Correspondence

Offering a Celebrity Experimental Treatment

N Engl J Med 1993; 328:1202-1203April 22, 1993

Article

To the Editor:

On November 29, 1992, Dennis Byrd, a football player with the New York Jets, suffered a serious spinal cord injury when he collided with a teammate during a game. According to reports by the news media, Byrd was treated at Lenox Hill Hospital in New York with an experimental drug1,2. These reports dismay researchers such as myself, who are conducting randomized, controlled trials of treatments for such injuries. The drug, GM-1 ganglioside, is an experimental therapy for spinal injury3. It has shown sufficient promise in a pilot study to warrant investigation in an ongoing phase III randomized trial. GM-1 is not approved by the Food and Drug Administration for routine clinical use.

Mr. Byrd was apparently treated with GM-1 outside a randomized protocol. This raises many questions. First, are all patients with spinal injuries being treated at the hospital with this experimental drug? If not, did this patient's celebrity status lead to a treatment not offered others? Second, will all patients with spinal cord injuries who are cared for at Lenox Hill Hospital be given GM-1 routinely in the future? Third, what human-subjects protocol was used to justify GM-1 treatment? The patient's approval was apparently, and not surprisingly, received. This however, is only part of the justification required for a compassionate-use protocol.

Furthermore, how will patients who are being asked to participate in placebo-controlled trials of GM-1 react to the treatment of a celebrity with the experimental drug? The implication is that celebrities are above participating in such trials. Moreover, experimental pharmacologic treatments can have untoward complications, including a worsening of neurologic recovery in some situations4,5. Were such risks discussed with this patient? Finally, unlike the patients in the original GM-1 study,3 Mr. Byrd was apparently treated with very large doses of methylprednisolone before receiving GM-1. There are no data to indicate what neurologic outcomes or sequelae might be expected from such a combined treatment protocol. Was the ignorance of physicians about this treatment approach conveyed to the patient?

Participants in controlled clinical trials are the real pioneers in research on spinal cord rehabilitation. I look forward to a future headline announcing that a celebrity has been enrolled in a randomized, controlled clinical study.

Michael B. Bracken, Ph.D.
Yale University School of Medicine, New Haven, CT 06510

5 References

1. 1

   Anderson D. Will mystery drug help Byrd heal? New York Times. December 3, 1992.
   

2. 2

   Drug could help Byrd. New York Times. December 3, 1992.
   

3. 3

   Geisler FH, Dorsey FC, Coleman WP. Recovery of motor function after spinal-cord injury -- a randomized, placebo-controlled trial with GM-1 ganglioside. N Engl J Med 1991;324:1829-1838
   Full Text | Web of Science | Medline

4. 4

   Bracken MB, Shepard MJ, Collins WF, et al. A randomized, controlled trial of methylprednisolone or naloxone in the treatment of acute spinal-cord injury: results of the Second National Acute Spinal Cord Injury Study. N Engl J Med 1990;322:1405-1411
   Full Text | Web of Science | Medline

5. 5

   Bracken MB, Shepard MJ, Collins WF Jr, et al. Methylprednisolone or naloxone treatment after acute spinal cord injury: 1-year follow-up data: results of the Second National Acute Spinal Cord Injury Study. J Neurosurg 1992;76:23-31
   CrossRef | Web of Science | Medline

Author/Editor Response

The above letter was referred to Lenox Hill Hospital, which offers the following reply:

To the Editor: We agree with Dr. Bracken that the articles in the press about Mr. Byrd left the impression that he received celebrity treatment. This, however, was not the case. At the request of the FDA in response to requests by physicians to obtain GM-1 after the publication of positive results of a clinical trial with GM-1 in 1991,1 the Single Investigator-Single Patient Use Protocol for the Treatment of Acute Spinal Cord Trauma with GM-1, an open-label protocol, was set up by the Fidia Pharmaceutical Corporation. This protocol provides access to GM-1 as well as information on safety, since there is no similar drug approved by the FDA to treat spinal cord injury. Approximately 60 patients have been enrolled in this protocol since June 1991. Mr. Byrd was the 54th patient admitted to the open-label protocol, hardly a celebrity status.

In March 1992, while the open-label trial remained in effect, a placebo-controlled study with GM-1 in acute spinal cord injury was begun. The protocol required that only patients admitted to 1 of 21 participating medical centers could enter the controlled study. Mr. Byrd was not admitted to one of the participating centers.

As in any experimental protocol, the patients enrolled in the open-label study gave informed consent and were advised of the potential risks and the experimental nature of the treatment without any guarantee of a favorable outcome. Mr. Byrd, his family, and advisers all took part in extensive discussions of informed consent with his treating physicians.

James A. Nicholas, M.D.
Patrick F. O'Leary, M.D.
Lenox Hill Hospital, New York, NY 10021

Kristjan T. Ragnarsson, M.D.
Mount Sinai Medical Center, New York, NY 10029

1 References

1. 1

   Geisler FH, Dorsey FC, Coleman WP. Recovery of motor function after spinal-cord injury -- a randomized, placebo-controlled trial with GM-1 ganglioside. N Engl J Med 1991;324:1829-1838[Erratum, N Engl J Med 1991;325:1659-60.]
   Full Text | Web of Science | Medline