A committee of international experts, assembled by the National Academies of Science and Medicine, released a highly anticipated report on human genome editing this past week.
The report, entitled "Human Genome Editing: Science, Ethics, and Governance" addresses three major applications of genome editing, but, has a focus on making changes in the DNA that can be passed down through generations - germline editing.
For germline editing, although interventions to treat or prevent diseases are far from being ready to be tried in humans, the committee placed an emphasis permitting the forward progress of this scientific technology, with the contingeny that proper oversight be in place.
The committee stated that "caution is needed but being cautious does not mean prohibition."
They laid out clear criteria and conditions that must be met before any clinical trial can be considered. In order to clear a path for germline editing, the following are necessary.
- There must be an absence of a reasonable alternative which may include a baby being born and treated after birth with somatic editing intervention
- The technology will be restricted to prevention of a serious disease or condition
- A gene may only be edited when there is a strong understanding of the particular gene that is going to be altered and that the gene may not be changed to a novel form, but, only to the form commonly found in healthy people.
- Credible preclinical data on the potential health benefits of performing the genome editing must be in existence
- An actual protocol requires a regulatory agency capable of ongoing oversight throughout the study - consistent with patient privacy and with maximum transparnency
The committee made it clear that clinical trials may not begin unless these conditions can be met, but, that the trails are permissable upon meeting them.
Stress was placed on the need for public conversation in places where it already exists, such as comment periods in federal agencies, and opening up new venues and opportunities
For the other, less controversial types of genome editing, existing in areas of basic research and somatic therapy (human genome editing that is not in the germline - therefore, not passed down through generations) the regulations that are already in existence were deemed adequate.
Basic research and somatic treatments are regulated by institutional biosafety committees such as the institutional review boards (IRB) and the NIH Recombinant DNA Advisory Committee (RAC). In addition, somatic therapy (clinical interventions that are non-heritable) is regulated by the US Food and Drug Administration. Somatic therapy is proving incredibly exciting with the advancements such as immunotherapy treatment for cancer where the T cells are genome edited to kill specific tumor cells or treating HIV by blood cell editing. This committee did, however, recommend that these should be limited to treatment and prevention of diseases and not used for other purposes such as enhancement.
It was made clear that enhancement - the use of technologies not for treatment or prevention of a disease or disability, but, to enhance human attributes - should not be performed using gene editing.
The take home messages were that genome editing is valuable and adequately regulated in areas where it is already being performed. Somatic therapy should be used for the treatment of prevention and disease - not enhancement of desired human traits. And, that heritable genome editing needs more research before it can be considered.
Lastly, the international aspect of the committee was stressed including members from all over the world - with an emphasis on the fact that this was an international endeavor and that the guidelines should apply to a global audience.