By Tom Still
MADISON – The decision by a California company to halt its study of a stem cell-based treatment for spinal cord injuries is less about whether such therapies will work than the monumental regulatory and financial hurdles that stand in the way.
In a move with implications for Wisconsin researchers and companies working with embryonic stem cells, Geron Corp. announced in November it will stop spending $25 million a year to bring a stem cell treatment to market.
The reasons are clear: It would have taken up to 10 years to develop a treatment that meets with U.S. Food and Drug Administration approval, so Geron’s executives decided to put their money behind other experimental products that could get to market faster.
Geron executives and researchers outside the company remain confident in the science behind the spinal cord treatment, which has worked in laboratory animals, but the long and arduous pathway to win FDA approval appeared to be too much to overcome.
The Menlo Park, Calif., company had long been viewed as the leader in stem cell therapies, thanks to patents on technology used to grow, manipulate and inject embryonic stem cells into the human body. Stem cells are cells capable of morphing into any one of more than 220 human cell types. Many of the patents licensed to Geron were the result of work performed at the UW-Madison, where researcher James Thomson and his team first isolated embryonic stem cells in 1998.
Is Geron’s decision a setback to the U.S. stem cell research industry? Not necessarily so, because most research is still funded through academic channels. Also, Geron was focused on a specific therapy – mending damaged spinal cords – while companies elsewhere are developing other uses for stem cells.
Cellular Dynamics International, a Madison company founded by Thomson and others, is a ready example. Thomson has long insisted that stem cell “cures” or treatments were years in the future, given the revolutionary nature of what is still a very young field of science. His company has focused instead on using stem cells for disease modeling, testing new drugs, diagnostics and other research applications.
“For us, it validates the basic business plan: Steer clear of therapeutics for the foreseeable future,” said Dr. Tim Kamp, director of the stem cell medical program at the UW-Madison and a co-founder of CDI.
Business plans for any U.S. company involved in seeking biotechnology cures, treatments or diagnostics must include seeking necessary approvals from the FDA, which regulates development of drugs, medical devices, biologics, vaccines and more. Increasingly, however, the bioscience industry has complained about the FDA’s regulatory pace. For example, the president of the national Biotechnology Industry Organization, former member of Congress Jim Greenwood, called on federal lawmakers to “build a 21st century FDA” or risk losing U.S. competitiveness in the life sciences.
“The Geron experience underscores how broken the FDA process has become in this country,” said Carl Gulbrandsen, managing director of the Wisconsin Alumni Research Foundation. WARF is the private, non-profit group that handles UW-Madison patents and licenses them to potential users.
Others say the FDA is simply being cautious in the wake of some high-profile drug failures and because its regulators, like others in the field, are new to stem cell research. Still, skeptics say the system has become so cumbersome that innovation is being stifled.
“Geron spent about $45 million to prepare its IND (Investigational New Drug application) alone. There aren’t many companies that can afford to do that,” Gulbrandsen said.
For many years, the biggest threat to stem cell research came from opponents who questioned the ethics of how the cells themselves were derived. Today, it appears, an emerging challenge may be the possibility that researchers and investors frustrated by paperwork and costs go outside the United States to develop stem cell therapies that should be kept home.
Still is president of the Wisconsin Technology Council. He is the former associate editor of the Wisconsin State Journal.