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Báo cáo khoa học: Structural and biological effects of a b2- or b3-amino acid insertion in a peptide Application to molecular recognition of substance P by the neurokinin-1 receptor ppt

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Structural and biological effects of a b2- or b3-amino acid insertionin a peptide Application to molecular recognition of substance P by the neurokinin-1 receptor Sandrine Sagan, Thierry

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Structural and biological effects of a b2- or b3-amino acid insertion

in a peptide

Application to molecular recognition of substance P by the neurokinin-1 receptor

Sandrine Sagan, Thierry Milcent, Rachel Ponsinet, Odile Convert, Olivier Tasseau, Ge´rard Chassaing, Solange Lavielle and Olivier Lequin

UMR 7613 CNRS-Paris 6, Universite´ Pierre et Marie Curie, Paris, France

Molecular mechanics calculations on conformers of

Ac-HGly-NHMe, Ac-b2-HAla-NHMe and Ac-b3

-HAla-NHMe indicate that low-energy conformations of the

b-amino acids backbone, corresponding to gauche rotamers

around the Ca–Cb bond, may overlap canonical backbone

conformers observed for a-amino acids Therefore,

Sub-stance P (SP) was used as a model peptide to analyse the

structural and biological consequences of the substitution of

Phe7 and Phe8 by (R)-b2-HPhe and of Gly9 by HGly (R)-b2

-HAla or (S)-b3-HAla [(R)-b2-HAla9]SP has

pharmacolo-gical potency similar to that of SP while [HGly9]SP and

[(S)-b3-HAla9]SP show a 30- to 50-fold decrease in biological

activities The three analogues modified at position 9 are

more resistant to degradation by angiotensin converting

enzyme than SP and [Ala9]SP NMR analysis of these SP

analogues suggest that a b-amino acid insertion in position 9

does not affect the overall backbone conformation Alto-gether these data suggest that [HGly9]SP, [(S)-b3-HAla9]SP and [(R)-b2-HAla9]SP could adopt backbone conforma-tions similar to that of SP, [Ala9]SP and [Pro9]SP In con-trast, incorporation of b2-HPhe in position 7 and 8 of SP led

to peptides that are almost devoid of biological activity Thus, a b-amino acid could replace an a-amino acid within the sequence of a bioactive peptide provided that the addi-tional methylene group does not cause steric hindrance and does not confine orientations of the side chain to regions of space different from those permitted in the a-amino acid Keywords: b2- and b3-amino acid; secondary structure; molecular mechanics calculations; substance P; neurokinin-1 receptor

Bioactive a-peptides often present conformational

equili-briums in solution but probably adopt one structure (or

family of related structures) when bound to their receptor

[1] A plethora of studies has been conducted on chemical

modifications of a-amino acids to stabilize this so-called

bioactive conformation that may be present as a minor

conformer in solution, and/or to design peptidomimetics

[2–8] The corresponding b-amino acids (Fig 1), should be

better building blocks to design peptidomimetics as

b-peptides are more resistant to degradation by mammalian

enzymes [9] However, each b-amino acid insertion in a

peptide sequence introduces additional degrees of confor-mational flexibility with the rotation around the Ca–Cb bond [10] Previous quantum mechanics calculations on protected b-dipeptides (mimics of tetra-b-peptides) have permitted the identification of many low-energy conforma-tions, namely six- and eight-membered ring hydrogen-bonded structures (C6, C8), extended structures (Ex) and helical structures (He) [11] The C6structure was found to be the most stable conformation by ab initio calculation [12] The Ex and He conformations observed for b-sheet, H14 and H12 helices (14- and 12-membered ring hydrogen-bonded structures) were less stable in the gas phase but gain stabilization in polar solvents [13] All these structures have also been found experimentally in b-peptides either in solution and/or in the solid state [14–17] An additional helix corresponding to the formation of successive , 10-, 12-hydrogen-bonded structures (C12/C10/C12) has also been detected [18]

The wide use of b-amino acids is prevented by the small number of enantiomerically pure b2-amino acids commer-cially available and the cost of synthesizing b3-amino acids Therefore, the design of heterooligomers made of both a- and b-amino acids could overcome this limitation, assuming that these chimeric a, b-peptides may keep the molecular recognition properties of a-peptides and the biological stability of b-peptides Only a few data have been reported in the literature on the structural properties of these heterooligomers However, it has been shown that the c-turn or C structure found with a-amino peptides can be

Correspondence to: O Lequin, UMR 7613 Paris 6-CNRS,

Laboratoire Structure et Fonction de Mole´cules Bioactives,

Universite´ Pierre et Marie Curie, Paris, France.

