Research Ethics Blog

Human Stem Cell Trial Approved in U.S.

From the Washington Post: First patient treated in stem cell study*

The first patient has been treated with human embryonic stem cells in the first study authorized by the Food and Drug Administration to test the controversial therapy.

A patient who was partially paralyzed by a spinal cord injury had millions of embryonic stem cells injected into the site of the damage, according to an announcement early Monday by the Geron Corp. of Menlo Park, Calif., which is sponsoring the groundbreaking study….

This Reuters story gives additional detail: First patient treated in Geron stem cell trial), including the fact that this is a Phase I trial, which will “not be aiming to cure patients but to establish that the cells are safe to use.”

An obvious question: what prior evidence justifies subjecting human subjects to such a clinical trial? As we’ve noted here before, the ethical standard for enrolling patients in a trial is provided by the notion of clinical equipoise — basically there has to be some legitimate disagreement in the expert community about the best treatment for the ailment in question. In this case, given that spinal injuries are generally not very treatable at all, what we’re looking for is some evidence that the embryonic stem-cell based treatment might well work. In that regard, an article in WebMD notes that:

In preclinical studies, animals with severe spinal injuries regained the ability to walk after treatment with OPC1 cells. The animals did not develop teratomas, reject the cells, or suffer nerve pain.

In addition to showing clinical improvement, treated animals’ damaged nerves became coated with new myelin and there was new nerve growth in the vicinity of the injected cells.

FYI, here’s Geron’s page about the trial: Phase I Trial of GRNOPC1

Interestingly (and sadly), of the handful of news reports I’ve seen so far, none of them has specifically mentioned the role of ethics review boards.

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*Note: the headline of the WaPo story would be more accurate if it specified embryonic stem cells.