Human Genome Editing Research Should Move Forward, Says Influential Hinxton Group

September 10, 2015
Press Release
For Immediate Release
Contact: Leah Ramsay, lramsay@jhu.edu, 202.642.9640

Human Genome Editing Research Should Move Forward, Says Influential Hinxton Group

Research involving editing the human genome, including research with human embryos, is essential to gain basic understanding of biology and germ cells and should be permitted, according to one of the first global meetings to debate the controversial new techniques.

The bold statement is published Thursday, September 10, by the Hinxton Group, a global network of stem cell researchers, bioethicists, and experts on policy and scientific publishing, who met in Manchester, England, September 3-4.

While firmly backing the need for gene editing research, the group makes a clear distinction between research and clinical application.

“We believe that while this technology has tremendous value to basic research and enormous potential for somatic clinical uses, it is not sufficiently developed to consider human genome editing for clinical reproductive purposes at this time,” the consensus statement reads.

According to Debra Mathews, a member of the Hinxton Group steering committee, discussions at the meeting focused on the use of gene editing in research and the most contentious aspects of these new technologies, primarily the implications for any children born with engineered genetic modifications, and also successive generations who would inherit those genetic changes; that is, the inheritable, or germline, nature of the modifications.

“While there is controversy and deep moral disagreement about human germline genetic modification, what is needed is not to stop all discussion, debate and research, but rather to engage with the public, policymakers and the broader scientific community, and to weigh together the potential benefits and harms of human genome editing for research and human health,” says Mathews, the Assistant Director for Science Programs at the Johns Hopkins Berman Institute of Bioethics.

The consensus statement addresses these ethical concerns, with the group agreeing that, “given all safety, efficacy and governance needs are met, there may be morally acceptable uses of this technology in human reproduction, though further substantial discussion and debate will be required”.

In the meantime, knowledge gained through basic science research is essential to human understanding of both ourselves and other life, the group says. “Much of our knowledge of early development comes from studies of mouse embryos, yet it is becoming clear that gene activity and even some cell types are very different in human embryos. Genome editing techniques could be used to ask how cell types are specified in the early embryo and the nature and importance of the genes involved,” says Robin Lovell-Badge, a member of the Hinxton Group steering committee and Group Leader, and head of the Laboratory of Stem Cell Biology and Developmental Genetics, The Francis Crick Institute.

The statement emphasizes the importance of “meaningful and substantial public engagement” to decision-making about genome editing. Policymakers are specifically addressed, stating that policy restraints on science should have justification that “that reaches beyond disagreements based solely on divergent moral convictions.”

“The relevant regulatory distinction should be not between using genome editing in somatic cells and using it in embryos, but between research and reproduction: whether those embryos are ever destined to be implanted, says Sarah Chan, another steering committee member and a Chancellor’s Fellow at the Usher Institute for Population Health Sciences and Informatics, University of Edinburgh.

“Restricting research because of concerns that reproductive application is premature and dangerous will ensure that it remains forever premature and dangerous, for want of better knowledge,” Chan says.

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Hinxton Group Consensus Statement on Genome Editing Technologies and Human Germline Genetic Modification, published online:http://www.hinxtongroup.org/Hinxton2015_Statement.pdf

About the Hinxton Group: http://hinxtongroup.org/

For more information or commentary from Debra Mathews, contact:
Leah Ramsay, Johns Hopkins Berman Institute of Bioethics
lramsay@jhu.edu 202.642.9640

Other contacts:

For commentary from experts who are NOT members of the Hinxton Group, contact: Robin Bisson, Genetic Expert News Service
robin@geneticexperts.org +1 (202) 833-4613

The Science Media Centre (UK)
fiona@sciencemediacentre.org +44 (0)20 7611 8300

Professor Robin Lovell-Badge, Hinxton Group Steering Committee member
Office: +44 (0)20 8816 2126; Mobile: +44 (0)7785 366987
robin.lovell-badge@crick.ac.uk

