prion disease & pentosan polysulphate in the uk richard knight ncjdsu university of edinburgh...
TRANSCRIPT
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PRION DISEASE &
PENTOSAN POLYSULPHATE IN THE UK
Richard KnightNCJDSU
University of EdinburghScotland
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PRION DISEASE & PPS
I GENERAL INTRODUCTION
II PENTOSAN POLYSULPHATE
III UK PATIENTS
IV CONCLUDING POINTS
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I
GENERAL INTRODUCTION
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DIFFERENT BACKGROUNDSDIFFERENT PERSPECTIVES
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TREATMENTS: HOW DO YOU EVALUATE THEM?
• IN THE ‘TEST TUBE’
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TREATMENTS: HOW DO YOU EVALUATE THEM?
• IN THE ‘TEST TUBE’
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TREATMENTS: HOW DO YOU EVALUATE THEM?
PROTEINS & CELLS ARE NOT ANIMALS
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TREATMENTS: HOW DO YOU EVALUATE THEM?
• IN ANIMALS
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TREATMENTS: HOW DO YOU EVALUATE THEM?
RODENTS ARE NOT HUMANS
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TREATMENT AT TIME OF INFECTION
IS NOT THE SAME AS TREATING CLINICALLY ILL ANIMALS
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TREATMENTS: HOW DO YOU EVALUATE THEM?
• IN THE ‘TEST TUBE’
• IN ANIMALS
• IN HUMANS
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X
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TREATMENT
REAL DISEASE BENEFIT SYMPTOM RELIEF TOXIC SIDE EFFECTS
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TREATMENT
REAL DISEASE BENEFIT SYMPTOM RELIEF TOXIC SIDE EFFECTS
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DISEASE PROCESS
SYMPTOMS
NOT ALWAYSEASY TO TELLTHE DIFFERENCE
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TREATMENT
REAL DISEASE BENEFIT SYMPTOM RELIEF TOXIC SIDE EFFECTS
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TWO TREATMENT SITUATIONS
CLINICAL ILLNESS PREVENTION
ANY SIDE EFFECTS MAY BE OF DIFFERENT SIGNIFICANCE
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DISEASE
TREATMENT
PEOPLE VARY
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PERSON SPORADICGENETIC
IATROGENIC VARIANT
TREATMENT
DISEASES VARY
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TREATMENT REQUIRES DIAGNOSIS
THE DIAGNOSTIC PROCESS IS NOT SIMPLE
NO SIMPLE ‘CJD TESTS’
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TREATMENT IDEALLY REQUIRES EARLY DIAGNOSIS
STOPPING BRAIN DISEASE PREVENTS FURTHER DAMAGE
REPAIR OF EXISTING BRAIN DAMAGE IS PROBLEMATIC
DIAGNOSIS OF CJD IS OFTEN ‘LATE’
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TREATMENT IDEALLY REQUIRES EARLY DIAGNOSIS
STOPPING BRAIN DISEASE PREVENTS FURTHER DAMAGE
REPAIR OF EXISTING BRAIN DAMAGE IS PROBLEMATIC
DIAGNOSIS OF CJD IS OFTEN ‘LATE’
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TREATMENT IDEALLY REQUIRES EARLY DIAGNOSIS
STOPPING BRAIN DISEASE PREVENTS FURTHER DAMAGE
REPAIR OF EXISTING BRAIN DAMAGE IS PROBLEMATIC
DIAGNOSIS OF CJD IS OFTEN ‘LATE’
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TREATMENT IDEALLY REQUIRES EARLY DIAGNOSIS
STOPPING BRAIN DISEASE PREVENTS FURTHER DAMAGE
REPAIR OF EXISTING BRAIN DAMAGE IS PROBLEMATIC
DIAGNOSIS OF CJD IS OFTEN ‘LATE’
MAY BE SEVERE, IRREVERSIBLE, DAMAGE
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II
PENTOSAN POLYSULPHATE
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PENTOSAN POLYSULPHATE: PPS
BEECH WOOD DERIVED
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PENTOSAN POLYSULPHATE: PPS
BEECH WOOD DERIVED
ESTABLISHED DRUGNON-PRION DISEASE
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PENTOSAN POLYSULPHATE: PPS
IN PRION DISEASE ?
