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From: Steven Victor MD eee Sent: 11/23/2012 1:06:38 PM To: cc: Subject: Report on Stem Cells future PUBLISHED: 22 NOVEMBER 2012 The view from the US: Stem cell therapy steps up a gear with first approval and improved political climate Special Report Peter Winter Positive clinical data, increases in federal funding and the first regulatory approval of a manufactured stem cell product mean momentum is building in the US. With the re-election of President Obama, this is likely to continue Share on twitterShare on linkedinShare on facebookShare on email Image: Bigstock When US President Barack Obama came into office he made good on his promise to overturn President George W. Bush’s executive order that - with the exception of a handful of existing stem cell lines - prohibited federal funding of human embryonic stem cell (hESC) research. Special Report - Stem Cell Research under Horizon 2020 Stem Cell Research in Horizon 2020: Experts debate what Europe must do to maintain its lead in regenerative medicine The view from the US: Stem cell therapy steps up a gear with first approval and improved political climate View from Europe: Delivering on the vision of regenerative medicine and stem cells Talking to the experts: Why the EU should support human embryonic stem cell research in Horizon 2020 OVERVIEW: Regenerate the future - human embryonic stem cell research is crucial to deliver health and growth This easing of restrictions on hESC research was expected to engender enough confidence to attract investors into the space and encourage pharmaceutical and biotech companies to build robust product pipelines based on stem cell therapies. However, a tougher regulatory climate for biopharmaceuticals in general and a protracted legal challenge to the relaxing of rules on hESCs has served to keep both big pharma companies and venture capitalists on the sidelines to date. Nevertheless, there has been progress, particularly at the research end of the development spectrum. Three and a half years on stem cells are no longer high on the political agenda as they once were. This speaks to the generally positive public acceptance of stem cell research in the US. A Research!America poll of likely voters in the presidential election, conducted in August 2012, found that 61 per cent of prospective voters were in favor of expanding funding for hESC research. Investor interest Looking back over his first term in office Obama can point to some major gains. These include funding increases for the National Institutes of Health’s (NIH) stem cell initiatives, and greater investor interest in the wider field of regenerative medicine including the use of adult stem cells and induced pluripotent stem (iPS) cells. However, it hasn’t all been smooth sailing. Although after protracted forethought, the US Food and Drug Administration (FDA) gave the green light for the Californian biotech Geron to begin the very first human clinical trial of a human embryonic stem cell-derived therapy in January 2009, the trial has not been without its problems. The Phase | trial, treating patients with acute spinal cord injury, was hit with an FDA clinical hold causing significant delays to its initiation in 2010. A year later, after 15 years’ effort, the Geron washed its hands of the programme, blaming its withdrawal from the stem cell space on capital scarcity and uncertain economic conditions. The decision wiped out a leading player in hESC translation and commercialisation. However, the regenerative medicine sector has been able to recover and is beginning to blossom. (See Public Markets section below). The prime movers in the Geron trial are now attempting to revive it. Research funding robust Federal funding for all forms of stem cell research has increased over the past four years. However, the NIH funding component for hESC has been dogged by litigation for the past three years. In Sherley v. Sebelius, researchers James Sherley and Theresa Deisher, who worked with adult stem cells, claimed the NIH guidelines violated the Dickey-Wicker Amendment, which prohibits the use of federal funds for research in which human embryos are destroyed or discarded. This overhang was not finally removed until August 2012 when a three-judge panel from the US Court of Appeals for the District of Columbia Circuit unanimously upheld the NIH 2009 guidelines that permit funding of hESC research. HOUSE_OVERSIGHT_013482