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Báo cáo khoa học: Alpha-fetoprotein antagonizes X-linked inhibitor of apoptosis protein anticaspase activity and disrupts XIAP–caspase interaction ppt

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We show here that AFP cancels XIAP-mediated inhibition of endogenous active caspases in cytosolic lysates of tumor cells, as well as XIAP-induced blockage of active recombinant caspase 3

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apoptosis protein anticaspase activity and disrupts

XIAP–caspase interaction

Elena Dudich1,2, Lidia Semenkova1,2, Igor Dudich1,2, Alexander Denesyuk3, Edward Tatulov2

and Timo Korpela4

1 Institute of Immunological Engineering, Lyubuchany, Russia

2 JSC BioSistema, Moscow, Russia

3 Department of Biochemistry and Pharmacy, A ˚ bo Akademi University, Turku, Finland

4 Joint Biotechnology Laboratory, Turku University, Finland

Apoptotic dysfunction plays a key role in cancer

pro-gression and leads to chemotherapeutic and

radio-therapeutic resistance [1–3] Many cancer radio-therapeutic

agents operate by inducing apoptosis and are

ineffec-tive in conditions of impaired apoptosis signaling

Novel strategies for cancer therapy are aimed at

dis-covering molecular targets involved in the induction

of apoptosis in normal and tumor cells, and at

selec-tively regenerating the apoptosis propensity in cancer

cells

Apoptosis is induced by two different mechanisms: the extrinsic or receptor-dependent pathway and the intrinsic or mitochondria-dependent pathway [4] Trig-gering of either pathway results in the initiation of caspase cascade activation events Caspases are gener-ally divided into two groups according to their func-tional hierarchy and substrate specificity The initiator caspase family includes caspases 2, 8, 9, 10 and 12, and is characterized by the presence of N-terminal prodomains DED or CARD, which are involved in

Keywords

apoptosis; apoptosome; caspases;

a-fetoprotein; X-linked inhibitor of apoptosis

protein

Correspondence

E Dudich, Institute of Immunological

Engineering, 142380, Lyubuchany, Moscow

Region, Chekhov District, Russia

Fax ⁄ Tel: +7 095 996 1555

E-mail: elena_dudich@mail.ru

(Received 28 February 2006, revised 3 May

2006, accepted 22 June 2006)

doi:10.1111/j.1742-4658.2006.05391.x

Previous results have shown that the human oncoembryonic protein a-feto-protein (AFP) induces dose-dependent targeting apoptosis in tumor cells, accompanied by cytochrome c release and caspase 3 activation AFP posi-tively regulates cytochrome c⁄ dATP-mediated apoptosome complex forma-tion in a cell-free system, stimulates release of the active caspases 9 and 3 and displaces cIAP-2 from the apoptosome and from its complex with recombinant caspases 3 and 9 [Semenkova et al (2003) Eur J Biochem

270, 276–282] We suggested that AFP might affect the X-linked inhibitor

of apoptosis protein (XIAP)–caspase interaction by blocking binding and activating the apoptotic machinery via abrogation of inhibitory signaling

We show here that AFP cancels XIAP-mediated inhibition of endogenous active caspases in cytosolic lysates of tumor cells, as well as XIAP-induced blockage of active recombinant caspase 3 in a reconstituted cell-free sys-tem A direct protein–protein interaction assay showed that AFP physically interacts with XIAP molecule, abolishes XIAP–caspase binding and rescues caspase 3 from inhibition The data suggest that AFP is directly involved

in targeting positive regulation of the apoptotic pathway dysfunction in cancer cells inhibiting the apoptosis protein function inhibitor, leading to triggering of apoptosis machinery

Abbreviations

Ac-DEVD-AMC, Ac-Asp-Glu-Val-Asp-7-amino-4-methyl coumarin; AFP, a-fetoprotein; IAP, inhibitor of apoptosis protein; IBM, IAP-binding motif; RFU, relative fluorescence units; XIAP, X-linked inhibitor of apoptosis protein.

