Addressing Unmet Needs of Narcolepsy Beyond Medication


Nancy Foldvary-Schaefer, DO, FAAN, director of the Sleep Disorders Center and staff in the Epilepsy Center at Cleveland Clinic, discussed the recent advancements in narcolepsy treatments and the importance of incorporating a holistic care approach for patients.

Nancy Foldvary-Schaefer, DO, FAAN, director of the Sleep Disorders Center and staff in the Epilepsy Center at Cleveland Clinic

Nancy Foldvary-Schaefer, DO, FAAN

Narcolepsy and idiopathic hypersomnia are chronic sleep disorders that negatively impact a patient’s alertness, mental and physical energy, functioning, as well as their quality of life.1 The current available treatment options for narcolepsy often do not meet the needs of the patients because of suboptimal efficacy, troublesome adverse events, development of treatment tolerance, and inconvenience.2 Over the last decade, the understanding of the neurobiology of narcolepsy has greatly improved with research, with progress slowly being made for additional therapeutics options for patients.

Low-sodium oxybate (LXB), an FDA-approved for treatment of cataplexy or excessive daytime sleepiness, is a promising long-term treatment for patients living with narcolepsy and idiopathic hypersomnia. In recent phase 3 clinical trials assessing LXB, the therapy demonstrated a consistent safety profile with that of sodium oxybate (SXB; Xyrem; Jazz Pharmaceuticals) in narcolepsy.2 In addition, findings revealed that the treatment could potentially have positive effects in multiple domains of functioning and quality of life, especially for younger patients with the condition.

Nancy Foldvary-Schaefer, DO, FAAN, director of the Sleep Disorders Center and staff in the Epilepsy Center at Cleveland Clinic, recently sat down in an interview with NeurologyLive® to discuss developments in narcolepsy treatment that have generated excitement among patients and families. She talked about how the emerging therapies in the field are aiming to improve the quality of life for patients with the condition, and the challenges that patients still face in their daily lives. In addition, Foldvary-Schaefer spoke about having a holistic approach to care, including the incorporation of psychological and behavioral support, that should be considered essential for these patients.

Clinical Takeaways

  • In the field of sleep disorders, narcolepsy treatments have experienced promising advancements with new approved therapies and clinical trials for improved therapies.
  • While a cure for narcolepsy remains elusive, the field is focused on enhancing patients' quality of life and energy conservation through these new emerging therapies.
  • Holistic care, including psychological and behavioral support, is recognized as a critical component in addressing the unique needs of patients with narcolepsy and idiopathic hypersomnia.

NeurologyLive: How would you assess the state of treating narcolepsy, based on your own clinical practice?

Nancy Foldvary-Schaefer, DO, FAAN: I think patients and families are very excited to see what the scientific community has been doing in recent years. In the last few years, we've seen several new drugs being studied and throughout the pandemic at Cleveland Clinic, we saw an increase in the number of new patients coming to our center. We've seen a couple of new medications FDA-approved for narcolepsy, and also for cataplexy. Our center also has participated in clinical trials for additional medications for the sleep disorder. At our center, we had an increase in the number of patients, a 3-fold greater, in those participating in trials. This is because patients are looking for new therapies, for places that have access to clinical trials, and looking for clinicians who have experience with some of the newer agents. I think all of that speaks to the fact that we're not there yet with therapies for narcolepsy to reduce sleepiness and improve other symptoms of the disorder.

We know that the medicines available don't normalize the brain of patients with narcolepsy and we don't have a cure. We're aiming to symptomatically make patients feel as close to normal as possible. We know that we're really not there yet. I think this is a very exciting time right now for patients and their loved ones because there's more new and emerging therapies coming into the field.

What else do you think is still an unmet need for these patients and what are you looking forward to in terms of treatments in the next coming months?

I would say that all brains are not alike, all patients with narcolepsy are not alike. Even though we have a few more drugs on the market, I continue to see patients who often don't feel that some of the newer drugs are working that well or that the old drugs don’t work that well. I know we at least we have challenges in Northeast Ohio with some of the insurance companies that really want patients to have used the generic drugs before we can get them newer brand name drugs. I think that's a challenge for both providers and for patients even with some of the major advances with the oxybate medications, and with pitolisant (Wakix; Harmony Biosciences), the first histamine receptor acting drug. There's still opportunity to find other mechanisms of medications that even further enhance wakefulness and treat the disturbed nighttime sleep that patients with narcolepsy experience with the disorder.

