The promise, the pitfalls and a paradigm shift, PART 2

The complex history of stem cell research yields hope for improved human health, unresolved concerns

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Much ado aboutnothing? 
Despite these challenges, Gilkey notes that the NCRM's workinvolving adult and pluripotent stem cells has generated more than $400 millionin federal, state and private funding. These dollars have been used to startnine Ohio-based companies and create nearly 600 Ohio jobs. The center has alsobeen awarded 19 patents and 10 U.S. Food and Drug Administration-approvedInvestigational New Drugs for stem cell studies. 
Thus, despite lingering questions and remaining debate, thework continues. To date, the U.S. Patent and Trademark Office has issued 11,552stem cell-related patents, 3,658 of which involve hESCs.
The researchers speaking to ddn note that some of the controversy being reported about hESCsmay not be in line with actual public perception. In 2010, the researcherspoint out, 70 percent of respondents to a Research!America poll said they favorexpanded funding for stem cell research.
But although Civin notes that "this is an issue that hasswayed an election or two," he says he believes "the controversy is pretty muchover" due to the iPSC discovery made by Yamanaka's lab.
"We are pretty much at the point where we almost don't needhESCs," Civin says. "What we do need them for are controls. We need to makesure our cells are good, that they aren't doing something flukey. People doingresearch usually now use iPSCs for various technical and repeatability reasons,so I think it is a non-issue. I'm not saying the debate will totally go away,but I do believe the controversy is dying."
Show me the money 
How much money do these researchers need? Scadden starts theprice point for an average research project at "millions of dollars."
"Most projects in this area take many people many years ofextensive work to do," he points out. "Every assay is four to six months ofevaluation in animals. It's extremely expensive work, and the procuring,culturing and storage of human cells is also very expensive."
At one point, Civin's lab had about $20 million in grants towork with: "A large lab like mine will have $1 million or $2 million a year infunding per year to keep it going," he says. "After that, our main source ofgrants is the NIH for basic and translational research. In Maryland, we arealso blessed by state funding. Beyond that, there are many foundations we workwith that are cancer-specific, like the American Cancer Society, or smallercharities for childhood cancer."
Civin points out that the NIH invests in projects that havea certain amount of feasibility data, so funding from private donors is oftenused for smaller pilot projects.  
"In general, researchers joke that an NIH grant is a rewardrather than an award," Civin laughs, "and you have to have half of the grantdone before it can be awarded. But as a scientist, I believe in shooting at alot of areas and taking the risk that I may not hit a target. There are certaingrants the NIH will award for those kinds of projects, but you need at least alittle feasibility data to get started."
Non-government funding for stem cell research may come withstrings attached. Some private foundations—such as the John Paul II Stem CellResearch Institute—will only fund projects that use adult or umbilical cordstem cells. Additionally, other organizations may stipulate that if theycontribute to the support of a project that eventually results in profits for auniversity or institution, they are entitled to a portion of it.
"For example, if a grant funds 10 percent of a researchproject that results in a profit of $1 million, the foundation may go to theuniversity and ask for 10 percent," explains Civin. "Most patents are held byinstitutions, and the inventor and inventor's lab share in those royalties, butthose who contribute to the research may have an agreement that entitles themto a certain percentage of that profit."
Good for business
A number of firms dedicated to stem cells have been launchedin the past few years, thanks to strong venture capital funding in geographicareas where government and science have favored stem cell research, accordingto Frost & Sullivan. California ranks highest, with a total of 10firms—dedicated to all kinds of stem cell research—receiving either venture orIPO-backed funding. Geron Corp., which works with hESCs, has the maximum market capital value at $605million, despite the fact that ASC research receives more funding.
And according to a 2010 report by the market research firm, manycompanies that provide the tools and technologies required for stem cellresearch have benefited from increased activity in this area. Specifically, themost common tools used by stem cell researchers are bio-imaging and microscopy;cell biology tools; immunochemicals; molecular biology tools; and proteinbiochemistry tools.
Top-ranking tool providers across these various areas,according to Frost & Sullivan, include: Abcam PLC; AbD Serotec; AbnovaCorp.; Active Motif; Amnis Corp.; Applied Biosystems and Invitrogen by LifeTechnologies; BD Biosciences; Bio-Rad Laboratories Inc.; Carl Zeiss Inc.; CellSignaling Technology Inc.; Clontech Laboratories Inc.; EMD Biosciences andMillipore; Kodak; Leica Microsystems GmbH; Lonza Group Ltd.; New EnglandBiolabs; Nikon Corp.; Olympus Corp.; Promega Corp.; QIAGEN; R&D SystemsInc.; Roche Applied Science; Rockland Immunochemicals Inc.; Santa CruzBiotechnology Inc.; Signalway Antibody Co.; Sigma-Aldrich Co.; STEMCELLTechnologies Inc.; and Thermo Fisher Scientific Inc./Cellomics and Pierce.
"I think there is always going to be a great opportunity forgrowth in this market in general as hESC research is allowed to blossom as anindustry," Witonsky says. "One thing to keep in mind is that with a lot ofresearchers we talk to, they are not necessarily looking solely at hESCs. Sosome of the controversy does not necessarily represent a major drawback forthese product categories, but at the same time, as research progresses, it willdrive more demand for these tools."
The promise and aparadigm shift 
Of all the promise that stem cell research holds for modernmedicine—to replace cells, tissue and organs, or to serve as disease models fordrug development—researchers tell ddnthat its greatest, and perhaps most imminent, potential is a major paradigmshift in the drug discovery process as we know it.
"The idea that one can generate cell populations that can betransplanted or implanted is something that is active in the clinic today,"Scadden says. "We do this all the time for cancer or blood disease therapy,burns, spinal fusions or corneal injury."
But the notion of using human cells in drug screens could bea major game-changer, Scadden says.
"There is a better way to develop and identify compounds,"he says. "Using human cells of the cell type that is affected by a particulardisease is a spectacular step forward."
"Imagine being able to do a clinical trial in a dishrepresenting hundreds of patients with a particular disease, and being able topredict which population will most benefit from a compound. This shifts theodds of success in your favor. This is the ultimate personalized medicine,"agrees Srivastava.
Just like stem cells, global research efforts with them aremany and varied
By Jeffrey Bouley, ddn Managing Editor
Stem cell research: Sorting fact from fiction

For the stem cell research arena, the main source of publicconfusion and debate centers on the use of cell lines derived from humanembryos. While society has yet to reach consensus on the ethical and moraldilemmas presented by hESC research, it's important to remember that it's notthe only form of stem cell research.

By Amy Swinderman, ddn Chief Editor

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