The Food and Drug Administration’s (FDA) approval of a clinical trial involving a human embryonic stem cell-based therapy is being hailed both as a triumph of science over politics, as well as heralding a much-needed financial shot in the arm for the nascent hESC biotech industry. In reality, it isn’t much of either.
The FDA’s green light in January to Geron Corp.’s (Menlo Park, Calif.) investigational new drug (IND) Phase 1 trial for patients with acute spinal cord injury is indeed significant, but for other reasons: it is the first such approval in the world, and it required an extraordinary effort to win. Geron’s application totaled 21,000 pages that described 24 separate studies conducted over half a dozen years, involving nearly 2,000 animals with spinal cord injuries, and requiring the production, and injection of more than five billion oligodendroglial progenitor cells.
Geron’s multi-center trial, which will involve up to 10 individuals paralyzed after suffering spinal cord injuries, is expected to begin enrolling patients early this summer at the rate of about one per month. Clinical data are not expected until at least a year later. “This marks the beginning of what is potentially a new chapter in medical therapeutics—one that reaches beyond pills to a new level of healing,” said Thomas Okarma, MD, PhD, Geron’s president and chief executive.
The trial is an essential step along the path of establishing the safety and efficacy of hESC-based therapies, which are—as yet—unproven. Nevertheless, scientists and financial analysts cheered Geron’s Jan. 23 announcement. “The neurosurgical community is very excited by this new approach to treating devastating spinal cord injury,” said Richard Fessler, MD, PhD, professor of neurological surgery at Northwestern University’s Feinberg School of Medicine in Chicago.
Researchers, who have complained about restrictions on federal hESC research funding since President Bush first authorized them in 2001, noted that FDA’s approval came only days after the inauguration of President Obama, who had vowed to increase the number of hESC lines eligible for funding from the National Institutes of Health (NIH). But the timing was coincidental, both Geron and the FDA said, with the decision coming within 30 days after the company’s last written submission to the agency, as required by law.
Geron has an exclusive license from the Wisconsin Alumni Research Foundation (WARF) in Madison, which holds several pioneering patents in the field, to develop therapeutic and diagnostic products from neural, cardiomyocyte, and pancreatic islet cells. Geron hopes to submit an IND next year for hESC-derived heart muscle cells that could generate functionally restored cardiac tissue responsive to standard drugs.
The company is one of many trying to develop hESC-based therapies. Advanced Cell Technology Inc. (Worcester, Mass.) teetered on the edge of bankruptcy last year as it struggled to develop hESC-based retinal pigment epithelium cells to treat age-related macular degeneration. ACT hopes to file its first IND by this summer, chief scientific officer Robert Lanza said. Novocell Inc., a San Diego-based company that merged in 2004 with two other stem cell firms, CyThera Inc. and Bresagen Inc., is seeking to produce hESC-derived pancreatic progenitors capable of developing into ductal, acinar, and endocrine cells that could produce insulin and other pancreatic endocrine hormones.
The FDA approval was clearly a milestone for hESC. Geron, and a handful of smaller stem cell companies, enjoyed a boost in their stock prices following the announcement. But the excitement may be hard to sustain, given the cost and long lead times required to develop successful biotech products. “You can’t copy off of other people’s homework,” Needham & Co. analyst Mark Monane told Dow Jones, noting that other stem cell companies will have to pave their own paths with the FDA to get their products into clinical trials.
Big Pharma has generally kept its distance from hESC research, but that began to change a couple of years ago, well before FDA’s approval of Geron’s IND. Novocell received financing in 2007 from Johnson & Johnson and other investors to support its preclinical development of diabetes and other disease cell therapies. Pfizer Inc. in December inked a nonexclusive collaboration with Novocell for access to its hESC-derived pancreatic progenitor cells.
Other recent investors and partners in stem cells include Roche Holding AG, Novartis AG, and GlaxoSmithKline. GSK last year entered into a $25 million, five-year agreement with the Harvard Stem Cell Institute to develop therapies for cancer, diabetes, and other conditions.
Despite the media and political attention focused on human embryonic stem cells, most of the action to date has involved better-understood and less controversial adult stem cells, from which therapies for diabetes and advanced kidney cancer have been demonstrated. Last June, Pfizer invested $3 million with San Diego-based startup EyeCyte Inc. to develop treatments for diabetes-induced retinal damage, a leading cause of blindness, using patient blood and bone marrow-derived progenitor cells.
For years, Pfizer and other Big Pharmas have been using adult and animal stem cells to screen potential new drug compounds. In what may be the clearest signal yet of confidence in stem cells, Pfizer announced last year it would spend $100 million over five years to develop therapies from stem cells, both adult and embryonic. Pfizer Regenerative Medicine, based in Cambridge, Mass., and in Cambridge, UK, will employ about 70 scientists and will enter into collaborations with academia and industry. The US team will focus on heart disease, diabetes, and cancer, while the UK team will focus on therapies for vision and hearing.
About the Author
Contributing editor Ted Agres, MBA, is a veteran science writer in Washington, DC. He writes frequently about the policy, politics, and business aspects of life sciences.
This article was published in Drug Discovery & Development magazine: Vol. 12, No. 3, March, 2009, pp. 6-7.
Filed Under: Drug Discovery