I think one of the real unmet needs in this patient population is having a therapy that improves quality of life as well as energy conservation among patients with narcolepsy. I know this because I'm collaborating with one of my colleagues, Alicia Roth, PhD, who is trained in behavioral sleep medicine, and started to do focus groups with our patients. She aimed to understand what support this population really needs, comparable with the cognitive behavioral therapy that is so prevalently given to patients with insomnia. She's piloted a group with cognitive behavioral therapy for hypersomnia instead of cognitive behavioral therapy for insomnia. She did this a little bit in line with what colleagues at Northwestern [University] have promoted as an additional therapy to address the challenges of getting through a day and planning your next day. I think this is very problematic for patients with hypersomnia. She's working on developing some interventions to treat that gap and I think that's very interesting.

I'm really privileged, personally, to be able to provide medical care for these patients. I have a psychiatrist in our group who can address comorbid mood disorders, which are very common among this patient population. Then I have Roth and her colleagues in behavioral sleep medicine that can provide some sleep, behavioral therapy for these patients. The patients really need more than a medication prescription and treating them holistically requires that we address these other things. I think we haven't done that so well in the past as we've done with other chronic disease populations, treating them in a holistic way with experts of different backgrounds coming together.

I'm really looking forward to Roth’s work. I think that will be a real novel, game changer for patients with hypersomnia who recognize that they're missing something in their quality of life and need some support beyond traditional medical therapy.

For specialists who see those with narcolepsy, what else would you like to mention?

I would tell them to think about the patient holistically because there's only so much that we can do on wake promotion. We've done better but there's no doubt that there have been significant scientific advancements in enhancing wakefulness as well as enhancing sleep quality. I think that many patients still struggle, particularly my younger patients. These are the ones that are older teens, and 20s, and 30s, who are still really trying to find their way from completing their education to employment. They are still trying to figure out what they can do realistically. In that regard, I'm so happy to have psychiatry support and psychology support. I think that's, at least for me, been the missing piece that has come together in our center nicely.

I think all patients with narcolepsy should have a psychosocial assessment. We should make sure we're optimizing for any mood disorders such as depression and anxiety because these things just get more complicated when we're using the wake-promoting medications to wake people up. I've had great experience collaborating with those types of individuals and oftentimes, it's those things that I found. It's the mood or the behavioral strategy that the patient needed. I was doing maybe all that was reasonable for a neurologist, using a week promoting therapy, but it was these other things that we had opportunities to help the patient better. I think patients really appreciate that and their families.

I think that education in general such as, employer education and teacher education, are critical things because these patients want to have the best quality of life. They want to outperform their colleagues who don't have narcolepsy, but they struggle. Every time I see a younger patient who's still in school or who's transitioning to school, I offer to provide some documentation. They can share the document with employers or deans of schools to give them some accommodation if that's appropriate and to provide a little bit of education for those individuals. This is done so they don't make the mistake of thinking these patients are lazy because they're not getting work done on time, or they need a little bit more time, or they're a little bit late. I think those types of things are critically important so that patients can feel that they have a shot, and that they're getting the support they need to succeed.

We’ve been talking about narcolepsy, but this also applies for patients with idiopathic hypersomnia since it is very similar in challenges and patient needs. Similarly, it is a delayed sleep phase disorder and young patients with the condition need a little bit more to be able to succeed in their life. Very often, their teachers and employers are very receptive and at first didn't understand and so providing a little bit of education goes a long way.

Transcript edited for clarity.

1. Schneider LD, Morse AM, Strunc MJ, Lee-Iannotti JK, Bogan RK. Long-Term Treatment of Narcolepsy and Idiopathic Hypersomnia with Low-Sodium Oxybate. Nat Sci Sleep. 2023;15:663-675. Published 2023 Aug 19. doi:10.2147/NSS.S412793
2. Wozniak DR, Quinnell TG. Unmet needs of patients with narcolepsy: perspectives on emerging treatment options. Nat Sci Sleep. 2015;7:51-61. Published 2015 May 22. doi:10.2147/NSS.S56077
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