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interactions with certain adapter molecules to form

death-inducing signaling complex or apoptosome [3–5]

Effector caspases 3, 6 and 7 exist in the cytosol as

inactive zymogens and are activated via a proteolytic

cascade started by the initiator caspases [5]

Molecular pathways leading to apoptosis are

evolu-tionarily conserved and are regulated by specific cellular

proteins Some, such as Bcl-2, control the release of

proapoptotic proteins from the mitochondria [6]

Others, including various cellular inhibitor-of-apoptosis

proteins (IAPs), bind directly to active caspases and act

as natural inhibitors of caspase activity [7] The IAP

family, currently identified in humans consists of

X-linked inhibitor of apoptosis protein (XIAP), ILP-2,

cIAP1, cIAP2, ML-IAP, NAIP, survivin, and livin

[7–9] All IAPs have so-called conservative BIR

domains, which are responsible for their interaction with

caspases XIAP is the most potent of all known IAPs

and contains three BIR domains The third BIR domain

(BIR3) selectively targets caspase 9, whereas BIR2 and

the linker region between BIR1 and BIR2 inhibit

effec-tor caspases 3 and 7 [5,10] This inhibition can be

relieved by IAP antagonists, which bind to IAPs

pre-venting caspase binding [5,8,9,11–13] Recent studies

have revealed that binding of IAP antagonists to IAPs

may stimulate their auto-ubiquitination and

degrada-tion, thereby preventing caspase inhibition [14,15]

Recognition of XIAP as a direct inhibitor of caspases

makes it an attractive therapeutic target This led to an

active search for any suitable molecular inhibitor

capable of easily penetrating a tumor cell to block

XIAP activity in the cytosol [8,9,16] The discovery of

endogenous regulators of IAP activity enhanced these

investigations Several intracellular inhibitory IAPs

have been characterized in humans, namely, Smac⁄

DI-ABLO, Omi⁄ HtrA2, GSPT1 ⁄ eRF3, ARTS, and XAF1

[17–21] However, only the first and best characterized

anti-IAP Smac⁄ DIABLO is currently known to be

directly involved in the regulation of apoptosis Other

anti-IAPs, such as Omi⁄ HtrA2 or GSPT1 ⁄ eRF3, seem

to have a primary physiological role that is not directly

related to XIAP⁄ caspase regulation [8,18,19] Smac ⁄

DIABLO is released from mitochondria into the

cyto-sol during apoptosis, wherein it can bind to XIAP [17]

The main highly conserved functional motif common

to all IAP antagonists, was termed the IAP-binding

motif (IBM), and became a target for finding novel

potential inhibitors of IAP [8,9,16] The motif ATPF⁄

AVPI was first characterized in caspase 9 and

Smac⁄ DIABLO was characterized as being responsible

for binding to BIR3 of XIAP [22]

Smac-derived peptides modeling their XIAP-binding

site, bind to recombinant BIR3 domains in vitro

[18–23] Recent studies have set out to design small molecular drugs carrying the IBMs [5,11–13] or artifi-cial chimerical peptides composed of the IBM sequence fused to a carrier peptide [23] The cell-permeable Smac peptides allowed the apoptosis resistance and chemoresistance of cancer cells with a high level of XIAP to be overcome in vitro and in vivo, as documen-ted [13,23] Despite the strong molecular basis for interaction with XIAP, natural Smac-derived peptides and other artificial IBM-based chimeric constructions have several intrinsic limitations (e.g poor in vivo stability and very low bioavailability) making them unsuitable for the treatment of cancer [8,9,23] The other known natural XIAP-binding proteins cannot act as anticancer drugs because of their exclusive intra-cellular location Therefore, the search for other XIAP-interacting and cell-membrane-penetrating drugs

is a highly desirable goal

Recently, it was discovered that the well-known oncofetal antigen a-fetoprotein (AFP) is able to induce apoptosis selectively in tumor cells without any toxicity towards normal cells and tissues [24–28] AFP is one

