Evaluating the Perspectives of Caretakers and Patients with MSA

Muscular system atrophy (MSA) is a rare, progressive neurodegenerative disorder characterized by the loss of nerve cells in the brain. This degeneration leads to symptoms resembling Parkinson disease, such as stiffness, slowness, and balance problems, along with severe autonomic dysfunction. Without disease-modifying treatments, clinical trials play a pivotal role in exploring potential therapies and rely heavily on the experiences and input of patients and their caregivers.

At the 2025 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held October 5-9, in Honolulu, Hawaii, researchers presented a study aimed toward gathering perspectives of people living with MSA (PwMSA) and their care partners on the design and operational characteristics of a phase 3 trial. Led by Beatrice Yang, MD, lead specialist for patent insights at Lundbeck in Valby, Copenhagen, the study found a consensus amongst interviewees that trial participation requires a significant time, resource, and emotional commitment.

In an exclusive interview with NeurologyLive®, Yang elaborated on these challenges, offering deeper insight into patient and caregiver experiences. Throughout the conversation, she highlighted the burdensome nature of clinical trials, for both patients and caregivers, and spotlighted the concern surrounding placebo treatments. Additionally, Yang emphasized the role technology plays in making trials more patient centered and easier for participants.

NeurologyLive: Provide some insights on your poster presentation, what were the greatest clinical takeaways?

Beatrice Yang, MD: Our poster is about how we at Lundbeck are incorporating feedback from patients and caregivers within the MSA space to inform the design of our ongoing phase 3 (MASCOT) trial (NCT06706622). Conducting MSA trials is very demanding, so we want to get it right. We conducted about 40 in-depth interviews with patients and care partners to really understand their perspectives.

What we learned was that all of them found trial participation very meaningful, but also a major commitment, with trial visits, complex logistics, and travel to the site. Many people are even bound to their homes. That duality was very insightful, it’s meaningful, but also a big burden. We also learned that although many understand the reasons for having a placebo arm, they find it frustrating not knowing whether they’re on placebo or treatment.

To address that, we’ve included an open-label extension, meaning that eventually all participants will receive active treatment. By listening to patients and caregivers, we identified practical changes that we’ve incorporated into our study design. For clinicians, this means more practical and accessible trials for their patients to consider participating in.

Was there anything in your poster that might not be obvious at first glance but offers particularly meaningful insight?

I think our work reminds us that the success of a clinical trial isn’t just about the efficacy of a drug. It’s also about how well you understand, support, and consider the needs of the people making the trial possible — the people living with the disease and their care partners. It’s easy to underestimate the time invested by individuals participating in research. It’s not just the time spent in clinic — there’s also significant time spent preparing, planning, coordinating, and traveling. Especially in a rare disease like MSA, even leaving the house and making sure the facilities along the way are appropriate is a big part of participation.

And adding to that is the caregiver perspective. We’re not just talking about one person — there’s the caregiver and the family. Our research shows that caregivers report a huge burden in all aspects of life. It impacts their social lives, relationships, work, even their mood and cognitive wellbeing. For people participating in a clinical trial, in this case, those living with MSA and their care partners, the time commitment goes far beyond just the trial visits.

How difficult has it been to incorporate caregiver feedback into trials, and why is it so essential today?

Historically, designing a clinical trial has been very research-led, with little or no systematic input from patients or caregivers. That meant we didn’t fully understand the real-life burdens of participation. But now, we’re in a different place and there’s growing recognition that patient-centered design isn’t just “nice to have,” it’s essential. There’s even an expectation from regulators. For example, the FDA in the U.S. has issued the Patient-Focused Drug Development guidance series, which informs us as drug developers how to engage with people living with and impacted by the disease, including caregivers, throughout drug development.

When it comes to clinical trials in general, especially from the biotech and biopharma side, how would you describe the current state of trials? Where are we now, and what challenges still remain?

I would say clinical trials have changed a lot. We live in a different time now, we have telemedicine, which became both encouraged and validated during COVID. People are also more connected and comfortable participating from home. That’s something we must consider when designing modern trials. Again, it comes back to being patient-centered and meeting participants where they are. By understanding their perspective, we can make participation easier and more inclusive. I think patient-focused drug development really got it right: we need to listen to those living with the disease.

From your perspective, what are the lingering unmet needs for MSA?

Well, there’s currently no treatment for MSA. Care is limited to symptomatic and supportive therapies, like speech or physiotherapy, but progression remains rapid. MSA is a quickly progressing neurodegenerative disorder, so patients and their families are still left devastated and the unmet need remains huge.

This is where I hope and believe that lenadogene nolibec and other investigational compounds offer hope to the MSA community. Lenadogene has the potential to be a first-in-class antibody treatment that actually addresses the underlying disease.

Our studies so far have shown promising and consistent trends toward slowing disease progression in MSA. Our confirmatory phase 3 trial is ongoing, so hopefully we’ll know more soon, but beyond treatments, we also need earlier diagnosis and better biomarkers.

Is there anything else you’d like to add about this topic or to the state of therapeutic development overall?

I’d really like to take this opportunity to thank all the people who participated in clinical trials because the progress we’re making simply wouldn’t be possible without them. For example, the interview study we did for this poster, learning directly from patients and caregivers, fed straight into our trial design. Hopefully this sets a precedent for how to design more patient-inclusive trials within neurology going forward.

Transcript edited for clarity. Click here for more MDS 2025 coverage.