As we all know, gene therapy is a kind of treatment which involves modifying the genes in body cells in order to stop disease. Genes are the carriers of DNA, which is the code that is in charge of the body’s form and function – be it making one so tall or regulating body systems. Diseases happen when these genes don’t work according to plan.
That is where gene therapy comes in to the rescue. It’s a method that involves replacing faulty genes or the addition of it as a way to cure disease or to improve the ability of the body to fight disease. Gene therapy has shown some promise in treating a large list of diseases such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
Up to this day, researchers are still studying how and when to make use of gene therapy. Conceptualized in 1972, its own authors have cautioned those who wanted to commence human gene therapy studies. In 1990, the first gene therapy experiment was approved by the US Food and Drug Administration. As of January 2014, 2,000 clinical trials have been conducted or approved.
Gene therapy experienced some early failures leading people to dismiss the treatment. However, since 2006, notable successes have regained the attention of researchers but as of 2014, it is still largely an experimental technique. Included in the list of diseases studied for gene therapy include retinal disease Leber’s congenital amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic lymphocytic leukemia, acute lymphocytic leukemia, multiple myelma, haemophilia and Parkinson’s disease.
Gendicine, the first commercial gene therapy, was approved for the treatment of certain cancers in China in 2003. Glybera, a treatment for a rare inherited disorder, was the first treatment to be approved for clinical use in either the US or Europe after it was endorsed by the European Commission in 2012.
Gene Therapy In the News
Gene therapy has been making the rounds in headlines recently as well. In New Scientist, it was reported that gene therapy can cure blindness by healing eyes and the brain. It’s known that injecting genes into the eyes of those with Leber’s congenital amaurosis reverses hereditary blindness. But it also causes the weakening of neuron connections and it wasn’t known whether the brains of those who have been blind would rearrange to adjust to new visual information. Thanks to the study, brain scans after two years of treatment show that sight pathways are similar to those with no vision problems at all. Meaning, the adult brain is restructured.
In The Washington Post, it was reported that gene therapy for hereditary deafness was also moving close to reality. New research showed that mice with genetic hearing loss were cured when faulty DNA was improved.
A separate but similar clinical trial backed by Novartis is also under way. This study, though, focuses on helping people who lost their sense of hearing because of damage or disease.
Given the potential benefits that gene therapy offers, there are still those who are not in favor of the treatment. What exactly are their reasons for being against this kind of technique? And what causes others to want to hear more about gene therapy?
List of Pros of Gene Therapy
1. It provides a cure for genetic disorders
As mentioned earlier, two studies on genetic blindness and hearing have shown positive results. Who wouldn’t want their loved one to be able to see the world they live in? Who wouldn’t want a friend to hear the wonders of this world? This forms the main argument for those in support of gene therapy: why wouldn’t we want to give the gift of sight and sound to those who haven’t experienced them since birth?
Cures for genetic disorders aren’t exactly that available. People who suffer are treated based on the symptoms they are experiencing. Meaning, there is no real cure for these diseases. But gene therapy is showing promise to be the answer to these issues.
2. It offers large scale treatment
Hereditary diseases are passed on from generation to generation. So if there is a history of a certain disease within a family, there is a chance that would get passed on and on. What if it could be stopped? What if it gets treated now so future generations won’t have to worry about it? That is the promise of gene therapy.
With gene therapy, the cells carrying the genetic disorders are altered. So fixing them before they could be passed on could end the line of family members getting the same illness that debilitated their relatives.
List of Cons of Gene Therapy
1. There are ethical and religious issues
Ethics and religious stances will always be a part of most things related to science. Although we have successfully cloned some animals, there is still backlash over whether we really should be doing it at all. It’s like the statement posed by Dr Ian Malcolm in Jurassic Park on the cloning of dinosaurs: “…your scientists were so preoccupied with whether or not they could that they didn’t stop to think if they should.”
The same train of thought applies to those who are opposed to gene therapy. Should we really alter the human gene just so we can produce perfect specimens who are free from genetic disease?
Also, some have a couple of reservations about the actual success rate of this form of treatment. Religious supporters are on the side of “this was not intended by nature.”
2. Not all clinical trials are successful
Although there are approved treatments, gene therapy is still largely experimental. It’s not like smallpox which was declared eradicated thanks to the invention of vaccines – and several others have officially been declared eradicated too. Whereas the smallpox vaccine has been proving itself for quite the number of years, gene therapy is still relatively new.
Who knows? Maybe when there are more and more reports of success with gene therapy then maybe the public will begin to embrace it. Then again, although the benefits of vaccination have been widely discussed, there are still a lot of those who are opposed to it.
So, only time will tell whether people start rooting for gene therapy and the number of benefits it brings.
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. If you would like to reach out to contact Natalie, then go here to send her a message.