of the major serum embryonic proteins involved in the regulation of growth and the development of immature embryonic tissues [29–31] The specific expression and internalization of AFP is restricted to developing cells, such as embryonic cells, activated immune cells and tumor cells, which suggests that it has an important regulatory role in cell growth and differentiation [32– 35] AFP expression is blocked completely after birth and is recovered only after malignant transformation [29–31] Various researchers have documented the existence of specific receptor-dependent mechanisms responsible for the active endocytosis of AFP by malignant cells [34–36] AFP has been well character-ized as a transport protein delivering natural ligands such as fatty acids, hormones, and heavy metals to developing cells [29] The specific expression and inter-nalization of AFP by developing cells, such as embry-onic cells or tumor cells, together with the properties

of the transport protein make AFP very attractive for tumor-targeting therapy [29,30,33] The growth-regula-tory activity of AFP and AFP derivatives has been demonstrated by various authors [24–28,37–43] Spe-cial interest has focused on the tumor-suppressive effects of AFP and its peptide derivatives [24–28,38, 39,42] The growth-suppressive activity of AFP can be realized by inducing apoptosis in many types of tumor

or activated immune cells [24–28,41] AFP can trigger apoptosis in tumor cells via activation of caspase 3, independent of the membrane-receptor signaling [26] AFP stimulates formation of the apoptosome complex, and enhances recruitment and activation of caspases 3

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and 9 by displacing cIAP-2 from the apoptosome

and from its complex with recombinant caspases 3 and

9 [28]

Based on the molecular mechanisms of

AFP-medi-ated apoptosis, we hypothesized that AFP might

inter-act with XIAP by displacing it from the complex with

caspases, and thus preventing caspase inhibition We

demonstrate here that AFP physically associates with

XIAP in cytochrome c-activated cellular lysates, and

that this complex does not contain the effector

caspase 3 We found that purified human AFP binds

to recombinant XIAP, disrupts the association between

XIAP and activated caspase 3, and antagonizes the

antiapoptotic function of XIAP Our data indicated

that AFP could also bind free XIAP to eliminate it

from the reaction area and prevent caspase binding

Results

AFP promotes caspase activation in cell-free

cytosolic extracts by blocking of XIAP-dependent

inhibition

Recent evidence has shown that AFP promotes the

processing and activation of procaspase 3 in the

pres-ence of low suboptimal doses of cytochrome c in

cell-free cytosolic extracts Simultaneously, AFP induced

the release of cIAP2 from the apoptosome complex

[28] Our recent experimental data allowed us to

hypo-thesize that AFP could operate as a XIAP antagonist

by affecting the interaction of XIAP with active

caspas-es, thus promoting their activity To determine whether

AFP can affect caspase 3 activity in HepG2 cytosolic

extracts in the presence of an inhibitory amount of

exo-genous XIAP, we monitored caspase activation in a

cell-free system Cytosolic cell extracts were activated

by the addition of cytochrome c⁄ dATP together with

AFP or human serum albumin (HSA) in the presence

of an inhibitory amount of rhXIAP Figure 1 shows

that addition of XIAP induced 50% inhibition of

caspase 3 activity in activated cell-free extracts

Addi-tion of AFP in the cytosolic extract induced significant

enhancement of the DEVD-ase activity The same

caspase activity was detected upon the simultaneous

addition of an inhibitory amount of exogenous rhXIAP

together with AFP The data clearly show that AFP

abrogated the inhibitory activity of exogenous rhXIAP

against endogenous caspases and failed to relieve

AFP-mediated caspase stimulation By contrast,

exo-genous HSA did not affect caspase activity in cell-free

extracts (Fig 1) Hence, AFP counteracts with XIAP

by abrogation its caspase inhibition in the

cyto-chrome c-activated cell-free cytosolic extracts

AFP promotes caspase 3 activity exclusively

by abrogation of XIAP-dependent inhibition

To study direct AFP⁄ XIAP ⁄ caspase 3 interaction we used recombinant proteins to form a reaction mixture

in order to avoid the influence of other active com-pounds, which are available in cytosolic extracts An effective amount of rhXIAP was added into the solu-tion of active recombinant caspase 3 to induce 50% decrease of its activity The kinetics of the DEVD-ase cleavage in the reaction mixture was monitored each

