Michael Levy, MD, PhD, associate professor at Harvard Medical School, spoke about the breakthrough of drugs for NMOSD and the challenges patients face to receive treatment.
This is a 2-part interview. To view part 1, click here.
Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune disease that causes repetitive inflammatory attacks on the optic nerves and spinal cord, with symptoms that can lead to blindness, paralysis, and death. Additionally, patients have reported that pain is among the most common and weakening of the symptoms that negatively impact their mood, mobility, and quality of life.1 Besides the 3 currently available FDA-approved therapies for NMOSD to reduce relapses, other nonpharmacological approaches to treatment have been recommended to patients.
One of those recommended treatments is called scrambler therapy, which is a noninvasive technology that was granted FDA approval in February 2009 for acute, chronic, and postoperative pain.1 Scrambler therapy, a type of transcutaneous electric nerve stimulation (TENS), is one type of approach to care to improve quality of life for patients with NMOSD among other examples such as physical therapy, exercising the body.
In a recent conversation, Michael Levy, MD, PhD, sat down with NeurologyLive® to talk about the current treatments available in NMOSD and the supporting data from the trials. Despite the success of the approved treatments, he spoke about the challenges and unmet needs patients still face to improve their quality of life. Levy, an associate professor at Harvard Medical School, shared his recommendations for other nonpharmacological approaches, especially for patients who may not have access to the approved therapies.
NeurologyLive®: What does the treatment landscape look like for patients with NMOSD?
Michael Levy, MD, PhD: We have three [therapies] that are currently approved. One is called eculizumab, which is a complement blocker. One is satralizumab, which blocks the interleukin-6 receptor, and the other is inebilizumab, which targets CD19 on B cells, and is a bleak B cell depleting drug similar to rituximab. All 3 of them have completed clinical trials between 2014 and 2020. In that time period, they all proved to be very effective in reducing the risk of relapse between 75% and 94% with, I would say, minimal risks. Especially when you compare those risks against the risk of a relapse from neuromyelitis optica (NMO), these drugs are very, very safe. I would say that the main problem with these drugs is access, as they are very expensive. Many patients in the US can't afford them, much less around the world. The job now is trying to get these drugs to aquaporin-4 seropositive patients around the world who need them.
Do you think there are any challenges still despite the treatments being safe and effective?
I’d talked with my patients, blue in the face about these wonderful drugs. The question they asked me as well, “is that going to get me out of my wheelchair now?” The answer is no. Because these drugs are really to prevent the next attack. They don't really do anything to improve regeneration and recovery of the nervous system that was damaged, or the optic nerves and spinal cords that have already been damaged by NMO. These drugs target the immune system and not helping the recovery process. Recovery proceeds in patients with NMO, but it's slow, incomplete, and not very good, to be honest.
I think the greatest area of unmet need in NMO is in treatments that encourage recovery and regeneration of damaged nerve tissue so that people can recover function, their eyesight, mobility and bowel bladder function, and reduce pain from spinal cord damage. There's so much to be done but at least we've been able to prevent the next attack with these wonderful drugs.
Are there any nonpharmacological approaches that you recommend to patients as well?
We recently completed a study using transcutaneous electrical nerve stimulation (TENS), and this is for treatment of pain and spasms in NMO. There's other approaches that we're pursuing, too. For example, using marijuana cannabinoid products and treatments that things like that, which are also pharmaceutical but not pharmaceuticalized. Also, other ways such as exercise, as we are working with a rehab program. Physical therapy also helps to improve not just mobility and strength, but also things like pain and constipation. These are techniques that we're employing. We're basically interested in every method that's available out there to improve the quality of life of our patients, and that includes medications, physical therapy, nonmedical therapies, anything out there.
Have there been any challenges with recruiting patients for clinical trials for those that have NMOSD?
Yes, the first 3trials were all placebo controlled, because there was nothing on the market already. There's nothing to compare to. The FDA insisted on a placebo control to prove that one of these drugs would be effective, that meant recruiting into a trial where your patient could be randomized into a placebo and receive no treatment. We already know how bad relapses are in an NMO and how bad recovery is, so that was a challenge. People had to understand in a fully informed way that they were taking a risk and they did. The result is that we now have 3 FDA-approved drugs and anything in the future can be compared to one treatment that's already effective.
Transcript edited for clarity.