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Geron's CEO, Dr. Thomas Okarma, says he understands the anxiety. "Nobody wants another Jesse Gelsinger here," he says, referring to the teen who died in a 1999 trial for gene therapy, another pioneering technique. The company, he says, has "lifted stones as far and high as we can to assure ourselves the safety of this product." The oligodendrocyte cells it is injecting (each patient will receive a syringe of 2 million cells) are "highly characterized," meaning it's seen no evidence of any outliers that might cause trouble down the road. Its tests found no tumors in rodents over the course of a year and no evidence of an immune response (patients will be given immunosuppressants for 45 to 60 days after treatment, but that's to be sure there's no inflammation in the injury site, which might destroy the injected cells).
Patients will be closely monitored with MRIs for the first year and followed for a total of 15 years. And trial volunteers will have complete injuries "with no hope of recovery," making the risk worth the potential payoff, says Okarma. Geron's mantra is "living cells will be tomorrow's pills," he says. And he is confident it will get there. So confident, he believes the oligodendrocyte progenitor cells might be applicable to other nervous-system disorders, like multiple sclerosis, Alzheimer's and stroke. Geron plans to publish more of its preclinical data, Okarma says, but in the meantime, the public should be assured that the company has done rigorous research. "Companies die when they're not successful, when a product is harmful or it doesn't work," he says. "The last thing we want to do is be wrong. The future of the company depends on this." Keirstead, for his part, says the first thing he asks when people say "slow down" is "Do you know anyone in a wheelchair?'"
Everybody wants to be cautious, but researchers, clinicians, patients and biotechs differ on how much risk they can live with and when to pull the trigger. Gearhart says his conservative approach butts up against others in the field. "Every time I say 'be circumspect,' my clinical friends are hitting me upside the head, saying, 'You've got to do it at some point'." That is the view of Dr. Gary Steinberg, a professor of neurosurgery at Stanford, a potential site for Geron's trial. "I'm highly enthusiastic," he says. Steinberg says he has faith in the FDA's review. Without effective treatments currently available, patients with spinal-cord injuries are left with little recourse. "There's a significant unmet need," he says. At the Miami Project to Cure Paralysis, scientific director W. Dalton Dietrich says he has concerns—this is a first, after all—but he also believes researchers will learn a great deal about how to use cellular therapies in humans, something that will help educate and advance the field.