5 min intervals rhXIAP in combination with HSA (Fig 2A) or alone (Fig 2B), induced twofold inhibi-tion of caspase 3 activity, but AFP⁄ rhXIAP pretreat-ment significantly reduced inhibition by rhXIAP (Fig 2A) Addition of AFP or HSA alone did not affect caspase 3 activity (Fig 2B) The results show that AFP does not directly affect caspase 3 activity, but targets XIAP by blocking its inhibitory activity against caspase 3 Therefore, AFP antagonizes XIAP function

AFP competes with caspase 3 and caspase 9 for XIAP binding

Functional interference of AFP and XIAP to examine their effect on caspase activity implied a direct physical interaction We further studied whether AFP can com-pete with caspases 3 and 9 for XIAP binding Pure recombinant His-tagged active caspases 3 and 9 were

Fig 1 AFP antagonizes XIAP-mediated caspase inhibition in cyto-chrome c-activated cell-free cytosolic extracts HepG2-derived cytosolic extracts were activated by 1 m M dATP and 5 l M cyto-chrome c and incubated with or without rhXIAP (250 n M ) with the addition of 400 n M AFP or 400 n M HSA for 1 h at 30 C Control lysates incubated without addition of HSA and AFP were taken as controls Caspase activity was measured by DEVD–AMC cleavage The mean data in RFU ± SD from three independent experiments are shown.

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incubated with rhXIAP and AFP or HSA, and protein

complexes were thereafter immobilized on

Ni–Seph-arose beads After extensive washing the supernatant

and pellets (beads) were blotted and probed with

anti-bodies to XIAP AFP, but not HSA, completely

abro-gated the association of rhXIAP with caspases 3 and 9

(Fig 3, pellet) Western blotting revealed the presence

of XIAP in the supernatants from Ni resin treated

with AFP, but only a negligible amount of free

rhXIAP was detected in the supernatants of

HSA-trea-ted samples (Fig 3) Western blotting of pellets using

antibodies against caspase 3 and caspase 9

demonstra-ted that neither AFP nor HSA was able to modulate

binding of His-tagged caspases on the nickel resin

(Fig 3) The data clearly demonstrated that AFP

cointeracted with XIAP by preventing XIAP⁄ caspase

complex formation

AFP coprecipitates with endogenous XIAP

in cellular extracts

We further studied the ability of AFP to interact with

endogenous XIAP in whole-cell extracts preactivated

with cytochrome c⁄ dATP ⁄ AFP Therefore, we

exam-ined whether AFP might be directly associated with

XIAP or⁄ and caspase 3 in cell-free extracts Protein

complexes were precipitated by the addition of

corres-ponding antibodies and protein A–Sepharose beads

Complex formation was detected by immunoblotting

of the proteins bound to the protein A–Sepharose with

anti-XIAP, anti-AFP, or anti-(caspase 3) IgG Figure 4

shows that AFP coprecipitated with endogenous XIAP

(Fig 4A, lane 3) but not with caspase 3 (Fig 4C, lane

2; C, lane 3), whereas XIAP coprecipitated with both AFP and caspase 3 (Fig 4A, lanes 2, 3) These results show that AFP associates physically with endogenous XIAP in activated cell cytosolic extracts (Fig 4A,

Fig 2 AFP abrogates XIAP-mediated inhibition of caspase 3 activity in vitro Active recombinant caspase 3 (3 n M ) was treated with: (A) a mixture of rhXIAP (200 n M ) with AFP (400 n M ) or HSA (400 n M ); (B) AFP (400 n M ), HSA (400 n M ), XIAP (200 n M ) or without additions Caspase 3 activity was measured by monitoring of DEVD–AMC cleavage at 5-min intervals Data were collected at 30 C for 30 min and expressed in RFU The mean ± SD of three independent determinations is shown.