Fax: + 33 1 44 27 71 50, Tel.: + 33 1 44 27 26 78,

E-mail: lequin@ccr.jussieu.fr

Abbreviations: NK-1, neurokinin-1; SP, substance P

(H-Arg-Pro-Lys-Pro-Gln-Gln-Phe-Phe-Gly-Leu-Met-NH 2 ); NKA, neurokinin A;

HGly, homoglycine; b2-HAla, b2-homoalanine; b3-HAla, b3

-homo-alanine; b 2 -HPhe, b 2 -homophenylalanine; Aib (aMeAla),

a-amino-isobutyric acid; Sar, sarcosine (N-MeGly); ACE, angiotensin

converting enzyme (E.C 3.4.15.1); PtdIns, phosphatidyl inositol;

PLC, phospholipase C; CHO, Chinese hamster ovary; CSD, chemical

shift deviation; Ex, extended structures; He, helical structures.

Enzyme: angiotensin converting enzyme (EC 3.4.15.1).

(Received 3 October 2002, revised 20 December 2002,

accepted 9 January 2003)

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stabilized as a C8 conformer in cyclic tetrapeptides and

pentapeptides containing one b-amino acid [19], whereas the

introduction of two adjacent b-amino acids induced a

10-membered hydrogen-bonded turn (C10) analogous to the

b-turn made of a-amino acids [20] This propensity of

b-amino acids to mimic canonical structures observed with

a-peptides (oligomers of a-amino acids) has recently been

applied to the syntheses of linear tetrapeptides of

somato-statin [20] and of cyclic tetrapeptide and pentapeptide

analogues of the RGD sequence [19] With the cyclic

compounds it is uncertain whether the C8 conformation

observed with a single b-amino acid is enforced by the cyclic

constraints The C10 conformation, initially identified in

cyclic b-peptides containing adjacent b2-, b3-amino acids,

has also been identified in solution with a linear

b-tetra-peptide analogue of somatostatin [20] These few examples

suggested that there might be spatial overlaps between some

three-dimensional structures of a-amino acids and those of

b-amino acids and posed the following questions Which

structures of b-amino acids overlap the canonical structures

of a-amino acids? Subsequently, is it possible to substitute

one a-amino acid in the sequence of a linear peptide for one

b-amino acid without drastically affecting the recognition

properties of the resulting peptide? To try to answer these

questions we chose SP (RPKPQQFFGLM-NH2) and the

glycine residue in position 9 of SP that has been extensively

analysed in structure–activity relationship studies In terms

of peptide affinity for the NK-1 receptor and biological

activity, this achiral amino acid can be favourably replaced

by sarcosine or proline, whereas a b-II¢ turn constraint

around residues 9 and 10 confers antagonist properties

[22,23] Therefore, this position associated with a plausible

conformational flexibility is considered as a switch point

between agonist and antagonist structures [21–23]

By molecular mechanics calculations the conformers of

Ac-HGly-NHMe, Ac-b2-HAla-NHMe and Ac-b3

-HAla-NHMe have been generated and compared to the

canonical structures of the corresponding a-amino acid

Ac-Gly-NHMe The corresponding SP analogues

substi-tuted in position 9 by these b-amino acids have been

synthesized and their conformational preferences analysed

by NMR spectroscopy These substituted SP analogues

were tested for their resistance to enzymatic degradation,

their affinity for the human NK-1 receptor and their

potency to stimulate adenylate cyclase and

phospho-lipase C (PLC) in CHO cells transfected with the human

NK-1 receptor [24,25] Modelling and superimposition of

the conformers of the different b- and a-amino acids

inserted in position 9 of SP was performed to analyze

structure–activity relationships

Experimental procedures Molecular mechanics calculations N-acetyl N¢-methyl amide derivatives of HGly, b2-HAla and

b3-HAla and of a-amino acids were built using INSIGHTII

(Accelrys Inc.) Molecular mechanics calculations were performed with theDISCOVERprogram and AMBER force field [26] The electrostatic potential energy was calculated with a distance-dependent dielectric screening of 4Ær and no cut-off was used

Minimum-energy conformers of b- and a-amino acids were generated by molecular dynamics at high tempera-ture followed by energy minimization Two thousand structures were generated by molecular dynamics at

1000 K, saving snapshots every 2 ps The time step used was 1 fs and the temperature was controlled by direct velocity scaling Each structure was then submitted to

2 ps of dynamics at 300 K and minimized using steepest descent, conjugate gradient and Newton–Raphson algo-rithms until the gradient was less than 0.001 kcalÆ mol)1ÆA˚)1

Conformational grid searches of b-amino acids were initially carried out at intervals of 30 for each torsion angle /, h, and w and were subsequently refined by varying /, w angles in intervals of 10 and setting h angle to) 60 ± 10 and 60 ± 10 Each /, h, and w-value was fixed by applying

a harmonic potential and the structures were minimized (adiabatic relaxation)

NMR spectroscopy Lyophilized peptides were dissolved in 550 lL of methanol (C2H3OH or C2H3O2H) at 1–2 mMconcentration NMR experiments were recorded at 298 K and 278 K on Bruker Avance spectrometers at a1H frequency of 500 MHz and were processed with the XWIN-NMR software 1D spectra were acquired over 16 K data points using a spectral width

of 5000 Hz Solvent suppression was achieved by presatu-ration during the relaxation delay or with a WATERGATE sequence [27] Proton assignments were obtained from the analysis of TOCSY (20 and 80 ms isotropic mixing times) [28] and NOESY (400 ms mixing time) experiments [29] Typically 512 t1 increments were acquired over a spectral width of 5000 Hz Prior to Fourier transformation in t2and

t1, the time domain data were multiplied by a 60–90 shifted square sinebell function and zero-filled Baseline distortions were corrected with a fifth-order polynomial.1H-13C HSQC experiments were recorded using pulsed field gradients for coherence selection [30]