Or: The Francis Crick Institute media team:
Katie Matthews: +44 (0)20 7611 2197 or +44 (0)7734 955308
Peter Zarko-Flynn: +44 (0)7525 053795

Study Finds Patients Give “Broad Endorsement” To Stem Cell Research

Media Contact: Leah Ramsay, lramsay@jhu.edu, 202.642.9640

In an early indication of lay opinions on research with induced pluripotent stem cells (iPSCs), which are stem cells made from skin or other tissues, a new study by bioethicists at Johns Hopkins University indicates that despite some ethical concerns, patients give the research “broad endorsement”.

During focus group discussions patients were largely in favor of participating in iPSC research even if personal benefit was unlikely, though they raised concerns about consent, privacy and transparency when considering donating tissue for this research. The bioethicists report their findings in the journal Cell Stem Cell.

“Bioethicists, as well as stem cell researchers and policy-makers, have discussed the ethical issues of induced pluripotent stem cells at length, but we didn’t have any systematic information about what patients think about these issues, and that is a huge part of the equation if the potential of this research is to be fully realized,” says Jeremy Sugarman, the senior author of the report and the Harvey M. Meyerhoff Professor of Bioethics and Medicine at the Johns Hopkins Berman Institute of Bioethics.

Unlike human embryonic stem cells, iPSCs are derived without destroying a human embryo. Research with human iPSCs is valuable for developing new drugs, studying disease, and perhaps developing medical treatments. Sugarman explains that, while far off, scientists are hopeful that iPSCs could someday be used to develop organs for transplantation that the body’s immune system will not attack, because they can be created from the person’s own cells.

The study reveals the importance of prior informed consent for those asked to participate in it. According to the report, consent was highly important for patients in all five of the focus groups that were convened.  Some patients even suggested that proper informed consent could compensate for other concerns they had about privacy, the “immortalization” of cells, and the commercialization of stem cells.

There was a “strong desire among participants to have full disclosure of the anticipated uses,” the report notes, with some participants wanting to be able to veto certain uses of their cells. The authors acknowledge the “practical difficulties” of this request but hope that their findings will “prompt investigation into creative approaches to meeting these desires.”

The study also revealed another side to some patients’ selfless motivations to participate in research as they might relate to eventual commercialization. The report quotes one participant as saying, “It won’t be just taken to become a money maker and the very people who need it the most will no longer be able to benefit from it” and another, “…it was a donation. It’s a humanitarian effort.”

The authors also note that the unique characteristics of their small study could have influenced the results; for instance, the fact that it was conducted in Baltimore, Maryland, among patients who have received care at Johns Hopkins, where the first immortal cell line was created from tumor cells taken from Henrietta Lacks, put related issues at the forefront of many focus groups.  “The idea that donated cells would potentially live forever was unnerving to some participants. In particular, the story about the creation of the HeLa cell line from Henrietta Lacks’ cervical cancer tissue, taken without consent, was raised in four out of the five focus groups,” the report states.

Additionally, the report indicates that the opinions that were expressed by patients may be influenced by their health, and whether or not they have personal experience with a debilitating illness, as some of the participants did.

“It seems fair to say that everyone experiences serious illness in their lives, whether themselves or through someone they know and care about, and this influences their opinions of healthcare and research,” Sugarman says. “This study is a first step in getting crucial information about what values are factored into a decision to participate in iPSC research, and what those participants expect from the experience.”

East Asia Faces Unique Challenges, Stem Cell Innovation Opportunities

New Consensus Statement From The Hinxton Group Focuses On Japan, China

Tension is the theme running through the new consensus statement issued by the Hinxton Group, an international working group on stem cell research and regulation.  Specifically, tension between intellectual property policies and scientific norms of free exchange, but also between eastern and western cultures, national and international interests, and privatized vs. nationalized health care systems.