Fig 3 AFP prevents XIAP ⁄ caspases complex formation Human recombinant XIAP was incubated for 2 h at 4 C with mixed His-tagged active recombinant caspase 3 and caspase 9 in the presence of AFP or HSA as described in Experimental procedures Protein complexes were precipitated by Ni–Sepharose beads Ni–Sepharose-bound proteins (pellet) and supernatants were ana-lyzed by SDS ⁄ PAGE ⁄ immunoblotting with anti-XIAP, anti-(caspase 3) and anti-(caspase 9) sera Input 1: rhXIAP (100 ng); Input 2: recom-binant tagged caspase 3 (50 ng); Input 3: recomrecom-binant His-tagged caspase 9 (50 ng).

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lane 3) As expected, endogenous caspase 3

coprecipi-tated with endogenous XIAP (Fig 4A, lane 2) No

interaction between caspase 3 and AFP could be

detec-ted (Fig 4B, lane 2; Fig 4C, lane 3) This indirectly

proved that AFP and caspase 3 interacted with the

same binding site of XIAP If AFP had been attached

to a binding site on the XIAP molecule other than that

responsible for caspase 3 binding, we would be able to

detect coprecipitation of all three proteins in this

experiment Hence, the results showed that AFP

act-ively binds to endogenous XIAP in cytochrome

c-acti-vated cellular extracts, but that it also prevents

complex formation of XIAP with active caspases The

data also suggested that AFP seems to bind to the

entire XIAP molecule only, because fragmented XIAP,

which was available in the cytosolic extracts (Fig 4A, lane 1), was not recovered on the immunoprecipita-tion⁄ western blotting pattern with anti-(caspase 3) and anti-AFP IgG within the limits of detection (Fig 4A, lanes 2, 3)

AFP physically associates with rhXIAP to form high molecular mass complexes

We then determined whether AFP and XIAP were able to form intermolecular complex rhXIAP was coincubated with rhAFP and then the protein mixture was subjected to native electrophoresis The complex formation was analyzed by western blotting with anti-AFP IgG (Fig 5A, lanes 1, 2) and with anti-XIAP IgG (Fig 5B, lanes 1, 2) Probing with anti-AFP revealed three AFP-specific bands (Fig 5A, lane 2), which correspond to the monomer, natural AFP-dimer and high molecular mass upper band corres-ponding to the AFP-specific macromolecular complex

To identify presence of XIAP in the AFP-specific com-plexes, we probed the blot pattern with anti-XIAP IgG This revealed presence of XIAP in the upper band, corresponding to the high molecular mass AFP-specific complex (Fig 5B, lane 2) We conclude that incubation of pure AFP with XIAP led to the forma-tion an intermolecular complex This AFP⁄ XIAP complex evidently contains more than two proteins, showing the ability of AFP to form multimolecular high-affinity complexes with XIAP

A

B

C

Fig 4 AFP associates physically with endogenous XIAP in the

cel-lular cytosolic extracts HepG2 cytosolic extract was activated by

the addition of 5 l M cytochrome c and 1 m M dATP for 30 min at

30 C in the presence of AFP (6 lg) and thereafter the specific

interaction of AFP, XIAP and caspase 3 was tested using

coimmu-noprecipitation with anti-AFP, anti-(caspase 3) or anti-(rabbit IgG) as

negative control Western blot analysis was carried out with

anti-XIAP (A), anti-AFP (B) and anti-(caspase 3) (C) sera Input: cytosolic

extract activated with cytochrome c ⁄ dATP (20 lg) Molecular mass

markers are indicated on the left.

Fig 5 Direct AFP ⁄ XIAP complex formation in vitro Recombinant human AFP and XIAP were coincubated for 1 h at 4 C and there-after subjected to the native nondenaturing PAGE followed by western blotting with anti-AFP (A) and anti-XIAP (B) IgG Lane 1 on the each pattern corresponds to rhAFP alone and lane 2 corres-ponds to AFP ⁄ XIAP.