Fig 1 Schematic representation of a-, b2- and

b 3 -amino acids with the torsion angles /, h and w For the b2and b3-amino acid substi-tuted-SP analogues, the methyl side chain of b-HAla exhibits the same orientation as the one in [Ala9]SP (CIP’s rule a-amino acid:

S configuration, thus (R) for b 2 -HAla and (S) for b 3 -HAla) (CIP, Cahn–Ingold–Prelog.)

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The chemical shift deviations of Ha and Ca were

calculated using random coil values determined in methanol

[31] and water [32], respectively No sequence correction

(proline effect, for example) was applied for this calculation

as only position 9 in the sequence of these analogues is

modified, the RPKPQQFF and LM-NH2 domains being

constant

Enzymatic degradation

Enzymatic cleavage of SP, [Ala9]SP, [HGly9]SP, [(R)-b2

-HAla9]SP and [(S)-b3-HAla9]SP by ACE was performed

as described previously, with slight modifications [33]

Briefly, peptides (10 nmol) were incubated in 100 lL Tris/

HCl 50 mM pH 8.3, NaCl 300 mM, with 0.01 U rat lung

ACE (Fluka) at 37C for 60 min Degradation was

stopped by addition of 1 lL trifluoroacetic acid and

followed by HPLC using a RP8 Lichrospher100 column in

isocratic mode (72% H2O, 28% acetonitrile, 0.072%

trifluoroacetic acid) at a flow rate of 1.5 mLÆmin)1

Retention times were 7.3 min for SP, 8.6 min for [Ala9]SP,

7.5 min for [HGly9]SP, 7.1 min for [(R)-b2-HAla9]SP and

7.7 min for [(S)-b3-HAla9]SP Enzymatic assays were

performed twice and yielded similar results All assays

were done in parallel experiments with control at t¼ 0 for

each peptide The percentage of degradation was calculated

by comparing the area of the peaks of the intact peptide at

t¼ 0 and t ¼ 60 min

Binding assays

Binding assays were carried out at 22C with either

[3H][Pro9]SP (0.2–0.5 nM, 65 CiÆmmol)1) for 100 min or

[3H]propionyl[Met(O2)11]SP(7–11) (2–5 nM, 95 CiÆ mmol)1)

for 80 min on whole CHO cells expressing the human

NK-1 receptor (6 pmolÆmg protein)1) as described

previously [24], in 200 lL Krebs-Ringer phosphate

solution consisting of 120 mM NaCl, 4.8 mM KCl,

1.2 mM CaCl2, 1.2 mM MgSO4 and 15.6 mM NaH2PO4,

pH 7.2 and containing 0.04% bovine serum albumin

(w/w), 0.6% glucose (w/v), 10 mMphenylmethanesulfonyl

fluoride SP peptide analogues (stored at )20 C in

water at a concentration of 1 mM) were diluted in the

binding buffer to the desired concentration just prior to

the assay

Phospholipase C and adenylate cyclase assays

PtdIns hydrolysis and cAMP accumulation were

deter-mined as described previously [25] Briefly, CHO cells

expressing the human NK-1 receptor (6 pmolÆmg

pro-tein)1) were labelled with [3H]adenine (0.2 lCi per well)

or [3H]inositol (0.5 lCi per well) for 15–24 h PtdIns

hydrolysis assay was performed in 500 lL Krebs-Ringer

phosphate buffer containing 10 mM LiCl and the peptide

to be tested for 10 min The accumulation of cAMP was

performed in 500 lL Krebs-Ringer phosphate buffer

containing 1 mM 3-isobutyl-1-methylxanthine and the

peptide to be tested for 10 min SP peptide analogues

(stored at )20 C in water at a concentration of 1 mM)

were diluted in the assay buffer to the desired

concen-tration just prior to the assay

Results Molecular modelling The conformational properties of b-amino acids and b-pep-tides have already been extensively analysed by ab initio quantum calculations [11,13] and molecular mechanics [12]

In the present study minimum-energy conformations of model b-peptides (Ac-HGly-NHMe, Ac-b2-HAla-NHMe and Ac-b3-HAla-NHMe) were first generated by molecular mechanics using AMBER force field The results were compared with those obtained previously from ab initio molecular orbital (MO) calculations and molecular mechan-ics calculations [11–13] The potential energy surfaces accessible to b-amino acids have plenty of local minima The minimum-energy conformers (data not shown) are similar to those obtained with CHARMm 23.1 force field [12]: six- and eight-hydrogen-bonded conformations with the methyl group in axial or equatorial orientations (conformers C6eq, C6ax, C8eqand C8ax), extended structure (Ex) with no hydrogen bonds and helical structures (H12and

