Progressive supranuclear palsy (PSP) is a devastating neurodegenerative disease. Symptoms are serious, ranging from motor impairments to dementia. The life expectancy for patients with PSP is only 5-7 years after symptoms start, and the disease develops randomly, with age being the only established risk factor.
PSP is caused by a buildup of the protein tau in certain brain regions, particularly those concerning movement. Tau is a protein that binds to the compound phosphate, but in PSP, tau binds to too many phosphates, protecting it from breaking down. This is harmful to our brains, which must clear tau to prevent buildups that can lead to disease. Neurodegenerative diseases caused by tau accumulation are collectively known as tauopathies. PSP is distinct from other tauopathies due to differences in the type of tau proteins and specific brain regions affected, among other features.
Unfortunately, treatment options are limited. PSP is currently incurable, with known treatments intended to help with symptoms rather than reverse the disease. Further, medications are generally ineffective at even treating symptoms, although some non-drug treatments like exercise may improve quality of life and safety. It has been difficult to develop effective treatments due to a number of factors – such as its quick progression and its frequent misdiagnosis as Parkinson’s disease (which has similar symptoms) – that make PSP difficult to study.
Thankfully, a 2025 clinical trial by the University of California, San Francisco, might get us closer to finding effective medications. The uniquely-designed trial will test three drugs simultaneously to reduce the time taken to find a treatment. The study will also immediately stop testing any therapies proven to be ineffective and replace them with other drugs as needed. Given the number of test subjects in this study, which will take place at 50 sites nationwide, only three drugs can be tested concurrently.
The upcoming clinical trial is bringing newfound hope to PSP patients.
Image Source: AzmanJaka
The trial follows a period of renewed interest in PSP; researchers are excited about how tau-related treatments for PSP might inform treatments for other, more common tauopathies, like Alzheimer’s disease. This and the fact that non-tau-related medications have been unsuccessful are reasons why only tau-related medications will be tested in this trial.
The researchers will publicly release the trial’s data, which is also innovative since such data is historically considered confidential. They hope other PSP researchers can use the data to help in developing more PSP treatments.
This clinical trial marks a step forward in the fight against PSP. Its innovative design gives it the potential to quickly identify treatments that could enhance the quality and length of life for patients. If successful, it could change the grim outlook for PSP patients and lead to a brighter future for them and their loved ones.
Featured Image: Nano Photos
