Advanced Cell Technology

Advanced Cell Technology (ACT) is a biotechnology company that specializes in developing cellular therapies to treat human diseases and was one of the first companies to run an FDA-approved clinical trial based on embryonic stem cells. The corporate offices and principal laboratory for ACT are located in Marlborough, Massachusetts, where the company is headed by Ted Myles (interim [Page 26]president, chief financial officer, and executive vice president of Corporate Development) who began at ACT in June 2013, and Robert Lanza, MD (chief scientific officer), who began working at ACT in 1999. Lanza is also an adjunct professor at the Wake Forest University School of Medicine in the Institute of Regenerative Medicine. ACT owns or licenses over 150 patents, including a broad patent for producing retinal pigment epithelial (RPE) cells for degenerative retinal disease.

ACT began working in animal cloning in the mid-1990s, but shifted its research focus to human cells in 1998, shortly after the first isolation of human embryonic stem cells (hESC). Michael West became ACT’s chief executive in 1998, and in 1999 bought the company. West recruited Robert Lanza to ACT, and the company focused on therapeutic cloning, a method of developing stem cell lines from a patient’s own cell, thus avoiding the potential for rejection of cells created from unrelated embryos.

In 2001, ACT scientists published a paper in the online journal Biomed describing a method for cloning a human embryo. Thereafter, West appeared on the television program Meet the Press to discuss the paper, bringing publicity to the company. However, according to Corie Lok in a 2012 article in Nature, that announcement may have backfired because the ACT scientists’ accomplishments were not considered significant by the scientific community (in part because the embryo they produced stopped dividing far short of what would be required to derive stem cells), but did trigger a public and political reaction that confused hESC research and cloning.

In order to raise funds, ACT merged in 2005 with the Utah-based company Two Moons Kachina and became a publicly traded firm. The company expanded and, in August 2006, Lanza and colleagues announced in Nature that they had developed a method to derive stem cells from a single cell removed from an embryo. This method could potentially allow stem cells to be produced without harming the embryo, but the embryos used in the reported study were destroyed in the process. According to Lok, this distinction was not made clear in the study or in press releases regarding the study and many news outlets reported that the company had developed a method to derive stem cells without destroying an embryo. If this claim had been true, ACT’s process would avoid some of the ethical concerns surrounding stem cell research. When it became clear that ACT did not have a method for deriving stem cells without destroying the embryo, it hurt the company’s reputation and made it difficult to attract investors.

ACT’s current research and development process has four main focuses, two of which are based on hESC. The first hESC-based therapy, RPE therapy, is intended to treat degenerative retinal disease. RPE therapy is currently undergoing Phase I/II clinical trials for adults (over 18 years) in the United States and the European Union as a treatment for Stargardt’s Macular Dystrophy and atrophic (dry) AMD (age-related macular degeneration). This phase of testing is primarily to determine the safety and toleration of the treatment, which involves the transplantation of hESC-derived RPE cells. The second hESC-based therapy in development is the hemangioblast platform for treatment of diseases and disorders of the circulatory and vascular system; this therapy, which is currently in the preclinical phase, is being developed by ACT in partnership with the Korean company CHA Biotech.

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