Massachusetts General Hospital

Massachusetts General Hospital (MGH, Mass General) is a Boston teaching hospital of Harvard Medical School and a founding member of the Mass General Brigham system. In prion disease it is the clinical home of the longest-running US cohort following people at risk of genetic prion disease (OBSERVE, NCT05124392), which it sponsors with the Broad Institute as collaborator. Its neurologist Steven Arnold also leads the MGH site of the PRiSM siRNA trial. The cohort's published biomarker work, much of it a joint MGH and Broad effort, shapes how prevention trials may be designed. Information only, not medical advice.

Key facts

  • MGH is described by its parent network as a founding member of Mass General Brigham, a Harvard Medical School teaching hospital and the largest hospital-based research enterprise in the US.
  • It is the lead sponsor of OBSERVE (NCT05124392), with the Broad Institute as collaborator and Steven E. Arnold, Professor of Neurology at MGH, as responsible party.
  • OBSERVE is registered with one site, the Alzheimer's Clinical and Translational Research Unit in Charlestown, with an actual start of 1 December 2017, an estimated enrollment of 150 and an estimated completion of 1 June 2027.
  • The 2024 Neurology paper reports a single-center cohort run entirely at MGH under an MGB institutional review board approval, with 62 participants (41 carriers of pathogenic PRNP variants and 21 controls) and four conversions to active disease.
  • That paper found CSF RT-QuIC seeding activity before onset in E200K converters and stable CSF prion protein over up to six years; authors came from the MGH McCance Center for Brain Health, the Broad Institute and Harvard Medical School.
  • Prion Alliance announced in July 2017 that it was sponsoring an MGH study led by Steven Arnold and Aaron Koenig, in collaboration with a Broad team, to prepare for future prevention trials; it was explicitly described as not a clinical trial.
  • MGH received an NIH R21 award (5R21TR003040-02, $212,829 in fiscal year 2020, 16 September 2019 to 30 June 2021) with Arnold as PI, to build biomarker evidence for antisense prevention trials in genetic prion disease.
  • Funders named for the cohort work are the Broad Institute, Ionis, Prion Alliance, the CJD Foundation and NIH (R21 TR003040, R01 NS125255).
  • MGH is one of five recruiting sites of PRiSM (NCT07444580, start 15 May 2026), with Arnold as site investigator; the sponsor is the Broad Institute with NINDS as collaborator.
  • The other PRiSM sites are Mayo Clinic Rochester, Columbia, University Hospitals Cleveland and Vanderbilt.

Timeline

  1. 1811MGH founded (T3 source).
  2. 2017Prion Alliance announces sponsorship of the MGH study led by Arnold and Koenig.
  3. 2017OBSERVE starts (actual start date in the registry).
  4. 2019NIH R21 TR003040 project period begins, ending June 2021.
  5. 2023Second data freeze of the MGH cohort, 62 people, preprint released 18 December.
  6. 2024Neurology publication of the fluid biomarker findings up to disease conversion.
  7. 2026PRiSM starts with MGH among five recruiting sites.
  8. 2026OBSERVE registry record last updated, still recruiting.

People

Mentioned on these pages

Last checked 1 November 2026

Sources for this page