The Business of Stem Cells
ACT has ongoing clinical trials, in which it is enrolling patients. On July 14, doctors at University of California, Los Angeles began treatment in two of its clinical trials. “One patient in each clinical trial, the Stargardt trial and the dry AMD trial, has undergone surgical transplantation of a relatively small dose (50,000 cells) of fully-differentiated retinal pigment epithelial cells derived from human embryonic stem cells,” explained Steven Schwartz, M.D., of the David Geffen School of Medicine at UCLA and retina division chief of its Jules Stein Eye Institute and the studies’ principal investigator. “Early indications are that the patients tolerated the surgical procedures well.”
Despite relatively small numbers of cells required to test its hESCs in these trials and its ability to monitor early-on efficacy signs, ACT doesn’t have a lot of cash. The firm ended this year’s third quarter with cash and cash equivalents of $13.9 million, compared to $16.1 million as of June 30, 2011. By contrast, Geron completed the same period with over $180 million on hand.
Geron, in trying to get to the clinic with its hESC therapy for spinal cord injury, faced a clinical hold focused on the FDA’s concern about the carcinogenic potential of hESC cells. The company also repeatedly said it was initiating a clinical trial over several years, a promise it couldn’t deliver on. Technical issues included the sheer mass of cells that had to be introduced into patients and a long wait time to see any indication of therapeutic results.
Waiting for the potential payoff from stem cell research proved too far off for Geron, which does not have any marketed products. It would have spent $25 million per year to continue its stem cell program. Joseph Pantginis, an analyst with Roth Capital Partners, told the Associated Press that it would have taken five to ten years before Geron’s lead hESC product reached the market. “This is still very much a fledgling space, and some people would even consider stem cells to be a science experiment, so there’s still a long way to go.”
There is no doubt that sufficient financing is key for conducting the trials needed to prove this new science and for navigating the regulatory path, which is predictably unpredictable for an emerging field. But the company with the most cash in the stem cell field—Geron—has left the arena.
Investors seem to be sitting the space out. Given that uncertainty over funding and patenting is rife in the U.S. and Europe, this is not surprising. If the revolving door for hESC therapeutic development lands Geron’s technology at a different company, that firm must have not just the money but also the will to further hESC-based therapeutic programs.