Will stem cell trials replay gene therapy's troubled past?

Remember Jesse Gelsinger? The name of the teenager, who died in a University of Pennsylvania gene therapy study in 1999, has become almost synonymous with "unsafe clinical trial." In a policy piece in last week's Science, James Wilson, who led the trial in which Gelsinger was a subject, cautions that some of the exuberance and haste which led gene therapy researchers to human trials too early is now afflicting the stem cell field.

"…I applaud President Obama for giving scientists longer leashes as they explore this exciting field. But in today's clamor of stem cell enthusiasm it is possible to detect haunting echoes of the early and ultimately troubled days of gene therapy," he writes.

The obvious implication of the piece is that stem cell research isn't ready for clinical trials. But are the two scenarios comparable? Or are these just the overcautious words of a man once burned?

-Alla Katsnelson, news editor, The Scientist

Not just Gelsinger has died or become extremely ill. Despite setbacks, gene therapy trial review procedures have not improved, they have instead gotten demonstrably worse.

Jolee Mohr died in 2007, from a trial that was even more rushed than Gelsinger's. The trial that resulted in Jolee Mohr's death did not even run through primate models first. (And primate models are imperfect.) That trial did not get a proper preliminary review from immunologists.

I think the concerns voiced by Wilson are extremely important.

Stem cells have the potential to create tumors as well as heal. We know that. So I think that proceeding forward, but with extreme caution, is wise. We need to do long-term studies on primates before moving to people. Or, if we do work with people, we need to make sure that they are very clear about the quite real risks.

The problem is not that a clinical trial is safe or "unsafe," but that research has risks. Gelsinger painful lesson for us was not "research (into new areas) is not safe." Research is NOT safe. Gelsinger was history repeating itself, showing the lessons we have not learned, such as research has risks, research may have unforeseeable risks, participants deserve to know those risks, and so on. There are many other examples, and there are sure to be others in the future. I think the issue is whether the possible benefit from stem cell research are worth the risks.

Wilson's policy forum piece in the May 8 Science discusses how the

hypereaccelerated translation to the clinic that occurred in the field of genetic therapy in the 1990s was driven by multiple factors

, including

unbridled enthusiasm of some scientists in the field.

Wilson had to have been one of those. He and his team's glaring lack of understanding of the basic physiology of the reticuloendothelial system must have contributed to Mr. Gelsinger's death.

While his warning to those in the field is important, it flys in the face of historical reality. The figure in the position piece shows that publications and animal studies have long preceded the very first clinical trials. The recent Experimental Biology 2009 meeting in New Orleans contained many posters and symposia with only non-primate pre-clinical studies.

Wilson is also preaching to the choir. Reputable scientists in the field have not rushed into anything because they know that what they can only working with, what the Bush administration allowed them to work with, is pretty meager.

After hearing the same, constant drone from the media of how stem cell research will help with Parkinson's, Lou Gehrig's, diabetes, heart disease, etc. for years now, the general public must be wondering where the cures are. Unfortunately, the new embryonic stem cell guidelines that the current Congress is reviewing are not much better than the previous administration's, so drone on media, drone on.