- Welcome back,
everyone, from lunch.
I will introduce
the first session
in the afternoon, which is
case studies, engineering,
and disability rights.
And for this session,
Pilar Ossorio,
who is a professor
of law and Bioethics
at the University of Wisconsin,
Madison, and bioethics
scholar in residence at
the Bioethics Program,
lead at the Morgridge
Institute for Research.
She's also the co-director of
the program on neuroscience
and law at the University
of Wisconsin, Madison,
and directs the University
of Wisconsin's ethics
and consultation service.
Again, these bios are pretty
dense because that's how
illustrious our speakers are.
Pilar will moderate this
session, and in this session,
we have the pleasure of
having Matthew Porteus.
But we also have
Rosemarie Garland-Thomson,
who is coming to us from
the University of Pittsburgh
through- oh, there she is.
Hi.
The wonders of technology.
So with that, and
since technology
is actually working
today, I will
have Pilar come to the
podium and start the session.
- So first, I would like
to thank the organizers
for this wonderful
event, and for inviting
me to be part of it.
So this morning we heard
that CRISPR-Cas technology
has been adopted very
rapidly, at least
in the research community.
It's used in plants,
animals, and humans.
It's being modified by
researchers around the world
to be used for many
specialized applications.
And I was happy to hear,
actually this morning,
that there was discussion
of international governance,
and how do we make that happen.
And so I think we also heard
that ethical, legal, and social
issues are being raised,
and perhaps with new urgency
because the scientific
and science policy
community view many types
of interventions on humans
as now becoming feasible in
the near future when, perhaps,
before they were
more theoretical.
So CRISPR-Cas gene editing
makes it relatively--
and I say relatively
easy, cheap,
and fast to make better
targeted genetic modifications
than we've been able
to do in the past.
So they're better
targeted, but of course,
one of the ethical
and regulatory issues
that we're still
grappling with in using
this technology as
a therapy in humans
is dealing with
off target effects.
How to minimize those, how to
identify them, and minimize
them if you're going to
actually use the technology
as a therapy in humans.
Setting that aside,
I think it's just
important to recognize
how rapidly this
has become a very widespread
technology, particularly
in the research community.
But even-- there's a lot
of public imagination
about this technology.
There are people
who have purported
to engage in do-it-yourself
gene editing, who have engaged
in attempts to bio-hack
themselves with gene editing
materials, let's say.
Whether they effectively
gene edited anything
is a whole other question.
I have no idea.
But today, we're primarily
talking about gene editing
of humans, right?
I think there was a
previous event that
talked about, maybe, gene
editing in crops and so forth.
Today we're talking
about humans.
And as Doctor Royal noted
earlier this morning,
gene editing in humans raises
numerous ethical and social and
legal issues, some of which
have been addressed in the past.
They're not all new.
And in particular, during the
1990s gene therapy era, or what
I like to call gene
transfer era, which
was really my
introduction to bioethics
as a baby bioethicist.
During that time, a lot
of issues were raised.
A lot of issues were
deeply analyzed.
We still have many
issues that have not been
fully or adequately analyzed.
And of course, there
are certain things
that capture the
imagination, but there
are many-- it's important
to understand, I think,
that there are many ethical and
regulatory ways of addressing
gene editing, especially somatic
cell gene editing in humans.
It's not like we have
to recreate the wheel.
It's not like we have nothing in
terms of ethics and regulation.
So a lot of it is not
particularly sexy.
For instance, we have ethical
and regulatory frameworks
for when it is acceptable
to do first-in-human trials
of new technologies.
Dr. Kimmelman, who
spoke this morning,
has really pioneered some
of the ethics work regarding
first-in-human trials.
We already have
those frameworks.
We need to think
deeply about how
to apply them, the details
about applying them
to somatic cell gene editing.
But we have the ethical
frameworks there,
we have the regulatory
frameworks there.
When we start talking-- so
thinking about gene editing,
we've heard the terms this
morning, somatic cell gene
editing and germline
gene editing.
So just to refresh
people's memory,
somatic cell gene
editing is done
with the intention of modifying
only the particular individual
who is being edited, and not
to have that genetic event pass
down to future generations.
Germline editing is
done with the intention
of modifying an oocyte,
sperm, or early embryo
with the intention that
the modification would
be passed down through a lineage
to future generations, right?
So we think about
germline editing,
I think it raises
some issues that
have been discussed
and analyzed,
especially in the 1990s.
But we haven't really had
to grapple with those issues
that much because making
intentional genetic
modifications to
future generations
was not really feasible
until, perhaps,
now or in the near future.
So what are some of
those issues, right?
Well, we think about
how do we balance risks
to people in the near
future, the next generation,
versus risk to people
in the far future?
Do we need to do some
balancing of that sort?
What do we owe to people
who don't actually exist?
Do we have obligations to them?
How can we have obligations to
them, especially if what we do
might change who is born, right?
That's a logical problem.
How will changing who
exists in the future
change the nature of society?
Those are very large
social questions,
and we haven't really had to
address them in any serious way
before.
And whether we're talking
about somatic or heritable gene
editing, we have some
questions like to what extent
do we expect, or
should we expect,
that people conform to
pretty narrow institutionally
defined norms of
behaviors and abilities.
Rather, as an alternative,
should we expect that our--
and even demand that our
institutions and norms
conform to a wider range
of variation among humans?
Would gene editing be used to
narrow the range of variation
or to expand the range of
variation, and in what ways,
right?
Because just as we
might use gene editing
to narrow the range of
variation with respect
to certain single
nucleotide polymorphisms
that lead to disease,
people might potentially
use it to expand human
variation in other ways.
So what should we demand of
society and our institutions
in terms of conforming to
the range of human variation?
How does that change
if we're intentionally
creating that variation or
limiting that variation?
And I think that
discussion this morning
highlighted the fact
that gene editing comes
into a social context in which
people all over the world
are doing this.
It is unlike the 1990s when
gene transfer was a technology
that not too many people
in not too many countries
really could,
realistically, undertake.
But now, gene
editing is something
that people all over the
world are already doing.
So we have different national
imaginations and priorities,
we have different cultural
concerns and considerations
about what kinds
of human variation
we value or don't value.
So this really does
raise a lot of issues
about negotiating governance
across national boundaries.
Because it's pretty clear, for
instance, from what we heard
this morning that
what happens in China
can have an effect on how
people in the United States
understand the technology
and its risks and benefits,
might affect how funding
for gene editing goes,
or the receptivity of the
public for gene editing.
So these are things that,
as we heard this morning,
a lot of people are
actually working on.
And I've been involved in
a little bit of that work.
But I think that's a discussion
that we can continue on, maybe,
in this panel and into
our afternoon panel.
So today, we have two
speakers for this panel.
As we heard, Dr.
Matthew Porteus,
who is a professor at
Stanford University.
He's in the Department
of Pediatrics
and the Institute for Cell
Biology and Regenerative
Medicine, and also in the
Maternal and Child Research
Institute at Stanford.
And he was a member of
the National Academy
of Science Engineering
and Medicine Committee
that studied issues related
to governance of gene editing,
and put out a report
that has been referenced
by several people this morning,
including Doctor Bonham.
And so he's going
to be talking today
about the current
technical limitations
on doing germline editing.
So this morning, I think even
though most of the panelists
were talking about germline--
or I mean, talking
about somatic editing,
still there was a
lot of, I think,
conversation-- there were a lot
of questions about germline.
