Guidelines for GM-1 in Acute Spinal Cord Injury

Abstract

To the Editor: The Guidelines Committee1 makes it a point that there is no new evidence for GM-1 since the 2002 previous guidelines,2 but it is they who have no new evidence. The summary they give for GM-1 is a brief paraphrase of the same account they gave in the previous Guidelines—and, indeed, both Guidelines documents essentially reflect discussions we gave in our original publications.3,4 But there is now a major difference in attitude: previously, the committee concluded, from the same identical evidence, that GM-1 should be "a treatment option" in acute spinal cord injury (SCI). Now, with nothing new, they conclude that GM-1 should not be given. How can they justify a different conclusion from the same evidence? If they are saying that the sheer length of time since the GM-1 trials were conducted invalidates them, then it's unclear what scientific principle makes the relation between evidence and conclusions change over time, absent new evidence. On the other hand, the length of time during which no other treatment has been approved only strengthens the "orphan drug" argument in favor of using GM-1. The actual approval status is that the US Food and Drug Administration on December 3, 2012, designated GM-1 as an orphan drug in acute SCI treatment, and GM-1 is now in the US Food and Drug Administration Investigational Device Exemption New Drug Application and approval process. In fact, GM-1 was proven in a pivotal, although small, randomized, double-blind prospective trial3 (the Maryland Study), which was completely successful, to meet its primary analysis and several secondary analyses and to have no negative suggestions. Then, the multicenter trial4 confirmed many of these positive results including that the GM-1 drug treatment group exhibited earlier recovery (and, therefore, a potential for more successful rehabilitation therapy). Because SCI is a devastating disease with no proven pharmaceutical therapeutic options, and because there is no safety concern over GM-1, it is hard to see a rationale for forbidding it as an orphan drug. Thus, the same evidence that the Guidelines Committee reviewed (and which has not changed over time) shows that GM-1 should be a treatment option, as they originally concluded. The committee's new contention that it should not be given is unsupported, gratuitous, and harmful. Disclosure The authors have no personal, financial, or institutional interest in any of the drugs, materials, or devices described in this article.