Baltimore, et al. propose path forward for human germline engineering in Science

In a new perspectives piece in Science, Nobel Laureate David Baltimore and co-authors including Jennifer Doudna and George Church, chart a potential path forward for human genomic engineering involving germline modification. See also accompanying Bioethics piece by Gretchen Vogel as well, “Embryo engineering alarm”.

human germline editing policy

In the piece, entitled “A prudent path forward for genomic engineering and germline gene modification” calls for further discussion and assessment of key potential benefits and risks to moving forward with this technology. The illustration included here is from the piece.

The piece is reflective to a large extent of conclusions from a recent meeting held in Napa on this issue.

The summary statement is as follows:  “A framework for open discourse on the use of CRISPR-Cas9 technology to manipulate the human genome is urgently needed.”

They make 4 more specific recommendations.

  1. Strongly discourage clinical application of this technology at this time.
  2. Create forums for education and discussion
  3. Encourage open research to evaluate the utility of CRISPR-Cas9 technology for both human and nonhuman model systems.
  4. Hold an international meeting to consider these issues and possibly make policy recommendation.

This statement seems mostly in synch with the recently released ISSCR statement, but perhaps not quite as strong as it does not call for a moratorium on clinical use as ISSCR does and instead “strongly discourages” such applications.

In addition, this piece by Baltimore, et al. conveys more of a sense of optimism and somewhat of a more relatively positive vision that eventually CRISPR-Cas9 human germline editing might have safe, effective and ethical clinical applications. Even so they are relatively cautious about that possibility:

“At present, the potential safety and efficacy issues arising from the use of this technology must be thoroughly investigated and understood before any at-tempts at human engineer-ing are sanctioned, if ever, for clinical testing.”

The full list of authors include David Baltimore, Paul Berg, Michael Botchan, Dana Carroll, R. Alta Charo, George Church, Jacob E. Corn, George Q. Daley, Jennifer A. Doudna, Marsha Fenner, Henry T. Greely, Martin Jinek, G. Steven Martin, Edward Penhoet, Jennifer Puck, Samuel H. Sternberg, Jonathan S. Weissman, and Keith R. Yamamoto.

One interesting thing to ponder is the potentially more diverse views amongst this list of scientists and bioethicists even though have come to a consensus clearly on key issues.

For example, in Vogel’s piece, Church is quoted with what might be viewed as somewhat of a dissent on at least one level:

“Those uncertainties, together with existing regulations, are sufficient to prevent responsible scientists from attempting any genetically altered babies, says George Church, a molecular geneticist at Harvard Medical School in Boston. Although he signed the Science commentary, he says the discussion “strikes me as a bit exaggerated.” He maintains that a de facto moratorium is in place for all technologies until they’re proven safe. “The challenge is to show that the benefits are greater than the risks.”

What are your thoughts on this new Science paper and the ISSCR statement, both out today?

ISSCR calls for moratorium on clinical application of human germline nuclear genome editing

The International Society for Stem Cell Research (ISSCR) just now released a new policy statement on human germline gene editing.ISSCR_logo

The statement calls for a ban on clinical application of human germline gene editing technology.

Here is the brief summary statement at the beginning:

The International Society for Stem Cell Research calls for a moratorium on attempts at clinical application of nuclear genome editing of the human germ line to enable more extensive scientific analysis of the potential risks of genome editing and broader public discussion of the societal and ethical implications.

These are very important issues that are raised.

Notably ISSCR is supportive of in vitro research in this area in the lab:

At this time, the ISSCR supports in vitro laboratory research, performed under proper ethical oversight, to enhance basic knowledge and to better understand the safety issues associated with human genome editing technologies, including their potential for application in somatic tissues. The ISSCR also calls for broad public and international dialogue on the capabilities and limitations of these technologies and on the implications of their application to the human germ line. The ISSCR is committed to playing a role in catalyzing both the scientific and broader ethical deliberations about germline genome editing.

The ISSCR also noted that it does not take a position on mitochondrial disease-related, so-called “3-person IVF” technology, which it distinguishes from nuclear genome modification.

This statement overall seems at a middle ground relative to the recent Lanphier piece calling for a broader moratorium.

Stay tuned for more scientists weighing in on this important issue very soon.

Intensifying dialogue on human germline gene editing

human egg editThe field recently saw Lanphier, et al. in Nature calling for a moratorium on human germilne genetic modification.

A number of additional stakeholders are expected to be weighing in soon on the issue of human germline gene editing.

For example, a Science paper from others in the gene editing community is anticipated soon and may well have somewhat different perspectives.

I’ve been polling on this issue and there seems to be rather broad, international support for some kind of moratorium.

Respectful, open and diverse dialogue will prove crucial to formulating a wise approach to human germline modification.

