In Depth Report from April 29th Paris Human Gene Edit Meeting

Editor’s note. Caroline Simons attended both the April 28th (see her report on that here) and 29th Paris meetings on human gene editing/genetic modification. Today, we have her in depth report on the April 29th meeting. I have posted her piece in full with only minor edits. If you are in a rush you can skip to the last page for Caroline’s top 10 takeaways from the meeting.

By Caroline Simons

The meeting of NAS and NAM which took place in Paris on 29 April was webcast live. A recording of the presentations will be posted in the coming weeks. This blog post is my record of the presentations and an attempt to give an overall impression of the discussions.

Paris Gene Editing Meeting

Credit, Caroline Simons

This meeting is one of a series which has been organized by the Committee to assist in its examination of the ‘scientific underpinnings as well as the clinical, ethical, legal and social implications of the use of human genome editing technologies in biomedical research and medicine.’ The Committee is examining both somatic and germline genome editing. It focuses on 7 areas in particular. These relate to the current state of the science, future directions and challenges, potential clinical applications, alternative approaches, and the efficacy and risks of gene editing in humans. The Committee asks if explicit scientific standards should be established to quantify off-target genome alterations, if current ethical and legal standards for human subjects research adequately address human gene editing technologies and what are the ethical, legal and social implications of the use of these technologies in humans. The Committee seeks to identify what principles or frameworks might provide appropriate oversight for these technologies and to assess the prospect of international harmonization of policies.

The Committee will prepare a report and make recommendations later this year. This report will ‘include a focus on advice for the United States’, and will ‘provide a framework based on fundamental, underlying principles that may be adapted and adopted by any nation’.

This 3rd Consensus Study Meeting was a larger gathering than the meeting of FEAM, the UK Academy of Medical Sciences and the Académie Nationale de Médecine France which was held the previous day. Attendees included European Commission representation, prominent scientists, philosophers, ethicists and lawyers from Europe, the US, Canada, China, Singapore and Malaysia. Three panels of speakers addressed the principles underlying governance, international governance perspectives and potential applications for germline editing. The meeting wrapped up with a moderated discussion among all of the attendees.

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Meeting summary of Paris human gene editing workshop

Editors note: This is a guest post from Caroline Simons who is attending the two Paris meetings on human gene editing. For more background on those meetings see here.

By Caroline Simons

There were just over a hundred participants at the workshop organized by the Federation of European Academies of Medicine, the UK Academy of Medical Sciences and the Académie Nationale de Médicine France. That number included experts in the fields of science, medicine, law and bioethics. They came from Europe, the US and China (and, I think I may have heard, one French politician).Caroline Simons

Some were engaged in active research, others represented national academies, policy making bodies, patients, research funders and industry. I noted one participant from the US represented DARPA, a reminder that gene-editing technologies may have harmful as well as therapeutic applications. There were about a dozen journalists, of whom two may cover this event in English – Anna McKie of Research Fortnight and Oliver Moody of The Times.

Académie Nationale de Médicine

Académie Nationale de Médicine, Credit Caroline Simons

The aim of the workshop was to consider current scientific activities in the European Union (EU) regarding genome editing and the regulatory landscape across the EU member states for this research and its clinical application in humans. The stated intention was to foster discussion between experts, provide information to the public and stakeholders and to consider whether an EU regulatory framework to govern the safe and acceptable use of human genome editing is desirable, and how it could be achieved. There were no agreed conclusions or recommendations from this workshop, but many interesting presentations and observations. A paper which will draw on the workshop discussions is to be published.

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GOP Congress seeks ban on CRISPR of human embryos

crispr nihGOP members of Congress want a ban on CRISPR of human embryos. They are moving to block genetic modification of human embryos using the new CRISPR gene editing technology.

