Cellular Purity: Key to the Stem Cell Race

“Rare is the union of beauty and purity”

The confluence of supply and demand is the engine of commerce. Today, the aging U.S. baby boom population is creating one of the fastest growing sectors for new product demand in our history. This 76 million-person strong segment of our population is facing a surge of degenerative diseases, many having no known cure. Examples would be Parkinson’s disease, osteoarthritis, heart failure, macular degeneration, and so on. On the supply side of the equation is the emerging field called “regenerative medicine.” Regenerative medicine was a term coined to refer to medicine’s new-found ability to manufacture any cell type in the human body, based on embryonic stem (ES) cells. Normally, the confluence of such powerful economic forces would generate enormous new industries to connect the tides of supply and demand. And yet, in the United States today, the industry of regenerative medicine is still in its infancy, and we are hearing about the difficulties some companies face in commercializing the new products. This leads us to ask, “Where are the bottlenecks, and how will industry rise to the occasion to deliver on these desperately-needed new products?”

The problems facing our nation in regard to an aging population and the rising national health care bill have received high visibility in the media. Less well known, perhaps, is the daily struggle behind the scenes, as scientists seek to bring the promise of regenerative medicine to the marketplace. As we have said, ES cells, and their related cells called induced pluripotent stem (iPS) cells, have the impressive ability to become all the cell types in the human body. What is not as well appreciated is that this protean power resident in the cells is also an enormous hurdle for people working in biotechnology who wish to actually produce these products on an industrial scale. The challenge is one of purity. How do we consistently manufacture only the cell type of interest when there are hundreds of cell types in the body? If we do not solve these technical challenges, preclinical development costs can rise into the hundreds of millions of dollars, squelching product development.

These difficulties in the first decade of regenerative medicine will likely be addressed in the second decade by new methods to completely isolate and scale up defined lineages from ES or iPS cells. BioTime is using a proprietary approach we call ACTCellerate™ which has already resulted in the isolation of >200 different cell types of the human body. The use of these purified cell types is expected to simplify the manufacturing process and ease the concern of regulators over product safety. BioTime’s announcement of a partnership with GeneCards is the beginning of our effort to steer regenerative medicine into a new era wherein many new human cell types can be manufactured to scale at an unprecedented level of purity and identity. This new generation of manufacturing technologies, or as we say Manufacturing 2.0, is expected to simplify product development and speed the transfer of supply to demand.

But for the research scientist, accessing for the first time the purified cellular building blocks of the human body, and being able to map out their gene expression profiles, is as much an appreciation of the beauty of human development as it is a quest for cures and products. Seeing for the first time in the laboratory dish the cellular components of the human body, and being able to map out the genes that cause the cells to weave themselves into human tissues, gives the bench scientist a vision of what life could be, how medicine could fashion new life-saving therapies. Our nation cannot afford to lag behind in the commercialization of regenerative medicine in such a critical time in our nation’s history. Never before have we faced such opportunity in medical research, and never before has it mattered so much for so many people.