Readers across the globe favor moratorium on gene editing of human germ cells in poll

In a still ongoing on-line poll, readers of this blog show a pattern of consistently favoring a moratorium on genetic modification of human germ cells.

Human germline gene editing poll

With more than 150 votes cast, the top two answers are ‘strongly favor’ and ‘favor’ a moratorium on human germline genetic modification. Only 15% strongly opposed a moratorium.

Overall those favoring a moratorium were 52.6% of respondents, while those opposed were 35,5%. These numbers could change over time and this poll is not scientific.

Notably, when I examined voting by country, the respondents from the vast majority of the 26 countries participating in this poll favored a moratorium, showing remarkable consistency across the globe.

The most notable exception was Canada where a majority of respondents strongly opposed a moratorium. Italy, Mexico, Romania, Russia and Malaysia each had 1-2 votes opposing a moratorium.

Could the readers of this blog just be somewhat conservative about cutting edge human modification technologies?

It doesn’t seem that way as in a past poll on 3-person IVF, another technology related to human genetic modification, readers were more inclined to favor legislation in the UK allowing that technology even if some were somewhat desirous of more data on it.

I’m still myself trying to sort out my own sense of where I stand on genetic modification of human germ cells and hope to blog about that in the near future.

Top 10 ways non-compliant stem cell clinic docs jeopardize their careers

Over the years I’ve often written about how unlicensed stem cell clinics and their physicians are putting patients at risk. In most if not all cases they are doing this simply for their own personal monetary gain. I do think that some of them truly believe they are helping patients, but still that doesn’t excuse risky behaviors that are throwing vulnerable patients into harm’s way.

The other reality here is that some of the stem cell clinic doctors are also putting their own practices and careers in serious jeopardy as well by potentially breaking ethical codes and even in some cases possibly the law. They aren’t doing this as some kind of ‘medical Robin Hoods’, but rather again for cash.

What are they doing that’s so risky?

Here’s a top 10 list.

  • 10. Selling stem cell interventions to patients for diseases that are outside the doctor’s area of expertise. Would you let a dermatologist do heart surgery on you? Would you as a plastic surgeon consider yourself qualified to treat patients with arthritis, MS, ALS, autism, etc. specifically for those disorders? Doctors treating patients outside their field of specialty are setting themselves up for big trouble. If literally anything goes wrong, can’t you just hear the trial lawyer saying, “Members of the jury, Dr. XYZ was practicing outside of his/her area of expertise when this problem occurred…”
  • 9. Conducting stem cell procedures without proper training. A weekend golf course-located stem cell “course” doesn’t quality a doctor to transplant stem cells into patients. Lack of training could easily be the basis for a malpractice suit or state medical board action.
  • 8. Not properly conducting informed consent with patients. As a doctor, you must tell patients about risks and alternatives.
  • 7. Not disclosing to patients that the doctor has a financial conflict of interest. At these clinics the doctors performing the experimental for-profit procedures are making money by convincing patients to buy the unapproved, experimental stem cell interventions aka “treatment”. They must tell the patients that in advance.
  • 6. Enabling many other doctors to do risky medicine. If you “train” other doctors to do stem cell procedures or you provide them a source of unapproved medical devices, etc. that enable them to put patients at risk, then you as a physician share in the responsibility and consequences that may come from that including litigation.
  • 5. Introducing stem cell drugs into interstate commerce. Doctors at stem cell clinics cannot legally be involved in transport of unapproved stem cell products from one state to another in the US, but sometimes they are. Importantly, keep in mind that introducing a stem cell drug “into interstate commerce” also more generally means pretty much any use of a stem cell drug product because of the much wider FDA definition that does not have to involve transport of the final product from one state to another. Simply anything used to make the drug need only come from another state.
  • 4. Exporting stem cell products out of and into the US. It is illegal for clinics to export or import unapproved human products/drugs across US national borders without permission in advance from the FDA and other agencies. Yet this happens all the time.
  • 3. Using investigational stem cell drugs that have not been approved the FDA. This may be a violation of federal law. Sure the FDA can be slow to act, but they will eventually come ‘a-knockin’ on your door if you are marketing and using an unapproved biological drug.
  • 2. Illegal service referrals. Some physicians doing stem cell interventions at dubious clinics may well have now set up illegal referral networks with other physicians. This is a very dangerous situation to be in as a stem cell clinic doctor as this is explicitly a criminal act.
  • 1. Treating children with risky, experimental stem cell interventions to make yourself money and sometimes using those children for PR. We see it happening more often. Parents whose children have autism, CP, or other issues are increasingly contacting me to ask about stem cell treatments and some tell me that their children have already gotten treated at clinics in the US. Yes, a stem cell clinic doc putting any patient at risk is not a good thing, but doing this with children to make money and for PR is more questionable and should something go wrong is far riskier to the doctor.