The new proposed spending bill part way through the approval process with the nugget of good news of a possible NIH spending boost after many years of losing ground against inflation, reportedly also contains language to block gene editing of apparently any human embryos even just for research. Update: More recent interpretations of the bill including by Hank Greely (quoted in media and in his comment on this post)  read things such that human embryo research in the lab using CRISPR would be permitted. Material in this post has been edited to reflect this additional new context.

The funding for the coming year for biomedical research thus would be restricted such that none of it could be used, “in research in which a human embryo is intentionally created or modified to include a heritable genetic modification.”

Bioethicist Art Caplan was quoted in a Business Insider piece that this ban is a bad idea since it would drive the research into strictly privately funded research labs…perhaps with less transparency.

I support continued research including in some limited cases using CRISPR in human embryos with appropriate ethical and institutional oversight and training for the researchers involved. You can see more on my views in my TED talk (also pasted above) and in my new book on human genetic modification.

Politico, reporting on the same development made it sound like the provision might be more specific just to the clinical sphere:

“the spending bill also blocks gene editing — denying the FDA any funds to review or approve clinical research in which a human embryo is intentionally created or modified to include a heritable genetic modification.”.

To be clear, I do support a moratorium on genetic modification of embryos, but only specifically when there is the intent to use them in a clinical setting to create genetically modified people. The Washington DC summit at the National Academy of Sciences two weeks ago (#GeneEditSummit) discouraged heritable clinic applications of CRISPR in humans, but did not go so far as to propose a moratorium.

Just freakin do it: patients voice impatience on CRISPR for genetic diseases

IMG_4221Both before and particularly now after the big human gene editing summit in Washington, D.C. at the National Academy of Sciences, I’ve talked with patients about their views on this new technology including at last week’s World Stem Cell Summit.

One of the most striking moments of the DC summit was when the mother of a pediatric patient made the comment after a talk about human gene editing that if it could be helpful with genetic diseases, “just freakin do it!” A lot of pain was evident in her voice and tears.

Some patients at the World Stem Cell Summit indicated very much the same perspectives. I’ve heard from patients on this via email over the past year as well.

There is a need for rapid, new approaches to genetic diseases.

Patients and their loved ones often have a higher degree of risk tolerance than scientists. I have seen this in the stem cell field too, where many patients ask me about unapproved stem cell treatments for themselves (“I have MS, ALS, etc.”) or their children (“he has autism, etc.”). It’s not that they want to take risks, but understandably they are willing because they are in an urgent, health-related situation in their family.

Antonio Regalado over at MIT Tech Review has a powerful recent piece on a case of Huntington’s Disease patients including Jeff Carroll who are in favor of human embryo editing:

“I have no compunctions about it,” says Carroll, who is a neuroscientist at Western Washington University, in Bellingham. “I am saying, please, please do mess with our DNA.”

What risks are acceptable when we are talking about making genetically modified human beings?

There isn’t going to be one “right” answer to such questions, but it is clear that patient voices need to be part of the discussion. At future meetings and discussions on germline modification more patient voice need to be heard.

Perspectives: no moratorium from organizers of #GeneEditSummit

Human gene editing green lightNicholas Wade Gene EditingI just got back from a historic summit on human genetic modification in Washington, D.C. New genetic modification technology, termed CRISPR-Cas9, has both made genetic modification a relatively simple matter for scientists and human genetic modification much more likely in the near future.

Heritable human genetic modification could prevent some rare genetic diseases so there is real potential there, but it also could open the door to serious problems such as unforeseen health consequences across generations, social justice issues, and eugenics. Both potential positives and negatives were discussed in depth at the summit. Keep in mind that most but not all genetic diseases already are preventable via existing technology that allows for genetic screening of unmodified human embryos.

I was there blogging the event (see posts here). My lab also works on genetics and genomics. We are using CRISPR for in vitro research on stem cells and cancer. The goal of the summit, held at the US National Academy of Sciences, was to chart a path forward on how science and scientists should handle the central question of whether to genetically modify humans and what considerations should go into such a decision.

