Published July 30, 2010
Buffalo medical researchers led by a team from the UB Department of Neurosurgery have embarked on a landmark prospective randomized double-blinded study to test the safety and efficacy of interventional endovascular therapy—dubbed “liberation treatment”—on the symptoms and progression of multiple sclerosis (MS).
Recent research has strongly associated chronic cerebrospinal venous insufficiency (CCSVI) with MS.
In a series of original studies, Paolo Zamboni, MD, of the University of Ferrara, Italy, found blockage of major venous outflow from the brain and spinal cord in patients with MS. Researchers from many institutions, including UB, have confirmed the association.
It is hypothesized that the narrowing in the large veins in the neck and chest might cause improper drainage of blood from the brain, resulting in eventual injury to brain tissue. It is thought that angioplasty—treatment commonly used by cardiologists and other endovascular surgeons to treat atherosclerosis—may remedy the blockages.
Zamboni has conducted preliminary studies that suggest the efficacy of venous angioplasty—“liberation procedure”—in the amelioration of MS symptoms.
The new study launched by UB researchers will explore whether correcting the blockages using endovascular intervention (balloon angioplasty) will improve MS symptoms or progression.
Called PREMiSe (Prospective Randomized Endovascular therapy in Multiple Sclerosis), the study is believed to be the first of its kind performed with Institutional Review Board (IRB) approval in the US. The IRB approval process ensures that there has been a careful determination of risks and benefits and that significant safeguards are in place.
PREMiSe is led by principal investigator Adnan Siddiqui, MD, assistant professor of neurosurgery, with coprincipal investigators Elad Levy, MD, associate professor, and L.N. Hopkins, MD, professor and chair of the UB Department of Neurosurgery.
Additional independent researchers from UB are participating in the evaluation and follow-up of study patients. An independent Data Safety Monitoring Board is ensuring the safety and effectiveness of the study on an ongoing basis.
In the first phase of the study, which took place June 29 and 30, ten MS patients from the United States and Canada who exhibit venous insufficiency underwent minimally invasive venous angioplasties to determine if the procedure can be performed safely.
In its second phase, the study will randomize 20 MS patients who will undergo either venous angioplasty or a “sham angioplasty” (i.e., a catheter will be inserted but there will be no inflation of the balloon). The treatment will be blinded in such 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 an appropriate safety profile and preliminary effectiveness, researchers will ask UB’s IRB for an extension of the protocol to study a larger number of patients in order to convincingly prove or disprove a causal relationship between CCSVI and MS.
Multiple sclerosis is estimated to affect more than 400,000 people in the United States and more than two million people worldwide. It is typically a disease of young adults characterized by either a relapsing or progressive decline in neurologic function resulting in significant disability. It is an inflammatory neurological disease widely considered to be autoimmune in nature, though its exact origins remain elusive.
If angioplasty is shown to be effective at improving MS symptoms, the implications for the future of MS treatment could be monumental. The physicians conducting PREMiSe are cautious but optimistic that initial findings will be promising.