Translation of stem cell research – great expectation met with some frustrations

by Alexey Bersenev on April 20, 2009 · 2 comments

in under discussion

 

Over the past decade there has been increasing pressure put on biomedical researchers to justify their projects by establishing short term time lines to produce technological or clinical applications. The drive for applicable or “translational” research is in many ways perfectly reasonable. For publicly funded research there are limited amounts of tax dollars to go around, and the public is looking for a return on their investment.

I cited Kevin Graham – author of “Connecting for kids” blog here, because his thoughts perfectly reflect what I’ve been wanting to discuss for a while. I’d like to talk about translation of stem cell research.

Translational research is no doubt extremely satisfying, seeing an idea progress from the lab bench to a patient or market place is a very tangible way of evaluating your success. Another contributing factor is that in order to highlight the importance of research to society research is almost always framed in terms of the human health issues that it might solve. As stem cell researchers we are certainly guilty of this.

Definitely the point of stem research is not only for the satisfaction of scientific interest, but mostly – translation. It should be translated to clinic or business. Translation is the hardest part on the way of justification of public demands. It should be “worth investment”. This part frequently fails, especially in embryonic stem cell (ESC) research. Just count the number of times we saw that technique perfectly working in mice but not in human. Recently I’ve been reading some interesting commentaries and articles about embryonic stem cell research translation failure. Let’s look at some of them:

Some frustrations from an expert’s mouth about huge money investment in stem cell research in California (San Francisco Weekly):

Almost five years after Californians approved Prop. 71, the field of stem-cell research is at a turning point. Many scientists like Kriegstein say their understanding of stem cells has not advanced sufficiently to benefit the severe degenerative diseases for which the field once held such promise, or to administer safe tests of therapies for those ailments in humans.

more from “Stem-cell stalemate: The push for cures may produce only disappointment – or worse

“I think we wish that they had found the cure yesterday or the day before,” Brown says. “It’s really hard after 12 years of being told, ‘Well, if we get enough financing, we’ll be able to have a cure in five years.’ You don’t know what is real and what is wishful at this point.”

One more from Kevin Graham – I’m curious. Are you?

Over this time we have come to realize that great medical and technological achievements springboard off of years of basic research, research that is often done with no awareness or indication of the payoff that will follow in the years to come.

citation from article: The ethos and ethics of translational research

The enthusiasm for stem cell research is an excellent case of the risks and potential benefits of endorsing a translational model.
We argue that contemporary stem cell research is being shaped by the translational ethos precisely because the research is developing at the same time as the demand for results. There is no longer the possibility of “pure” or “curiosity-driven” exploration of stem cell science because of the nature of nonfederal government funding accompanied by exalted expectations for results.

So we have to admit that the last ten years of stem cell research, since the first isolation of human embryonic stem cells (ESC), didn’t bring this technology to regenerative medicine and didn’t justify public and medical expectations. But do you think ten years is enough to make critical conclusions? Do we need more time for research or should we reduce the ESC research budget and focus on other types of cells for regenerative medicine?

I think the frustrations about clinical potential of embryonic stem cell research, which were perfectly shaped by the “translational model”, left some important messages for us. It’s not that easy as we (scientists) and public thought and probably we should reduce scale of research in terms of clinical translation in favor of adult stem cells and ESC-based business models.



Another side of “expectations-met-frustrations” is translation to a business. Adult stem cell research has much better clinical translation compared to ESC, but it still struggles from problems of commercialization and lack of profitability.

citations:
from commentary to blog post: Stem cells and the politics of the ethics of the few:

This research to create such a wonderful stem cell from ESCs by the sequenced cytokine soup method is going to eventually work, no doubt. However, it is going to cost huge amounts of money. This cost will eventually be passed on to consumers, as are all medical development costs.

Todd McAllister about cell-based medicine industry failure in the article: Cell-based therapeutics from an economic perspective: primed for a commercial success or a research sinkhole?

Despite widespread hype and significant investment through the late 1980s and 1990s, cell-based therapeutics have largely failed from both a clinical and financial perspective. While the early pioneers were able to create clinically efficacious products, small margins coupled with small initial indications made it impossible to produce a reasonable return on the huge initial investments that had been made to support widespread research activities.

Dr. Paul Martin from the report about failure of commercialization and adoption of stem cell therapy in UK:

Although cell therapy is now established as an important branch of medicine, innovative firms struggle to make money, putting the UK industry in a very vulnerable position in the short term. Unless the situation changes the industry will contract and the progress needed to develop important cell therapies will be adversely affected.”
… However, the sector suffers from a high level of company turn over. As a consequence, the industry is dominated by small, young companies lacking the resources to bring products easily and successfully to market and those that do struggle to make sales.

What is your opinion about translation of stem cell research? Has it really progressed forward in the last few years? Is the embryonic stem cell research worth the investment or waste of money and fooling of public expectations? How long should we do research and what are the criteria to conclude about applicability of technology in clinic or industry? And what is the future?

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{ 2 comments… read them below or add one }

Chris April 20, 2009 at 8:19 pm

It’s premature to call the California experiment with stem cell research a failure for at least four reasons.

1) The institute charged with giving out the money, CIRM, fought a lawsuit brought by religious conservatives for two years. The first grants (training grants, not research grants) were issued in 2007. So we’ve only had a year or so of the ten year project underway.

2) CIRM funds all types of stem cell research, not just embryonic. Many of the last round grants were for iPS cells.

3) CIRM did something the NIH would never do: fund translational research to the tune of $500-$700m. These are proof of concept and early phase clinical projects in the “valley of death” where funding is hard to come by.

4) The field is only 10 years old! True, many people overhyped what stem cells can do, but the progress, scientifically speaking, has been very rapid.

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Alex April 21, 2009 at 12:26 pm

to Chris –
thank you for clarification about CIRM.
I agree that field developed very rapidly and asked whether 10 years enough or not to expect results from clinical translation. Adult stem cell translation doing much better actually and have some examples of 5-7 years way from mouse to trial phase II-III. That’s even better then we can see in other fields of biotech/pharma.

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