The promise, the pitfalls and a paradigm shift, PART 2
Much ado about nothing?
Despite these challenges, Gilkey notes that the NCRM's work involving adult and pluripotent stem cells has generated more than $400 million in federal, state and private funding. These dollars have been used to start nine Ohio- based companies and create nearly 600 Ohio jobs. The center has also been awarded 19 patents and 10 U.S. Food and Drug Administration-approved Investigational New Drugs for stem cell studies.
Thus, despite lingering questions and remaining debate, the work continues. To date, the U.S. Patent and Trademark Office has issued 11,552 stem cell-related patents, 3,658 of which involve hESCs.
The researchers speaking to ddn note that some of the controversy being reported about hESCs may not be in line with actual public perception. In 2010, the researchers point out, 70 percent of respondents to a Research!America poll said they favor expanded funding for stem cell research.
But although Civin notes that "this is an issue that has swayed an election or two," he says he believes "the controversy is pretty much over" due to the iPSC discovery made by Yamanaka's lab.
"We are pretty much at the point where we almost don't need hESCs," Civin says. "What we do need them for are controls. We need to make sure our cells are good, that they aren't doing something flukey. People doing research 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 the price point for an average research project at "millions of dollars."
"Most projects in this area take many people many years of extensive work to do," he points out. "Every assay is four to six months of evaluation 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 to work with: "A large lab like mine will have $1 million or $2 million a year in funding per year to keep it going," he says. "After that, our main source of grants is the NIH for basic and translational research. In Maryland, we are also blessed by state funding. Beyond that, there are many foundations we work with that are cancer-specific, like the American Cancer Society, or smaller charities for childhood cancer."
Civin points out that the NIH invests in projects that have a certain amount of feasibility data, so funding from private donors is often used for smaller pilot projects.
"In general, researchers joke that an NIH grant is a reward rather than an award," Civin laughs, "and you have to have half of the grant done before it can be awarded. But as a scientist, I believe in shooting at a lot of areas and taking the risk that I may not hit a target. There are certain grants the NIH will award for those kinds of projects, but you need at least a little feasibility data to get started. "
Non-government funding for stem cell research may come with strings attached. Some private foundations—such as the John Paul II Stem Cell Research Institute— will only fund projects that use adult or umbilical cord stem cells. Additionally, other organizations may stipulate that if they contribute to the support of a project that eventually results in profits for a university or institution, they are entitled to a portion of it.
"For example, if a grant funds 10 percent of a research project that results in a profit of $1 million, the foundation may go to the university and ask for 10 percent," explains Civin. "Most patents are held by institutions, and the inventor and inventor's lab share in those royalties, but those who contribute to the research may have an agreement that entitles them to a certain percentage of that profit."
Good for business
A number of firms dedicated to stem cells have been launched in the past few years, thanks to strong venture capital funding in geographic areas where government and science have favored stem cell research, according to Frost & Sullivan. California ranks highest, with a total of 10 firms— dedicated to all kinds of stem cell research—receiving either venture or IPO-backed funding. Geron Corp., which works with hESCs, has the maximum market capital value at $605 million, despite the fact that ASC research receives more funding.
And according to a 2010 report by the market research firm, many companies that provide the tools and technologies required for stem cell research have benefited from increased activity in this area. Specifically, the most common tools used by stem cell researchers are bio-imaging and microscopy; cell biology tools; immunochemicals; molecular biology tools; and protein biochemistry tools.
Top-ranking tool providers across these various areas, according to Frost & Sullivan, include: Abcam PLC; AbD Serotec; Abnova Corp.; Active Motif; Amnis Corp.; Applied Biosystems and Invitrogen by Life Technologies; BD Biosciences; Bio-Rad Laboratories Inc.; Carl Zeiss Inc.; Cell Signaling Technology Inc.; Clontech Laboratories Inc.; EMD Biosciences and Millipore; Kodak; Leica Microsystems GmbH; Lonza Group Ltd.; New England Biolabs; Nikon Corp.; Olympus Corp.; Promega Corp.; QIAGEN; R&D Systems Inc.; Roche Applied Science; Rockland Immunochemicals Inc.; Santa Cruz Biotechnology Inc.; Signalway Antibody Co.; Sigma-Aldrich Co.; STEMCELL Technologies Inc.; and Thermo Fisher Scientific Inc./Cellomics and Pierce.
"I think there is always going to be a great opportunity for growth in this market in general as hESC research is allowed to blossom as an industry," Witonsky says. "One thing to keep in mind is that with a lot of researchers we talk to, they are not necessarily looking solely at hESCs. So some of the controversy does not necessarily represent a major drawback for these product categories, but at the same time, as research progresses, it will drive more demand for these tools."
The promise and a paradigm shift
Of all the promise that stem cell research holds for modern medicine—to replace cells, tissue and organs, or to serve as disease models for drug development—researchers tell ddn that its greatest, and perhaps most imminent, potential is a major paradigm shift in the drug discovery process as we know it.
"The idea that one can generate cell populations that can be transplanted 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 be a 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 particular disease is a spectacular step forward."
"Imagine being able to do a clinical trial in a dish representing hundreds of patients with a particular disease, and being able to predict which population will most benefit from a compound. This shifts the odds of success in your favor. This is the ultimate personalized medicine," agrees Srivastava.
Just like stem cells, global research efforts with them are many 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 public confusion and debate centers on the use of cell lines derived from human embryos. While society has yet to reach consensus on the ethical and moral dilemmas presented by hESC research, it's important to remember that it's not the only form of stem cell research.
By Amy Swinderman, ddn Chief Editor