H14) The C6and C8structures were found to be the most stable conformations Most low-energy structures corres-pond to h torsion angles around 180, 60 and)60 Then, in order to compare the conformational spaces of a and b-amino acids, grid searches were performed and the generated structures were superimposed with canonical conformations of a-amino acids The atoms used in the rmsd calculation are those involved in the two peptide bonds and the two N- and C-terminal methyl groups, in order to compare the backbone orientations on both sides

of the b-amino acid The systematic fits indicate that structures with h torsion angle above 90 have large rmsd values In particular, the low-energy extended structures, corresponding to h 180, cannot overlap any conforma-tion of a-amino acids In order to visualize on the potential energy surfaces the conformers of b-amino acids that fit with canonical conformations of a-amino acids, two (/) w) Ramachandran maps corresponding to h torsion angle values of +60, gauche(+), or)60, gauche(–), were calculated (Fig 2) The calculated conformers are only a part of the representative statistical ensemble, due to the h angle restriction HGly has a wide range of accessible conformations, whereas b3-HAla has a more limited conformational space (Fig 2) For both gauche(–) and gauche(+) conformers, regions of the (/ – w) diagram can overlap canonical b-sheet, a-helix and reverse c-turn conformations found in a-amino acids (rmsd lower than 0.05 nm) The gauche(+) conformers which fit to a-helical, C7 and b-sheet conformations have approximate (/, w) torsion angles of ()100, )110) ()130, 20) and ()170, 80), respectively, and correspond to favourable regions For the gauche(–) conformers, the conformations that fit to a-helix have high energies and only two low-energy regions

of the diagram can fit to C7 or b-sheet conformations, with (/, w) torsion angles of ()30, 120) and ()70, 180), respectively

Secondary structure of the SP analogues [(R)-b2-HAla9]SP, [(S)-b3-HAla9]SP as well as [(R)-b2 -HPhe7]SP and [(R)-b2-HPhe8]SP have been synthesized

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by solid phase methodology and obtained with purities and

yields comparable to SP and [Pro9]SP [34]

Due to its inherent flexibility, SP is largely unstructured in

water but helical conformation of the 4–8 domain is induced

in lower dielectric constant solvents such as methanol

[35,36] The1H-NMR spectra of all the SP analogues in

methanol are relatively well-dispersed and have been

completely assigned using conventional techniques [37]

The chemical shifts of a carbon (Ca) have been assigned

from HSQC spectra (data not shown) The chemical shift

deviations of Ha protons and Ca carbons (CSDHa and

CSDCa), corresponding to differences between observed

chemical shifts and random coil values, are commonly used

to detect secondary structures in peptides and proteins [38]

They are reported in Fig 3

Stronger upfield shifts of Ha (Fig 3A) and downfield

shifts of Caresonances (Fig 3B) in the 4–8 sequence are

observed for [Aib9]SP compared to SP The positive variation observed for CSDCain methanol is weak when compared with CSDHa Because the random coil values of

Ca were determined in water, it is possible that Ca chemical shifts are sensitive to solvent variation, causing an under-estimation of calculated CSDs These CSDHaand CSDCa variations demonstrate the formation of more stable and abundant helical structures for [Aib9]SP than for SP By restrained molecular dynamics, [Aib9]SP has been shown to adopt a stable helix from residues 4–10 while the 4–8 domain of SP adopts a more flexible helix [36] Taking SP as

a reference, CSDHaand CSDCaindicate that the introduc-tion of one methyl (Ala) or two methyl groups (Aib) on Ca carbon of Gly9 increases progressively the 4–8 domain folding into helical structures An opposite effect due to the helix breaker property of Pro is observed for [Pro9]SP, this decrease in helical structure is limited to the adjacent Phe8

Fig 2 (/ – w) maps of b-amino acids with indication of the conformational space common with that of a-amino acids The (/ ) w) maps corresponding to h-values of )60 and 60 are indicated for HGly (A), b 2 -HAla (B) and

b3-HAla (C) The contours are drawn within

21 kJÆmol)1(5 kcalÆmol)1) of the global minimum and are gradually coloured from blue to red (the energy difference between each level is 2.1 kJÆmol)1) The structures that exhibit rmsd values smaller than 0.05 nm with canonical structures of a-amino acids are indicated with squares of different colours: blue, a-helix; green, reverse c-turn; red, anti-parallel b-sheet The corresponding (/, w) values of the canonical structures are ( )57, )47) ()90, 68), and ()139, 135).