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PENTOSAN POLYSULPHATE: PPS
IN PRION DISEASE ?
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PENTOSAN POLYSULPHATE: PPS
IN PRION DISEASE ?
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PENTOSAN POLYSULPHATE: PPS
IN PRION DISEASE ?
?
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PENTOSAN POLYSULPHATE: PPS
ORAL or IV: DOES NOT ENTER BRAIN
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PENTOSAN POLYSULPHATE: PPS
ORAL or IV: DOES NOT ENTER BRAIN
NEED DIRECT ACCESS TO BRAIN
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INTRA-VENTRICULAR ADMINISTRATION
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INTRA-VENTRICULAR ADMINISTRATION
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INTRA-VENTRICULAR ADMINISTRATION
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INTRA-VENTRICULAR ADMINISTRATION
CURRENT PPS TREATMENTOF PRION DISEASE
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POTENTIAL PPS PROBLEMS
PROBLEMS WITH CATHETER
SURGERY: DAMAGE / BLEEDING
POST SURGERY: INFECTION
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INTRA-VENTRICULAR ADMINISTRATION
PROBLEMS WITH PUMP&
CONNECTING TUBE
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POTENTIAL PPS PROBLEMS
PROBLEMS WITH PPS
BLEEDINGSEIZURESOTHER TOXICITY
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III
PPS TREATMENT IN THE UK
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UK PPS TREATMENT
• NO ORGANISED CLINICAL TRIAL
• COLLECTION OF INFORMATION
ON A FEW INDIVIDUALS
WHO CHOSE TREATMENT or
WHOSE FAMILIES CHOSE TREATMENT
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ONE ORGANISED OBSERVATIONAL STUDYPublished 2008
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INTRAVENTRICULAR PENTOSAN POLYSULPHATE IN HUMAN PRION DISEASES: AN OBSERVATIONAL STUDY IN
THE UKI Bone, Belton L, Walker AS, Darbyshire J
European Journal of Neurology 2008, 15:458-464
www.mrc.ac.uk/Utilities/Documentrecord/index.htm?d=MRC003453
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MRC STUDY PATIENTS
• 2 hGH CJD NO OBVIOUS BENEFIT
• 2 GSS NO OBVIOUS BENEFIT
• 3 vCJD 2/3 POSSIBLE BENEFIT
(ALIVE LONGER)
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MRC STUDY PATIENTS
• 2 hGH CJD NO OBVIOUS BENEFIT
• 2 GSS NO OBVIOUS BENEFIT
• 3 vCJD 2/3 POSSIBLE BENEFIT
(ALIVE LONGER)
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MRC STUDY PATIENTS
• 2 hGH CJD NO OBVIOUS BENEFIT
• 2 GSS NO OBVIOUS BENEFIT
• 3 vCJD 2/3 POSSIBLE BENEFIT
(ALIVE LONGER)
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MRC STUDY PATIENTS
• 2 hGH CJD NO OBVIOUS BENEFIT
• 2 GSS NO OBVIOUS BENEFIT
• 3 vCJD 2/3 POSSIBLE BENEFIT
(ALIVE LONGER)
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MRC STUDY PATIENTS
• 2 hGH CJD NO OBVIOUS BENEFIT
• 2 GSS NO OBVIOUS BENEFIT
• 3 vCJD 2/3 POSSIBLE BENEFIT
(ALIVE LONGER)
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MRC STUDY PATIENTS
• SOME PROBLEMS DUE TO INTRAVENTRICULAR ADMINISTRATION (NO MAJOR ONES)
• NO PROBLEMS DUE TO PPS ITSELF
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MRC STUDY PATIENTS
• SOME PROBLEMS DUE TO INTRAVENTRICULAR ADMINISTRATION (NO MAJOR ONES)
• NO PROBLEMS DUE TO PPS ITSELF
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PRESENT UK SITUATION
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Intra-ventricular PPS Cases Treated in the UK
Disease Treated Currently alive
vCJD 5 4
sCJD 1 1
GSS 2 0
hGH 2 0
