November 7, 2011 | Posted By Ricki Lewis, PhD

Gene therapy is experiencing a renaissance, with many of the recent successes in children. For some conditions, the younger the child, the better the genetic correction, because affected tissues degenerate with time. This is the case for adrenoleukodystrophy (ALD), the “Lorenzo’s Oil” disease that strips the insulation from brain neurons. One goal of the not-for-profit Stop ALD is to team gene therapy with newborn screening, to help boys before they begin to lose abilities. 

Should gene therapy be attempted even earlier? Before birth?

Fetal gene therapy is already being done in non-human animals, presumably in preparation for phase 1 clinical trials. Gene therapy is technically more challenging than inserting a shunt to drain a hydrocephalic brain or repairing an open spine, because it entails delivering gene-carrying viruses to affected cells and not anywhere else. It is fetal medicine on a different scale. 

Carson Strong, a professor of human values and ethics at the University of Tennessee Health Science Center, argues in Human Gene Therapy that a phase 1 clinical trial on human fetuses is probably too risky, because this part of the FDA drug approval process tests safety, not efficacy. The fetus likely has nothing to gain. However, Strong describes two ways to address the problem by adjusting not the benefit part of the ethical equation, but the risk: use doomed fetuses. 

Why not experiment on fetuses so sick that they wouldn’t survive long after birth, if they even make it that far, or on fetuses scheduled for termination? The latter were indeed used to develop amniocentesis, chorionic villus sampling, and cord blood collection, technologies that have benefitted many. Using these fetuses without a future, Strong suggests, offers the possibility of examining their tissues to track the gene therapy. I envision the sliced eyeballs of some of the dogs in my gene therapy book, a necessity before people with Leber congenital amaurosis received the gift of sight.

I have a problem with gene therapy before birth. Yes, it would be wonderful to correct a genetic defect before birth, but I'm uncomfortable with how we get there. At the risk of sounding pro-life, I do not think that fetuses in utero – unable to live ex utero, yet metabolizing, transcribing genes, their neurons firing and muscles contracting – should be subject to such experimentation, even if doing so could ultimately save lives.

It isn’t the idea of “therapeutic misconception” – expecting phase 1 trials to debut miracle cures – that bothers me. Instead, I sense a more fundamental disconnect: the healing intent of gene therapy coupled with the difficult decision to end a pregnancy. 

The situation isn’t the same as studying development gone awry in extra embryos genetically destined for disease. That’s not an intervention, it’s observation, and it occurs during the first two weeks of the prenatal period, before the notochord has appeared and certainly before it has blossomed into a nervous system. Early embryos and fetuses are part of a continuum, yes, but in terms of what they can and cannot possibly feel, they are not the same.

To paraphrase the mathematician Ian Malcolm in Jurassic Park: just because we can doesn’t mean we should. Let’s focus gene therapy research on children who need it now.

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The Alden March Bioethics Institute offers graduate online masters in bioethics programs. For more information on the AMBI master of bioethics online program, please visit the AMBI site.

1 comments | Topics: Bioethics in the Media , Ethics and Morality , Genetics , Patient Autonomy , Pharmaceuticals , Reproductive Medicine , Stem Cell Research


Michael Roberts

Michael Roberts wrote on 03/04/13 4:57 AM

Thanks for the this really interesting article. We would be happy to publish your commentary on the <a href=""; title="Gene Therapy Review">Gene Therapy Review</a>. Please contact me if you are interested.

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BIOETHICS TODAY is the blog of the Alden March Bioethics Institute, presenting topical and timely commentary on issues, trends, and breaking news in the broad arena of bioethics. BIOETHICS TODAY presents interviews, opinion pieces, and ongoing articles on health care policy, end-of-life decision making, emerging issues in genetics and genomics, procreative liberty and reproductive health, ethics in clinical trials, medicine and the media, distributive justice and health care delivery in developing nations, and the intersection of environmental conservation and bioethics.