So Dr. Porteus is going
to talk about germline,
and then I'm also just going
to say a little bit more
about Rosemarie at
this moment, too.
So as you heard, she's
our second speaker.
She will be coming
to us from remotely.
And she's actually a professor
of English and Bioethics
at Emory University where
she teaches and works
in disability studies
and in particular
on questions of justice
related to disability.
She also teaches American
Literature and Culture,
bioethics, and she works and
teaches in feminist theory.
And she's going to
speak on the core topic
of this session, which has
to do with disability rights.
And I think, this morning,
we heard some questions
around justice and
access to the technology.
You might this
afternoon hear, perhaps,
a different vision of--
a different
conception of justice
relating to disabilities.
Thanks.
[APPLAUSE]
- Thank you, Pilar,
for that introduction.
Thank you to the organizers for
putting this really outstanding
day together.
Thank you to my prior
speakers this morning
who did a lot of the legwork
setting up what we're going
to talk about this afternoon.
And finally, thank you
all for being here.
It's clear that this is an
audience that's engaged and is
asking immediately, I think,
insightful and incisive
questions that we're
all grappling with.
So what I'm going to try to
do over the next 20 minutes
is use somatic cell editing,
and our advances in somatic cell
editing, to treat diseases
like sickle cell disease
as a way of highlighting
the challenges we're
going to have to do germline
inheritable editing.
And I'm going to,
actually, start
with the criteria that the
committee that Pilar just
mentioned that I was on, in
terms of what might be required
before one would proceed to
germline or heritable editing.
I realize I left
heritable out of my title
because I think heritable
is the key feature here,
not so much germline.
So as was mentioned,
while many of the issues
that we're all very interested
in and want to grapple with
have been around for 20,
30, or 40 years or more.
The reality is is the
CRISPR-Cas9 system
gave a level of precision
to modifying the genome that
was unprecedented, and was
highly active as was discussed
before, and raised
the possibility
that once what was considered
infeasible was now feasible.
And in fact, the quick
application of the Cas9 system,
and again, I think
it's better called Cas9
because that's the scissors.
We don't actually
use a clustered
regular intersperse palindromic
repeats in anything we do.
But CRISPR is such a cool
name that we all use that.
But it's really Cas9,
that's our enzyme.
Quickly was applied to make
genetically modified organisms.
And again, it was
highlighted today
how many organisms within a
couple years of the discovery
the technology was raised,
including a publication showing
that modifications of
human tripronuclear zygotes
was possible.
Although those
publications actually
highlighted the limits to it.
So out of this came a call to
have the National Academies put
together a study committee
along with the Royal Society.
And I think for the first
time, and Pilar will probably
correct me, was the first time
a formal committee actually
said there might
be a path forward
for heritable or
germline entity.
I think prior was really
considered off-limits because
of the lack of specificity.
And listed here are number
are all the criteria that we
developed, and the report
is available online.
Now, I'm not going to
read through all of these
because they're available.
Now, just before-- but
I am going to split them
into two different categories.
I just want to
point out that this
was a relatively large committee
for the National Academies.
They tell me they usually
like to have committees
about half this size.
But given the need
to have stakeholders
from a wide range of countries,
and you can read here--
actually, it's not
listed their countries,
but they came, I think,
from all the continents
that people live on
other than Antarctic.
And a wide range of
fields, including
lawyers and bioethicists
and scientists
and human geneticists.
And out of this, I think,
pretty good committee--
it's obviously not a
perfect committee--
came these criteria.
And the key social and
governance criteria,
I best that's paraphrase
as no secrecy.
That whatever goes on, if
you're going to do this,
you should be
transparent about it,
you should be public about
it, you should disclose it,
and you should engage the public
about what you want to do.
And I don't want
to minimize these
because they're
really important,
but I'm a scientist, so I'm
going to focus now a little bit
more on the technical criteria.
And there were several
different technical criteria,
some of which have been
touched upon earlier today.
The first was is that
there would be no reason
to do heritable
germline editing if one
could have a genetically related
child through other methods.
Healthy, genetically related
child through other methods.
And those other methods could
include in vitro fertilization,
with a pre-implantation
genetic diagnosis.
Or it might, actually,
mean if we develop
real good somatic
cell therapy, there
would be no need to
then to use this method,
and there would be, then, no
reason to cross that rubicon.
I, personally, believe
that using editing
to increase the number of
potentially implantable embryos
is not a compelling rationale
to cross this rubicon.
I also think,
technically, it's not
likely to increase the frequency
because you're putting embryos
through a manipulation,
and thereby increasing
the chance they'll be inviable.
I think it's possible--
so in the United Kingdom under
the National Health Service,
IVF with PGD is
available and paid
for couples that are at
risk of having children
with genetic diseases as long as
you meet the list that they've
pre-approved.
And I think that has interesting
regulatory consequences,
and it prevents suffering
because you're not
bringing children into the
world with genetic diseases.
And as I wrote on the
slide, it has a side effect
of being economically
cost effective.
The second technical
criteria is it
should be restricted to
serious diseases or conditions,
and to genes with
clearly proven causality.
And the extension of that
was that the committee said,
you should only be
able to take a variant
and change it back to a
nucleotide sequence that
is present and
common and healthy
in the human population.
And so what that
means is that you
can take pathologic
sequences and revert them
to normal sequences,
but what it also
means is you don't take
healthy sequences, or even
pathologic sequences, and
introduce new sequences that
has never been in the
human genome before.
And I would actually argue
even shouldn't be people--
and here's where Karen and I had
a little discussion at lunch--
is if you can find a few
patients in the world
with a certain variant,
does that truly
mean that that
variant is healthy?
Or do I believe that the
gene is there for a reason,
and there's probably a healthy
function that it's there for,
and we can have a
discussion about that.
So with that as a
background about,
OK, if we believe
in these criteria,
we have to figure out a
way of reverting simply
to a normal sequence.
What is the challenge
in doing that?
And the challenges
can be listed--
or some of the challenges
can be listed here.
We need to make sure that
the process is specific.
We need to make sure
that we don't create
a chimeric embryo or human.
We want to figure
out how to generate
only those common and
healthy sequences.
And I want to propose, actually,
that zygote editing may not be
the solution to these problems.
But instead, what we may
end up seeing if this ever
does become
widespread, is editing
will occur at a stem cell
stage or at [INAUDIBLE]
pluripotent stage, and
you will the generate
sperm or eggs that will then
create a human by standard IVF.
So let me first rule
out this pathway.
So the standard way of
doing genome editing
is to design a nuclease
that makes a break.
We discussed that the Cas9
system has really democratized
and revolutionized the field
because of its ease of use
and because of this activity.
And when you make
a break it, can
be repaired by the cell
in a mutagenic fashion,
creating genes that
are now inactivated.
And again, we heard some
discussion this morning
about how that might be
used in somatic cells.
But almost by definition,
those new indels
are sequences that
have never been
seen in the human population.
They may mimic
mutations that have
been seen in the
human population,
but they are not identical
to the sequences that
are seen in human populations.
So to think about doing
this in the germline then,
we really need to harness the
other pathway that one might
use genome editing,
which is the homologous
recombination pathway
or homology directed
repair pathway.
And in this pathway, you
initiate the process exactly
the same way, with
an engineer nuclease
that makes a break in the
target site you want to modify.
And then, you
provide a donor DNA
that serves to
repair that break,
and you can introduce
exactly this sequence changes
that you want to make.
So you have both spatial
and nucleotide precision.