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Search for the potential IBM in the structure of

the AFP molecule

The AFP protein contains a putative IBM-like

sequence ATIF(29–32), which fits the IAP binding

tetrapeptide consensus [9,44] (Fig 6) Similar to the

other IBM proteins, Smac, Omi and GSPT1 [17–19],

AFP requires N-terminal processing to expose the

IBM motif at the newly generated N-terminus

Processing of the first 28 amino acids could allow

exposing of N-terminal motif ATIF that is highly

reminiscent of IBM of caspase 9 and other IAP

antag-onists (Fig 6) In common with other IBMs, the

IBM-like motif of AFP bears Ala at its N-termini The Ala

residue within the IBM is highly conserved (Fig 6)

and has been shown to be essential for the interaction

between XIAP and mature Smac⁄ Diablo [45] This

sequence displays a high degree of similarity to the

IBM of caspase 9 with a single replacement of Pro3 in

caspase 9 to Ile3 in AFP (Fig 6) [22] This position is

variable in different IAP antagonists and does not

seem to be critical in forming the XIAP-binding site

(Fig 6) The AFP IBM-like sequence has Phe at the

P5 position, as in Drosophila Sickle and Grim and

Xenopsis Casp-9 (Fig 6) [22,46] As shown previously

[46], Phe at P5 position is clearly favored for BIR2

binding The requirement for proteolytical processing

of AFP to expose the N-terminal IBM may explain why only part of the total amount of AFP, that which has undergone proteolytical processing, participates in complex formation with XIAP (Fig 5A) Proteolytical processing of AFP is usually observed in cyto-chrome c-activated lysates [28] It has been shown that proteolytical cleavage of pure AFP results in AFP fragments exposing different destabilizing N-terminal residues [27] However, our results show that pure recombinant AFP and XIAP can interact without any requirements for the presence of active caspases in the reaction mixture We tentatively suggest the existence

of another IAP-binding site in AFP, one which does not require N-terminal processing to be activated for XIAP binding

Structural modeling of the AFP(dimer)⁄ BIR2–3 complex

Our results show that AFP can bind to an entire XIAP molecule but not to its fragments It has also been demonstrated that AFP displaces caspase 3 from its complex with XIAP, suggesting that the BIR2 domain

is involved in this interaction The data also suggest that AFP uses at least two different XIAP binding sites to form the AFP–XIAP complex Previous studies suggest that AFP [27], as well as XIAP [47], is able to

Fig 6 Sequence alignment of IBM-bearing proteins Collinear alignment of the N-terminal sequence 1–55 from HSA and 1–60 from human AFP (upper) Sequence alignment of IBM-bearing proteins: human AFP, caspase 9–p12, caspase 7–p20, Smac ⁄ DIABLO, GSPT1; Omi ⁄ Htr2; mouse caspase 9–p12; Xenopus caspase 9–p12; Drosophila ICE, Reaper, Grim, Hid, Sickle, Jafrac2, GSPT1; C elegans GSPT1 Identical res-idues are highlighted in black Resres-idues conserved in several IBM proteins are indicated in grey IBM-like sequence is boxed Protein sequence data have been taken from the Protein Data Bank [63].

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dimerize, which could create many possibilities for

interaction stoichiometry We suggest that AFP dimer

forms a complex with XIAP by interacting with both

BIR2 and BIR3 domains The involvement of BIR3 in

the interaction with AFP is supported by recent studies

showing that AFP induced the release of both

caspase 3 and caspase 9, as well as cIAP-2, from the

apoptosome complex [28] It is possible that caspase 9

cointeracts with AFP⁄ XIAP complex similarly to

caspase 3 It is expected that AFP dimer interacts with

the BIR2 and BIR3 domains of XIAP and forms a

2 : 1 stoichiometric complex Native electrophoresis

indicates that the AFP⁄ XIAP complex is formed by

more than two molecules and includes at least three

members (Fig 5) Taking into account that both AFP

and XIAP tend to dimerize, a few interaction models

can be proposed We suggest a simple complex

com-posed of AFP dimer and XIAP monomer

The 3D molecular structure of the AFP molecule

remains unsolved Human AFP and HSA exhibit 39%

amino acid sequence homology [44] The authentic

structural homology of AFP and HSA allowed us to

predict the tertiary structure of AFP based on the

atomic coordinates obtained by X-ray crystallography

for HSA [48] Using the NMR structures of the BIR2

[49] and BIR3 [50] domains, the AFP(dimer)⁄ BIR2–3

complex was constructed (Fig 7) In this complex, the

BIR2 and BIR3 domains show the same local twofold symmetry as two AFP monomers in the AFP dimeric structure Moreover, the IBM-interacting grooves of the BIR2 and BIR3 domains lie close to the ATIF-end

