Upcoming UCSF Trial to Evaluate Effectiveness of Three Drugs for PSP

An upcoming clinical trial conducted by the University of California, San Francisco (UCSF) will simultaneously evaluate multiple drugs to determine their effectiveness against progressive supranuclear palsy. The trial has been designed with an emphasis on diverse enrollment and will offer financial support to participants.
The study is being funded by a five-year grant of up to $75.4 million from the National Institute on Aging.
Evaluating Drugs for Other Disorders in PSP
Progressive supranuclear palsy (PSP) is one of two FTD disorders that primarily affect the motor cortex, causing symptoms centered around movement difficulties. While there are currently no approved treatments for PSP, experimental drugs are being tested for disorders like ALS or Alzheimer’s disease. PSP is primarily driven by dysfunction of the tau protein, which also plays a crucial role in the development of Alzheimer’s and ALS, meaning that drugs developed for these disorders could potentially have an effect against PSP.
Scientists at UCSF will conduct what is known as a “platform trial,” a type of clinical study where multiple treatments are evaluated side by side against the same control group (which will receive a placebo in the UCSF study). Researchers have noted that at least three therapies will be reviewed over 12 months, with a potential 12-month open-label extension where participants who initially received a placebo will be given active treatments. The trial will be open to people diagnosed with Richardson’s syndrome (PSP-RS), which makes up 70% of diagnoses, with enrollment expected to begin in fall 2025.
“Unlike typical clinical trials, platform trials can remain open with multiple new therapies tested in successive cycles if the first ones don’t work,” said Adam Boxer, MD, PhD, a principal investigator of the trial. “This means there are more opportunities to identify effective treatments in a faster timeframe, with lower cost and less burden to participants. Patients on the PSP trial will have a 75% chance of treatment with an active drug, and after one year, all participants will have the opportunity to receive a drug.”
The effectiveness of the drugs will be determined using clinical measures such as the PSP Quality of Life scale. In similar studies, drugs are considered effective if scores indicate that people treated with experimental drugs slowed or halted diseased progression. Cerebrospinal fluid samples will also be collected to evaluate changes in PSP-associated molecules and help identify new biomarkers.
UCSF Focuses on Enrollment from Underrepresented Groups
Researchers are increasingly calling attention to the need for greater inclusion of underrepresented groups in FTD research. Because many existing studies are based on cohorts primarily made of white Westerners, there is a disconnect between culture/language and best care practices, which can make providing care more difficult. Changes in cultural context can change how FTD symptoms present, and many current diagnostic tools and interventions lack adaptations to account for this.
However, UCSF noted that a core focus of the study was the inclusion of participants from communities who have been underrepresented in previous studies.
“We will build on experience from other community-engaged research programs and develop relationships with Spanish-speaking communities,” said Julio Rojas, MD, PhD, a principal investigator. “These participants would be served by Spanish-speaking clinicians, including a neuropsychologist conducting assessments in Spanish. Similar efforts will be made in the African American and other medically underserved communities.”
The study plans to eliminate barriers to participation for underrepresented groups by covering hotel and transportation costs to and from study sites. UCSF and other trial sites are collaborating with CurePSP, who will assist in recruiting participants for the study.
FTD researchers are bringing experimental interventions and diagnostic tools to the clinical trial stage at an increasing pace. If you or a loved one are interested in helping drive FTD research, visit AFTD’s Studies Seeking Participants page to find opportunities to participate. The FTD Disorders Registry can help you keep up to date on the latest research opportunities and let you contribute your lived experiences of FTD to guide research efforts.
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