Upbeat prospects for some California clinical trial efforts from CIRM

Over at the California Stem Cell Report, David Jensen is reporting on some good news from CIRM for California on the stem cell clinical trial front.

Stem cell biotechs Asterias and Capricor have stem cell trials supported by 20+ million in CIRM funding each and have been hitting milestones. These trials are progressing and so far have good safety profiles. Asterias and CIRM have mentioned some possibly encouraging early hints at efficacy as well in its trial, and apparently there are hopeful hints from the Capricor trial too.

See the posts from CIRM here (a weekly summary kind of post that begins discussing Asterias) and here. For background, also see past posts I’ve done on both companies here and here in the archives, and see especially my interview with Asterias leadership from a few months back.

It’s early days for these trials and at these phases they are not really designed to look for efficacy so a conservative approach to discussing such trials is in order given the stage, but at this phase of the game for early clinical trials the news has been all one could hope for so far in both cases.


Asterias cells

The Asterias and Capricor trials are for spinal cord injury and Duchenne muscular dystrophy, respectively. The latter trial utilizes the Capricor CAP-1002 product, which is a cool allogeneic cardiosphere technology made from donor human heart tissue. A beating cardiosphere from a different source (IPSCs) can be seen in the video above. Asterias’ trial employs their OPC product made from hESCs, which is also inherently allogeneic. The idea of potentially repairing the injured spine via stem cells is intriguing.

I’m hoping in the next month or so to do a broader update on the stem cell and regenerative medicine biotech arena. By way of disclosure, I do not have any financial stake in either company discussed here.

TGIF weekend reads: stem cells, GMOs, #CRISPR, cats, cloning and more

Sounds recommended weekend reads for you to enjoy since it is Friday and almost the weekend.

Holy GMO, China wants to buy Syngenta.

cat stem cellsRetractionWatch says that Macchiarini will soon be out at Karolinska. For more on this “super surgeon” who used stem cells, see here regarding recent Vanity Fair Expose.

David Jensen on CIRM and human genetic modification: no germline work funded now, but possible in the future.

This is pretty awesome. Canadian ‘dream team’ to probe stem-cell link to brain cancer

If you mix cats and stem cells, it’s got to be good, right? Cat stem cell therapy gives humans hope

And now onto mice. Suicide of aging cells prolongs life span in mice

Human gene editing. For the first time in history, a government has approved genetically editing human embryos

And for laughs…

Mix GQ magazine and a cloning conspiracy = good for a laugh. Forget the ‘Flat Earth’ Stuff, B.O.B Thinks Celebrities Are BeingCloned

TGIF links to tip-top weekend science reading

Some stuff to read, think about, and do.

Weekend science reading: fantastic found links


Glioblastoma, image from Wikipedia

David Jensen reports on the future of CIRM as a “beautiful machine”.

CIRM funds brain cancer research in a big way: Funding a clinical trial for deadly cancer is a no brainer. Glioblastoma is a fatal tumor that needs new clinical approaches so this is exciting. One of the goals of my lab is to find new pathways to treat glioblastoma in children.

Jacob Corn of IGI has a Wish List for Science on CRISPR science, but also science more generally.

The case for possibly retracting a retraction over at RetractionWatch.

Take another look at last year’s Takaho Endo paper, which was a major step toward debunking STAP so why did the new paper in Nature debunking STAP using similar approach not cite Dr. Endo’s 2014 paper? I’m glad to see that the other new Nature paper did cite Dr. Endo’s paper.

Pete Shanks over at BioPolitical Times from CGI voices concerns about balance at upcoming NAS meeting on human gene editing.

Zhang’s lab reports on Cpf1, a useable new CRISPR partner protein in place of Cas9 and Antonio Regalado has the backstory: New CRISPR Protein Slices through Patent Problems

The Node on Enabling research with human embryonic and fetal tissue resources

The tiny fingers that touch stem cells from David Kent on Signals Blog

Caplan Article on Bogus Stem Cell Research: Some Different Views

When bioethicist Arthur Caplan talks about stem cells, people pay close attention and for good reason.

Art Caplan

Caplan has provided important perspectives on the stem cell field over the years. For example, you can see a guest post on this blog here about human cloning.

