The trial did suggest that the therapy might be safe. But it was not designed to determine whether it worked, said Dr. Joanne Kurtzberg, a pediatric hematologist-oncologist at Duke University Medical Center and president of the Cord Blood Association, who led the trial.
Dr. Kurtzberg has also led two subsequent Phase 2 clinical trials — one on the efficacy of cord blood transfusions as a treatment for autism, and another on cerebral palsy. In both cases, while the results have been encouraging, they are still preliminary; it would take larger, Phase 3 trials in more patients to prove that a treatment is truly safe and effective.
“I think that some of the companies you’re referring to leverage and take advantage of our studies in a way that is premature,” Dr. Kurtzberg said about private cord blood banks.
Some private cord blood banks are also floating vague possibilities of regenerative stem cell medicine as a cure for heart disease, Alzheimer’s, diabetes and other conditions. Not one of these therapies has made it past a Phase 2 trial, or is approved by the F.D.A.
Morey Kraus, chief scientific officer at ViaCord, said that he understands that more research on cord blood is needed for use in unapproved conditions, but that even if the research doesn’t pan out, parents will have been glad they had the option of banking and trying it as a therapy.
Not everyone agrees: “The data suggest there might be a little help there, but I find it not at all convincing,” said Dr. Steven Joffe, a pediatric oncologist and bioethicist at the University of Pennsylvania Perelman School of Medicine. The cost of private banking “would be hard to justify at this point,” he said, unless someone in the family has a known blood disorder that might require a stem cell transplant.
According to Dr. Knoepfler, cord blood stem cells should not be thought of as “some kind of panacea” for all diseases. “People are in desperate situations,” he said, “they’re looking for hope, and giving them false hope has definite downsides.”