of the first and second AFP monomers, respectively, allowing for the possibility that they belong to the same XIAP molecule

Discussion

Recent evidence has broken the main paradigm of apoptosis, stating that the release of cytochrome c is the point of no return in the apoptotic program [17,18,51,52] It has been shown that certain tumor cells are able to recover after cytochrome c release and survive despite the constitutive presence of cyto-chrome c in the cytosol in the absence of any signs of apoptosis [53] Moreover, caspase activation does not always result in cell death [16–18,52] The ability of the cell to die at the postmitochondrial level depends mainly on the activity of endogenous inhibitors of apoptosis, such as IAPs, sHSPs, or Bcl-2 [6,7,54,55] There is further evidence of a high level of apoptotic activation and the upregulation of IAPs in tumor tis-sue [8,16] Inactivation of XIAP or the cancellation of XIAP inhibition appears both necessary and sufficient for cytochrome c to activate caspases and trigger cell death [9,16] The activity of IAPs is regulated by a group of IAP-regulatory proteins that bind to IAPs and inhibit their antiapoptotic function [17–21] These factors are important research targets to search for new nontoxic drugs with selective pro-apoptotic activ-ity for tumor cells The identification of protein drugs, which can overcome the tumor defense system

by preventing the realization of apoptosis in tumor cells, will have a great potential as tumor therapeutic agents [9]

In this study we showed that AFP could participate

in the regulation of apoptosis in tumor cells by coun-teraction with the most potent endogenous inhibitor of mammalian caspases XIAP Our results show that AFP binds to XIAP and disrupts its interaction with caspase 3 These results are in harmony with the fact that AFP can bind to cIAP-2 and disrupt its interac-tion with caspase 3 and caspase 9 [28] Because pure AFP can bind XIAP in vitro, this interaction appears

to be direct The binding seems to be highly specific, because it did not occur with the nonapoptotic protein HSA, structure and function of which is closely related

to AFP In addition to the direct association with XIAP, AFP could also relive the XIAP-inhibitory effect on the activity of the mature recombinant caspase 3 Moreover, AFP shows the ability to enhance

Fig 7 Hypothetical molecular model of the AFP(dimer)⁄ BIR2–3

complex Each monomer of the AFP dimer is shown in blue and

red, respectively The BIR2 and BIR3 domains are shown in green.

The dashed yellow lines connect the ATIF peptides of each AFP

monomer and the IBM-interacting grooves of the BIR2 and BIR3

domains The figure was produced using MOLSCRIPT v 2.1 [62].

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cytochrome c-dependent activation of caspase 9 and

caspase 3 in the presence of an inhibitory amount of

exogenous XIAP In this respect, AFP behaves in a

similar manner to the IAP antagonists This group of

proteins is characterized by the presence of the

N-ter-minal conserved BIR-binding motif (IBM), which is

required for IAP binding [20] The presence of IBM in

the intracellular protein allowed us to recognize its

possibility of serving as an IAP antagonist [9,13,23]

Mammalian proteins Smac⁄ DIABLO, Omi⁄ HtrA2

and GSPT1⁄ eRF3 are released from mitochondria

upon triggering apoptosis and require processing to

reveal the IBM at the newly generated N-terminus

[17–19] However, other proteins, such as ARTS and

XAF1, which do not contain an IBM-like motif, were

also seen to antagonize IAP function via an unknown

mechanism [20,21] The search for the potential

IBM-like sequence in the structure of AFP revealed the

presence of a similar amino acid sequence ATIF in

human AFP at position 29–32 [44] It can be proposed

that processing of the first 28 amino acid residues

gen-erates the AFP fragment with an N-terminal motif

ATIF that is highly reminiscent of IBM of caspase 9

and other IAP antagonists (Fig 6)

