A multi-drug clinical trial offers renewed hope for patients with progressive supranuclear palsy (PSP), a rare and currently incurable neurodegenerative disease. Led by UC San Francisco (UCSF) and conducted across up to 50 sites nationwide, the trial will simultaneously evaluate three different drugs, with the potential to add more therapies as the study progresses. The goal is to identify treatments that can slow or halt the progression of PSP, which typically leads to death within seven years of symptom onset.
The trial is supported by a five-year grant of up to $75.4 million from the National Institute on Aging (NIA), part of the National Institutes of Health (NIH). According to Julio Rojas, MD, PhD, of the UCSF Department of Neurology, Memory and Aging Center, and a principal investigator of the trial, the study has the potential to "transform the type of care that patients with PSP receive." He added that even a modest slowing of disease progression, such as 20% or 30%, would represent a significant improvement for patients.
Understanding PSP and the Innovative Trial Design
PSP is characterized by the buildup of tau protein in the brain, leading to the weakening and death of brain cells. The condition, which affects approximately 30,000 Americans, is often misdiagnosed as Parkinson's disease. The most common form of PSP is Richardson’s syndrome, which presents with symptoms including slowness, stiffness, balance issues, and difficulty with eye movement.
The clinical trial employs a platform design, similar to that used in amyotrophic lateral sclerosis (ALS) research. This approach allows for the continuous evaluation of new therapies, reducing the time and cost associated with traditional clinical trials. Adam Boxer, MD, PhD, a principal investigator in the trial, explained that this design offers more opportunities to identify effective treatments faster and with less burden on participants. Patients in the PSP trial will have a 75% chance of receiving an active drug, and all participants will have the opportunity to receive a drug after one year.
Focus on Diversity and Collaboration
The trial places a high priority on enrolling participants from diverse and underrepresented populations. Researchers plan to engage with Spanish-speaking and African American communities, providing services such as Spanish-speaking clinicians and neuropsychological assessments in Spanish. Practical and financial barriers to participation will be addressed by covering transportation and hotel costs.
The nonprofit organization CurePSP is collaborating with UCSF and other trial sites to facilitate participant recruitment. Kristophe Diaz, PhD, Executive Director and Chief Science Officer of CurePSP, emphasized that the trial represents a "pivotal step forward" and brings renewed hope to patients and families affected by PSP.
Enrollment is anticipated to begin in the fall of 2025 and will be open to patients with Richardson’s syndrome who have experienced progressive symptoms for fewer than five years and are accompanied by a care partner.
