TORONTO—Like electrical wires, neurons are insulated. But in multiple sclerosis (MS), the insulation (called myelin) is stripped or worn down, slowing conduction along the axons—the wires of the nervous system. MS is widely considered to be an autoimmune disease, in which the myelin is attacked by the body’s own T cells. But Paulo Zamboni, director of the Vascular Diseases Center at the University of Ferrara in Italy, thinks a blockage in the veins that drain blood from the brain is the part of the problem. In his pilot study, published December 2009 in The Journal of Vascular Surgery, MS patients who had such blockages (called chronic cerebrospinal venous insufficiencies) fixed surgically, including Zamboni's wife, showed a significant improvement.

News of the treatment traveled fast and MS patients around the world, eager for a remedy, began asking for the experimental procedure. But doctors outside the study remain skeptical. The main limitation to Zamboni's pilot trial is that it was "open-label," meaning patients knew they were receiving the treatment and so did the researchers. And when hopes are high in both parties, bias can occur. But despite doctors' cautionary words about Zamboni's preliminary findings, some patients have journeyed to clinics in Poland and beyond and paid more than $10,000 for the procedure.

Zamboni's controversial treatment, which he presented here as an invited speaker April 14 at the American Academy of Neurology's annual meeting, highlights the need for properly designed clinical trials in which patients are randomly assigned to treatment and placebo groups and both patients and researchers are unaware of who has received treatment or placebo. Promising results from open-label pilot studies can prompt patients, desperate for a cure, to seek out risky and costly experimental treatments that might not work as expected.

Image: iStockphoto/dpmike