professor brain austen 6w* hrujh¶v1 … · 74 mv 326 mv 349 mv 559 mv 1266 mv 1038 mv imagnob...

17
Novel peptides for anti-amyloid therapy and MRI diagnosis in Alzheimer’s Disease St George’s Neurodegeneration Unit www.sguk.depts/ndu Professor Brain Austen Balpreet Matharu Duncan Hiscock Nick Spencer Franklyn Howe Brian Austen

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Page 1: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

Novel peptides for anti-amyloid therapy and MRI diagnosis in Alzheimer’s Disease

St George’s Neurodegeneration

Unit

www.sguk.depts/ndu

Professor Brain Austen

Balpreet Matharu

Duncan Hiscock

Nick Spencer

Franklyn Howe

Brian Austen

Page 2: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB
Page 3: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

Many (35%) MRI scans on patients are performed with the use of contrast agents

which enhance selective tissues by increasing relaxation of adjacent water protons. Gd

is the most common contrast agent, chosen as it contains 7 unpaired electrons which increase

T1 longitudinal relaxation (protons realigning with the external field) and reduce T2

transverse relaxation (time for protons to exchange energy with other nuclei). Free Gd is

toxic unless complexed with a stable ligand.

Page 4: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

NH2------VKMDAEFRHDSGYEVHHQKLVFFAEDVGSNKGAIIGLMVGGVVIATVIVITLVMLKK-------COOH

NL GQ VI X

Swedish double

mutation 670/671

Flemish

mutation

692

Dutch

mutation

692

Florida

mutation

717

London

mutations

716

Australian

mutation

723

G

F

-Secretase -Secretase -Secretase

Membran

e

A Extracellular domain Cytoplasmic tail

N C

APP

K

Italian

mutation

692

40 42

--

Aggregation

domain

Inversion FFVLK

Retroinversion ffvlk (D-amino acid residues)

Anti-toxicity properties published in Peptides (2010) 31;1866-1872

Page 5: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

DOTA- Gly-DArg-DPhe-DPhe-DVal-DLeu-DLys-Gly-DArg-Gly-Pentadiamine

Gd

Amyloid-binding Transport Contrast

R1

Synthesised MRI contrast agents for neurodegenerative disease

DOTA –Gly-DArg-DPhe-DPhe-DVal-DLeu-DLys-DArg-rlsysrrrf-NH2

Gd

SynB Amyloid binding Contrast

R3

R2

DOTA –Gly-DArg-DPhe-DPhe-DVal-DLeu-DLys-Gly-hexaDArg

Gd

Amyloid-binding Transport Contrast

Contrast Amyloid binding Transport

R5

DOTA-Gly DArg-DPhe-DPhe-DVal-DLeu-Lys(Biot)-r-Ser(Glc)-Gly-NH2

Gd

R4 DOTA-Gly-Darg-Lys-DGly-DThr-DThr-DVal-DAla-DThr-Arg-NH2

Page 6: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

0

50

100

%Int.

500 1000 1500 2000 2500 3000 3500 4000 4500 5000

Mass/Charge

1[c].L1

2[c].L1

3[c].L1

4[c].L1

5[c].M

6[c].M

74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV

ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB FrB neat0001, ImagSynB FrB neat plus Gd0001

Shimadzu Biotech Axima CFR 2.7.2.20070105

173

2343

2633380

2786

19246

2183

173

327

1702

1548

1317 2588

23441394

2139

1172

2825

2441

1504

1724

R3

R2

R1

R1 +Gd

R2 + Gd

R3 + Gd

O

NH

NH2

N

NH

O

NH

O

NH

O

NH

O

NH

O

NH

O

NH2

NH

O

NH

NH2

N

NH

O

NH

O

O

N

NN

N

O

OH

O

OH

O

OH

NH N

HNH

2

MW = 1579

O

NH2

NH

NH2

N

NH

O

NH

NH2

N

NH

O

NH

NH2

N

NH

O

NH

NH2

N

NH

O

NH

NH2

N

NH

O

NH2O

NH

NH2

N

NH

O

NH

O

NH

O

NH

O

NH

O

NH

NH2

O

N

NN

N

O

OH

O

OH

O

OH

NH O

N

MW - 2160.6

NH

NH2

N

O

NH

NH2

N

NH

O

NH

O

NH

O

OH

NH

O

OH

NH

O

OH

NH

NH2

N

NH

O

NH

NH2

N

NH

O

NH

NH2

N

NH

O

NH

O

NH2O

NH

NH2

N

NH

O

NH

O

NH

O

NH

O

NH

O

NH

NH2

O

N

NN

N

O

OH

O

OH

O

OH

NH O

N

MW = 2630

R1 R2 R3

R5

R5+Gd

Page 7: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

FC2-1 100uM R1-R4 binding to amyloid 1-40 fibrils

Interaction analysis on Biacore

Page 8: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

Binding of biotinylated reagents to Abeta40 oligomers (o) and fibrils(f).

Page 9: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

Preliminary data has validated the function of these agents, but further

work is needed to optimise their effectiveness at highlighting -amyloid

and to validate the correlation of MRI to histopathology (A) AD section plus R2 (B) AD section plus R2

(C) Young control plus R2 (D) AD section plus antibody

In AD post-mortem sections, R2 stained plaques (A) and vascular amyloid (B), in a similar fashion to A antibodies (D). Sections froma control young patient did not bind R2 (C). SW-APP 293 cells transfected with the amyloid precursor protein, treated with

proteasome inhibitor, lactalysin, to accumulate A, took up and retained R2. Visualised via incorporated biotin in R2.

Page 10: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

Amyloid deposition with age

5X FAD mice

Page 11: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

R1

Non-TG TG TG

Reagent

R2

R3

TG

TG

Pre-incubation

Ex-vivo MRI

Page 12: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

0 10 20 30 40 50 60 700

10

20

30

40

50

60muscle

blood

kidney

bladder/urine

lung

liver

stomach

SI/LI

total brain

tibia/femur

skin

thyroid

carcass

injection site

time (min)

%ID

By Duncan Hiscock, GE Healthcare

Distribution of R1 in mouse

Page 13: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

Compound 2 minute brain uptake

(%ID-kg/g) Brain Clearance (2:30

minute ratio)

153Gd-(DOTA)-rGffvlkGrG-pentadiamine 0.0046 3.3

153Gd-(DOTA)-rGffvlkKrrrrrrr-NH2 0.0058 7.9

153Gd-(DOTA)-Grffvlkrrlsysrrrf-NH2 0.0052 2.1

PET gold standard 0.22 9.0

SPECT gold standard 0.09 12.2

Page 14: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

US/MR SYSTEM

Page 15: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

0 10 20 30 40 50 60 70

0

200

400

600

800

1000

1200

Time after injection (min)

T1 (

ms)

T1 change after injection

Page 16: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB
Page 17: Professor Brain Austen 6W* HRUJH¶V1 … · 74 mV 326 mV 349 mV 559 mV 1266 mV 1038 mV ImagNoB FrA0001, ImagNoB Plus Gd 0001, imagRNoB FrA 0001, ImagRNoB Gd complex 0001, ImagSynB

500/14 ms TR/TE

2000/40 ms TR/TE