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residue Homologation of Gly9 in [HGly9]SP does not

decrease the helix proportion Methylation of HGly in

position two, in [b2-HAla9]SP, has no effect on the helical

structure when compared to SP, whereas methylation of

HGly in position three, in [b3-HAla9]SP, induces a helical

folding of the 4–8 domain similar in amplitude to that

observed for [Ala9]SP Whatever the chemical modifications

carried out in position 9, the3JNH-Hacoupling constant of

Gln5 remains close to 5 Hz, indicating that Gln5 always

adopts a helical conformation This invariable folding of

Gln5 is related to the helix initiator propensity of Pro4 and

decreases from Gln5 to Gly9 In methanol, the helical

structures in the 4–8 domain increase with [Pro9]SP < SP

 [HGly9]SP  [b2-HAla9] < [b3-HAla9]SP < [Ala9]SP

< [Aib9]SP The presence of helical structures was

con-firmed by the observation of NN (i, i + 1), aN (i, i + 2), aN

(i, i + 3), aN (i, i + 4) and ab (i, i + 3) NOEs along the 5–8

domain of SP and [Aib9]SP The aN (i, i + 4) and ab (i, i

+ 3) NOEs were not detected in [b2-HAla9]SP, [b3

-HAla9]SP and [HGly9]SP, indicating more flexible helical

structures

The C-terminal part of SP undergoes a complex

con-formational equilibrium between more or less extended

structures On the basis of CSDHa, mono- or di-methylation

of Gly9 (Ala, Aib) appears to induce helical structure for

Leu10 This effect is also observed for [b3-HAla9]SP The analysis of coupling constants and NOEs indicate that the local conformation of residue 9 is not well-defined in the b-amino acid-substituted SP analogues

The lack of NOEs prevented three-dimensional structure calculations by restrained molecular dynamics Neverthe-less, the similar patterns of Ha and Ca CSDs suggest that the different SP analogues may adopt conformations of their peptide backbone close to that of SP, which has been previously described [36] The decreased number of observed NOEs in [HGly9]SP, [b2-HAla9] and [b3-HAla9]SP

is consistent with a higher flexibility around the b-amino acid-substituted position

Potency of the SP analogues The binding potencies of these analogues for the two specific binding sites, NK-1M and NK-1m, associated with the human NK-1 receptor have been measured with transfected CHO cells [24,25] The more abundant binding site NK-1M (85%) is labelled by [3H][Pro9]SP and is coupled to cAMP production, whereas the less abundant binding site NK-1m (15%) is labelled by [3H]propionyl[Met(O2)11]SP(7–11) and associated with the production of inositol phosphates The binding and agonist potencies of the SP analogues are

Fig 3 Chemical shift deviations of Ha (A) and Ca (B) for the SP analogues modified in position 9 The CSD Ca are not shown for [Pro9]SP Residue X

is either Gly (SP), Ala, Pro, Aib, HGly, b 2 -HAla or b 3 -HAla.

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expressed as Kifor NK-1M (major site) and NK-1m (minor

site), and EC50values for the cAMP pathway and for the

inositol phosphates pathway

SP, [Ala9]SP and [Pro9]SP are almost equipotent at the

major binding site NK-1M (Kibetween 0.64 nMand 1.6 nM

and EC50 values between 4.8 and 8 nM) [HGly9]SP and

[(S)-b3-HAla9]SP are 30 to 45 times less potent than SP (Ki

and EC50values) [(R)-b2-HAla9]SP is 10 times more potent

than the corresponding b3-analogue and is only three to five

times less potent than SP (Table 1)

Regarding the minor binding site NK-1m, SP and

[Pro9]SP show a 10-fold increase in affinity compared to

their affinity for the NK-1M binding site, with Ki in the

subnanomolar range (0.13 nM) and EC50 values in the

nanomolar range (1.0 and 0.7 nM, respectively) Surprisingly,

[Ala9]SP exhibits one of the highest affinity ever found for

the NK-1m specific binding site (Ki¼ 7 pM), as it is almost

20 times more potent than SP and [Pro9]SP and even 10

times more potent than [Gly9(wCH2CH2)(S)Leu10]SP

[HGly9]SP and [(S)-b3-HAla9]SP present similar Kivalues

for the NK-1m specific binding sites (3.2 and 2.3 nM,

respectively), being 20–30 times less potent than SP [(R)-b2

-HAla9]SP is only 4.5 times less potent than SP and

[Pro9]SP, as observed for the NK-1M binding site The

EC50 values for inositol phosphates production of these

three b-amino acids-substituted SP analogues [HGly9]SP,

[(S)-b3-HAla9]SP and [(R)-b2-HAla9]SP are almost

identi-cal (EC50 2 nM) For comparison, the affinities and

potencies of different analogues of SP substituted at

position(s) 9 or/and 10 are also reported in Tables 1, i.e

[Aib9]SP [36], [Pro10]SP, [Gly9w(CH2CH2)Gly10]SP,

[Gly9w(CH2CH2)(S)Leu10]SP and [Gly9w(CH2CH2)

(R)Leu10]SP

In contrast, [(R)-b2-HPhe7]SP and [(R)-b2-HPhe8]SP

are very weak competitors for NK-1M and NK-1m

specific binding sites, being 2000 times less potent than

SP

Table 1 Affinities of b-amino acid-containing peptide analogues of SP for the NK-1M (labelled with [3H][Pro9]SP) and the NK-1m (labelled with [3H]propionyl[Met(O 2 )11]SP(7–11)) binding sites and their related potency to stimulate adenylate cyclase and phospholipase C in CHO cells expressing the human NK-1 receptor All experiments have been performed in triplicate in at least three independent experiments Numbers in parentheses refer

to structures in Fig 5.

