Miracle Cure or Ethical Quandry?

By Donovan Makus


In late November, while many of us were distracted by end of term work, a bombshell announcement appeared that created quite a stir. Located halfway around the world in Shenzhen, China, an academic researcher, Dr. He Jiankui, who is affiliated with the Southern University of Science and Technology, has claimed to have successfully edited the genomes of twin baby girls to confer AIDs resistance upon them. While Dr. Jiankui’s intention may have been to create a positive reaction, the actual result was quite different. Researchers and groups around the world condemned him, and his university is currently investigating his research. To understand why a good thing, the introduction of AIDs resistance, can lead to such a strong negative reaction, it’s useful to consider the importance of good research design and ethical guidelines.

In the fight against disease, few technologies show as much promise as gene editing. Some diseases are passed on through “faulty” genetic code; examples include Cystic Fibrosis, a disease which impairs breathing, and hemophilia, a disorder which leads to difficulty in clotting blood. If scientists could repair the changes from the typical genes that cause these diseases, then the end result would be vastly superior to a lifetime of doctor’s visits, medications, and temporary treatments. For those suffering from these diseases, a potential gene editing solution offers a panacea that would save them a lifetime of medical interventions. Many researchers are already examining the use of gene editing; thousands of trials are either underway or have already been completed. In fact, the use of gene editing to experiment with managing HIV/AIDs is not a new area. A search of a major scientific database using the search terms “CRISPR HIV,” CRISPR being a major method of editing genomes, reveals several hundred research articles. Evidently, the reason for the ruckus caused by Dr. Jiankui isn’t attempting to find a gene-editing solution for AIDs. No, the reason that he received a great deal of criticism comes down to his lack of research ethics oversight and the dubious nature of his experimental design.

To understand the issues with Dr. Jiankui’s work, it’s useful to examine it in detail. His project involved a group of 8 couples, all consisting of an HIV-positive male and an HIV-negative female. Five of these couples chose to go ahead with his research. Dr. Jiankui used eggs and sperm from each couple to create an embryo or two and then proceeded to edit their genomes, using the common CRISPR, to edit genes responsible for the CCR5 protein, in a way to make the embryos resistant to HIV. It is important to note, though, that this is not a complete fix, and HIV could still infect someone with an “immune” CCR5 protein. Additionally, his editing method did not lead to a complete swap of the genes; in fact, both edited embryos still retain some original CCR5 proteins. Currently, he is formally on a leave of absence to do research from his home institution. However, in the past, Dr. Jiankui received funding from his university as well as the Chinese government and a local hospital, as well as reportedly using some of his own funds. While he states that he is currently in the process of formally publishing his results, Dr. Jiankui’s decision to announce to the world the successful birth of twin girls with the edited gene, Nana and Luna, infuriated many of his fellow researchers and funding agencies who have strict ethical restrictions about human gene editing. Some of his colleagues went so far as to pen an editorial in Nature, the prestigious scientific journal, denouncing his work and stating he broke numerous ethical guidelines. It didn’t help matters when he announced that these were only the first two edited babies to be born; another pregnancy was also underway. In fact, while Dr. Jiankui’s informed consent statement to the couples in his study was highly technical and rather light on details about what he was doing, it was quite explicit in stating he would have the right to use images of any successfully modified children in promotional materials. It is exactly these ethical concerns as well as concerns over Dr. Jiankui’s motivation (given his slick PR work) as well as issues with the actual effectiveness of the treatment, that led to a negative reaction.

Concerns about the ethics of gene editing are well-founded. While gene editing of somatic cells, ones that are not involved in reproduction, continues at a rapid pace, the pace of editing of germ line cells, associated with eggs and sperm, and the editing of embryos, faces ethical challenges. For one, without proper guidelines, the technology could be used in socially disruptive ways. Inequality could be reinforced as the wealthy pay to have their children “edited” to remove negative traits, as well as add potentially positive traits related to physical appearance or other socially desirable traits. Naturally, this is only one view of this debate, with some claiming that there should be no children born with any diseases we could theoretically “edit out” and others suggesting that there should be no human developmental gene editing at all, as an embryo cannot offer informed consent. The whole notion of pre-birth consent also leads to highly contentious debates about when exactly “life” begins. These views, of course, assume gene editing works correctly in the first place. Given the complexity of the human genome and our incomplete understanding of our own biology, the potential exists to inadvertently affect other biological processes. In Dr. Jiankui’s case, the genes he edited code for a protein called CCR5, which, when modified to reduce the risk of HIV infection, also increases the risk of West Nile infection should the specific mosquitos appear. In light of these challenges, the path forward for gene-editing of germ line cells, which Dr. Jiankui did, seems more complex than for other body cells. Additionally, there are concerns that Dr. Jiankui did not fully educate his research subjects on the nature of his work until it was well advanced, and that offering IVF to couples as well as paying for some of his own research violated ethical norms about scientific research consent and funding. Academics are not supposed to become so deeply engrossed in their work, going so far as to put their own resources towards it, as it may lead to the tweaking of experimental results or poor research methods. Additionally, it’s nearly impossible to see if Dr. Jiankui’s work actually did any good, as testing it would require attempting to infect the twin girls with HIV, something that would lead to universal condemnation.

While the future for gene-editing is definitely bright, there remain issues associated with editing human germline cells, which go beyond the scientific field. The history of science is filled with pioneers who defied conventional thinking and made great discoveries. Names such as Curie, Watson, and Cricks come to mind, but science is also filled with cautionary tales of experimentation gone wrong, such as the shameful case of the Tuskegee Syphilis Experiment. Science can tell us what works and doesn’t work for gene editing, and improvements can be expected over time, but it cannot answer the philosophical questions surrounding the ethics of editing the very blueprints of life.

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