Clinical trial in Buffalo to test 'liberation treatment'
Published Monday, June 28, 2010 3:20PM EDT
Researchers at the University at Buffalo are about to launch North America's first clinical trial to test the "liberation treatment," an experimental therapy designed to halt the symptoms of multiple sclerosis.
The landmark prospective, randomized, double-blinded study will test both the safety and efficacy of the endovascular therapy on MS symptoms and progression.
The researchers will examine whether dilating blocked neck veins in MS patients using angioplasty remedies the blockages and improves MS symptoms or progression.
The study follows the work of Dr. Paolo Zamboni of the University of Ferrara, Italy, who theorizes that blocked neck veins slow the outflow of blood from the brain and contributes to MS. He's called the vein condition CCSVI, or chronic cerebrospinal venous insufficiency.
The first leg of the University at Buffalo trial will take two days and will begin Tuesday. Ten patients have already been selected to receive the treatment. They will then be followed for 30 days of analysis to measure the side effects and possible risks of the procedure.
The study will be led by Dr. Adnan Siddiqui and colleagues at the University at Buffalo's Department of Neurosurgery.
Depending on the results, researchers will then embark on Phase 2, which will randomize another 20 MS patients to undergo either angioplasty or a "sham angioplasty" (i.e. a catheter will be inserted but there will be no inflation of the balloon).
Hospital officials say the treatment will be blinded and performed in a way that "neither the patient undergoing the procedure nor the clinicians evaluating the patient will be aware which procedure was performed."
If results suggest the procedure is safe and effective, researchers will apply to study a larger number of patients.
Meanwhile, editors of the Canadian Medical Association Journal have joined the growing debate over CCSVI and demands by patients for rapid access to treatment.
CMAJ Editor-in-Chief Dr. Paul Hebert and Deputy Editor Dr. Matthew Stanbrook note that several MPs in Ottawa have recently lobbied the federal government to invest in research into the controversial treatment. They note, as well, that patients have held well-publicized demonstrations, demanding access to the liberation procedure -- which they applaud.
"Patients should insist on evidence. They should also insist on having their views represented when decisions about where to spend research dollars are made," the editors write.
But they add that research funding should be allocated based on evidence, not political or patient pressure.
"Good health policy decisions should not be based on hope and desperation," they write. "Equally, scientists and skeptics should avoid discounting novel ideas prematurely. It is precisely the unexpected scientific discovery that often leads to major advances in care."