Peptide K i , NK-1M (n M ) EC 50 , adenylate cyclase K i , NK-1m (n M ) EC 50 , phospholipase C

SP (1)a 1.6 ± 0.4 8 ± 2 0.13 ± 0.02 1.0 ± 0.2

Propionyl[Met(O 2 )11]SP(7–11)a 1900 ± 450 >5000 10 ± 2 37 ± 4

[Pro9]SP (2) a 1.1 ± 0.1 10 ± 2 0.13 ± 0.02 0.7 ± 0.1

[Aib9]SP (3) b 44 ± 4 125 ± 30 c 3.8 ± 0.4 5.5 ± 1.5

[Pro10]SP 24 ± 2 375 ± 50 3.7 ± 0.5 3.0 ± 1.0

[Gly9(YCH 2 CH 2 )Gly10]SP (4) 190 ± 30 1250 ± 50 3.0 ± 0.7 1.2 ± 0.5

[Gly9(YCH 2 CH 2 )(S)Leu10]SP (5) 2.6 ± 0.5 24 ± 6 0.07 ± 0.01 0.6 ± 0.1

[Gly9(YCH 2 CH 2 )(R)Leu10]SP (6) 73 ± 8.5 1130 ± 370 4.6 ± 0.8 2.5 ± 0.7

[Ala9]SP (7) 0.64 ± 0.070 4.8 ± 1.2 0.0070 ± 0.00065 0.69 ± 0.03

[HGly9]SP (8) 64 ± 3.5 290 ± 74 3.2 ± 0.4 2.0 ± 0.2

[(R)-b2-HAla9]SP (9) 5.2 ± 0.70 40 ± 5 0.54 ± 0.12 2.0 ± 0.4

[(S)-b3-HAla9]SP (10) 53 ± 6 360 ± 55 2.3 ± 0.3 2.9 ± 0.7

[(R)-b 2 -HPhe8]SP 2400 ± 300 >10 000 240 ± 25 215 ± 25

[(R)-b2-HPhe7]SP 2500 ± 400 >10 000 230 ± 30 210 ± 30

a, b

results already published in [24] and [36], respectively.cEfficacy 73% that of [Pro9]SP taken as the peptide of reference.

Fig 4 Relation between (A) affinity for the NK-1M binding site and potency to activate adenylate cyclase and (B) affinity for the NK-1m binding site and potency to activate phospholipase C of b-amino acid-containing peptide analogues Symbols are the experimental results obtained with data in Table 1 Dotted lines represent theoretical values obtained from equations previously determined with 53 (A) and 22 (B)

SP analogues, respectively [39].

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We have previously established that a good correlation

exists between EC50values and the corresponding Kivalues,

i.e EC50 for cAMP production and Ki for the NK-1M

binding site (log(EC50)¼ 0.8 log(Ki) – 0.6), and EC50for

inositol phosphates production and Kifor the NK-1m site

(log (EC50)¼ 0.9 log (Ki) –1.0), respectively [39] The data

obtained in this study (Fig 4) square with the equations

determined previously with the exception of SP, [Pro9]SP,

[Ala9]SP, [Gly9w(CH2CH2)(S)Leu10]SP and [(R)-b2

-HAla9]SP, which show apparently abnormal behaviour in

their affinity for the NK-1m binding and their potency to

stimulate PLC (Fig 4B) Whatever the affinity measured

for the NK-1m binding site for these agonists (from 7 pMto

0.54 nM), the corresponding potency to stimulate PLC is

always close to 1 nM (from 0.6 nM to 2 nM), the highest

EC50/Kiratio being close to 100 for [Ala9]SP (Table 1) This

apparent discrepancy between the varying affinities and the

nonvarying response of these agonists could be explained by

the number of receptors to be occupied by these agonists to

get activation of the second messenger cascade as reported

[40]

Enzymatic degradation of SP analogues

ACE is a dipeptidyl carboxypeptidase known to hydrolyze

the peptide bonds between residues 8–9 and 9–10 of SP [33]

The peptide cleavage after one hour incubation with ACE

was monitored by HPLC Percentages of degradation were

found to be 54 ± 1% for SP, 55 ± 3% for [Ala9]SP,

33 ± 5% for [HGly9]SP, 26 ± 3% for [b2-HAla9] and

14.5 ± 2.5% for [b3-HAla9], thus showing that these b-amino acid-containing peptides have increased stability towards cleavage by ACE compared to the corresponding a-peptides

Discussion Computational studies confirm that the conformational space of b-amino acids is larger than that of a-amino acids, but low-energy conformations of the b-amino acids backbone, corresponding to gauche rotamers around the Ca–Cb bond, can overlap canonical backbone conformers observed for a-amino acids Therefore the addition of a backbone methylene group could have minor effects on the overall conformation and biological activity of the peptide