Now, before I go into how
this has been developed,
it was again mentioned
earlier today
that there are now a number
of different genome editing
systems that are not based on
making a double stranded break.
And it's possible in the future
that as these systems get
more and more developed,
they will actually
solve some of the
problems that I'm
going to say that currently
exists in nuclease based
systems.
So I'm going to
focus on the system
that we have found to be most
active in an ex vivo setting,
and that's to purify
our cell of interest,
to deliver the Cas9
guide RNA, the GPS,
as a protein RNA complex.
And then, follow that by
delivering the donor vector
on a virus.
And I won't go into
a lot of the reasons,
but what we found
is using this system
we can introduce the
genome editing machinery
in a way that doesn't
perturb the cellular function
significantly.
We're applying that to
sickle cell disease.
As Vence Bonham
mentioned earlier today,
this is a disease caused
by a single nucleotide
variant in the betaglobin gene
leading to a single amino acid
change in a protein, causing
red blood cells to sickle,
but leading to a multi
systemic disease.
And the median lifespan, as
was mentioned earlier earlier,
has not changed in
the United States
over the last three decades.
But the median lifespan
for patients in Africa,
where we estimate there may
be 200,000 births per year,
is in the first decade of life.
So using the RNP/AAV6 system,
where now in the blood
stem cells from sickle
cell disease patients
were able to correct the
mutation in up to 60% to 70%
of the alleles.
So frequency far
above what is thought
to be therapeutically
necessary of about 15%.
So we are now trying
to take this system
and move it from the
laboratory into the clinic.
And if in the Q&A you're
interested in our challenges
in that, I can say we
can talk about it more.
But maybe I can
quickly summarize
that that process
probably cost $10 million
to do in a lean
academic environment,
and would cost even more
if you were doing it
in a biotech or pharmaceutical.
Now, even though
we're able to correct
a large number of the genes,
in the process of correcting
some of these genes we
break the other allele.
And let me just go back.
To achieve this
level of correction,
we correct the
underlying mutation,
but we also introduced
silent changes
to facilitate that high
correction frequency.
And so what I'll point out
is those silent changes
are not present in
the human population,
and therefore, would not
be permissible under what
the criteria I just said.
Moreover, 70% of the cells
have at least one allele
with a non-homologous
end joining sequence.
And so again, that's
not a sequence
we'd want to introduce
in the germline.
And what's shown
in the data here
is within the
population of cells.
It's highly chimeric.
So some cells have
both alleles modified,
some cells have one
allele modified,
some cells have two
alleles modified
by insertions and deletions.
So this issue of chimerism
is really important.
So what about specificity?
So one way of thinking
about specificity
is that you're more likely to
get a highly specific reaction
if you don't have the nuclease
around for a long time.
So having it come in,
do its job, and go away
is one way of
increasing specificity.
And that has been shown by
a number of different labs.
But the example I like to
use was from Andy May's group
in which he showed that a
guide RNA that was targeting
a gene involved in a
hereditary bone marrow
failure, a rare hereditary
bone marrow failure syndrome,
looked very non-specific
in a test tube.
But when we use the RNP system
that I just described to you,
it was completely specific.
There were detectable
off-target indels.
But people want to
get even better.
And so then, the second strategy
beyond just using an RNP
was to improve the biochemical
properties of the Cas9 protein.
And we got to collaborate
with a company,
and we were able to show
that this high fidelity Cas9
improved the specificity of
our process by over 10-fold.
So we reduced our
off-target indels
so that they only occurred
at one site in the genome,
and at a frequency
of about 1% or less.
I'll put into context that
our genome is not sacrosanct.
It does not stay stable,
and that everyone's genome
in this room is different.
And I will say, thankfully,
diversity is strength,
and diversity gives
us robustness.
But moreover, our genome--
every single cell in our
body has a different genome
than its neighboring
cell because there
is ongoing mutations that
occur in every cell, every day.
Some of us fly on
airplanes, and we probably
shouldn't do that if we
want to not all drowned.
But that just exposure
to the ionizing radiation
is creating a
burden of mutations.
And so I'll argue that I think
that high fidelity ex vivo
RNP based genome editing is
safer than bacon, meaning
that we expose ourselves
to mutations all the time.
And that if you calculate how
specific our RNP is compared
to what we do, it's
actually much more specific
than what we do standardly.
Now, how efficient is this
RNP/AAV6 system in zygotes?
So it's, of course, has not
been used in human cells
or published in human cells.
I think it is being tested.
But in mouse cells, it's
a highly efficient system
in terms of creating
targeted mutations,
but it still results
in chimerism.
So what you end up with
is a mouse that has--
a fraction of the animal
has the changes you want,
but another fraction
of the animal
has changes that you don't want.
And so again, this is
not efficient enough
as it stands to think
about applying it
to the human population.
So what I mentioned
earlier, I don't think,
actually, zygote
editing is the way
that this is ever
going to really work.
And the reason I
believe that is if you--
the way you're going
to do zygote editing
is you'll inject either at the
one cell or two cell stage,
you'll let that cell
grow into a blastocyst,
you'll pluck one or
two cells, you'll
analyze the genome of
those one or two cells,
but we know that
those one or two cells
don't predict what's going
on in the six or seven cells
that you leave behind.
And I just can't get my
head around how you would
solve this technical challenge.
But I think you can get around
this technical challenge
by thinking about
modifying a stem cell ,
which you could then purify
to whole genome sequencing
to assure that you've
got only what you want,
and then derive your sperm and
eggs from those primordial stem
cells.
So for the sperm,
you could think
about modifying
spermatogonial stem cells.
For egg, you could think
about modifying an IPS cell,
and then differentiating
it into eggs.
And I won't show you the
data, but the ability
to turn IPS cells into true
eggs has been done in mice
by groups in Japan.
And I'm going to
show you the data
that being able to edit
spermatogonial stem cells
has also been done in mice.
So again, the RNP/AAV6 system
works very well in IPS cells.
And we can achieve up to 60%
bi-allelic gene correction
in IPS cells, which means
identifying a clone that
had both alleles corrected would
be a relatively simple process.
Now, in terms of editing
mouse spermatogonial
stem cells, that has been
done both with zinc finger
nucleases, which we did several
years ago, and more recently
with the Cas9 system.
And again, quite efficiently.
So in the last couple
minutes I just want
to summarize some
personal conclusions.
And in the last slide I'm
going to, actually, throw out--
I won't call it a strawman,
a proposal that you guys can
shoot down about how this
translational path forward
might work.
So the first is
that there are only
a very few examples in which
heritable or germline editing
would be needed to
prevent a child from being
born with a serious disease.
Karen, actually,
outlined one such couple.
A mom who has both alleles
having an autosomal dominant.
But this is still a very
few number of people.
As a pediatrician who takes care
of N of one diseases though,
I don't think this is
a reason not to do it.
I think the idea of
restricting edits
to sequences that are known
to be common and healthy
protects us from having
leakage into applications
that don't address disease.
There's no reason--
basically, you're
just going to do it to
make lots of normal people.
It also limits our hubris
in terms of thinking, well,
we're smarter than evolution.
I'm going to create this
variant in the human population
because I think I'm smarter
than what human evolution
and human genetics
has showed us.
And I think you could use
the UK National Health Care
System to generate
a list of sequences
that you could edit to, and say,
this is the permissible list.
There, obviously, has to be
quantitative public disclosures
in international
review, and I'll
talk about that
in the next slide.
You want to do it in
a non-chimeric way.
