Letter of Support Issued by FDA for α-Synuclein Biomarker Testing

The Food and Drug Administration (FDA) issued a Letter of Support (LoS) for the use of α-synuclein (α-syn) seed amplification assay (SAA) in human cerebral spinal fluid (CSF) samples to improve clinical trial efficiency for conditions with underlying α-syn pathology, including Alzheimer disease (AD), Parkinson disease (PD), and dementia with Lewy bodies (DLB). The LoS was requested by the Critical Path for Parkinson’s Consortium (CPP) of the Critical Path Institute (Tucson, AZ), a nonprofit organization working to improve the drug development process.

Biomarker testing with α-syn SAA has demonstrated accuracy in detecting the early biology associated with PD, AD, and DLB before the emergence of symptoms. The technique therefore has applications in screening and selecting participants for clinical trials for the evaluation of early interventions for neurodegenerative diseases, which aim to delay or prevent symptom onset. Applications for α-syn SAA also include tailoring therapy for people living with PD, AD, DLB, and other conditions. The LoS also includes discussion of the use of the neuronal α-synuclein integrated staging system (NSD-ISS) definition and staging framework in therapeutic development.

“This FDA Letter of Support is a transformative moment for the field, promising to speed clinical trial design in Parkinson’s and related disorders,” said Diane Stephenson, PhD, Executive Director of the CPP at the Critical Path Institute. “The cross-collaboration among patients, researchers, clinicians, regulators and patient advocacy organizations demonstrates the critical role every player holds in moving today’s achievement forward.”