Further to the therapeutic outlook on first-in-human studies at the Brugge meeting was Adrian Thrasher's thoughtful presentation on his own X-SCID study at Great Ormand Street Hospital. Thrasher's study was able to restore immune function in nearly all volunteers. Recently, however, his team reported a lymphoproliferative disorder like those seen in a very similar Paris study.Thrasher stated clearly "The purpose [of X-SCID protocol] is therapeutic effect; it is not a safety study." Fair enough: the study was in a pediatric population (standard research ethics requires clear therapeutic warrant for such risky studies), and Thrasher's protocol did not range doses the way typical first-in-human studies do. And I should add, there is some grounds for thinking of the study as having therapeutic warrant, not the least because it was supported several unsuccessful X-SCID human studies and a successful one in Paris). Still, putting the therapy before the learning- this made me somewhat uncomfortable. Therapy might have been his (and his hospital's) intent, but to describe the study as ontologically "therapeutic" and not "research"? Intent only gets us so far...
Thrasher revealed some unusual properties about the molecular events leading to this leukemia (see? told you it could be construed as a safety study). And now, here's the compassion part. Thrasher was circumspect about this particular leukemia, because the patient who developed the leukemia had originally been ineligible for the protocol because he had a matched unrelated bone marrow donor. The regulatory agency made a "one-time" exception to waive the normal risk-benefit balance.
Of course, one should be very careful generalizing from this one case where "compassion" seems to have led authorities astray. And presumably, the boy's parents were thoroughly informed about the risks going in to the protocol. Still, the example is somehow instructive. (photo credit: missinguigga 2008).
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