But I think that some of the
technical problems I outlined
can be solved between
the advances in stem cell
biology, germ cell biology,
and somatic cell editing.
And so I think if we hide behind
the technical limitations,
we actually aren't
forward-thinking about,
well, when this is
technically feasible,
is this something we should
do, and how should we do that?
And while I have focused
on the technical concerns,
I do not want to leave
you with the impression
that I don't think the societal,
ethical governance issues
are not important.
They are probably
even more important
because the technical issues, as
I said, probably can be solved.
So here's my--
again, I'm not going
to call a straw man
proposal because it
might be a real proposal.
Is I think the
translational path forward
is that a large, diverse
international committee
consisting of a lot
of different people
is convened to regulate germline
editing to create pregnancies
worldwide, not to regulate
germline editing for research
or somatic cell editing.
This committee would
supplement, but not replace,
existing regulatory
processes and authorities.
And I want to also point
out, the reason this
has to be international is if
one country, for example, we
have the example in China,
we have no restriction
on migration across borders.
And so if one country does
it, it affects actually
all of us in the world.
The committee might
adopt the strict criteria
that I outlined that the 2017
study committee proposed.
And I, again, like this idea
of only editing two sequences
that are known to be
common and healthy.
Now, of course, a committee
has no statutory authority,
so how is this going to
get a statutory authority?
It will only come if
countries actually
pass laws that say it is
only legal in our country
to perform heritable editing
and create a pregnancy if it
has been reviewed and
approved by this international
committee.
And the committee
would only approve,
or not, after a full public
disclosures with time
for a public comment period the
rationale, preclinical data,
including efficacy,
safety, and chimersim,
a review of the consent form,
a view of the clinical trial
design.
And for specific protocols in
which the committee did not
have the appropriate
stakeholders, of course,
you would want to bring
those stakeholders
onto the committee as
voting members, but as an ad
hoc basis.
Finally, I want to say, we
should never get to a point
where having the
children in quote,
"normal way" is considered
either unethical or frowned
upon.
And I think one of the
dystopian futures that people
have discussed is that, well, if
we were doing germline editing,
it would be expected that
we would all do this.
And that would be a
tragedy in my mind.
Barry Coller was
on the committee,
and I realize I'm one minute
over, but to summarize he said,
we want healthy babies,
not designer babies.
And I paraphrase that
a little bit to say,
we want healthy humans,
not designer humans.
And so I'll stop there, and
pass it over to Rosemarie.
Thank you very much.
[APPLAUSE]
- Thank you very much,
Matt, and the organizers
for the opportunity to
participate and contribute
to this really
important symposium.
And I'm particularly
appreciative
about the opportunity to try
to do this using communication
technologies, all
the time realizing
the limits of
those technologies.
So what I'm going to do here
is I have prepared a PowerPoint
presentation that
I'll be sharing
with you as a kind of
substitute for my live presence.
And I am garrulous, and
on top of that I also
am offering some fairly
counter-intuitive claims
and pieces of information.
So I will make this PowerPoint
available, it's long,
to anyone who wants to
take a look at it afterward
as a record of my
contribution here.
And I'll be moving
through it, maybe
jumping around a
little bit, which
I hope is not too
disconcerting, to try
to respect the time
that we have together,
and to generate some
important conversations
in our conversation
period afterward.
So most of the conversations
and presentations
about genetic editing, or
genetic engineering, center
around, as Matt's really
important presentation
just did, how these
technologies work,
and what we ought to be
using these technologies for.
And perhaps, how we ought
to be regulating them.
And that's a very
important conversation.
I'm going to offer something
quite different from that
here by focusing,
instead, more fully
on the social, the cultural,
and to a certain degree
the historical context
and the implications
of genetic engineering.
And so I'm hoping
that what I present
here will be a complement
to what Matt has presented,
and the other parts of the
conference that took place
this morning that I wasn't
able to participate in
because of my other
obligations here in Pittsburgh.
So let me begin
with my first slide.
I want to have us
together here in my time,
and I'm going to try to
work with 20 minutes here,
maybe 25 minutes, with asking
us to consider a paradox.
So this paradox has two
elements, as do all paradoxes.
So the first part
of this paradox
is that I want to make the
claim that this time now,
and place, where we
are gathered together
is the best time in all of
human history for people
with disabilities to have
a high quality of life.
The second part-- if we
can go back one slide--
of this paradox that relates
to the first claim I'm
making here, is that
modern liberal societies,
and I'll talk more
about this, this
is why this is the best time
for people with disabilities
to have the opportunity to
have a high quality of life.
It's because modern
liberal societies
aspire to assure
justice, equality,
and inclusion for all
members of the social order.
So these two assertions
relate to one another.
That's the first
part of the paradox.
The second part of the
paradox is the idea--
and I'll return to this
paradox more fully--
that modern liberal societies
at the very same time
are developing
medical technologies
for eugenics selection in the
interest of procreative liberty
and population health.
So I want us to hold
this paradox in our head
together when I consider some
of the points I'm going to make
in the rest of my presentation.
So the next slide,
thank you, was a repeat
of the first part of
my paradox, that this
is the best time in all of
human history for people
with disabilities to
have the opportunity
to flourish and to have
a high quality of life.
And let me give
you some examples.
In the next slide,
for example, I'm
showing a picture of the
actor Peter Dinklage, who
is a person who lives with
one of the conditions that
is part of the conversation
about heritable genetic
engineering that we're having
in public and in medical circles
and in the kinds of
conferences that I'm going to
and the kinds of papers that
I'm reading and contributing to.
And Peter Dinklage has a
genetic heritable condition,
which we consider to
be a genetic disease.
And that is he has
achondroplasia, which is
a heritable genetic condition.
So let me take a look at
some of the other people
that I want to mention who
live with these conditions that
are part of the conversation
about heritable genetic
engineering, and that we are
considering whether to select
for or to select against.
OK, next slide.
This is a picture of the
disability rights advocate,
Haben Girma, who is
a woman who lives
with hereditary
congenital deaf blindness.
And I'm showing here
a picture of her
with President
Barack Obama in 2015
when they were able
to meet in Washington
DC for a celebration
for the Americans
with Disabilities Act.
And Haben Girma is the
first deaf blind person,
if we can imagine this, to
graduate from Harvard Law
School.
Next, I want to show an image of
a person who is the first sign
language using deaf Rhodes
scholar, a person whose name is
Rachel Kolb.
She's featured here in a very
short film, which is terrific
if you have an opportunity
to take a look at it,
called Can You Read My Lips,
which is about how she uses
communication
technologies effectively
to attain a high quality
of life and be integrated
into the social fabric,
and be able to operate
as the first sign language
using deaf Rhodes scholar.
The next slide shows a
picture of John Kemp,
who is the president and
CEO of this Viscardi Center.
He is born with significant limb
reduction, which is understood
generally as genetic.
In other words, he was born
both armless and legless,
and uses a configuration
of prosthetic technologies
and mobility
technologies in order
to carry out his
work in the world
to be an attorney
and a public figure
and the president and CEO
of the Viscardi Center.
Next slide would be an
example of a woman who
is a business woman.
Her name is Kathy D. Woods.
She is a pioneer of
disability fashion.
She has a company in which
she designs and manufacturers
clothes for small adults.
And I'm showing her
a picture of her
with her apparel company logo.
She is an African-American
woman of small stature,
or a small woman with
a genetic condition
that can be identified in utero
and genetically increasingly.
