CurePSP Publishes Voice of the Patient Report Following February EL-PFDD Meeting

CurePSP has published the Voice of the Patient Report, the formal summary of everything gathered at our February 6 Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting on PSP. Drawing together prerecorded testimonies, live community discussion and written feedback, the milestone report offers a comprehensive picture of life with PSP and the urgent unmet therapeutic needs. The report is intended for the FDA, other government agencies, regulatory authorities, medical product developers, academics, clinicians and anyone else working toward a future with better treatments for PSP.

It is dedicated to the members of the PSP community already lost to this disease, and made possible by those living with PSP and their care partners whose testimony fills its pages.

More than 200 community members joined the meeting virtually, with over 20 sharing testimony directly by phone or video. An additional 90 written comments were submitted from our global community of people impacted by PSP.

Many of the findings will be familiar to anyone who has lived with PSP. Poll respondents reported an average of 8.5 different PSP-related health concerns each, a reminder that the disease often shows up as a cascade of symptoms. Balance and mobility problems topped the list, with 97% of respondents reporting falls, closely matched by choking and swallowing difficulty and by speech loss. The community described the profound social isolation that can result from these symptoms, both for the person living with PSP and their care partner.

The community also described the urgent unmet therapeutic needs, with current treatment options limited to symptom management. No approved therapy slows or stops PSP's progression, and most respondents said their current medications and therapies work only "somewhat" or "very little."

Short of a cure, what the community wants most is treatment that actually changes the disease's course. Physical, speech and occupational therapy remain the tools people rely on most, and Parkinson's medications the most common drug tried, but families were candid that these only offer management, and often just temporarily. Community members also indicated interest in treatments that would maintain or improve the ability to swallow, communicate, move, balance or remain independent as long as possible.

Clinical trials came up almost as often as treatment itself. Written comments were full of families volunteering to take part. "There are no barriers, as we would love to participate in a trial," one care partner wrote. Another said their family would do anything to take part in research that could spare others what they've been through.

Alongside that willingness came specific asks: more trials, trials open to every stage of PSP, not just those newly diagnosed, trials open to all subtypes of PSP, trial sites people could actually get to and honest answers about what participation would mean for daily life, not just for the data. The potential benefits of a trial would need to outweigh risks.

The community’s feedback is meant to directly shape how research moves forward. “Hearing the daily impacts of PSP directly from the community ensures that their lived experiences and priorities are incorporated into the drug development process,” said Jennifer Brummet, PhD, Director of Scientific Affairs and Partnerships at CurePSP.

For CEO Kristophe Diaz, PhD, the report's value extends beyond documentation. “The Voice of the Patient Report is a roadmap for better drug development,” said Dr. Diaz. “It gives researchers, regulators and medical product developers direct insight into what people living with PSP and their care partners experience daily and what meaningful treatment success looks like from their perspective. As more therapies enter development, integrating these lived experiences into every stage of the process will be essential to delivering treatments that truly matter.”

The Voice of the Patient Report is now available on CurePSP's website and will also be published on the FDA's website, where it becomes a lasting resource for the agencies, researchers and drug developers working to bring PSP treatments to the people who need them.

Thank you to our community for participating in this event. We are also grateful for our sponsors, Ferrer, GemVax & Kael, and Novartis, that made this project possible.

Read the full report