I have RRMS. I don't want to take medications. What's the biggest risk if I don't?
Disease modifying therapies (DMTs) for MS are 1) expensive, 2) associated with a variety of side effects and potential safety risks, and 3) are partially effective. With this understanding, your question is one I hear frequently in clinic. The major risk of deferring DMT for relapsing-remitting MS is an increased likelihood of clinical relapses in the future that can disrupt your day-to-day function, and short- and long-term disability. Let me explain…
We have a great deal of natural history data on MS (this is information on the average experience of patients with MS who were not exposed to therapy). This information is available to us because it wasn’t until the mid-1990’s that we had our first approved DMT for clinical use. What we have learned is that patients with early and frequent clinical relapses are at a higher risk of developing walking disability (for instance, the average individual with greater than 5 clinical attacks in the first 2 years of MS will need a cane to walk by 6 to 7 years into their disease course; while the average patient with 0 or 1 clinical attack will need a cane by the 20 year mark). We have also learned that the initial MRI brain scan after the first clinic event of MS can tell us about the likelihood of disability in the future—the more scars of MS that are seen on this first scan, the greater the probability for disability sooner rather than later. Keep in mind that these numbers reflect MS patients who did not have DMT options.
At the time of this blog post, we have ten FDA-approved DMTs, with more being reviewed. In nearly every case, these therapies have repeatedly shown effectiveness in 3 main measurements: 1) less clinical relapses or attacks compared to placebo, 2) reduced number of new, enlarging, or enhancing scars on the brain MRI scan, and 3) a reduction is short-term disability compared to placebo.
Critics have long pointed out that the DMTs are not cures, are expensive, have variable safety risks, and have not been proven to make a difference in the long-term. While it is true that all DMTs are partially effective—meaning that the data does not show that they cut down new relapses, MRI lesions, and disability by 100%—they have recently demonstrated effectiveness in the long-term. In 2012, a paper was published in the journal Neurology that presented long term follow-up data on the first approved DMT called betaseron. This 21-year study followed the originally randomized study participants (an early treatment group that was randomized to betaseron from the beginning, and a late treatment group that comprised individuals that were originally randomized to placebo but were given the opportunity to take betaseron after the initial clinical trial ended 3 years later). This paper showed that there were more people alive from the early treatment group 21 years later compared to those in the late treatment group. You can read more about this at http://msj.sagepub.com/content/19/5/522.full. This study confirmed our suspicions that DMT does help in the long term and does underscore the need to treat early rather than waiting—because there appears to be a window of opportunity to alter the course of the disease for the better.
Many of the barriers to initiating a DMT for relapsing-remitting MS patients have been removed. There are financial and co-pay assistance programs in place to make DMTs affordable for patients, risk mitigating protocols for DMTs, and experienced MS clinicians that can help you in choosing a reasonable DMT that can fit your goals and lifestyle, and outline a plan to make sure the medication is doing what we expect it to do and make it tolerable for the patient.
While the majority of my patients ultimately decide to start a DMT after considering this information, some choose to defer treatment. That is certainly each individual’s prerogative. Before making the decision, everyone should recognize the fact that we do not yet have a therapy that can reverse or repair tissue damage already incurred due to MS inflammation, nor do we have a therapy at the moment that reverses sustained disability. What we are left with are DMTs whose purpose is to get in front of the disease and impact it for the better, and hopefully avoid significant disability in the future.