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Intra-ventricular PPS Cases Treated in the UK
Disease Treated Currently alive
vCJD 5 4
sCJD 1 1
GSS 2 0
hGH 2 0
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vCJD DURATION OF ILLNESS > 20 MONTHS
September 2009
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vCJD DURATION OF ILLNESS > 20 MONTHS
0
1
2
3
4
5
15 20 25 30 35 40 45 50 55 60 65 70 75 80 85 90 95 100
Duration of illness (months)
Num
ber
of c
ases
dead alive (treated)
September 2009
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ALL UK vCJD DURATION OF ILLNESS
02468
1012141618202224
0 5 10 15 20 25 30 35 40 45 50 55 60 65 70 75 80 85 90 95 100
Duration of illness (months)
Num
ber
of c
ases
dead (untreated) dead (treated PPS) alive (treated PPS)
September 2009
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Intra-ventricular PPS Cases Treated in the UK
Disease Treated Currently alive
vCJD 5 4
sCJD 1 1
GSS 2 0
hGH 2 0
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sCJD DURATION OF ILLNESS
020406080
100120140160180200
0 5 10 15 20 25 30 35 40 45 50
Duration of illness (months)
Num
ber
of c
ases
dead alive (untreated) alive (treated)
September 2009
+
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IV
CONCLUDING REMARKS
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PPS
NOT A CURE
• HIGHLY PROBABLE: PROLONGS DISEASE IN VARIANT CJD
• NO GOOD EVIDENCE FOR BENEFIT IN OTHER FORMS OF CJD
• NO EVIDENCE OF TOXICITY FROM PPS ITSELF
• INTRAVENTRICULAR ADMINISTRATION IS NOT EASY
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PPS
NOT A CURE
• HIGHLY PROBABLE: PROLONGS LIFE IN VARIANT CJD
• NO GOOD EVIDENCE FOR BENEFIT IN OTHER FORMS OF CJD
• NO EVIDENCE OF TOXICITY FROM PPS ITSELF
• INTRAVENTRICULAR ADMINISTRATION IS NOT EASY
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PPS
NOT A CURE
• HIGHLY PROBABLE: PROLONGS LIFE IN VARIANT CJD
• NO PRESENT EVIDENCE FOR OTHER FORMS OF CJD
• NO EVIDENCE OF TOXICITY FROM PPS ITSELF
• INTRAVENTRICULAR ADMINISTRATION IS NOT EASY
![Page 68: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/68.jpg)
PPS
NOT A CURE
• HIGHLY PROBABLE: PROLONGS LIFE IN VARIANT CJD
• NO PRESENT EVIDENCE FOR OTHER FORMS OF CJD
• NO EVIDENCE OF TOXICITY FROM PPS ITSELF
• INTRAVENTRICULAR ADMINISTRATION IS NOT EASY
![Page 69: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/69.jpg)
PPS
NOT A CURE
• HIGHLY PROBABLE: PROLONGS LIFE IN VARIANT CJD
• NO PRESENT EVIDENCE FOR OTHER FORMS OF CJD
• NO EVIDENCE OF TOXICITY FROM PPS ITSELF
• INTRAVENTRICULAR ADMINISTRATION IS NOT EASY
![Page 70: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/70.jpg)
FURTHER RESEARCH ON PPS
IF POSSIBLE: RCTs
? OTHER ANIMAL RESEARCH
![Page 71: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/71.jpg)
FURTHER RESEARCH ON PPS
IF POSSIBLE: RCTs
? EASIER ADMINISTRATION METHODS
![Page 72: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/72.jpg)
TREATMENT TRIALS WITH A STRUCTURED FRAMEWORK
INTERNATIONAL COLLABORATION
TRIALS WITH UNIFORM METHODS
EUROPE: ‘THERAPRION’
![Page 73: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/73.jpg)
EARLIER TREATMENT
EARLIER DIAGNOSIS
![Page 74: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/74.jpg)
EARLIER TREATMENT
EARLIER DIAGNOSIS
![Page 75: PRION DISEASE & PENTOSAN POLYSULPHATE IN THE UK Richard Knight NCJDSU University of Edinburgh Scotland](https://reader036.vdocuments.net/reader036/viewer/2022062516/56649e165503460f94b01bcc/html5/thumbnails/75.jpg)