One more slide to show you
to give you an example.
This is the comedian
Maysoon Zayid.
In her 2013 Ted Talk,
she is a comedian
with a significant
congenital disability.
It's not entirely clear
the genetic nature
of her disability, but
she advertises herself
as one of the first
disabled sit down as opposed
to stand up comedians because
of her mobility impairment.
And finally, I wanted
to share an image
of an older person
who has achieved
quite a high quality of life.
This is perhaps the most
well-known philosopher
in the world.
Jurgen Habermas, a
German philosopher
born in the late
1930s in Germany who
was born with a cleft palate,
which is not understood,
of course, entirely now
is a genetic condition,
although we're discovering new
genetic diseases, of course,
every day.
And we're not entirely sure what
a variety of genetic conditions
are produced by
genetically, but Habermas
writes in his acceptance speech
for The Kyoto Prize, the way
that growing up at the time that
he grew up with a cleft palate
and enduring many
corrective surgeries when
he was young for
the cleft palate
that he was born with as a
congenital disability, actually
shaped his work as a philosopher
of public communication,
and as a philosopher,
and that he would not
have done this work
in his life had he not
had the experience
of being born with
and living with a cleft palate.
So I want to go on to the next
part of my assertion here,
and that is that modern
liberal societies--
this is part of my paradox--
aspire to assure justice,
equality, and inclusion
for all members of
the social order.
And I'm going to introduce
here a bit of history
to talk to you a little bit
about the human and civil
rights movements
that began in 1948
and continue through
2017 and beyond.
And this is a bit of a
history of the development
of bioethics, and its
concurrent development
of the human and civil
rights movements.
So next slide please.
I'm going to begin with a
quote from Hannah Arendt,
the philosopher who wrote
in Eichmann in Jerusalem:
a Report on the Banality
of Evil in 1963,
that she believed
political regimes ought not
to determine-- and this
is a very famous quote--
who should and who should
not inhabit the world.
And this concept
is the concept that
forms a set of covenants, which
is what I call them, that is
to say declarations, treaties,
covenants, laws, policies that
constitute the Civil and
Human Rights Movement,
which I think--
and my history is that it begins
in 1948 with-- next slide--
the Universal Declaration
of Human Rights.
And I'm showing here an
image of Eleanor Roosevelt
holding this
Universal Declaration
of Human Rights in 1948.
And I'm going to quote from that
declaration in the next slide.
And that is, again, in 1948,
and I'm quoting directly,
all human beings are
born free and equal
in dignity and rights.
And this summarizes the impulse
behind the Civil and Human
Rights Movements that swept
in the mid 20th century all
of liberal social orders.
Another quote directly, from
the Universal Declaration
of Human Rights, talks about,
very often, the dignity
and the worth of
the human person.
And so this sense
of human rights,
this sense of human equality
and dignity and equal rights
inspires the Human and
Civil Rights Movement
of the mid 20th century.
And a part of this, of
course, is-- next slide--
the Disability Rights Movement.
And I'm showing here an image of
the Disability Rights Movement.
We have a protest
here with a number
of wheelchair-using
people with disabilities,
who are disability rights
advocates, who are protesting
in the mid 1970s and early
1980s and even in the late 1960s
for an accessible
built environment
and for equal rights and access
for people with disabilities.
And the banner here
that's being held up
shows the interrelation
of these rights movements
by quoting Martin
Luther King who says,
injustice anywhere is a
threat to justice everywhere.
So the Disability
Rights Movement
gives us the legislation
and the treaties
that we have in place
in the mid 20th century.
And later than mid 20th century,
and that is-- next slide--
primarily in the United
States the Americans
with Disabilities
Act, the ADA, of 1990.
And again, there's an
amended ADA of 2009,
which assures that people with
disabilities in the United
States will have equal
access and the means
to protest against
and take legal action
against discrimination, and
the legal capacity to request
accommodations.
So that's what the Americans
with Disabilities Act
does and aspires to do.
And of course, all justice
legislation is aspirational.
So there is a gap
between the aspiration
and the realization of the
Americans with Disabilities
Act.
But nonetheless, it is in
place to transform this society
and to make this time and
this place the best time
for people with disabilities
to flourish, certainly
in liberal democracies.
Another, of course, one of
these implements or treaties--
next slide--
is the Convention on the Rights
of Persons with Disabilities
enacted in 2006.
The United States has not
signed on to this treaty
because it has the Americans
with Disabilities Act.
But many other countries
around the world
have signed on to this treaty,
which is the United Nations
Convention on the Rights of
Persons with Disabilities.
We have it concomitant with
the Disability Rights Movement.
We also have what I call
knowledge development
about disability rights
and disability culture,
and the next slide gives
you an example of this.
This is the deaf bioethicist,
Jackie Leach Scully,
who is English, working now
in Melbourne, Australia,
who has written one of the
first and most important books
in the knowledge development
about disability bioethics.
And this is called Disability
Bioethics Moral Bodies
and Moral Differences.
So we have not just treaties,
but other knowledge documents,
if you will, that
help bring forward
the context that makes
it possible for people
with disabilities to
flourish most fully right now
in this place and in this time.
So I want to talk
for a moment, and I
have a lot of the things
I want to say on slides
so that you can have access to
them later if you'd like to.
This is a rather dense
presentation, so next slide.
I want to assert that
human rights covenants,
and this is, of
course, a summary
of what I've been saying in the
presentation I've been making
so far, carry out the principle
of active accommodation
that is fundamental to the
disability rights movement.
And those principles
of active accommodation
are change the environment,
not the person,
protect rather than regularize,
abide rather than improve,
and accommodate
rather than eliminate.
So this is my summary of the
work of the Disability Rights
Movements and its covenants
and their enactment.
OK, next slide.
If we are to enact the work of
these covenants effectively,
and when we do that
in our social order,
these disability
rights covenants
that I've very quickly
summarized here
for you will strengthen
support for disability culture
for the presence of people with
disabilities, and for awareness
about disability
inclusion and justice
across all civic institutions.
Another way of saying
this-- next slide--
would be that implementing these
disability rights covenants
does four things.
Does many things, but I want
to enumerate the four things
that it can do.
One is to change
attitudes, another one
is to increase access, a
third is to build community,
and the fourth is to
cultivate leadership.
And I've given you some examples
of some of the leaders--
people with disabilities leading
the way in public culture
for people with disabilities
to enter into public life
and to flourish most fully.
I want to spend just
a few more minutes,
and we'll move on a
little bit quickly.
If we can move from--
next slide.
And I'm not going to talk about
that to get to the next slide.
Because I want to talk
about the second part
of this paradox a
little bit, and that
is that at the same time that
this flourishing of people
with disabilities is
possible now in a way
that it never has been
in all of human history,
we are developing in
modern liberal societies
medical technologies
for eugenics selection
against conditions that we
consider to be disabilities
in the interest of procreative
liberty and population health.
And this is where genetic
engineering comes in.
Let me offer a
definition of eugenics
here, which is the practice
and the ideology of shaping
a particular human
community according
to the values of that community
by controlling who reproduces,
how they reproduce, and
what they reproduce.
So I want to suggest that
genetic engineering is
part of a pattern of
eugenic technologies
that are reducing
human variation.
I'm going to skip over
some of the history
of negative eugenics
that I've included here.
People can take a look at
that a little bit later.
I was going to talk a bit
about the old eugenics.
Eugenics being imagined
as the self-direction
of human evolution.
