Testing iPS cells in people? Not yet

This past week a story unfolded that ended with the dismissal of a researcher from the University of Tokyo who had claimed to be the first to test a therapy based on reprogrammed iPS cells in humans.

It all began last week when CIRM grantee and UC Davis scientist Paul Knoepfler blogged about research being reported by Hisashi Moriguchi. He chronicled the story on his website. New Scientist describes the situation:

In a poster presented at a meeting of the New York Stem Cell Foundation, Moriguchi – who claimed to work at Harvard Medical School and the University of Tokyo – described results from a trial in which cardiac muscle cells were grown from induced pluripotent stem (iPS) cells, and transplanted into six US patients with severe heart failure.

… This was surprising, given the safety concerns that surround iPS cells – adult cells that have been reprogrammed to an embryonic state. Support for the claim quickly disintegrated: within hours, Harvard released a statement noting that Moriguchi had no current affiliation with the university, nor any ethical approval to run a clinical trial.

The supposed results got a lot of attention at the time because Shinya Yamanaka had just shared the Nobel Prize for his role in discovering how to make those very iPS cells that Moriguchi was claiming to have tested. Yamanaka works part time at the CIRM-funded Gladstone Institutes.

The problem is that he never did inject those patients. This is a big deal in part because CIRM and other organizations have been very critical of clinics claiming to be offering stem cell “therapies” based on no clinical trial data (here’s more information about stem cell tourism). In the U.S., new therapy ideas have to be tested in trials approved by the Food and Drug Administration, and those trials also have to be approved by an Institutional Review Board at the institution participating in the trial. In this case, Harvard claimed that their IRB had never approved the research.

CIRM requires IRB approval for any clinical trial that involves our funds, just like we require institutional animal review board and stem cell review board approval for animal research or research involving human embryonic stem cells. We get those assurances before we issue funding and in our yearly progress reports from grantees.

These steps – institutional reviews, Food and Drug Administration reviews, controlled clinical trials – do take time, but they also ensure that once a therapy reaches patients it is likely to be safe. While we would all love to find a cure for any number of diseases tomorrow the hard fact is that good science takes time, there are no short cuts. In science as in life, if something seems too good to be true, it usually is.

A.A.

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