Money and stem cell ‘miracles’: Pluristem questioned on ethics

Pluristem Therapeutics Inc. is a publicly traded (NASDAQ) biotech Israeli company that is developing stem cell treatments based on the use of expanded placental stem cells called PLX cells.

The Pluristem PLX drug is not approved for treating patients at this time in any country, however Pluristem has treated a handful of patients via something called “compassionate use”, a mechanism for treating patients who are desperately ill for whom there is no other available treatment.

Bloomberg news reports that Pluristem’s compassionate use treatment of patients has raised serious ethical questions. Press Releases and statements issued by the company, curiously timed stock sales by Pluristem leaders, lack of patient follow up, and an unreported child patient death together lead Bloomberg to paint a picture of potentially conflicted interests at the company.

Bloomberg reporter David Wainer, reporting on the story, suggests a possible motivation for some of Pluristem’s actions:

The flurry of press releases and lack of follow-up shows the pressure early-stage drug companies are under to generate investor excitement. As with most biotechnology companies that have no drugs on the market, raising money through stock sales is Pluristem’s main way of financing product development.

A case of a 7-year-old girl treated in May of this year with PLX by Pluristem has in particular raised ethical questions. The girl had a life threatening condition called aplastic anemia in which a person’s bone marrow stops working normally.

After her PLX treatment, a press release was issued by Pluristem that made very dramatic statements such as (emphasis mine):

“Compassionate Use of Pluristem’s PLX Cells Saves the Life of a Child After Bone Marrow Transplantation Failure.”

Investors took note and the price of Pluristem stock shot way up.

A second patient was treated in August to much fanfare as well. The patient’s doctor publicly stated that PLX saved her life.

Pluristem stock shot up again. Within days, members of the Pluristem leadership team sold stock in the company making major profits (for some over $100,000).

According to the Bloomberg article, a third compassionate use occurred the next month in September of this year for a leukemia patient.

Sadly, it was around the same time of the third treatment that the girl who was the first compassionate use patient died. But Pluristem did not report this publicly even though Bloomberg quoted a Pluristem statement that suggests a culture of openness:

“Pluristem is a publicly traded company and must maintain transparency, as they are obligated to share information with the public,” the company said in an e-mailed statement.

Why no report on the patient’s death then?

Wainer writes about this issue as follows quoting an ethicist:

Even if the law is less than crystal clear on the subject, the company should have disclosed the girl’s death, said Paul Root Wolpe, a bioethicist and director of the Center for Ethics at Emory University in Atlanta.

I asked Associate Professor Leigh Turner of the University of Minnesota Center for Bioethics about this case and the lack of disclosure:

Transparency is important, but it appears that the company is being “transparent” about dramatic improvements in health of individuals receiving Pluristem’s PLX Cells while failing to issue press release disclosing case of a child who died after undergoing this experimental intervention.

Dr. Turner is interested in and studies ethical issues related to clinical use of stem cells. I asked him to comment on the “saved a child’s life” press release by Pluristem:

Such press releases risk misleading investors by creating overly optimistic account of scientific research. More importantly, press releases describing miracles and lifesaving cures are harmful because they give seriously ill individuals an unrealistic account of effectiveness of experimental stem cell interventions. Instead of being provided with a balanced, accurate account of risks and benefits generated from meaningful clinical trial data they risk being persuaded that a company has developed an effective intervention when in fact research is at a very preliminary, inconclusive stage. I worry that science-by-press-release betrays vulnerable individuals by offering hope that is not justified. Many companies avoid this problem by reporting findings at the conclusion of clinical studies instead of issuing breathless press releases based upon short-term results in individual research subjects.

I agree with Turner that science-by-press-release is dangerous for biotech companies, their investors, and their patients on many levels. Compounding that dangerous complexity further are stock sales by company leadership that occur not so long after press releases.

Turner also has a broader take on this case that I believe is very interesting and helpful:

There are some valuable lessons here both for reporters as well as for consumers of news coverage of stem cell research and other areas of science that are routinely overhyped. Whose interests are served when press releases are issued? Is there something significant about timing of press releases? Are independent voices included in news coverage or do all quoted individuals have vested interests in promoting positive account of study results? Why is a press release being issued if results are based upon a single research participant or merely a few research subjects? And finally, what financial interests are swirling beneath the language of “miracles”, “cures”, and “lifesaving” interventions? These are questions reporters should ask and they are also questions those of us who read and otherwise consume the news should consider whenever we are confronted with dramatic claims floated on anecdotes, testimonials, and research “findings” based on meaningless sample sizes.     

Part of what is so disappointing about this case to me is that the PLX therapy might have real promise as a drug, promise that has been at least temporarily clouded by actions by the company. I hope the case does indeed provide lessons to all interested parties including other biotech companies working on stem cell treatments. I also hope that PLX goes on to be shown to be successful as a safe, effective treatment in clinical trials, a path that would be facilitated in my opinion moving forward by the company avoiding even the appearance of ethical issues.