Last week, Columbia, Maryland-based Osiris Therapeutics boasted the world’s first approved stem-cell drug.
It isn’t, but the drug, Prochymal, used to treat graft-versus-host disease following transplant procedures, is still a rare regulatory success for a stem-cell therapy, and one might have expected some excitement about it at the World Stem Cells and Regenerative Medicine Congress in London this week.
It was rarely mentioned. They had other things to be excited, and worried, about. Amid a predictably buoyant mood for a meeting of industry hands, delegates discussed some successful regulatory approvals, large investment deals, a few cell-based therapeutics that are making revenue and the advancement of clinical trials over the last couple years — all of which, taken together, points to a ripening of the field, they say.
The conference covered not just stem cells, but all cellular therapies: anything in which biological cells are use for therapy. One of the meetings organizers, Chris Mason, chair of regenerative medicine bioprocessing at University College London, is keen to stamp out the distinction of stem cells. “I don’t like the word [stem cells],” he says. “They are all just living cells as therapies.”
Mason, who doubles as a partner in the investment and advisory firm Proteus Regenerative Medicine, says that growth has been strong across the industry. There are now 2,700 clinical trials in cellular therapies, 700 companies with mature technologies, and the 8 cell-based products approved in the United States and Europe now account for a more than US$1-billion industry, which he expects to grow to more than $3 billion by 2014. The lion’s share of that revenue is accounted for by three drugs, each with revenue of more than $200 million: the cancer drug, Provenge, and two skin grafts used for diabetic ulcers, Demagraft and Apligraf. The conference itself, in its seventh year, drew 350 people, its largest showing yet.
Hot topics were deals, like the $750 million that Dublin-based Shire paid for Dermagraft proprietor, Advance BioHealing in 2011 and Teva’s $6.8-billion purchase of Cephalon. A slew of others, having tentatively proven themselves effective, are entering phase III trials.
Representatives discussed the status of their pipelines relatively openly, largely because they are facing common enemies. One is the difficulty they face in getting health-care systems to agree to pay for the treatments once they are approved. The main target, however, are the regulatory agencies, many of which had representatives at the meeting. These agencies are alternately cajoled and condemned, with some stressing the importance of “getting in with the FDA [US Food and Drug Administration] early in the development process”, and others, usually behind the scenes, describing their own experiences as Kafkaesque attempts to navigate an endless maze.