West Coast Editor
SAN FRANCISCO - Just before a unique meeting on stem cell research got under way here, preclinical news from the UK boosted hopes for the field as applied to stroke.
ReNeuron Group plc aims to file an investigational new drug application and begin dosing humans this year, based on findings from a second study with ReN001, a therapy for chronic disability after stroke.
"Everything is on track," said John Sinden, Guildford, UK-based ReNeuron's chief scientific officer, pointing out that data from the second study in rodent models reinforce results from the first study, with delivery method and implant location selected to mimic as much as possible the protocols likely to be followed in human trials.
The new data back up the first set in another way, he told BioWorld Today.
"We've used cells that have been banked - they're derived from GMP lots, equivalent to the clinical lots that will go into the patients," Sinden said. "The original studies were done with, as it were, early materials."
What's more, the latest results show a dose-ranging effect, suggesting that a higher dose has the potential to reverse both the sensory and motor symptoms from stroke. In September, ReNeuron held a formal pre-investigational new drug application meeting with the Center for Biologics Evaluation and Research component of the FDA.
The company's headlines hit just ahead of the one-day International Symposium on Stem Cell Collaboration today at the University of California at San Francisco. Sponsored by the Women's Technology Cluster (WTC), a business incubator dedicated to women leaders building technology-driven firms, the event is intended to "help get the dialogue going early" between academia, companies and would-be investors, said Cori Gorman, president of DNA Bridges, a San Francisco-based consulting firm for biotech and pharma.
"We're scientists with business experience," said Gorman, who serves on the board of trustees of the WTC. She spent 10 years at South San Francisco-based Genentech Inc., and co-founded the gene therapy firm Valentis Inc., of Burlingame, Calif.
Although Valentis recently reported stock-boosting Phase IIb data with its lead candidate VLTS 934, a nonionic block copolymer that acts as a vascular lubricant in patients with peripheral arterial disease, gene therapy mostly has struggled, and that struggle partly inspired Gorman to find ways of advancing the dialogue.
Planning for the stem cell event began in the fall of 2004, but the WTC decided "it was a little early then," Gorman said. "We needed a few more successes on the basic research side" - successes like ReNeuron's.
"What's beautiful about their work is that they have found a way to make the cells last a long time in the undifferentiated state," Gorman told BioWorld Today. "They understand that you need to have them stable and reproducible, and then be able to remove whatever trigger that is letting them remain in a stem cell-like state. Geron [Corp., of Menlo Park, Calif.] is also trying to do this."
ReNeuron's GMP ability with cell lines makes the company more ready for FDA scrutiny, too, and "that's something gene therapy missed out on a little bit. They weren't thinking about what the FDA would want."
Ultimately, Gorman said, "some of the things going on in academia" tipped the scale in favor of putting the symposium together. She mentioned work on rodent spinal cord regeneration in the lab of Brian Cummings at the University of California at Irvine, and research on heart regeneration by the likes of Nadia Rosenthal in Italy and scientists at the University of Minnesota.
StemCells Inc., of Palo Alto, Calif., in July entered a licensing deal with ReNeuron that gives the former exclusive access to the latter's c-mycER adult human stem cell immortalization technology for use in developing therapies for lysosomal storage diseases, spinal cord injury, cerebral palsy and multiple sclerosis. In return, ReNeuron got exclusive access to StemCells' adult neural stem cell patent estate for the development of its own c-mycER conditionally immortalized neural stem cell therapies for stroke, as well as Parkinson's disease and Huntington's disease.
As part of that arrangement, StemCells has taken a 7.5 percent fully diluted equity interest in ReNeuron, and the agreement provides for reciprocal royalties and milestone payments.
"That [deal] is a key one for us," Sinden said, calling StemCells "a very big player in the U.S."
After neural applications, he said, ReNeuron's "next stage is to work with [the technology] in other types of stem cells, and we already have some projects under way," at the preclinical stage.
His firm's approach is "different, because [ReNeuron uses] clonal cell lines, so there's an element of genetic modification, but all of that is understood from the regulators' point of view," Sinden said. "They like what they see, in terms of how we're operating."