One of the main goals of California’s $3 billion stem cell research agency is to draw companies into the state so they can vie for a share of the funding.
With a recently funded $32 million initiative, the California Institute for Regenerative Medicine (CIRM) has attracted two of the biggest US players in stem cell banking to Novato, CA, to form one of the largest biobanks of induced pluripotent stem cells (iPS cells) in the world.
Cellular Dynamics International (CDI) of Madison, WI, co-founded by stem cell pioneer James Thomson, has already established a fast-growing business by selling induced pluripotent stem cells—and specialized cells derived from them such as neurons—to big pharmaceutical companies and other research customers. CDI, which recently filed a registration statement with the SEC for an initial public offering, plans to use part of the $57.2 million it hopes to raise from investors to build out a lab for the CIRM-funded iPS cell biobank.
Like embryonic stem cells, induced pluripotent stem cells can be coaxed into forming any of the specialized cells of the body. But iPS cells, which can be used for drug assays, among other purposes can be made from a skin or blood sample from any individual. This bypasses the ethical and practical constraints of deriving embryonic stem cells from very early stage human embryos, which are destroyed in the process.
At its new California outpost on the campus of the Buck Institute for Research on Aging in Novato, CDI will create iPS cells from 3,000 individuals under a $16 million grant from CIRM. Its partner, the non-profit Coriell Institute for Medical Research of Camden, NJ, will manage the repository of cells and distribute them to investigators worldwide. Coriell, which was awarded a $10 million grant from CIRM, already operates a bank of 75 iPS cell lines created in-house and by outside researchers. The balance of the $32 million will go to academic centers that will recruit donors of the tissues used to create the iPS cells.
While CIRM also supports startup companies that are trying to use stem cells to repair damaged hearts or nerves, its iPS biobank will mainly be a boost to disease investigators rather than a source of therapeutic cells.
“I look at it as opening up the human body for research,” says Coriell CEO Michael Christman. The CIRM cells will be shared with researchers, whether they work at non-profit research centers or for-profit companies. Non-profits will pay only a service charge to Coriell, but for-profits may be subject to other fees, Christman says.
From its own iPS cells, Cellular Dynamics now churns out billions of specialized cells such as cardiomyocites, neurons, and epithelial cells at its Wisconsin headquarters. Its business is based on persuading drug companies that these fully human cells make a better testing platform for the potential of their drug candidates than traditional assay methods using live animals or manipulated cell cultures from humans and animals. CDI’s customers have included Hoffman-La Roche, Eli Lilly and AstraZeneca.
CIRM’s biobank will be different from CDI’s current products because it will provide a way to study the drug responses of diseased cells, rather than “normal” ones. CDI will create iPS cell lines from people who have been diagnosed with common disorders, such as autism, Alzheimer’s disease, and cardiovascular defects. Researchers can then produce neurons from the iPS cells of an autistic child in a lab dish, for example. Those cells are expected to express at least part of the underlying abnormalities that lead to autism. Tests on these never-before-available cell types could give drug researchers a way to screen for compounds that might correct the problem, says CIRM scientific officer Uta Grieshammer.
“You can’t take brain cells from living people,” says Grieshammer. Using the cells, drug developers can also test for toxicities that might sink an otherwise promising drug candidate.
CIRM’s biobank is arising at the same time that two European consortia are also setting up large iPS cell banks: the academic-industry partnership StemBANCC, and HipSci, which will serve researchers in the UK. Each will create iPS cells from both healthy people and those diagnosed with illnesses.
Grieshammer says these biobanks will target some of the same diseases as CIRM’s, but each will probably add something unique to the research mix.
Banking and supplying iPS cells to the growing number of researchers who want to use them is increasingly becoming an industrial-scale activity rather than an informal exchange network among individual labs. Like many biobanks that facilitate this cell-sharing among scientists, Coriell originally began its New Jersey repository by
