Genetics and Gene Therapy: A New Frontier
Public Domain image of gene therapy delivery
As I was researching this post, most of the blogosphere was abuzz about President Obama’s executive order allowing federal funding for stem cell research (see the IAmBiotech article here). It has been largely received by the community as an important first step towards treating and possibly curing many debilitating diseases that exist today. While I think it sounds very exciting and I understand what it means in terms of what kind of resources are now available to those using stem cell research, I’m not yet sure what specific diseases will be targeted using methodologies developed around stem cells. Perhaps I’ll work on a collaborative article with IAmBiotech in the future, or some of you can provide your own thoughts here in the comment field below!
Today, however, I wanted to focus a little on gene therapy. I was prompted to look at this after reading “Gene therapy gets closer to a ‘cure’” on the Boston.com site. At its very base, gene therapy attempts to cure disease by attacking defective genes in the human body that allow the attack in the first place, replacing them with healthy ones grown in a controlled environment. When research first began in earnest 20 years ago, it showed promise for a new generation of therapies that could help us live more healthy lives. As time has passed, however, that promise had grown stale, and with the high profile death of a teen patient, it seemed that the continued development of this area of study was unlikely.
Recently, however, several landmark treatments have scientists excited about the field again and using the word “cure” for instances where the word would have been unthinkable a few years ago. Earlier this year, researchers reported in the New England Journal of Medicine that gene therapy had been successfully used to cure children of “bubble boy disease,” an immunodeficiency which leaves its victims vulnerable to a severe reaction to or even death from the simplest of infections. And unlike in previous trials, all of the patients in this trial were still alive at the end of a two to eight year period.
Similarly, a form of hereditary blindness was treated successfully using gene therapy in a small number of patients, and their vision was at least partially restored. Separately, Corey Haas’ blindness was caused by a rare disease called Leber’s congenital amaurosis, and was also treated using gene therapy. Even though the therapy is presumably risky because it’s so new, Ethan Haas indicated that the risk was worth it because his son can now see things he couldn’t before.
Gene therapy wasn’t so promising at first. The accepted method of delivery for therapy is a modified version of a virus that can cause the body’s natural defenses to activate and attack the virus and that, if the virus delivers the treatment to the wrong place in the genome, can cause cancer. One patient, Jessie Gelsinger, died in a highly-publicized case in which his immune system responded severely and fatally to the introduced antigen. In another French trial, several children developed leukemia as a result of the treatment for “bubble boy disease.” As Xandra Breakefield, a professor of neurology at Massachusetts General Hospital recalled for the article, the failed trials were so discouraging she wouldn’t even admit she was doing gene therapy.
Since that time, however, new methods and knowledge have helped propel the research along slowly but surely, and scientists are cautious but newly optimistic about what promise treatment can provide its patients. From what I’ve read, many of the diseases that gene therapy would help cure cause such a reduced quality of life that patients may be willing to risk the ultimate sacrifice, their death, for the chance that the therapy would help them lead better lives.
We’re certainly treading on risky ground with gene therapy, especially considering that we may inadvertently create some disease that could be worse than any Mother Nature has unleashed during our time on this earth. However, the potential gene therapy offers may outweigh the risks in the case of curing disease.
I think where we may take therapy a step too far is when we use genetics to select traits that we like or dislike for our children, as a Los Angeles clinic is proposing to do in this Wall Street Journal article, “A Baby, Please. Blond, Freckles — Hold the Colic.” Granted, it is enticing to be able to select your child’s traits, but aside from the practical issues, like the expense, is it really moral to deny existence to a child that would have been selected naturally? One might argue that we get to select color and features of a car or a house, but those things are material, and are more about us than they are about the object. With a child, we’re again being selfish because it would show that we worry about what the child’s traits say about us rather than how they define that new person.
The new discoveries on the horizon of gene therapy and genetic engineering are both exciting and scary: exciting because they open new possibilities and knowledge for the human race, but scary because they bring a new set of responsibilities and moral issues with which we have to wrestle. What do you think? Leave your thoughts, and perhaps answer our polls below!
Which do you think are an appropriate use of our understanding of the human genome?
- Genetic therapy (100%, 1 Votes)
- Trait selection (0%, 0 Votes)
- Both (0%, 0 Votes)
- Neither (0%, 0 Votes)
Total Voters: 1
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