Boston's biotech community leads wave of stem cell consolidation, deal making

Boston Business Journal, Ron Leuty, July 8, 2009

Today at the ISSCR Meeting in Barcelona, the merger of two stem cell companies, IZumi Bio and Pierian Inc was announced, with the new company being named IPierian. According to the new company's website iPierian is

"...a pioneering biopharmaceutical company that is taking the cutting-edge technologies of cellular reprogramming and directed differentiation to an entirely new level to harness the power of induced pluripotent stem cells to advance the understanding of human diseases and accelerate the discovery of more effective therapeutics for patients"

The two precursor companies, iZumi and Pierian, both had synergistic skills in the area of inducible pluripotent stem cells (iPS), a type of "artificial stem cell" that is created from skin or other tissues. The use of iPS cells for therapeutics development is more attractive to scientists than embryonic stem cells for several reasons. Firstly, iPS cells can be generated to be patient-specific, thus overcoming problems with need for taking of immune suppressants. Currently embryonic stem cells can not be used in patients for several reasons, and the few times that their use is contemplated, the patient is sentenced to taking life-long immune suppression so that they do not undergo rejection. Secondly, iPS cells can be generated under highly defined conditions. Embryonic stem cells that are currently used have been developed years ago and face various problems such as the fact that many of them have previously been grown on mouse cells or using animal products. In contrast, iPS cells can be generated with relatively little effort.

iZumi was supported by the venture capital groups Kleiner Perkins Caufield & Byers and LExington, Mass.-based Highland Capital Partners with a $20 million investment, whereas Pierian was founded by MPM Capital managing directors Ashley Dombkowski and Robert Millman as well as Harvard University scientists. The new company, which will be led by John Walker as CEO and Corey Goodman, as Chairman, raised an additional $10 million from Boston-based MPM Capital and $1.5 million from FinTech Capital Partners.

Initial goals of the company will be use of the iPS cells to address disease affecting the central nervous system that have no effective treatment such as spinal muscular atrophy, Parkinson's Disease, and ALS. In the long-run the company plans to investigate conditions such as heart failure, liver failure, and diabetes. As part of the new company's strategy, it will seek synergistic collaborations with established market players.

Despite the aggressive goals the company has set for itself, there are several drawbacks that one must consider. Firstly, pluripotent stem cells, regardless of whether they are iPS or embryonic stem cells, all cause cancer when administered into animals. iPS may be especially dangerous since oncogenes (genes that cause cancer) are needed for the creation of these cells. In order for iPS to be used safely, it will be necessary to make sure that the cells being made for injection are completely the cells that one wants, and no contamination with the original iPS cells. In other words, if one is treating Parkinson's Disease, one can not simply inject iPS cells into the area of the brain that is damaged, since this conceptually will form a tumor. In contrast, one would have to "teach" the iPS cells to become the specific cell that is damaged in Parkinson's Disease, called the "dopaminergic neuron", one will have to concentrate these cells outside of the body, and then inject them directly where they are needed. Once the cells are injected, they will have to form connections with the existing cells and subsequently integrate and take over their function. This is in contrast to the present-day clinically available adult stem cell therapies, where in many cases adult stem cells are injected either intraviously or intrathecally, and the natural signals of the body instruct them to differentiate into the needed tissue. Although differentiation efficacy of adult stem cells may be lower on a per cell basis, of the thousands of people that have been treated with adult stem cells no reports of tumor formation exist.

iPierian's scientific leadership comes from the respected embryonic stem cell experts Dr. George Daley, Douglas Melton and Lee Rubin who are faculty at Harvard. The scientific advisory board (SAB) of the company will be chaired by Dr. George Daley, and will include Amy Wagers, Kevin Eggan, Benoit Bruneau, and Matthias Hebrok.