and donor DNA is that the off-target rate is much lower than CRISPR, the most well-known gene-editing tool. The other advantage is that they can be administered in vivo (in a living person or living animal), by simple intravenous injection of the nanoparticles. You don't have to take the cells out of the body, because the nanoparticles provide a very effective and safe delivery method. We have been using material in the nanoparticles that is already FDA-approved, therefore it is already recognized to be a safe material" ("Gene Editing for Cystic Fibrosis: A Q&A with Peter Glazer, Ph.D., M.D.").
The benefit of moving forward with these advancements in gene editing is that, based on the history of gene editing, there have already been improvements to when Cystic Fibrosis genetic research began in 1964 ("Cystic Fibrosis Research Milestones"). And while risks, casualties, and mistakes have been made, that is normal for big research projects. Nothing is guaranteed, but it is the responsibility of researchers and medical specialists to find the most effective way to provide a healthy lifestyle for patients, including those with Cystic Fibrosis. The same goes for cancer research; not every method has worked the first time. Genetic editing for Cystic Fibrosis will, no doubt, take trial and error. Yet, if it were to change and improve the lives of small children who struggle to breathe while playing outside with their friends, or even adults who have trouble enjoying a meal with their families, it would be worth it.
Cystic Fibrosis is a fatal disease that started by taking children from their parents, and has progressed to taking parents from their children. While this development shows that the life expectancy of those afflicted with Cystic Fibrosis has increased dramatically in the last century, the fact that people are still dying from this disease is motivation enough to continue researching and moving toward a cure. Cystic Fibrosis is indeed a genetic disease, which means that researchers, scientists, and medical professionals will have to dance the fine line of ethical and unethical. Genetic editing, while very new, seems to be the most effective treatment in the experimental stages. It has been shown through genetic therapy using Ivacaftor and Lumacaftor has been the most beneficial to patients on the market, but it is not a permanent treatment that genetic editing provides. This is controversial due to its stereotypes involving designer babies and altering the divine plan, but it is proven that genetic editing targeted at diseases does not affect physical or personality traits. Genetic editing does have known risks and is of course, still in its experimental stages, but the developments outweigh the costs exponentially. It is common for there to be risks in all medical experiments, including common drugs and procedures being tested for cancer treatment. Furthermore, there are genetic editing treatments for Cystic Fibrosis that are already supported by the Food and Drug Administration. These advancements have progressed too far to turn around now. It is now up to the Food and Drug Administration, ethics boards, scientists, and medical professionals to create awareness for genetic editing to treat Cystic Fibrosis. If the common misconceptions can be dispelled, and the experimental process to be more transparent to the public, it will allow for more support of this massive movement. If this is achieved, perhaps one day Cystic Fibrosis will be an archaic disease of the past and will no longer steal men, women, and children from their families, friends, and loved ones.
Works Cited
Boseley, Sarah. "Cystic Fibrosis Treatment Found to Improve Lives of Sufferers in Trials." The Guardian, Guardian News and Media
Buckland, Danny. "Recent Advantages in the Management of Cystic Fibrosis." Onlinelibrary.wiley
"Cystic Fibrosis - How Is Cystic Fibrosis Treated?" National Heart Lung and Blood Institute, U.S. Department of Health and Human Services
"Cystic Fibrosis: Discovery of a Key Molecule for Improving Treatments." ScienceDaily, ScienceDaily
"Cystic Fibrosis Research Milestones." CF Foundation
Edmondson, Claire, and Jane C. Davies. "Current and Future Treatment Options for Cystic Fibrosis Lung Disease: Latest Evidence and Clinical Implications." Advances in Pediatrics., U.S. National Library of Medicine
"Gene Editing for Cystic Fibrosis: A Q&A with Peter Glazer, Ph.D., M.D." National Heart Lung and Blood Institute, U.S. Department of Health and Human Services
Sample, Ian. "Gene Editing - and What It Really Means to Rewrite the Code of Life." The Guardian, Guardian News and Media