We'll move through the
next slide, the next one,
into what is sometimes called
the new eugenics, which
is generally understood
as liberal eugenics.
Let me offer a definition
of liberal eugenics.
So liberal eugenics is a
contemporary conversation
among philosophers
and bioethicists
that debates conflicting
liberty interests,
and it makes a moral
argument asserting
that reproductive autonomy
should include both
the obligation and
the right to select
against bringing disabled
children into the world.
So this is a definition
of the conversation that
is known as liberal eugenics.
And let me give you an
example of liberal eugenics
that comes from one of
its major philosophers.
Next slide.
Next slide.
Whoops, I'm sorry.
Go back one.
Thank you.
So this is a quote
from the philosopher
at Oxford University, Julian
Savulescu, who has put forward
a concept called procreative
beneficence in which Savulescu
argues that parents
in liberal societies
have the moral obligation to
create children with the best
chance of the best life.
And Savulescu goes on to argue--
next slide-- that couples
should select embryos or fetuses
which are most likely to
have the best life based
on available
genetic information,
including information
about non-disease genes.
So what Savulescu is doing here
with procreative beneficence
is endorsing what we call
enhancement genetics to be used
for enhancement in
order to provide
future persons with the
most advantageous traits
to have the best life.
So this is the concept
of liberal eugenics.
And I want to move on to one
more slide, a couple of slides
down, to talk a little bit
about reproductive selection
technology and practice.
Now, genetic editing
is only one element
of the practice of
liberal eugenics
in the reproductive environment.
And I want to simply suggest
several of these practices,
and then I'm going to
end they so we can talk
about other kinds of things.
But just to give you
a list here of where
these reproductive selection
and technology practices occur
in genetic counseling,
genetic editing,
selective genetic
testing and screening,
embryo screening, imaging
technology screening,
prenatal fetal diagnosis and
prognosis, prenatal information
delivery and framing, and
withholding newborn care
and sustenance.
Now, whether we agree
with these technologies
and the use of them or
not is really not what
I'm bringing forward.
I'm being descriptive
here in saying
that these are technologies
that are in play now
that have the effect of what
liberal eugenics is carrying
out.
So I will end with
one slide, which
makes the argument that I
would like to leave you with,
and that is the effect of these
multi-stage eugenics election
practices is to
increasingly standardize
modern liberal communities
by reducing human
biological and social
diversity, and along with it
social tolerance for
people with disabilities.
So I'll end there.
There's lots more if you
would like to have access
to the PowerPoint presentation.
Thank you very much.
And I'm happy to participate
in the conversation.
[APPLAUSE]
- Wow, thank you, Rosemarie.
I think that talk just
raised a whole bunch
of questions for us.
- It sure does.
- It's fabulous.
So I guess I want to push
on your very last claim,
and first of all say that seems
to be as an empirical claim
as much as a normative claim.
And as an empirical
claim, is it consistent
with your observation
that, at the same time
we've been developing
these technologies,
it is also the best time
to be alive as a person
with a disability at
least in this country.
Because I suppose one
could make the argument
that, in fact, perhaps
something like allowing parents
to have this kind of
reproductive liberty
results in people making choices
to have the children they want,
and the people who make
the choices to have
the children with down
syndrome or other conditions
that they could have screened
for but chose not to.
Really invest a lot
in those children,
and work to ensure that those
children have a very good life,
and those children are in our
society adding to the diversity
and richness of the society.
So I just want to push on
your very final claim there.
- When I make the
claim that this
is the best time,
at this very moment,
to live in modern
liberal social orders
if you are a person
with a disability,
I don't think it
conflicts with the premise
that we're reducing human--
well, not the
premise, the reality
that we're reducing
human variation
at the exact same time.
Take down syndrome,
for example, people
born over the last several
years with down syndrome
have the best opportunities for
education, for reemployment,
for a high quality of life,
better than any other time
that such people have
been born into the world.
And it is also exactly
at the same time when--
and the statistics vary a little
bit on this-- that worldwide,
and certainly in
the United States,
we are reducing that
population by up to 90%.
- Did you have any--
- I think one of the
key statements when
you were describing
liberal eugenics was the--
I think I'd like to you to
just explore a little bit more
about giving couples
the opportunity
not to have a child
with a disability
versus the obligation to have
a child without a disability.
Because I think that really
gets at the heart of the matter
whether you have a liberal
society in which there's
a freedom of choice, or whether
you have a liberal society
that is obligating
you to do something
that you wouldn't normally do.
And, actually, my
last statement was
saying that we
should not ever feel
like you should be
obligated to have
a child in an artificial way.
So [INAUDIBLE] obligation
and choice or opportunity.
- This is one of the really
crucial points here about
liberal eugenics as Julian
Savulescu and several other
utilitarian philosophers
and thinkers--
sometimes there are other terms.
Transhumanist,
for example, who--
I mean, Savulescu
makes the boldest claim
by saying that parents have
a moral obligation as opposed
to an opportunity.
So he's putting pressure
on the possibilities
for procreative choice or
procreative liberty in order
to make a moral argument that we
ought to have the best persons.
So that's why I like to
quote that because there
is, of course, a fine
distinction between what
we might think of as
free choice and a kind
of overdetermined
choice, which exists
in the reproductive environment.
And the over determination
of this free choice
is created in part by
the series of conditions
that we have decided to
test for with the potential
to select against.
- As I've been thinking
about what you're saying,
I'm reflecting on my own
training and practice
as a pediatric
oncologist, which is
we have families who come in
with a child who's diagnosed
with a cancer for which
there is known therapy,
but it's not guaranteed to
cure their child with therapy.
And if the parents
refuse chemotherapy,
in the US at least, there
can be the court order
forcing of the removal of the
child and the chemotherapy
administered.
So do you disagree with that,
or why is it OK to force
a family to have
chemotherapy for cancer,
but not OK to force--
first of all, I agree
that we should not force
couples to have an obligation.
I'm with you, but
I'm wondering how
you make the distinction
between post-natal provision
of optimal care versus,
say, prenatal, what you're
calling liberal eugenics?
- The ethical problem
is the expansion
of what we consider to be--
of the human variations
that we are now considering
to be disease categories.
So take something like down
syndrome or achondroplasia.
These are understood
as diseases.
These are understood as
negative health conditions
that can be tested for in a
variety of different ways.
They are also conditions
that people can live with,
and that people do live with,
and that live flourishing lives
with these conditions.
So when you identify such a
wide variety of human variations
as disease, and when you have
a very narrow understanding
of what we think of as health
and healthy and a healthy baby,
you are establishing
sets of criteria
that influence the
kinds of choices
that people make in
exercising procreative liberty
in the reproductive environment.
- Thank you.
That was wonderful.
I would like to change
just a little bit
and direct a question
to you, which is--
so you set forth the criteria
that the National Academy
Committee had
specified for when it
might be acceptable
to go forward
with germline gene editing
or heritable gene editing.
And those criteria seem very--
it's a very, very high bar.
And I wanted to put that in
conversation with the comment
that Dr. Bonham had on
one of his slides from one
of his patients
that he interviewed.
The person who said, well,
if you can do somatic cell
editing to take
care of sickle cell,
why would you let more
people be born with this?
Why wouldn't you
just do editing that
would prevent this
from happening
to people in the future?
And so I wanted to put
that in conversation.
- Well, I think that quotes--
Vence is using that for
a very particular reason
because it's
challenging a lot of us
to think about just that issue.
