PSPA Talk Alistair Church Dept Neurology Royal Gwent Hospital
What are you going to do about it ? How long have I got? What is going on in my Brain? Who is going to help me? Are you sure? PSP What is that? What is going to happen to me? What else could it be? What can you do for me?
“A chilled, slow, earthy, fixed old man. A cadaverous man of measured speech. An old man who seemed as unable to win, as if his eyelids had been nailed to his forehead. An old man whose eyes – two spots of fire – had no more motion than [sic] if they had been connected with the back of his skull by screws driven through it, and riveted and bolted outside, among his grey hair.” “He had come in and shut the door, and now he sad down. He did not bend himself to sit, as other people do, but seemed to sink bolt upright, as if in water, until the chair stopped him.”
Hx of PSP “It looks like a funny kind of Parkinsonism” - Professor Gilbert H. Glaser, 1963 April 1964 Progressive Supranuclear Palsy A Heterogeneous Degeneration Involving the Brain Stem, Basal Ganglia and Cerebellum With Vertical Gaze and Pseudobulbar Palsy, Nuchal Dystonia and Dementia JOHN C. STEELE, MD; J. CLIFFORD RICHARDSON, MD; JERZY OLSZEWSKI, MD
PSP-RS PSP-P PSP-PGF PSP-F PSP-CBS PSP-OM PSP-SL PSP-C PSP-PLS
What is that? • Progressive Spranuclear Palsy • Neurodegenerative disorder • Disorder of: gait, balance, eye movement, bulbar function, cognition. • Parkinsonism poorly responsive to L- dopa • Tauopathy
Why me? • Unlucky 5/100,000. male>female. Over 40yrs • Sporadic. Less than 1% monogenic related to Chromosome 17. • Genome wide association studies (GWAS) • 3 genes. (STX6, EIF2AK3, MOBP) • H1/ H1c Haplotype MAPT • Environment • Epigenetics • Guadeloupe • Northern France
How do you know? • Clinical diagnosis • Diagnostic criteria • No good biomarkers • Imaging • Pathology
What else could it be? • Review of 60 patients with a clinical diagnosis of PSP • 47 PSP (78%) • 3 PD with additional Lewy bodies • 1 Alzheimer’s • 4 Multisystem atrophy • 1 each of CBS, Picks, MND, CVD, FTD with PD
• Tau deposited in • Basal ganglion • Brain stem • Cortex • Cerebellum • Spinal cord
What’s going to happen to me? • Progressive • Variable • Balance • Bulbar dysfunction • Vision • Cognition
What can you do for me? • Diagnosis • Education • Support • Management • Research
Diagnosis • History • Falls • Unresponsive to L-dopa • Progressive • Examination • Parkinsonism- symmetrical • Up-right posture • Eye-lid apraxia • Eye movement disorder • Postural instability
Support services • PSP society • Physiotherapy • Occupational Therapy • General Practice • Social services • Dietician • Speech and language therapy • Gastroenterology/ Respiratory medicine • Palliative care
Management - Motor • Physiotherapy/ Occupational Therapy • L-dopa • Amitriptyline • Amantadine • Botox
Management - Vision • Lubrication • Prisms • Dark glasses • Botox
Management - Mood • Depression • SSRI/ SNRI • Impulsivity • Epilim
Management – Nutrition/Communication • SLT • Swallow • Hyper-salivation • Communication • Dietician • PEG feeding
Management – Advance Care • Palliative care • Advanced Directives • Power of attorney • Will • Caregiver support
How long have I got? • 8.7yrs • 12.8yrs (PSP –P, PSP-PGF
What are you going to do about it? • Correct diagnosis • Support • Education • Coordinate care • Research
What are the challenges? • Ascertaining patients with rare disease at an early stage • Firm diagnosis at an early stage for PSP and CBD • Monitoring the disease progress • Understanding disease biology • Research Infrastructure
Research Network / PROSPECT Study
Research Network / PROSPECT Study Sites
PROSPECT PSP Aims ims Natu atural al h histo istory: : up to 7 years Clinical Assessment/MRI/CSF/Blood biomarkers/Psychology. Cr Cross ss-se sectio ctional al: (“one-stop/light touch”) Clinical Assessment / DNA / Consent to re-contact Lo Longitu itudin inal al: 1yr follow up, biomarkers, CSF 862 p patie atients ts an and co countin ting
Drug Research • Co enzyme Q10 • Riluzole • GSK inhibitors • Lithium • Tideglusib • Microtubule stabilizers • Davunetide
Passport Monoclonal antibody BMS-986168 Completed 2 phase 1 studies Plan RCT 459 PSP patients worldwide Closed for recruitment Sept 2018
ARISE Trial Monoclonal antibody 378 patients Open Jan 2018 Stopped July 2019
Thanks very much
For more information Website site www.pspassociation.org.uk He Helplin ine 0300 0110 122 Email mail helpline@pspassociation.org.uk Write ite to to us s at at Freepost PSPA
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