Jonathan Kimmelman
STREAM (Studies of Translation, Ethics and Medicine)
McGill University
Guidelines 2.0
lab
studies
therapeutic
trials
assisted
reproduction
Principles
Principles
Integrity of the Research Enterprise
Respect for Subjects
Social Justice
Transparency
Primacy of Patient Welfare
gene editing
?
hEmbryo
1. Process
2. Substance
EMRO
review
approval
ongoing monitoring
1. Process
2. Substance
criteria
scientific merit
expertise of investigators
ethical justification
ISSCR
nDNA
modification
hEmbryo
in vitro
>14d;
primitive
streak
nDNA
modification
hEmbryo
ISSCR
in vivo
nonhuman
>14d;
primitive
streak
nDNA
modification
hEmbryo
ISSCR
implantation
gamete
ISSCR
nDNA
modification
hEmbryo
implantation
Recommendation 2.1.4:
Such research will enhance fundamental
knowledge and is essential to inform any
thoughtful deliberations about the potential
safety and use of nuclear genome editing in
strategies aimed at preventing the
transmission of genetic disorders
Recommendation 2.1.4:
until further clarity emerges on both
scientific and ethical fronts, the ISSCR holds
that any attempt to modify the nuclear
genome of human embryos for the purpose
of human reproduction is premature and
should be prohibited at this time.
Principles
Integrity of the Research Enterprise
Respect for Subjects
Social Justice
Transparency
Primacy of Patient Welfare
3 unresolved points :
1) biosafety
It would be irresponsible to proceed with any clinical use of germline editing unless and until
(i) the relevant safety and efficacy issues have been resolved, based on appropriate
understanding and balancing of risks, potential benefits, and alternatives, and
(ii) there is broad societal consensus about the appropriateness of the proposed application.
Moreover, any clinical use should proceed only under appropriate regulatory oversight.
At present, these criteria have not been met for any proposed clinical use: the safety issues
have not yet been adequately explored; the cases of most compelling benefit are limited; and
many nations have legislative or regulatory bans on germline modification.
However, as scientific knowledge advances and societal views evolve, the clinical use of
germline editing should be revisited on a regular basis.
Statement of Task: The study will examine 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. .
What is the current state of the science of human gene editing, as well as possible future directions
What are the potential clinical applications
What is known about the efficacy and risks of gene editing in humans, and what research might increase the specificity and
efficacy of human gene editing while reducing risks?
Can or should explicit scientific standards be established for quantifying off-target genome alterations
Do current ethical and legal standards for human subjects research adequately address human gene editing What are the
ethical, legal, and social implications of the use of current and projected gene-editing technologies in humans?
What principles or frameworks might provide appropriate oversight for somatic and germline editing in humans? Are there
examples of how these issues are being addressed in the international context?
What are the prospects for harmonizing policies? What can be learned from the approaches being applied in different
jurisdictions?
The committee will address these questions and prepare a report that contains its findings and recommendations. The report
will provide a framework based on fundamental, underlying principles that may be adapted and adopted by any nation that is
considering the development of guidelines. The report will also include a focus on advice for the United States.
Public Meetings
Meeting #1
The committee held a brief open session in which they discussed the charge with the study sponsors.
December 3, 2015 (4:00 pm eastern) Washington, DC
Meeting #2: The committee will hear input from select stakeholder groups.
When: February 11, 2016 (8:00 am eastern)
Where: Washington, DC, Keck Center of the National Academies
Meeting #3: The committee will hear input from the international community. This will be the final public meeting of the
consensus committee.
When:
April 29-30, 2016
Where:
Europe (city TBD)
Committee
Members
100
50
0
20
07
20
08
20
09
20
10
20
11
20
12
20
13
20
14
20
15
Number of donors
150
38%
30%
Research
preferences
at
2me
of
consent
Kalista
et
al.,
Cell
Stem
Cell
2013
Improving
IVF
outcomes
Improving
Stem
cells
and
Regenera2ve
medicine
Fidelity
of
CRISPR
in
human
embryos