And I'll add to that as well,
which is the argument, well,
wouldn't it be much easier just
to modify something at a one
cell stage than try to modify
an entire body, or even
an entire organ
system like the blood.
So there's a technical argument
that people will make as well.
And I think the broad public
discussion with a broad range
of stakeholders is
about whether crossing
that boundary of modifying
genes that will be passed
on future generations,
which I do believe
is something that is
a line, is it worth
crossing that line
for that purpose
when the characteristic--
I'll call it a disease--
could be treated in other ways?
I'll just reflect
on what Rosemarie
is saying is that
disease always affects
the shape of that person.
But I don't think
that mitigates giving
the opportunity of that
person to cure themselves
of the disease.
Just because somebody is
shaped by their disease
does not mean we have
to enforce that they
live with that disease.
And you're nodding your
head, so you clearly agree.
But I think this is an
important thing to bring out.
Where I think we
might get to is--
one of the things that
has come up today,
and I think a lot of
people thought about,
is this concept of risk benefit.
So would you use a
first-in-human therapy
for something that
had an alternative?
Probably not.
Would you use first-in-human
therapy in a couple
that this was their only
way, and they chose to do it,
they weren't
obligated to do this,
to have genetically related
child, or healthy genetically
related child?
Supposing 30 years
or 50 years, there's
now been 100 or
200 people brought
into this world
that way, and it was
shown to be safe and effective.
Now, does the risk
benefit change?
And you start to say,
well, you're right.
Maybe we should do this
for sickle cell disease.
Then we get into the
issue, of course,
of I think it was implied in
the question about malaria,
is a lot of these variants do
exist in the human population
because they confer protection
against certain environmental
conditions.
And we would have
to continue, really,
get even more
knowledgeable about
human genetic environment,
social environment
interactions.
- So I think it's now time
to invite the audience up
for questions.
Great.
Jonathan.
- So I'm just interested in
this demarcation problem.
So clearly, conditions like
deafness, achondroplasia--
- Excuse me, would it
be possible for people
to say their names?
- Sure, yeah.
Jonathan Kimmelman,
McGill University.
So I'm interest in
this demarcation issue.
Clearly, one can
look at conditions
like down syndrome,
achondroplasia, deafness
as not necessarily disabilities,
but actually affirmative
aspects of human flourishing.
There's another category
of biological phenomenon,
that humans encounter,
that are clearly
in the pathological state
that have practically
nothing affirming about them.
Canavan disease,
Huntington's, et cetera.
And I guess what
I'm wondering, I
want to hear what
you have to say about
whether I'm misconstruing this.
To me I'm thinking about
this as a demarcation issue.
You have some conditions
that are just categorically
pathological and
others that aren't.
Am I wrong to think of this
as a demarcation issue?
Or if it is a
demarcation issue, tell
me more about how
we draw the boundary
around the conditions that
are part of human flourishing?
They're just different and
we haven't built societies
around recognizing that
flourishing, versus conditions
that are just scourges.
- Yeah.
Maybe I'll take a
first shot, Rosemarie,
and you can do second.
Absolutely, you're talking
about what another person--
another way of framing that is--
well, I hate to use the
term slippery slope,
but what's disease
and what's trait?
I'll tell you what
you don't want,
is you don't want me
deciding, and you probably
don't want you deciding.
That's why I propose
that the decision should
be made by a enormous group of
people, including the patient--
we won't call them patients,
people and family members
with that condition.
And I think that one way of
thinking about it is, say,
deafness.
Some families feel like that
defines them as a person,
and they want to have
children with deafness.
Other families feel like
that's not something
they want to have children for.
So I think that my
answer would be twofold.
One is that it's
not me to decide
that, it's the
families of communities
with the disease that weigh in.
But then I would say, and there
should never be an obligation,
but I think there should
be an opportunity.
- Rosemarie.
- Thank you.
This, of course, is a
really important question.
And in laying out my description
of eugenics and liberal
eugenics and the practice
of liberal eugenics
in the interest of
reproductive liberty,
I don't want to make
an argument that we
should outlaw these practices,
or to oversimplify it
in any way.
The important development
of medical technologies
and genetic editing is
only one medical technology
that will help human beings
have better lives and flourish.
I want to point out that
it's, well, complicated.
One good-- well, let me
bring up two examples.
So one example is Huntington's.
So the conversation about
genetic editing often turns
to what kinds of conditions
should we work to eliminate
with heritable
genetic editing now,
and one of the nominations
is always Huntington's.
And I work quite closely with
the historian Alice Wexler,
who has researched and
written about Huntington's.
And Alice says that
if you ask people
who have Huntington's
in their family,
her thinking is that
overwhelmingly these people
would say Huntington's is
an absolute scourge that
ought to be eliminated from
the human condition for once
and for all.
But at the same time, there
is also a complexity to this.
One complexity is that there is
a treatment being developed now
that is in trials
for Huntington's that
may be successful.
That's one thing to think about.
Huntington's is a very late
onset genetic condition.
Lots of things can
happen between conception
and the onset of Huntington's.
Huntington's can manifest in
a variety of different ways,
even though it is
a genetic condition
that if you have
the gene for it,
you will develop the
disease, or at least that's
the understanding of it now.
But what's most interesting
is that for about 20 years
we've been able to identify the
gene for Huntington's so that
people can, if they
suspect Huntington's is
in their family, they can have
the genetic test and find out
whether they carry the
gene for Huntington's.
And what has happened
is only about 20%
of people who may have
Huntington's in their family
have actually taken the test.
And this says something about
how we understand ourselves
as people with specific
genetic identities,
and how we understand ourselves
as navigating lives in relation
to our genetic profiles.
I'm not sure, nor is Alice
Wexler, what this says,
but it's something worth noting.
This conflict that is apparent
between the number of people
that actually want to
find out whether they have
the genes for Huntington's,
and the idea that Huntington's
might be edited out of
the human condition.
Those are not necessarily
contradictory situations,
but rather, they're
instructive for us
to think more clearly about
how we understand ourselves
as human beings with
genetic complexities
and genetic identities.
- Thank you, Rosemarie.
We have a line of people who
are waiting to talk to you.
So next please.
- I actually have
a question for Matt
about the last slide, the
gigundo committee that--
the International
Committee that's
going to solve all
these problems, and then
get all their countries to
sign on to them and pass laws.
By the way, I'm Jennifer
[? Hopeshal, ?] I'm sorry.
I'm a political scientist
in government department
of Harvard.
So I'm going to ask about
the politics of this,
not surprisingly, which is it
who sets up such a committee?
How does this happen?
I mean, that's not
your job to answer,
of course, but the
broader question
that this evokes for me is,
there are a lot of people
in this room, probably
including myself, who
think that scientists have
some responsibility, more
or less, but more than
zero, to engage seriously
and importantly in
the public arena.
And other people
who say, well, no,
actually scientists have
a distinctive comparative
advantage, and it's
not the public arena.
That's the job of people who
teach politics or whatever.
So the particular question is
about your gigundo committee,
that sounds wonderful.
The broader question
is how do we
get from here to there,
but in particular,
how do we think about the role
of scientists, clinicians,
front line people?
How much responsibility
should they have to take,
and how much of it is
just not their job?
- All right, could
I just say, let's
let Matt answer that
question, and try and keep
our questions
short, and remember
that if we don't get a
chance to answer everything,
there is another
panel after this
that maybe can take on
some of these questions.
- I think I'll give
a very short answer
to that, which is I absolutely
think scientists have
an obligation to participate
in the evaluation
and interpretation
of the science we do.
