Patient advocates—often suffering from incurable diseases—have become increasingly influential in pushing for the development of new therapies, and that influence has been multiplied by the internet’s ability to quickly spread information and connect like-minded individuals. Conversely, physicians and scientists, who have seen therapies come and go, tend to harbor an innate skepticism toward newly proposed treatments.
The two groups often find themselves at odds, with patients citing a lack of compassion and understanding of the urgency for treatment and physicians insisting patients don’t comprehend the importance of conducting rigorous science to prove safety and efficacy before offering a treatment. This oft-repeated scenario is playing out in the endovascular world surrounding a controversial issue: The existence of chronic cerebrospinal venous insufficiency (CCSVI) as a proposed cause of multiple sclerosis (MS), and the use of endovascular therapy to treat it.
Among the featured speakers at next year’s ISET meeting (Jan. 16-20, 2011) is Paolo Zamboni, MD, the Italian vascular surgeon who first proposed and is now testing the CCSVI theory. The Town Hall gathering will include patient advocates, skeptics and clinicians who were initially doubtful, but eventually were convinced by desperate patients to provide the therapy. All will be discussing Zamboni’s contention that stenoses of the principal pathways of extracranial venous drainage are strongly associated with MS, symptoms of which may be alleviated through endovascular therapy, including percutaneous transluminal angioplasty and the placement of stents.
It’s easy to dismiss patient advocates as naïve and unwilling to consider the risks and lack of scientific proof of the validity of a therapy. Let’s not forget, however, patient advocacy can be an extremely important contributor to the advancement of medicine. An excellent example is that of uterine fibroid embolization (UFE), a proven therapy that was entirely patient advocacy driven. In the case of CCSVI, there is no doubt that research would not be moving forward without the push from patients. Whether it bears out remains to be seen.
In the meantime, we must continue to stress the importance of conducting good science. A therapy—even one eventually proven to be beneficial—can get ahead of itself. For example, many early UFE patients were treated without the benefit of data, before the most optimal approach was scientifically established.
It’s important to recognize that MS patients, given few options and offered no cure, are often desperate for something, anything that might provide some relief. And they’re not naïve or ignorant of the scientific process. Smart, educated advocates have formed CCSVI Alliance, a 501(c)(3) not-for-profit organization that includes a scientific advisory board.
Clinicians must be compassionate with patient advocates, in this case, helping MS patients understand that the CCSVI diagnostic workup is extensive and expensive (and is not covered by insurance), there is no proof at this point that treatment works and the treatment—even if it is proven to be beneficial—comes with risks and may not work for everyone.
It’s also important to remember that while the initial skepticism for new therapies often is borne out (laetrile cancer treatment and left ventriculoplasty for cardiomyopathy), sometimes it is not (hand washing before surgery to reduce infection and the use of “Lorenzo’s oil” in the treatment of presymptomatic adrenoleukodystrophy patients).
Patient advocacy is here to stay. For the benefit of all, we must learn to work together, balancing compassion with skepticism, supporting the science that will give us the answers we all crave and appreciating that pioneers often must swim upstream.
Dr. Katzen is course director of the International Symposium on Endovascular Therapy (ISET) and founder and medical director of Baptist Cardiac & Vascular Institute in Miami, Fla.