On Thursday, April 13, 2023, The Michael J. Fox Foundation (MJFF) announced a historic breakthrough1 in the development of Parkinson’s disease detection and diagnosis. The new tool is an α-synuclein seeding amplification assay (αSyn-SAA). It is engineered to detect abnormal alpha-synuclein in the cerebral spinal fluid, both in people diagnosed with Parkinson’s as well in those who have not yet been diagnosed or displayed symptoms but who are at high risk of developing symptoms in the future.
This discovery is novel in that it allows researchers to see ante-mortem whether or not aberrant alpha-synuclein is present in the brain, something that was never possible before. Aggregated alpha-synuclein is considered the pathological hallmark of Parkinson’s disease – the ability to measure this prior to symptoms developing makes early disease intervention possible on a larger scale.
Identifying and treating the disease as soon as possible has always been the goal of drug development for neurodegenerative diseases. The earlier a therapeutic can intervene in the disease, the better chance it has to have a disease modifying effect. By identifying patients before they have developed ‘ symptoms, future therapeutics could be administered as a prophylactic, essentially protecting the neurons from losing their function and stopping the disease progression altogether.
Gain’s lead candidate GT-02287 works by correcting misfolded forms of the GCase enzyme at the very start of the disease cascade. As a result, the compound has shown a strong tendency to deplete toxic forms of alpha-synuclein in preclinical models. More importantly, GT-02287 has shown to be neuroprotective preclinically, exhibiting a tendency to protect neurons from losing their function.
This breakthrough could offer the possibility to administer GT-02287 very early in a patient’s disease progression, potentially eliminating further advancement of the disease and pathological symptoms. This development is a major step in the direction of having a cure for Parkinson’s disease in the not-so -distant future.
To read the announcement from MJFF click here: Breaking News: Parkinson’s Disease Biomarker Found
The full publication can be found here.
1“Assessment of heterogeneity among participants in the Parkinson’s Progression Markers Initiative cohort using α-synuclein seed amplification: a cross-sectional study” Siderowf, Andrew et al. The Lancet Neurology, Volume 22, Issue 5, 407 – 417