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Stem cell vet ACT aims for pioneering human trial of iPS-cell-derived therapy

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This year scientists shared a Nobel Prize for their part in reprogramming adult cells into a stemlike state, a major advance for stem cell science. Now Advanced Cell Technology (ACT) wants to take the science a step further, seeking approval to trial platelets derived from reprogrammed adult cells in humans.

The Wall Street Journal cites experts who say that it could be the first human study using induced pluripotent stem (iPS) cells to make the test therapy. With hopes to get the green light for the iPS-cell-derived platelet study by next year, ACT ($ACTC) seems to be moving toward trials of such therapies faster than some anticipated. The problem with using the cells as therapies has been that methods needed to reprogram adult cells to a pluripotent state could introduce genetic abnormalities that could be dangerous to patients.

As the WSJ points out, however, platelets don't carry DNA and therefore the therapy from ACT could overcome that hurdle. The Marlborough, MA-based biotech, which has explored numerous approaches over the years to advance stem cell therapies, hypothesizes that patients who are treated with platelets made from their own reprogrammed cells could be treated without the risk of resistance to platelets from donors that occurs over time. Clot-forming platelet therapy aids patients with anemia and leukemia.

"You have to manipulate the cells quite a bit, so I think you'll have to overcome the safety concerns of the FDA and other regulatory bodies," James Thomson of Morgridge Institute for Research in Madison, WI, told the WSJ.

Induced pluripotent stem cells provide supplies of stemlike cells that do not require the destruction of embryos, a major perk for researchers seeking to advance stem cell science without the weight of ethical objections to embryonic stem cells to impede their progress. Yet many pharma companies have just begun to warm up to using iPS cells as tools to test drugs and in preclinical studies of diseases.

ACT clearly wants to leap ahead with human therapies based on the young stem-cell science.

- get more from the WSJ article (sub. req.)

Special Report: Stem cell research advances in fits and starts

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