Steven E. Arnold

Key facts

• Principal investigator of OBSERVE and of the MGH PRiSM site.
• Single site (Alzheimer Clinical and Translational Research Unit, Charlestown, Massachusetts), recruiting; PI Steven E. Arnold (MGH); derived publication Vallabh et al. Neurology 2024 on fluid biomarkers up to disease conversion.
• Symptomatic arm: probable prion disease by CDC criteria, positive CSF RT-QuIC or PRNP genetic test, MRC Prion Disease Rating Scale score 15 or more (no more than moderate impairment), a study partner; excludes pregnancy, lumbar puncture contraindication, recent participation in another prion trial; five US sites (Massachusetts General Hospital with PI Steven Arnold; Mayo Clinic Rochester with PI Vijay Ramanan; and three others); adults 18 and over.
• The OBSERVE registry lists Steven E. Arnold as responsible party, Professor of Neurology at Massachusetts General Hospital, with the Broad Institute as collaborator.
• The study was sponsored by Prion Alliance from July 2017 with his clinical team at MGH, funded by donations and later by the CJD Foundation, an NCATS grant and Ionis.
• He is senior author on the 2020 BMC Medicine report of 27 carriers and 16 controls and on the 2024 Neurology report of four conversions over six years.
• The cohort reached 62 people (41 carriers, 21 controls) by December 2023 with more than 150 CSF and plasma samples.
• The registry shows annual visits in Charlestown, optional MRI, travel support and a stipend, and an estimated completion date of 1 June 2027.

Timeline

1. 2017Study announced.
2. 2020BMC Medicine biomarker report.
3. 2023Second data freeze reported.
4. 2024Neurology paper on fluid biomarkers up to conversion.