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The gene therapy revolution is coming. Will the US get left behind?

Gene therapies are coming — and if you sign up for human testing, in many cases, they’re here already. The question isn’t whether there’s potential in gene therapy, or even whether we’ll realize that potential, but how quickly the potential will be fulfilled, and who will benefit as a result. Last year, the United States federal budget included a rider banning certain types of gene therapy research, and that has some in the scientific community worried. Will it be American scientists who benefit from the coming explosion of gene therapy spending, exploiting the country’s lead in funding and size, or will other nations (perhaps less ethical ones) reap the rewards of this incredible technology?

There are basically two types of gene therapies in the works right now: somatic (body-cell) modifications applied to developed people (infant and up), and so-called embryonic modifications that will affect every cell throughout development. Only the latter can have effects that are totally ubiquitous throughout a patient’s body, or which are passed from generation to generation — and that’s the type of gene therapy that was banned earlier this year.

crispr headHere’s the relevant bit of the budget: “(Sec. 749) Prohibits the FDA from acknowledging applications for an exemption for investigational use of a drug or biological product in research in which a human embryo is intentionally created or modified to include a heritable genetic modification. Provides that any submission is deemed not to have been received, and the exemption may not go into effect.”

Right off, this issue evokes the similarly US-led bans on embryonic stem cell research — but this is an imperfect comparison. For one, embryonic stem cell research was mostly opposed through funding bans, but more importantly the arguments against it centered on moral and religious arguments, not practical or scientific ones. The idea wasn’t that it was too dangerous to use embryonic stem cells, but simply wrong to do so, while germ-line gene editing is being opposed largely on the basis that we cannot bear the risks of the practice — both to the individuals arising from these engineered germ cells and for the population into which these individuals are released.

The embryonic stem cell issue is largely dead at this point because it has been made irrelevant by technology. Scientists can now create highly pluripotent stem cells from adult cells like skin cells, and there is an ever-shrinking proportion of research that still requires the full pluripotency of real embryonic cells. Some in the anti-camp hold this up as proof that science can proceed in a way they see as ethical while still achieving what it wants — and honestly, fair enough. This is something we accept quite readily in other contexts; it’s wrong to cure a million people by using even one person as an unwilling subject, for instance, no matter how terrible the diseases. Right or wrong, that’s the level of moral certainty that exists on the stem cell issue.

Yet with gene editing, the issue is slightly different. There are definitely moral qualms about gene editing being wrong, about it “playing God” or something equally meaningless, but there’s also a more unique aspect of the argument: a belief that gene therapies simply won’t have the intended effect. The ban is an expression of the simple, mostly non-fact-based idea that research into human germ-line modification will be fraught with deformed babies and countless still-births. In the eyes of mostly conservative legislators, this is an unacceptable risk.

There are also worries about a flawed synthetic gene spending several generations silently working its way deep into the population, harming a great number of people. These ideas are not inherently irrational or stupid, really, but they are also totally speculative. We just don’t know if these are valid fears, but they are inherently fears that can be addressed, in a way that the questions of whether an embryo in a human being cannot. On the surface, that would seem to imply that we should allow at least some level of research to go forward and try to find out.

Even so, statements like those of bioethicist James Hughes in Motherboard earlier this week are more than a little flawed. In that interview, he claims that it is “morally untenable” to tell a parent that their child can’t receive these therapies — but that misses the point. As mentioned, the other side of the argument equally appeals to the idea that nobody should be sick if it’s preventable, especially those we make sick with our foolhardy scientific ambition. These two moral arguments cancel each other out in theory and have to be put head to head in practice, with actual scientific results. Neither side can do that at present.

More to the point, it is possible for a method of discovering therapies to be more immoral than voluntarily not developing those therapies. What if the Nazi use of prisoners of war really had produced a cure for hypothermia — would that imply that we should throw out our rules about involuntary human testing? The argument to be made for gene therapy can’t simply reply on its medical potential. It has to be proven to be capable of supporting truly ethical research or simply give up on the idea of convincing conservatives.

This is a new issue, but there is some recent precedent to look at. So-called “three-parent children” could soon become common in the UK, based on the replacing an egg cell’s diseased mitochondria (and the unique stretches of DNA those mitochondria contain) with those of a different, healthy egg. Right now, the data going into that project show there’s little reason to think that germ-line genetic manipulation is inherently, unacceptably dangerous.

Given the wording of the ban, it would be interesting to see a challenge that worked by editing the egg or sperm cells in an adult patient, probably through a viral vector. In other words, it’s one thing to ban “creating” a genetically modified embryo, but doesn’t a woman have the right to modify any cells in her body? And once that modification has gone on, does her natural, healthy reproductive process then become illegal? I’d guess, and hope, not. So will germ cells become illegal targets for modification in reproductively healthy adults? By my reading of the ban, such modifications would currently be allowed since they are not directly creating a modified embryo — nature does the creating as normal.

One thing that does have precedent, however, is the behavior of the biomedical industry. Embryonic stem cell research around the world did not disappear or even lag all that far behind during the US funding ban, despite having arguably a much lower profit potential than gene therapies overall. Can the United States really hold back this research? Americans will not sit back and endure diseases that have cures, simply because those cures involve mitochondrial replacement, or because some of the research that produced the cure came about through techniques that are illegal in the United States.

House1We also need to remember that gene therapies are going to be big, to the point that they could cure certain diseases that currently require lifetimes of medicines. If the some of the most profitable medicines in the world start to be made obsolete, and the therapies replacing them are illegal inside the United States, you can expect it to have a major effect on the economy. And as with most medical bans, both justified and otherwise, it will almost certainly lead to a black market that advantages the very wealthy, and provides sub-par or dangerous care to the poor.

In the end, these are purely practical arguments, met with the simple but iron-clad conservative reply: “So what? If it’s immoral, it’s immoral. Let other countries pursue unethically dangerous breakthroughs. We’re better than that.”

If the goal really is to open the minds that populate a bipartisan congress, that’s the point of view that scientists and activist legislators need to meet head on. Otherwise, they’ll have to wait for the science to start producing real, world-changing cures before they can get the American industry started — and by then it may be far too late to catch up.

Now read: What is gene therapy?

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