Thus, SP was used as a model peptide to analyse the structural and biological consequences of a single b-amino acid incorporation When Phe7 or Phe8 are replaced by

b2-HPhe, the corresponding analogues are weak competi-tors of specific NK-1 binding sites These amino acids are in the helical domain of SP which extends from residues 4–8 [35] Any modification of Phe7 causes a dramatic loss in receptor affinity for the corresponding peptide [21,41] Indeed, the backbone conformation, the aromatic ring and the orientation (v1and v2) of this phenylalanine have to be conserved for full biological potencies of the peptide [41] It

is possible that the side chain of b2-HPhe may not fit in the binding subsite devoted to the aromatic ring of Phe7 Yet, molecular calculations indicate that low-energy structures of

Fig 5 Schematic representation of the amino acid sequence 9–10 of the SP analogues 1–10 (pharmacological data in Table 1) Rectangles under analogues indicate compounds that have affinity for the two (NK-1M/NK-1m) binding sites similar to those of SP Ovals under analogues point out compounds compared to SP that lose at least a factor 20 in affinity for the two (NK-1M/NK-1m) binding sites (1) SP; (2) [Pro9]SP; (3) [Aib9]SP; (4) [Gly9(YCH 2 CH 2 )Gly10]SP; (5) [Gly9(YCH 2 CH 2 )(S)Leu10]SP; (6) [Gly9(YCH 2 CH 2 )(R)Leu10]SP; (7) [Ala9]SP; (8) [HGly9]SP; (9) [(R)-b 2 -HAla9]SP; (10) [(S)-b3-HAla9]SP.

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b2-HPhe could fit the bioactive conformation of Phe7

around the peptide bond and the aromatic ring Because

Ca-methylation of Phe7 leads to an inactive compound [36],

it is likely that the additional methylene group of b2-HPhe

causes steric hindrance within the receptor as does the

methyl group in Ca-MePhe Although position 8 of SP can

accept larger aromatic substituents than position 7 [41],

Ca-methylation is also prohibited, suggesting that steric

hindrance can again be evoked to explain the lack of affinity

of [(R)-b2-HPhe8]SP for the NK-1 receptor

Gly9 in the sequence of SP constitutes a hinge between

the helical domain and the C-terminal residues which adopt

more or less extended conformations, Phe7, Phe8, Leu10,

and Met11 being key elements of the SP pharmacophore

[21] N-methylation of Gly9, i.e [Sar9]SP, as well as proline

substitution, i.e [Pro9]SP, yield potent and selective NK-1 agonists [21] Therefore Gly9 should be a more favourable candidate for b-amino acid substitution than Phe7 or Phe8 The SP analogues modified in position 9 are schematically represented in Fig 5 Their binding potencies for the two binding sites (NK-1M and NK-1m) associated with the NK-1 receptor in CHO transfected cells [24,25] have been classified in two groups In one group, peptides are as potent

as SP, or even more potent, whatever the binding site considered, i.e [Pro9]SP, [Gly9(wCH2CH2)(S)Leu10]

SP, [Ala9]SP and [b2-HAla]SP In the second group, SP analogues are more than 20 times less potent than SP, whatever the binding site considered, i.e [Aib9]SP, [Gly9(wCH2CH2)Gly10]SP, [Gly9(wCH2CH2)(R)Leu10]SP, [HGly9]SP and [b3-HAla9]SP Previous structure–activity

A

B

C

Fig 6 Superposition of selected conformers of

b 2

-HAla (A), b 3

-HAla (B) and Aib (C) with Pro The (/, w) values of the selected Pro conformer are ( )72, 153) The low-energy conformers of b-HAla generated in the grid calculation have been selected on the basis of the best fit with Pro They correspond to (/, h, w) angles of ( )110, 70, 100) The energy differences relative to the global energy mini-mum are 0.88 and 0.86 kcalÆmol)1for b2-HAla and b 3 -HAla, respectively Nitrogen atoms are coloured in blue, oxygens in red, hydrogens in grey Carbon atoms of Pro are coloured in cyan, those of other amino acids in green The side chain methyl carbon of b-HAla and the pro R methyl carbon of Aib are coloured in magenta.

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relationship studies [42] have established that the leucine

