PHILADELPHIA — Two Penn Medicine research teams have received a total of $1,450,000 in funding commitments from CurePSP, the Foundation for PSP | CBD and Related Brain Diseases, to study rare neurodegenerative disorders that cause motor, balance and cognitive impairment.
The team of John Trojanowski, MD, PhD, and Virginia M.-Y. Lee, PhD, MBA, will leverage recent work uncovering how tau, an essential protein of nerve cell structure, becomes toxic and spreads from cell-to-cell in diseased brains. Using this discovery, they aim to develop disease-modifying tau immune therapies for progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD), in animal models at first. The most promising anti-tau antibody candidates could then be pursued in industry-sponsored clinical trials for patients with PSP and CBD.
The $600,000 to the Trojanowski and Lee team has the potential to increase through a novel challenge program. Penn Medicine researchers will team up with CurePSP to raise funds that will be directed back to Penn Medicine research efforts.
A team led by geneticist Gerard Schellenberg, PhD, professor of Pathology and Laboratory Medicine, received $850,000 from CurePSP to perform whole exome sequencing to detect rare inheritable genetic changes that cause or increase susceptibility to developing PSP. This follows up on the 2011 Nature Genetics study in which Schellenberg’s team identified three new genes associated with risk for PSP and confirmed two additional genetic variants affecting risk for PSP. PSP is primarily caused by an abnormal accumulation of tau protein, which can be influenced by both inherited factors and environmental insults (e.g. repetitive brain trauma).
PSP, a movement disorder and form of frontotemporal degeneration, affects around 30,000 Americans, and after Parkinson's disease, is the second most common cause of degenerative parkinsonism. The disease is characterized by a difficulty coordinating eye movement, imbalance and gait instability, stiff movements, difficulty swallowing and speaking, along with mood and emotional changes. There is currently no treatment or cure for PSP.
An estimated 3,000 Americans are affected by CBD, which includes similar symptoms to those found in Parkinson’s disease, such as poor coordination, rigidity, and impaired balance, along with speech and cognitive impairments, difficulty swallowing and stiff or uncontrolled movements.
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