Let the Experiment Begin: Impact of ECJ Stem Cell Ruling on Synthetic Blood Development

By Kevin E. Noonan --

In the aftermath of the European Court of Justice's decision last month that patent claims encompassing human embryonic stem cells (hESCs) were patent-ineligible in Europe on public order and morality grounds (see "European Court of Justice Renders Stem Cell Decision"), some commentators argued that the decision had a "silver lining":  hESC development unhampered by stem cell patents.  This view was challenged at the time, by several comments on an e-posting of the argument in a Nature News article, and today with regard to efforts to produce synthetic blood in the U.K.

The Nature News article, by Ewen Callaway, cited several European stem cell researchers on the negative impacts of the ban, including Oliver Brüstle at the University of Bonn, Germany (it was his patent that was invalidated by the ECJ's ruling) ("European ban on stem-cell patents has a silver lining").  This reaction was characterized as akin to saying that "the sky is falling," and the article contrasted this sentiment with the "more moderate view" that the ruling had positive aspects; for example, a "physician scientist," Chris Mason of University College London was quoted as saying that "[i]f anything the ruling is an opportunity.  It's not the end of stem cells in Europe."  A genuinely more moderate view was voiced by Nick Bassil, from Kilburn and Strode, who simply said that "[t]ime will tell how serious [the impact of the ruling] is going to be."  In addition, the article cited alterative aspects of stem cell technology that remain patentable including "[g]rowth media, equipment and chemicals that help scientists work with stem cells," citing Julian Hitchcock of Field Fisher Waterhouse, as well as methods for using stem cells rather than the stem cells themselves (although patent claims on such methods may also be suspect under the Court's ruling, depending on how such methods are claimed).  Finally, other types of intellectual property protection, such as trade secret, was cited, depending on the complexity of the techniques and methodologies needed to properly grow hESCs (paradoxically advocating non-disclosure of the "manufacturing processes" used as a beneficial alternative to the disclosure of such methods in patents and neglecting the societal consequence that non-disclosure promises to impede rather than promote progress).  And finally, the article suggests that many of the hESC patents will have expired by the time the therapies are ready for the clinic, again citing Dr. Mason, who suggests that the lack of patents could promote an "anything goes" climate for stem cell research.

Comments on the article rebutted this rosy view, noting that the ban was "far-reaching" and extended to "any downstream products" of hESCs (Comment #28580).  Others noted that the principles invoked by the ECJ in banning hESC patenting (the dignity of the individual) could be applied to protecting the dignity of other individuals, patients (Comment #28589), and that the Court "invent[ed] moral standards" for Europe unnecessarily (Comment #28617).

But the question remains whether the ruling will foster or hinder innovation, and while it will take perhaps a generation to understand the full extent of the ban, it is worthwhile noting its effects as they develop.  One such effect involved efforts by U.K researchers to create synthetic blood from stem cells.  As reported last month by FierceBiotech, The Daily Mail, and The Independent, the effect on the ban on these efforts may not be as beneficial as some have hoped (or posited).  The goal is to produce O-negative blood cells, which as the "universal donor" type would satisfy the unmet demand for blood.  Despite some successes, notably by Edinburgh University's Marc Turner, it is reported that researchers are considering "moving away from embryonic stem cells to iPS" (induced pluripotent stem cells, derived from mature human cells) in light of the ECJs ruling.  The reason is simple "[w]ithout patents . . . no private company would ever touch their work, let alone gamble the money needed to launch commercial operations."  As Dr. Turner told The Independent:

The NHS is not in the business of developing products and to see this technology going forward we would need private investment.  In the broadest sense, the patent ruling will have an impact on possible investment by the private sector.

As noted by Fiona MacRae in The Daily Mail, artificial blood is "the holy grail," having the potential to alleviate chronic blood shortages (particularly in acute situations like battlefields and car crashes) as well as providing an assured pathogen-free blood supply.  While teams from Edinburgh and Bristol Universities have made "thousands of millions" of red blood cells from bone marrow stem cells, this is mere proof of concept in view of the "2.5 million million" red blood cells contained in the amount of blood used in a typical blood transfusion.  The promise is in hESCs, which "are easier to multiply in large numbers," she says.

These efforts are threatened by the ECJ's ruling; as quoted in The Independent, Professor Sir Ian Wilmut (who cloned the sheep Dolly) expressed the widely-held opinion that "[u]nfortunately [the ruling] will make it less likely that companies in Europe will invest in the research to develop treatments to use embryonic stem cells for treatment of human diseases."  The Independent article also noted that over 100 "spare" in vitro fertilization (IVF) embryos from fertility clinics have been used to generate hESC lines and that one of them, RC-7, had been used to produce mature human red blood cells (albeit not O-negative cells).

This is just one data point in what can be expected to be a long experiment on the effects of patenting on innovation.  It has the benefit that these effects can be evaluated in "real time" and not retrospectively.  Whether the negative effects of the ECJ's ruling outweigh those benefits cannot be predicted.

For additional information regarding this topic, please see:

• "No More Patents for Cells Derived from Human Embryos in EU," October 18, 2011
• "European Court of Justice Renders Stem Cell Decision," October 18, 2011
• "European Court of Justice Considers Embryonic Stem Cell Ban," May 11, 2011

Comments

[6]

"Whether the negative effects of the ECJ's ruling outweigh those benefits cannot be predicted."

Sure it can Kev. Watch. I predict that the "negative effects" of the ruling, if they exist, will not outweigh the benefits.

[Robert Cook-Deegan]

On a more dour, but probably realistic, note: it seems quite unlikely that "time will tell" about the effects of the ECJ ruling. I doubt we'll ever know. There are so very many confounds in policies about hESC reearch and its applications, and patent are just one, possibly a minor one at that (compared to regulatory data exclusivity, other forms of exclusivity...). Other factors at least as important: whether stem cells actually work, level of R&D funding, whether policies about such products have higher or lower barriers to market entry due to regulatory pathways (that are not well trammeled or clearly marked), alternative cell-based methods that don't involve hESCs at any step... The odds of our being able to sort out a causal pathway of what policies mattered most are very low, even in retrospect, let alone prospectively.

[6]

"On a more dour, but probably realistic, note: it seems quite unlikely that "time will tell" about the effects of the ECJ ruling."

In other words, he agrees with me, if there are any negative effects, which there very well may not be, will not outweight the benefits.

We realists think alike.

[Kevin E. Noonan]

Dear Bob:

Actually, we might be able to tell, if the effects are similar to the effects of the Bush stem cell ban. While I agree that there are confounding variables, if stem cell companies flock to more hospitable shores (to paraphrase Chief Judge Rader) we will have a good indication. But of course there is no way to predict.

Thanks for the comment.