The Atlanta Journal and Constitution October 24, 2001 Wednesday, Home Edition SECTION: Business; Pg. 5E HEADLINE: Atlanta Tech: Stem cell lab wrestles with questions; OK for federal funding no guarantee of success for Athens-based BresaGen BYLINE: KATHY BRISTER " Athens --- The phone is silent in BresaGen's small office. It's a relief to John Smeaton, chief executive of the biotechnology firm. For weeks after President Bush's Aug. 9 announcement that BresaGen's four embryonic stem cell lines were among 64 approved for federally funded research, Smeaton found it tough to get any work done. Reporters kept calling to ask how BresaGen harvested its stem cells --- starter cells that develop into cells with specific functions --- from human embryos. They wanted to know what the company would do with the potentially disease-fighting cells. They quizzed him on how BresaGen planned to build a business from its research. After Sept. 11, they moved on to other stories. Sitting in the cluttered, white-walled calm of BresaGen's laboratory on the University of Georgia campus, Smeaton still has questions of his own. Just how many of the 64 designated stem cell lines are viable for research? Will politics play an ongoing role in BresaGen's business? Will the company's start-up cash last two years, as planned? "The biggest concern is the time frame --- whether we can afford to finance [stem cell research] as this area develops," said Smeaton. "We're hoping the financial markets will project that this is the area where the next big success will come." It's a valid concern, said Frank DiLorenzo, a biotechnology analyst with Standard & Poor's. Even companies on the president's list will have to look for more money than they'll get from federally funded research, he said. Building a viable business based on stem cells is a "long way off." Even on a biotechnology timetable --- which generally expects investors to wait a decade or more for a return on investment --- using stem cells to treat patients is far down the road, DiLorenzo said. "Public funding will help, but it's not going to be enough." BresaGen got off the ground late last year with a $12.5 million cash infusion from its Australian parent, BresaGen Limited. That money is supposed to last until early 2003. Then the Athens operation may have to ask BresaGen Limited for more, or it may explore going it alone through an initial public offering, Smeaton said. The latter may be difficult, even if the slouching stock market rights itself, DiLorenzo said. Biotech is risky. Investors see embryonic stem cell therapies --- in very early stages and facing long-term regulatory and political hurdles --- as riskier still. Nonetheless, stem cell work seemed promising enough for BresaGen Limited to establish a U.S. operation to isolate embryonic stem cells. The work couldn't be done in Australia. While it is legal to do research on embryonic stem cells there, it is illegal to experiment on human embryos, and, therefore, illegal to harvest stem cells. After shopping for U.S. sites, BresaGen chose Athens late last year because it is home to renowned cloning and stem cell researcher Steven Stice and because it is affordable. The company's laboratory --- being moved in stages from UGA's Animal and Dairy Science building to the university's new bio-business incubator --- costs about a third of what similar space would go for in California, said BresaGen Chief Scientific Officer Allan Robins. In addition, the state of Georgia wooed BresaGen with a $1 million equipment grant awarded by the Georgia Research Alliance, a public-private technology economic development group. Smeaton expects BresaGen's $12.5 million to last longer in Georgia than it would have in biotech hot spots like San Diego or Boston. BresaGen isn't rushing to create revenue. BresaGen Limited doesn't expect the Athens operation to contribute directly to its bottom line, Smeaton said. Rather, it wants BresaGen's stem cells for its own research. Smeaton often carries stem cells back to the the parent company's headquarters in Adelaide, Australia, in his pocket. His body temperature creates an ideal incubator during the 32-hour flight. BresaGen Limited also wants to use the stem cells the Athens operation isolates to gain access to research being done by other scientists. These researchers may use BresaGen's stem cells at very little cost, but they must agree to give BresaGen Limited first dibs on buying or licensing intellectual property gained through their work. BresaGen's inclusion on the list for federal research dollars is key to this business plan. Still, the business of biotechnology is a gamble. Paying high-powered scientists and providing them with cutting-edge equipment is expensive. There's no guarantee the research will lead to breakthroughs in patient treatments. Even if the science works in a laboratory, the Federal Drug Administration may deem biotechnology-based drugs or therapies too risky for broad medical use. BresaGen executives have more to worry about. Research on embryonic stem cells is controversial and politically charged. Medical researchers say embryonic stem cells could unlock treatments for cancer, Alzheimer's and other diseases. But many anti-abortion groups oppose the research because stem cells often are obtained from aborted fetuses. A compromise between these forces led Bush to designate embryonic stem cell lines isolated before Aug. 9 as the only ones that would qualify for federal research dollars. BresaGen hasn't borne the brunt of any anti-abortion protests, Smeaton said, but he won't reveal the location of the clinic from which BresaGen gets the embryos. UGA officials --- worried about jeopardizing projects funded with other federal research dollars --- wouldn't let BresaGen bring embryonic stem cells into its campus laboratory until Bush's list was released. The release of the federal-funding list was a relief, but Smeaton still has questions. He and others in the field aren't sure 64 stem cell lines --- some of which may not have been fully isolated before Aug. 9 --- will be enough. Smeaton also wonders whether later-stage research using heart, pancreatic or liver cells produced from embryonic stem cell lines not on Bush's list will be eligible for federal funding. "If the answer is no, there may be a lot more political pressure for things to change to get to the goal of curing disease," he said. Smeaton may have to employ the patience of a biotechnologist in his wait for answers. Since Sept. 11, a Senate hearing on these issues has been postponed twice. GRAPHIC: Graphic: STEM CELL TECHNOLOGY Embryonic stem cells are basic, blank cells that can change into any one of the more than 200 specific tissue and organ types that make up the human body. These cells also can proliferate indefinitely, giving doctors potentially unlimited options in treating various ailments, including diabetes, Parkinson's disease, kidney disease, chronic heart disease, liver failure and cancer. Here is how embryonic stem cell technology works: Where are stem cells? Once the egg is fertilized it develops into an embryo or blastocyst. Within the embryo, stem cells are formed and eventually will create the various parts of a human being.* A pre-implantation embryo of 30 to 150 cells. Stem cell removal After several days scientists take the stem cells from the embryo. The cells are then cultivated in great quantities in a lab. Treatment Two ways stem cells can aid patients: Stem cells can be changed by a scientist into a particular type of cell, for example a skin cell, then transplanted into the patient. Stem cells can be transplanted into diseased bone, organ or tissue and then change into that specific cell to aid healing. Scientists also can grow stem cells into tissue in a laboratory petri dish, then apply it to the patient. Stem cell types Embryonic -- Stem cells found only within the embryo. These cells are less specialized than adult stem cells. PAGE 4 The Atlanta Journal and Constitution October 24, 2001 Wednesday, Adult -- These specialized stem cells are found in various parts of a mature human. Adult stem cells are needed to replenish the supply of cells in our body that normally wear out. They are often present in only minute quantities, are difficult to isolate and purify, and their numbers may decrease with age. Embryonic stem cells can be harvested three ways 1. Frozen embryos -- Spare embryos from fertility clinics. 2. Fresh embryos -- Embryos created specifically for research. 3. Cloned embryos -- Skin cells from an adult are injected into a donated egg cell stripped of its DNA. Stem cells are extracted from the resulting embryo clone, which does not keep growing. * Stem cells can be found in all humans at any point in the life cycle. Sources: National Institutes of Health, Associated Press / MICHAEL DABROWA / Staff ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn