I have PPMS. I was diagnosed 8 years ago. Three years ago my neurologist put me on Gilenya. I took it for 18 months but had no results positive or negative so I was told to discontinue. Now I am seeing a new neurologist who after one visit wants to change my diagnosis to RRMS so he can prescribe Tecfidera (He also travels and speaks on behalf of the drug company). I am skeptical to begin a new drug for RRMS when I am sure I have PPMS. Any advice on how to speak with my neurologist?
Great question. This is the bind we find ourselves in today with all the medications approved for relapsing MS. The truth of the matter is that very few patients who we see in clinical practice, even those with relapsing remitting MS, would even qualify for the clinical trials that are done to get the drugs approved by the FDA. This means that we are stretching the indication for treatment in many patients and using our professional judgement. There is nothing wrong with this and the regulatory authorities permit this approach to practice. Often this experimentation in every day practice leads to new discoveries such as the use of fumarates (like Tecfidera) for MS. But when you take this approach to treatment it is important to be open with patients about what is known and not known.
Generally, all doctors must establish their boundaries for treatment recommendations. For instance, if I saw a woman with optic neuritis associated with good recovery and a virtually normal MRI scan and spinal fluid evaluation, I would not recommend a DMT, but would continue to monitor the patient . Similarly, if I saw a patient with many years of secondary progressive MS who has not benefited from prior DMTs, I may even discontinue therapy depending on the circumstances.
The evidence suggests that primary progressive patients do no respond adequately (in a clinically meaningful way) to most disease modifying therapies. One exception seems to be that younger patients (under age 50) with PPMS may respond to rituximab. But until the studies are done we often use available drugs in the hope that patients may benefit. Gilenya is a good case in point for you; when you started on this drug, we did not know the results of the Gilenya primary progressive clinical trial. Now we know there was no benefit.
I typically recommend that patients with primary progressive MS participate in clinical trials when they are available to them. This is definitely the best approach to finding meaningful treatments for you or other patients in your circumstance. Discuss this with your doctor or check out available clinical trials at clinicaltrials.gov
Revere (Rip) Kinkel MD
Director of the Multiple Sclerosis Program
Professor of Clinical Neurosciences
University of California San Diego