But we don't have the--
we're not the only voice
in the conversation.
And I think the fact that
we have many people today--
many scientists participating
today shows many of us agree.
I also think that some
scientists are terrible at it,
and they probably
shouldn't participate.
And just like I probably
should not be a politician.
So who should make
this committee?
Again, as you said, I don't have
to decide this, fortunately.
But I hope that the
two committees that
were discussed earlier, the
National Academy Committee
with the Royal Society
Committee, as well as the WHO
Committee, may come
to the conclusion
that this committee I
propose needs to be formed.
And then, I think it
would be something
like the WHO or
the UN that would
make this sort of
committee, and we
know how well that worked
with climate change.
So I understand
all the weaknesses
with such committees.
- Let me say that there is
a International Bioethics
Committee, I have
learned recently,
that is constituted and
run by UNESCO that exists.
It's an International
Bioethics Committee.
And I was just at
a meeting in Geneva
about disability bioethics.
And it was interesting
because several members
of this committee were present
at the meeting, the conference.
And they were very eager to
talk about disability bioethics,
but several of them said that
they didn't know anything
about disability or
disability rights.
So one of the things
that would be important
is to have a broad
representation
or, and a educational
component for this committee
to bring forward perspectives
and histories and knowledge
bases outside of medical
science and medical technology--
- Absolutely
- On this committee.
- Thank you.
That is-- I totally agree.
I think we all agree.
- We all agree.
- Next.
- Ricardo Cortez, the
Institute [INAUDIBLE]..
This question is for Matthew.
On the technical criteria
that you were listed as far as
you're allowing this type
of technology to be applied.
You said, it was restricted to
serious diseases or conditions,
but when you see that there
is about, I don't know,
30 million rare diseases in
the world, and 80% of those
are genetic, is that maybe
just too broad over restricts?
- The question is is the
restriction too broad?
Well, it goes I
think a bit to what
Pilar asked me earlier is why
not for sickle cell disease.
And I guess I would answer
your question is start strict.
If things are safe and effective
and there is global consensus,
then expand to more
and more conditions.
But I think in
2019, I would rather
err on being strict
rather than being loose.
My opinion, right?
- Next.
- Hi, my name is Amy Steiner.
And I guess my question
comes from both
being a clinician
as well as a parent
of a child with a disability.
One of the things, or a couple
of the factors that struck me
in the presentation related to
people with disabilities that
weren't really
highlighted was one,
geography plays
an important part
in your ability to be
successful with a disability.
We actually moved 3,000
miles to get access
to appropriate care for
a child with Asperger's,
high functioning, here
today because he has
an interest in gene editing.
So I do hear the
part about there's
an ability for them to
have thriving lives,
but at the same time,
I think geography
plays an important
part in access to care.
Socioeconomics is also an
incredibly important part.
I spend about 25 to $30,000
a year out of pocket
just so he can get
the therapies that
aren't covered by insurance.
And then, to your point
about down syndrome,
there are varying
degrees of disability
and physical suffering
that come with many
of these different types
of genetic disabilities.
And so while one may be
incredibly high functioning,
another one may be
very catastrophic.
Bedsores, infections, chronic--
ventilator use and so forth.
And so one of my questions
is related to the covenants
that were established.
In what year were
those established?
Was that before a
lot of this type
of technology and
discussion had started
related to gene editing?
And then, my other
question is who
should carry the
responsibility of making sure
that other children have access
to the same things my son did?
- I'd like to address
that because one
of the most important
parts of this conversation
has to do with distribution
of resources, and just
distribution of resources.
So when I offer the
real provocation that,
right now, right here,
perhaps in Cambridge,
people with disabilities
have the best opportunity
for the highest quality of life
ever in all of human history.
That provocation is based
on access to resources.
And part of the
conversation about the use
of these medical
technologies needs
to focus on resource
distribution.
The amount of money that
goes into the development
of medical technologies
to eliminate disability,
and people with
disabilities, could
be redirected to
increase and support
the quality of life of people
with disabilities, children
with disabilities,
people with disabilities
throughout the life course
in important and just ways.
- In regard to the degree
of physical suffering
or potential physical suffering
as a result of a disability?
- I think this is a
complicated question
because it is ethically
inappropriate for anyone
to evaluate the
suffering of anyone else.
And we have had
these conversations
more fully around the politics
and ethics of aid in dying,
but to assess in advance what
kind of suffering someone
may occasion in a life
is I think, actually,
unethical for us to
do in the same way
that it is basically unethical
for us to, as a community,
impose the preferences of
some people on future persons.
And yet, of course,
that's the responsibility,
the parental obligation,
and the obligation
that we have as a community
to ensure and provide health
for everyone and
for future persons.
So I'm talking about
a paradox here.
I'm talking about a conflict
of ethical imperatives
that we want to be
able to really think
about quite clearly.
- Hi, I'm David [? Risen. ?]
Here's my question,
it's directed to
Professor Garland-Thomson.
As a physician and the father
of an institutionalized adult
with severe autism who
is intellectually very
compromised, I could
not fail to notice
what I thought was
a certain selection
and bias in the group of
disabled adults you showed us,
all of whom were of normal or
above average intelligence,
and who were functioning
in the world.
Could you comment?
- Yes, thank you
for that question.
A great deal of very
interesting work
has been done about or
around family members
with what we consider to
be significant or severe
disabilities.
Some of that work is done by
the philosopher Eva Kittay, who
has a daughter whose
capacities and life
might be somewhat
parallel to your son.
That is to say, Eva's daughter
has lived in an institution
and thrived in an
institution her whole life.
And she could be understood
as having a severe cognitive
impairment of some sort.
And Eva Kittay's entire
work, as a philosopher,
has been to make the argument
that her daughter, and people
like her daughter, can have
a high quality of life,
and can, and often are,
loved and appreciated
as family members.
And to bring forward these
stories, there are many of them
in memoir written by
people with disabilities
who have family
members, children
with significant disabilities,
who love and appreciate
these children, and want them
as members of their families
as they are.
As counter-intuitive as
that may seem to many of us,
these are the narratives that
are brought forward that we
need to pay some attention to.
- Can I just make
one comment and--
- Of course.
- Which is-- and I
think, Rosemarie,
you should have said this,
which is I'm sorry to hear about
your son.
It sounds like while he can
have a high quality of life
as you just argued,
I think that reducing
suffering in this world
is all of our goals.
And if you believe
your son is suffering,
then I think it's up to us
as physicians and scientists
and the society to find ways
to minimize that suffering.
And that's what the
conversation is about without--
[APPLAUSE]
--labeling human beings who are
alive as somehow less worthy.
So that's the balance
we're looking for.
- I wanted to add one thing.
I think that the suffering
and toll taken on his mother,
on me, and on his
siblings is also
part of the equation because
of the enormous demands placed
on a family system by a
child who is born this way.
- Yeah, I agree.
And I call that the
echo of the disease
because it really
echoes through even more
than just your family.
I am sure it echoes
broad, and that's also
what we want to improve.
- Thank you.
[APPLAUSE]
- Thank you, Pilar,
Matthew, and Rosemarie.
To say that was a very powerful
session is an understatement.
And not surprisingly,
as someone said today,
the technical we can do, we
can work around the technical.
But this is where
the challenge lies.
And we will take
a 15 minute break.
And please come back
because Sharon Begley
will have all the
moderators back,
and we will reflect on
all the sessions today.
Thank you.
