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Gearing for nitty-gritty questions on making safe products from embryonic stem cells
On Thursday, April 10, the FDA held its first public advisory committee on assessing safety risks of cell therapies derived from embryonic stem cells. In a hotel ballroom just outside Washington, more than two dozen committee members and invited specialists weighed in. Separated from the discussion area by a yellow plastic chain, were about 200 prominent academics, consultants and industry representatives, and members of the press.
Prior to the meeting, the FDA had released a 12-page briefing document outlining the safety questions to explore, particularly how cells should be characterized and assessed for safety before transplantation and how patients could be monitored afterwards.
The biggest concern is that once placed in human subjects, cells could proliferate and differentiate in ways that are harmful and uncontrollable, and that studies of human cells in mice and rats won’t reliably predict their safety in human patients. A transcript of the session should be available from the FDA in a few months. Here’s about 2,000 words of what I found salient. (Disclosure: I was fresh off a red-eye from San Francisco, and I’m writing this on the plane back.)
Previously, I ran around asking attendees for their top-level thoughts. You can read that here. The consensus of the advisory committee for cellular, tissue, and gene therapies seemed to me to be both “Onward!” and “Careful!”
The sections that follow are 1) company presentations 2) wanted: fortune-tellers for teratomas 3) why can’t a mouse be more like a patient? 4) cell products are special and 5) once a trial enrollee, always a subject?