A highly anticipated report says heritable genome editing, using tools like CRISPR to create edits that can be passed down to future generations, is not yet safe enough for clinical use.
But the 224-page report, “Human Heritable Genome Editing,“ precipitated by the birth in 2018 of twin girls in China whose embryos had been edited to resist HIV infection, also provides specific guidance on how nations should prepare for the eventual use of CRISPR or other gene editing tools. It maps out a translational pathway from research to the clinic, including the specific criteria and standards that must be met to permit medical use.
The International Commission on the Clinical Use of Human Germline Genome Editing from the U.S. National Academies of Medicine and of Sciences, and the U.K.’s Royal Society, was behind the report. It brought together 18 experts from 10 nations who were tasked with “developing a framework for scientists, clinicians, and regulatory authorities to consider when assessing potential clinical applications of heritable human genome editing.”
A separate WHO committee is examining the scientific, ethical, social, and legal issues related to human genome editing and is expected to advise on governance mechanisms later this year.
Dr. Bartha Maria Knoppers (LLB, PhD), Canada Research Chair in Law and Medicine and Director of the Centre of Genomics and Policy at McGill University, served on the international commission. Heritable genome editing has been a topic of concern since the human genome was mapped, she told Research Money, but international dialogue on the state of the technology, its potential uses, as well as the ethical, moral, and social aspects of human genome editing, has been insufficient. The new report aims to change that, recommending “extensive societal dialogue” before the clinical use of heritable genome editing is permitted.
“I think this scientific analysis, together with what’s coming from the WHO, is saying, ‘No matter what the results are, we need an ongoing discussion. We cannot wait and react. We’ve got to anticipate and govern,’” Knoppers said.
In an email to Research Money, Dalhousie University philosopher Dr. Françoise Baylis (PhD) commended the report’s authors “for recognizing that the permissibility or impermissibility of heritable human genome editing is too important an issue to be left to scientists alone. The challenge for policymakers is how to effectively engage civil society in important debates and discussions about the ethics of attempting to take over the evolutionary story.”
In total, the committee made 11 recommendations covering research on human genome editing and its translation to the clinic, as well as scientific governance and oversight.
Should the technology advance to be safe, reliable, and efficient, the committee proposes that its initial medical use should be strictly limited to the prevention of serious monogenic diseases, such as cystic fibrosis, thalassemia, sickle cell anemia and Tay-Sachs disease. (Monogenic diseases are the result of mutations to one or both copies of a single gene.)
“Any initial uses of HHGE should proceed incrementally and cautiously, and provide the most favorable balance of potential benefits and harms,” said commission co-chair Richard Lifton, president of the Rockefeller University in New York City in a press release.
“The report documents in very stark terms how few prospective parents might benefit from this technology,” wrote Baylis, University Research Professor at Dalhousie University and author of Altered Inheritance, a book about the implications of human genome editing released last year. “This is important background information for policymakers tasked with making difficult resource allocation decisions in the public interest. The data provided can usefully inform policy choices about how best to invest limited resources, including limited health research dollars taking into consideration possible opportunity costs if investing in heritable human genome editing.”
On governance and oversight, the committee calls for the establishment of an international scientific advisory panel to monitor and assess data on the development of genome editing technologies and their clinical use, an international body to anticipate and evaluate new uses of the technology, and a whistleblowing mechanism through which concerns about research or conduct on human genome editing could be reported.
By the time Chinese researcher He Jiankui revealed the birth of the CRISPR twins, he had consulted with dozens of scientific leaders in the U.S. and elsewhere about his research. But no one went public about it.
“We realized that there’s no such mechanism where you can go and where the procedures are secure and confidential, so that the ‘accused’ is as fairly treated as the person raising concerns,” said Knoppers.
The WHO is piloting a global registry to track clinical trials using human genome editing and has called on relevant R&D initiatives globally to register their trials.