Fall/Winter 2015 Vol. 15 Number 2

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Initiative on Human Gene Editing

©Alexander Vasilyev/Thinkstock

Hundreds Attend International Summit; Comprehensive Consensus Study Gets Underway

New gene-editing technologies such as CRISPR-Cas9 are being heralded as "revolutionary" because of the great promise they offer for advancing biomedical research and curing genetic diseases like cystic fibrosis and muscular dystrophy. The technologies could also deepen our understanding of Alzheimer’s disease, cancer, and other illnesses with more complicated genetic connections.

However, the potential for gene editing to one day be used to make fundamental genetic changes that could be passed onto future generations -- known as human germline editing -- is of widespread concern, even to many of the scientists who are involved in such research. A group of prominent researchers who recognized how quickly this area of science is advancing approached the National Academy of Sciences and National Academy of Medicine for guidance. In response, the two academies launched a comprehensive initiative on human gene editing to provide researchers, clinicians, policymakers, and society with a thorough understanding of these
Participants at the International Summit on Human Gene Editing, photos by Pam Risdon Participants at the International Summit on Human Gene Editing, photos by Pam Risdon Participants at the International Summit on Human Gene Editing, photos by Pam Risdon Participants at the International Summit on Human Gene Editing, photos by Pam Risdon
technologies in order to inform decisions about human gene-editing research around the world.

The first major component of that initiative, the International Summit on Human Gene Editing, was convened in December in Washington, D.C. Co-hosted by NAS and NAM together with the Chinese Academy of Sciences and the Royal Society – the science academy of the United Kingdom – the summit attracted approximately 500 attendees and participants from more than 20 nations for three days of discussion about the scientific, ethical, and governance issues involved in human gene-editing research. In addition, more than 3,000 people in 70 countries participated via a live webcast of the summit, and there was widespread international news coverage of the event.

At its conclusion, the committee of experts that organized the summit issued a statement with its conclusions on basic and preclinical research, somatic cell research, and human germline research. "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," the committee said. "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."

The statement calls upon the four academies that convened the summit "to take the lead in creating an ongoing international forum to discuss potential clinical uses of gene editing; help inform decisions by national policymakers and others; formulate recommendations and guidelines; and promote coordination among nations." In response, the presidents of the four academies said they would work with academies around the world and in coordination with other international and scientific medical institutions "to establish a continuing forum for assessment of the many scientific, medical, and ethical questions surrounding the pursuit of human gene-editing applications."

Following the summit, NAS and NAM began moving forward with the second major component of their initiative, a study of the scientific underpinnings of human gene-editing technologies, their potential use in biomedical research and medicine -- including human germline editing -- and the clinical, ethical, legal, and social implications of their use.

Over the next year, the committee conducting the study will perform its own independent and in-depth review of the science and policy of human gene editing by reviewing the literature and holding data-gathering meetings in the U.S. and abroad to solicit broad input from researchers, clinicians, policymakers, and the public. The committee will also monitor in real-time the latest scientific achievements of importance in this rapidly developing field. While informed by the statement issued at the international summit, the study committee will have broad discretion to arrive at its own findings and conclusions, which will be released in a peer-reviewed consensus report expected in 2016. The report will provide a framework based on fundamental, underlying principles that may be adapted by any nation considering the development of guidelines for human gene-editing research, with a focus on advice for the U.S.

-- Molly Galvin

More information and updates on the initiative are available at <nationalacademies.org/gene-editing>.

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