The organizers of the summit tasked themselves more specifically with deciding whether to propose a moratorium on heritable human genetic modification. Several of them had in the previous months seemed to indicate support for something like a moratorium in public statements and interviews. However, at the end of the summit, the organizer’s statement did not take a decisive step. They only discouraged heritable human genetic modification. There was no recommendation for a ban or moratorium.

GMO Sapiens Front CoverIn fact, David Baltimore who served as Chair, said at the end of the meeting that they specifically were not endorsing a moratorium and that was a conscious decision. It’s not entirely clear though why they made this decision, which seems to leave the door somewhat open to making genetically modified humans. More on that in a bit.

My own perspective is that we need a moratorium of at least several years on clinical use of heritable human genetic modification technology so I am somewhat disappointed in the final summit statement.

Why am I concerned enough to be in favor of a clinical moratorium? I mentioned some of the risks earlier in this piece. You also can see my concerns articulated in more depth in my new educational book on human genetic modification (here; note that it is written for both lay and scientific audiences and if you are interested in getting it you can use discount code WS15XMAS30 to get 30% off) and in my new TEDxVienna talk.

The summit organizers had several options available to them on their statement. You can think of it with a stoplight analogy. They could have proposed a moratorium, or a red light. They could have wholly endorsed human genetic modification and given it a green light. In fact the headline of a news story (see image above) seems to suggest that they did do this, but that is incorrect and the actual body of that article correctly reflects that the organizers only endorsed continuing basic research, which I also support.

Another option was to make a yellow light statement something along the lines of proceed with caution. They didn’t do this either…at least not exactly. The organizers’ statement was more like an “orange light“, somewhere in between yellow and red. While they wrote that any attempts in the immediate future at heritable human genetic modification would be “irresponsible” they did not go so far as to say via a moratorium, “don’t do it”.

The vagueness to the public of the statement is further reflected in the fact that the headline of a story on the summit on the front page of the NY Times by Nicholas Wade got the gist of the summit statement exactly wrong and incorrectly said that the organizers did endorse a ban (see above).

Why did the organizers go for an orange light approach to germline human genetic modification?

Perhaps as a group this best represented their range of opinions. In other words, they themselves did not reach a consensus to have a moratorium. I didn’t sense that there was such a consensus overall at the whole summit either. Reaching a consensus in science can be extremely challenging so by their nature consensus statements may tend not to be decisive. I get that.ABCD Plan Human Germline Modification

One potential more practical reason for not proposing a moratorium is that the organizers firmly believe that germline human genetic modification will someday prove useful and desirable. I got that vibe from some of their talks as well as from those of other very influential parties at the meeting. In that hypothetical scenario, a moratorium today could be hard to reverse tomorrow (in the future). Perhaps they didn’t want to risk impeding the clinical translation of the technology in the future with a moratorium. However, a pause in human genetic modification need not have been onerous or long-term.

Another possible consideration for the organizers is that a clinical moratorium could have hypothetically also unintentionally discouraged human embryo gene editing research in the laboratory so this may be another reason for not pursuing a moratorium. Again like the organizers, I also support such research, but for me it should be on a limited basis with appropriate bioethics training, transparency, and oversight (see my ABCD plan).

In the end, the statement from the organizers would have been more effective if it had been far shorter, clearer, and understandable to the lay public. Perhaps they were most focused on sending a message to scientists who might be more likely to get the key points of the statement, but even so it would have been best to be understandable to all.

I hope that with continuing dialogue and meetings, which the organizers also rightly proposed, that this issue can be clarified further and that the public can be engaged at a far deeper level. However, there is strong urgency for action and clarity here, and the lack of a decisive statement from this unique meeting was a missed opportunity in that regard.

Time is short. The technology in this arena is advancing at warp speed, it is so ubiquitous, and there is such strong enthusiasm that we do not have the luxury of years to have more meetings and discussions, as much as they may be very important, without taking a clear stance.

The number one question I’m hearing today after the meeting is concerning: isn’t human heritable genetic modification now already inevitable?