In a first for Monash University and The Alfred, researchers have received an orphan drug designation by the European Medicines Agency (EMA) for sodium selenate in the treatment of progressive supranuclear palsy (PSP), and are needing patients with the debilitating disease to test the drug.
PSP is a rare neurological condition that affects walking, balance, eye movements and swallowing, and is caused by a protein build-up in the brain called tau.
The phase 2 randomised controlled clinical trial uses sodium selenate as a world first initiative as a disease modifying treatment for PSP, and involves patients having a PET scan to measure tau in their brain.
Received from the European Medicines Agency, orphan drug designation is granted when a drug shows promise in the treatment of a rare disease, and sodium selenate has shown promise in clearing tau build-up in preclinical testing.
PSP is often misdiagnosed as Parkinson’s disease, and currently there is no cure, or any specific treatments available. PSP is a fatal illness, with current survival rates ranging from five to eight years from the onset of symptoms.
Lead researcher Dr Lucy Vivash, from Monash University’s Department of Neuroscience, and The Alfred’s Department of Neurology, said this clinical trial is among the few worldwide focusing on this disease.
“The trial is investigating whether our drug, sodium selenate, reduces disease progression in patients with PSP,” Dr Vivash said.
“There are currently no treatments that alter the underlying cause of PSP, accumulation of the toxic protein tau. Our drug acts to prevent or even reverse the build up of this protein, which we hope will halt the progression of symptoms in participants treated with the drug. Given this is a rare disease, new treatments, and treatment trials are rare, we need participants in order to find a treatment and one day a cure.
“Beyond investigating the drug, we also hope to be able to use the data collected in this study to better understand PSP, to improve diagnosis as well as identifying potential new treatment targets.”
Ramila, a former kindergarten teacher and participant in the study said, “the care and support I receive from the trial staff is amazing.”
“I have enjoyed the social interaction and meeting new people.”
The trial needs participants, (aged 40 years or over), who have been medically diagnosed with PSP. The trial is being run at six participating locations in Melbourne, Brisbane, Sydney and Adelaide.