However, in my post today I respectfully discuss how I disagree with several parts of this week’s piece by Caplan on why there are allegedly so many ethical problems in the stem cell research field.

Caplan’s article (see screenshot from video at right) is focused on a question articulated by the title:

Why so much Fake, Unduplicable Stem Cell Research?

One might start off the bat by challenging the article’s title and intrinsic question above, since in reality that the vast majority of stem cell research is quite real and replicable.

David Jensen over at California Stem Cell Report, writing about Caplan’s article, pointed out that serious research issues are not unique to the stem cell field as, for example, there have been disastrous issues in the cancer field too:

There is no doubt some spectacular fraud has surfaced in stem cell research. But the problem of replication within stem cell research may not be entirely out of line with problems elsewhere in science. Los Angeles Times columnist Michael Hiltzik wrote last fall about a study by Amgen that examined 53 “landmark papers” in cancer research and blood biology. Only five could be proved valid, a shocking result, according to Amgen. Similar results were turned up byBayer in Germany, Hiltzik said.

On the other hand, there have been some truly terrible stem cell research fiascos of late so let’s focus on Caplan’s reasoning for why the stem cell field has had these problematic events.

Caplan begins by talking about the STAP cell fiasco in Japan involving allegedly faked research reported in Nature that powerful stem cells could be made by simple stressors such as low pH.

Caplan writes that the researcher in question, Haruko Obokata, “confessed that she had made it up.” In fact, quite the opposite is true. Dr. Obokata says that her study is correct overall and that she did not make it up. To my knowledge, she only admitted to careless errors arising innocently from lack of experience that she says do not affect the conclusions of her papers.

Getting back to the central question of why stem cell research sometimes runs into ethical problems Caplan argues that lack of funding is one reason for the problems. I wish there was indeed more stem cell research funding, but I do not believe this is a clear reason for ethical problems in the field. I don’t see this playing a significant role and funding woes certainly aren’t specific to the stem cell field.

A second reason given for trouble is what one might call the “stem cell hero temptation” effect. In other words, breakthroughs in stem cells might gain a researcher the world’s attention  (“being a hero to the world” is how Caplan describes the attraction ) so there may be perceived incentive to fudge or outright fake stuff. From some of the cases we’ve seen in recent years, this reason seems accurate.

An additional Caplan assertion for explaining the ethical issues facing the stem cell field can be boiled down to a lack of people to provide oversight. Caplan writes:

Another major problem in the stem cell field is that the number of people doing research in this area has shrunk…That may mean that there are fewer people to watch one another.

I’d be interested to see if he has any data to back up this claim. In fact, my sense is the opposite about the size of the stem cell field. If anything the number of people working on stem cells seems to continue to grow overall. I do not believe that the stem cell field lacks sufficient people power to adequately review itself.

So if I disagree with two out of three of Caplan’s reasons, why then do I think that there are sometimes ethical challenges in the stem cell field such as the STAP cell problem?

First, let me say again that he’s right about a few unwise research folks chasing international fame at any cost.

However, another issue here is that a heck of a lot more people around the globe are paying attention to the stem cell field. As a result, ethical problems that are also present in other fields of science (e.g. image manipulation, non-reproducible papers, etc) get noticed far more if they are in stem cell papers. To sum it up, there are more eyes on stem cell papers looking for troubles after publication.

As I blogged before, I also believe that in the specific STAP case, the reviewers and probably editors too were unduly positively biased by the addition of some stem cell big wigs to the authors list on the STAP Nature papers. This points to another contributing problem to broader problems in the field: a small number of stem cell bigwigs have way too much power as reviewers. In other words, journal editors rely on too few eyes to review the highest profile manuscripts. Big journals and their editors need to diversify their stem cell reviewer lists and the review process needs to be more about data and less about names.

In the end the stem cell field is likely to continue to run into a few bumps and even land mines as it proceeds. Addressing recurring problems in an open, expeditious manner would be wise. Training in ethics for researchers seems to be in need of a boost. The journal review process also is a logical place to focus. Is it naive to hope that Nature might take the lead on reform of the review process?