side chain orientation is crucial for full binding potency,

[Gly9(wCH2CH2)(S)Leu10]SP being even more potent than

SP whereas [Gly9(wCH2CH2)(R)Leu10]SP is a weak

com-petitor at the NK-1 receptor (at that time NK-1M and

NK-1m binding sites were not differentiated) The amide

bond between residues 9 and 10 is not involved per se in any

stabilizing interaction within the NK-1 receptor because

[Gly9(wCH2CH2)(S)Leu10]SP is as potent as SP More

important is the length of the spacer between residues 8 and

10 Indeed, [Gly9(wCH2CH2)Gly10]SP is a weak

compet-itor of specific NK-1 binding sites while homologation

of one carbon, such as [Gly9(wCH2CH2CH2)Gly10]SP

(D Loeuillet & S Lavielle, unpublished data), led to

an analogue completely devoid of binding potency for

NK-1 binding sites Interestingly, the presence of an amide

bond such as in the homologated analogues [HGly9]SP,

[b2-HAla9]SP and [b3-HAla9]SP restores part of the

potency to recognize the NK-1 receptor In view of this

observation it can be proposed that the improved affinity

is indicative of an energetically favoured bioactive

conformation stabilized by the amide function In

[Gly9(wCH2CH2CH2)Gly10]SP, the backbone of the

ami-nohexanoic moiety must be unable to adopt two

consecu-tive gauche(+) rotamers

The pyrrolidine ring of proline does not hamper the

correct positioning of Leu10 and Met11, because [Pro9]SP is

as potent as SP Ca monomethylation of Gly9 drastically

increases the affinity for the NK-1m binding site of

[Ala9]SP, supporting the formation of a new stabilizing

interaction between this methyl group and a hydrophobic

subsite within the specific NK-1m binding site The CH2b of

the pyrrolidine ring of [Pro9]SP probably fits within this

hydrophobic subsite and thus compensates destabilizing

interactions due to CH2c and CH2d of the pyrrolidine ring

or non optimalF-value However, the introduction of a

second methyl group on the Ca of Gly9 in [Aib9]SP induces

a strong repulsive interaction Although the three b-amino

acid substitutions in position 9 of SP are tolerated by the

NK-1 receptor, the substitution of the flexible Gly by the

even more flexible HGly causes a 30- to 40-fold decrease in

affinity and biological activity Substitution by b3-HAla has

similar effects Only the b2-HAla substitution yields an

analogue that is nearly as potent as SP Thus, a peculiar

orientation of the methyl group in b2-HAla-substituted SP

might be at the origin of a stabilizing interaction

Modelling studies and NMR analysis suggest that the

three b-amino acid-substituted SP analogues [HGly9]SP,

[b2-HAla9]SP and [b3-HAla9]SP may adopt

conforma-tions around residue 9 that are analogous to those

adopted by a-amino acids (Gly, Ala, Sar, Pro) To explain

the slightly higher biological potency of [b2-HAla9]SP

compared to [HGly9]SP and [b3-HAla9]SP, the structures

(backbone and side chain) of the different a and b amino

acids were superimposed Pro, the most constrained

residue, was used as a template for the superimposition

and an extended conformation was chosen, in accordance

with structure–activity relationship studies [21,24]

Low-energy structures of the b-amino acids that best fit are

shown in Fig 6 They all correspond to gauche(+) values

of the h angle The methyl group of b2-HAla occupies a

position close to that of CHb of Pro or the pro S methyl

in Aib or Ala Conversely, the methyl group of b3-HAla occupies a position similar to the pro R methyl of Aib, on the opposite side of the pyrrolidine ring of Pro A parallel can be drawn regarding the differences in biological activities between [Ala9]SP and [Gly9]SP on the one hand, and [b2-HAla9]SP and [HGly9]SP on the other hand In-deed, the higher pharmacological potency of [b2-HAla9]SP compared to [HGly9]SP suggests that the methyl group of

b2-HAla9 may fit within the hydrophobic subsite devoted

to the methyl group of Ala9 The similar biological potencies of [b3-HAla9]SP and [HGly9]SP indicate that even though the backbone of [b3-HAla9]SP may adopt the bioactive conformation, the methyl group of b3-HAla9 may not be orientated towards this hydrophobic stabi-lizing subsite

Finally, as shown herein with the use of ACE that has been reported to cleave SP between residues 8–9 and 9–10 [33], the peptides containing a b-amino acid substitution in position 9 have increased stability compared to the corres-ponding a-amino-acid-containing peptides Therefore it is possible to increase peptide stability at the expense of a minimal decrease in its activity

HGly (named by the authors b-alanine) has been previously introduced in the sequence of the C-terminal heptapeptide of NKA, another peptide of the tachykinin family that binds the NK-1 and NK-2 receptors [HGly8] NKA(4–10) is as potent as NKA and [Ala8]NKA on rabbit pulmonary artery and rat portal vein, two NK-2 receptor bioassays [43] More recently, the same Ala substitution was reported to cause a significant decrease in biological activities of [Ala8]NKA measured in human tissues [44] Indeed, [Ala8]NKA(4–10) was shown to be a weak partial agonist ([HGly8]NKA(4–10) was not tested in this study) These discrepancies have been attributed to the differences

in sequences of rabbit, rat and human NK-2 receptors ( 85% homology)

In conclusion, a b-amino acid could replace an a-amino acid within the sequence of a bioactive peptide provided that the additional methylene group does not cause steric hindrance and does not confine orientations of the side chain to regions of space different from those permitted in the a-amino acid Thus, insertion of a single b-amino acid in

a bioactive peptide could be favourably applied to improve both biological potency and enzymatic stability of the original peptide, as already shown with the empiric design of

a metalloendopeptidase tripeptide inhibitor [45]

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