Phase 1 Trial Set to Begin For Experimental Therapy For Multiple System Atrophy and Progressive Supranuclear Palsy

According to a story from, the company Prana Biotechnology Ltd recently announced that it is beginning the recruitment process for its Phase 1 clinical trial of its investigational product PBT434. This Phase I trial will evaluate the safety and tolerability of PBT434 in healthy volunteers. The therapy is in development for the treatment of multiple system atrophy and progressive supranuclear palsy.

Multiple system atrophy is a neurodegenerative disorder characterized by symptoms such as muscle stiffness, slowed movement, tremors, ataxia, and postural instability. Other symptoms include dry mouth, inability to sweat, impotence, and incontinence. The cause of this disease is unknown, with treatment primarily being supportive. Multiple system atrophy progresses quickly; most patients are disabled within five years and are dead about eight years after diagnosis.

Progressive supranuclear palsy is a similarly degenerative disease. Early symptoms include slow movement and changes to personality. Other symptoms include poor coordination, lunging forward suddenly when moving, falling, fast walking, slurred speech, difficult swallowing, loss of vertical eye movement, and a backward tilt of the head caused by stiff neck muscles. There is no known cause, and it does not appear to have a genetic basis. Treatment for progressive supranuclear palsy is mostly supportive. Survival after diagnosis varies widely, with an average of seven years.

The manner of degeneration and presentation of symptoms in these two diseases are similar to the more well known Parkinson’s disease, with significant variations in symptoms and presentation that define each one. PBT434 has a unique mechanism of action is meant to inhibit the buildup of alpha-synuclein and tau. These proteins have been linked to the degeneration that occurs in Parkinson’s, progressive supranuclear palsy, and multiple system atrophy. PBT434 has shown potential in mouse models in reducing the accumulation of these proteins by maintaining a normal balance of iron in the brain.

The volunteers will receive a single dose of the therapy and will be monitored for 72 hours. In addition, volunteers will later receive eight days of dosing in order to monitor any effects from repeated use.

Multiple system atrophy and progressive supranuclear palsy have no known treatments that can slow down the progression of the disease. Hopefully PBT434 will be able to make a difference.

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