It Has Arrived: Gene Editing Recommendations Published

February 28, 2017 • Posted in Blog

D. Joy Riley, M.D., M.A.
Executive Director

The committee appointed to advise our government regarding the editing of genes, including editing the genes of the human embryo, has published a draft report (see the title page screen shot above). We at The Tennessee Center for Bioethics & Culture are studying the document’s 261 pages, and have a few caveats to share with our readers. Germline gene editing was given cautious approval by the committee:

Heritable germline genome editing trials must be approached with caution, but caution does not mean they must be prohibited. (p. 102)

and

If the technical challenges are overcome and potential benefits are reasonable in light of the risks, clinical trials could be initiated, if limited to only the most compelling circumstances, subject to a comprehensive oversight framework that would protect the research subjects and their descendants; and have sufficient safeguards to protect against inappropriate expansion to uses that are less compelling or less well understood. (p. 102)

The recommendations regarding heritable genome editing are summarized as:

RECOMMENDATION 5-1. Clinical trials using heritable germline genome editing should be permitted only within a robust and effective regulatory framework that encompasses

  • the absence of reasonable alternatives;
  • restriction to preventing a serious disease or condition;
  • restriction to editing genes that have been convincingly demonstrated to cause or to strongly predispose to that disease or condition;
  • restriction to converting such genes to versions that are prevalent in the population and are known to be associated with ordinary health with little or no evidence of adverse effects;
  • the availability of credible pre-clinical and/or clinical data on risks and potential health benefits of the procedures;
    during the trial, ongoing, rigorous oversight of the effects of the procedure on the health and safety of the research participants;
  • comprehensive plans for long-term, multigenerational follow-up that still respect personal autonomy;
    maximum transparency consistent with patient privacy;
  • continued reassessment of both health and societal benefits and risks, with broad on-going participation and input by the public; and
  • reliable oversight mechanisms to prevent extension to uses other than preventing a serious disease or condition. (pp. 102-3)

Many questions arise from the reading of this list of recommendations; a few of them are:

  1. Are “reasonable alternatives” being sought now, or will most/all of the attention of our laboratories be focused on gene editing?
  2. Who decides what is a “serious disease or condition”?
  3. At what level are genes considered to “strongly predispose” to a disease or condition? Does that mean genes which may predispose to breast or colon cancer in mid-life will be targets of gene editing, akin to the licensing of preimplantation genetic diagnosis in the UK?
  4. How does a researcher maintain “rigorous oversight of the effects of the procedure on the health and safety of the research participants” if the participant is an embryo, a child, or an adult whose genome has been changed?
  5. To what kinds of follow-up will these participants be subjected? How will one’s personal autonomy as well as privacy be “respected” if, as an embryo one’s genome was tinkered with, and now inquiring scientists want to know?

Who created these recommendations? The list of creators includes a number of scientists who already do gene editing, so such recommendations are not surprising. Public input is now recommended by the committee.

Public input is needed. The final paragraphs in the document chapter regarding germline editing show the committee’s concern:

As of late 2015, the United States is unable to consider whether to begin germline genome editing trials, regardless of whether the criteria laid out above could be met. As noted above, a provision (in effect until at least April 2017) was passed in a budget bill, in which Congress included the following language:

None of the funds made available by this Act may be used to notify a sponsor or otherwise acknowledge receipt of a submission for an exemption for investigational use of a drug or biological product under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or section 351(a)(3) of the Public Health Service Act (42 U.S.C. 262(a)(3)) in research in which a human embryo is intentionally created or modified to include a heritable genetic modification. Any such submission shall be deemed to have not been received by the Secretary, and the exemption may not go into effect.

The current effect of this provision is to make it impossible for U.S. authorities to review proposals for clinical trials of germline genome editing, and therefore to drive development of this technology to other jurisdictions, some regulated and others not.

Perhaps a call to your representative and senators during the month of March would be helpful. Let them know of your opinion regardingĀ germline gene editing of embryos and future generations. It would be your public service.

Additionally, if you desire your own copy of the committee’s report, it can be purchased here, or downloaded without charge here.