Cautious investors don’t slow progress in regenerative medicine
Regenerative therapy has been hanging out with the wrong crowd, gaining a bad reputation in the process. That’s changing now, however, as cancer vaccines prove effective and as the retiring population of Baby Boomers flatly refuses to go gently into that good night.
Given those drivers, it’s no surprise that the regenerative medicine session of Biotech Showcase™ 2011 in San Francisco attracted an overflow crowd.
“People view regenerative medicine as a far-off objective, but that is not true. There’s a tremendous amount of ongoing work,” noted Gil Van Bokkelen, Chairman and CEO of Athersys, and Chairman of the Alliance for Regenerative Medicine.
Alan Trounson, Chairman of the California Institute for Regenerative Medicine concurred, citing promising results in diabetes, cancer, neurodegenerative disease and other conditions. He admitted, however, that much of the work is in early clinical trials. “In animal studies, we have a cure for HIV,” he said. “In diabetes, we expect to file an IND application in about two years.”
The goal for Richard Insel, CSO of the Juvenile Diabetes Research Foundation, is to find a cure for diabetes by accelerating discovery and development. The Foundation is funding projects to restore beta cell function, either by transplanting encapsulated stem cells, or alternatively, by regenerating the beta cells in the hosts themselves. He reported that one of the programs the Foundation supports is producing beta cells at the same rate as mature cells, and this is reversing diabetes.
Geron focuses on embryonic stem cells and, according to Tom Okarma, President and CEO, “We have robust animal data for glial cells, cardiomyocytes, dendritic cells, etc.” For example, GRNOPC1, a potential therapy for spinal cord injury, is in human trials where it has induced new blood vessels and myelination, and improved locomotor activity. The progenitor cells expand ten to fifteen fold in animal models, and migrate. “They’ve been injected up to two weeks after the initial injury,” he pointed out.
To recoup the USD 45 million spent to generate the data for an IND involving six animals, Okarma said Geron will expand this platform for Alzheimer’s disease and thrombotic stroke applications.
Advanced BioHealing has launched perhaps the largest trial ever for venous leg ulcers, involving 500 patients in seven countries, Dean Tozer, Senior VP, said. His technology relies upon seeding neonatal fibroblasts onto biodegradable scaffolds. It also applies to diabetic foot ulcers.
Chris Calhoun, CEO of Cytori Therapeutics, reported on the regenerative potential of using stem cells from fat tissues to reconstruct breasts after lumpectomies. “With three needle sticks, we can reconstruct the breast in one hour,” restoring normal appearance to a situation not otherwise served.
The question broached by Mark Monane, Managing Director of Equity Research Biotechnology and Life Sciences at Needham & Company, was, “What are we getting?” Despite new therapies and even a few blockbusters, “There’s a less than optimal return on investment,” he concluded.
That said, “One of every three patients could be helped by regenerative medicine,” Monane continued. Therefore, to increase the potential return, he advised companies to develop and leverage platforms to reduce the risk and thereby attract investors. “Regenerative medicine companies are founded on hope, but Wall Street wants deliverables.”
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