The consensus, titled Statement on Data and Materials Sharing and Intellectual Property in Pluripotent Stem Cell Science in Japan and China, was released on the Hinxton Group’s website Monday, November 19, 2012.

“China and Japan are among the world’s leading nations in stem cell research, but because of challenges distinct from western nations, they are dramatically underrepresented in terms of patents and licensing,” says Debra Mathews, PhD, MA, assistant director of Science Programs at the Johns Hopkins Berman Institute of Bioethics and a founding member of the Hinxton Group. Mathews was one of 22 co-signers of the consensus statement.

“We thought it was crucial, with the science advancing incredibly rapidly, and as intellectual property policies evolve in East Asia, to examine our 2010 global recommendations for proprietary issues in stem cell research in that regional context,” Mathews says.

Strengthening national stem cell innovation was the top goal articulated in Kobe, the statement says.  Whereas in the West there is a robust and mature infrastructure for encouraging and supporting the development of intellectual property rights such as patents, East Asian nations like China and Japan have comparatively less well-developed, younger systems, the group observes.  While this can make it more difficult to bring new inventions to international markets, the statement says, the opportunities created by the regional environment in Japan and China provide valuable lessons for the global development of this field.

“For example, as noted in the statement, Japan and China each have a large and highly qualified scientific workforce, paired with substantial national investment in stem cell research,” says Mathews. “This combination of factors means that both countries are well-situated to take the kinds of collective action that will be required to move the field forward efficiently and translate basic science discoveries into products and therapies.”

An area where Japan and China exercise strong state control to the possible benefit of stem cell-based invention is their national health care systems, the statement notes.  In the West, strong intellectual property rights have encouraged the “development of stand-alone blockbuster products,” the group says, whereas the national health systems in East Asia may allow patients access to more individualized, innovative treatments.  This, the group posits, could be a model for stem cell-based therapies.

“Innovation in China and Japan occurs in the context of national commitments to public health, and as a practical matter that should make access to cell-based therapies more equitable,” Mathews says.

The statement also notes the significant cultural differences that contribute to challenges — and opportunities — with intellectual property policy, practice and stem cell research in the region.  The group notes that Japan and China are “markedly less litigious” than western nations, and recognition for scientific work and publication priority are highly valued.  “Secrecy appears to be a relatively more common mode of protecting researchers’ raw [intellectual property rights], as opposed to more formalized legal systems of protection, such as patenting,” the statement says. In light of this, an appropriate incentive to sharing data and materials among scientists in the region would be the protection of their interests and rights, perhaps through a grace or priority period, the group says, during which the data is public but the original scientists have exclusive rights to publish.

The statement also discusses the challenges of sharing data and materials internationally, noting an “underlying tension between national and international interests.”  In China, for example, samples donated by citizens are considered intellectual property of the state and are governed by strict polices that create roadblocks to international sharing and access. Such policies, varying country by country, may present significant challenges to the Hinxton Group’s goal of creating an internationally coordinated stem cell bank, the statement says.

“There will always be tensions between national and international and between public and private,” as far as innovation and the protection of that innovation, the group says.  “Within the biomedical sciences, the key is to strike a balance that both promotes innovation and improves global health,” the statement concludes.

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Consensus Statement: www.hinxtongroup.org/consensus_hg12_final.pdf

Japanese and Chinese translations will be posted at www.hinxtongroup.org as soon as they are available.

About the Johns Hopkins Berman Institute of Bioethics
One of the largest centers of its kind in the world, the Johns Hopkins Berman Institute of Bioethics is the home for collaborative scholarship and teaching on the ethics of clinical practice, public health and biomedical science at Johns Hopkins University. Since 1995, the Institute has worked with governmental agencies, nongovernmental organizations and private sector organizations to address and resolve ethical issues. Institute faculty members represent such disciplines as medicine, nursing, law, philosophy, public health and the social sciences. More information is available at www.bioethicsinstitute.org.