Our results indicate that only entire XIAP could

bind AFP (Fig 5) This means that AFP binds

simul-taneously to at least two BIR domains BIR3 binding

is preferential for an IBM-like motif similar to that

available in caspase 9 Taking into account that AFP

competes with caspase 3 for complex formation with

XIAP, we consider that both BIR2 and BIR3 may be

involved in complex formation with AFP A similar

model has been described previously [56] for a complex

of dimeric Smac protein with recombinant XIAP

frag-ments containing both BIR2 and BIR3 domains, or

for a complex of XIAP with active caspases 3 and -7

[10] Another model, which involves the entire AFP

molecule, could be also proposed, and will introduce

other parts of the molecule in their interaction with

XIAP To identify the molecular mechanism of the

AFP⁄ XIAP interaction additional structural studies

are needed

Although our results show that AFP interacts

phys-ically with XIAP and protects activated caspases from

IAP-induced inhibition, they do not reveal how it

operates There are several possibilities However, a

functional preference of AFP for tumor cells seems

evi-dent [30–41] It has previously been shown that AFP

selectively penetrates tumor cells via specific membrane

AFP receptors expressed on the surface of tumor cells

but not on normal adult cells [32–36] Unlike other

anti-IAPs, such as Smac or Omi, which have an

exclu-sively mitochondrial localization and become available

to interact with IAPs only after apoptosis has been triggered by cytochrome c release, AFP was available whenever it entered into the cell via cellular membrane receptors or was synthesized inside the cell Thus, AFP

is able to regulate the IAP level in the cytosol inde-pendently of whether cell is undergoing apoptosis or not Under conditions of constitutively high levels of XIAP expression in tumor cells [57], AFP could reduce its protein level, presumably by proteosome-mediated degradation

Comparison with normal cell lines and tissues has shown that many tumor cell lines and tissues have constitutively higher levels of active caspase and free cytochrome c in the absence of apoptotic stimuli and yet are not undergoing apoptosis [58,59] Simulta-neously, tumor cells have high levels of expression of survivin and XIAP [57,60] Survival of cancer cells is possible under conditions of pacific equilibrium between pro- and antiapoptotic signals Taking into account that normal cells and tissues do not overex-press apoptotic stimuli and IAPs, whereas cancer cells and tissues do, IAP-targeting drugs will have highly selective proapoptotic activity for cancer cells and lit-tle toxicity towards normal cells [8,9,16] The general obstacle preventing the design of apoptosis-regulating drugs on the basis of known natural anti-XIAPs is their intracellular localization and the inability to use them as internal regulating factors Considering that AFP can penetrate selectively into tumor cells via specific membrane receptors, the molecular mechan-ism of AFP-mediated targeting regulation of apopto-sis could be suggested to be as follows: (a) AFP selectively penetrates tumor cells via specific AFP re-ceptors; and (b) formation of the AFP–XIAP com-plex prevents its binding to activated caspases, increases XIAP instability against ubiquition⁄ protea-somal destruction and reduces the XIAP level to pro-mote apoptosis induction This function of AFP may serve to sensitize tumor cells to weak proapoptotic stimuli by inducing a tumor-specific response to che-motherapeutic or radiotherapeutic treatments The selectivity of the AFP-mediated proapoptotic activity for tumor cells may be explained by its counteraction with IAPs, which are shown to be dominantly over-expressed in tumor cells under conditions of the sim-ultaneous existence of high levels of various active proapoptotic factors

Normal cells do not undergo AFP-induced apoptosis because they do not express high levels of IAPs, do not contain constitutively activated caspases and do not express membrane AFP receptors AFP seems to

be directly involved in targeting positive regulation of the apoptotic pathway dysfunction in cancer cells by

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inhibition of IAP function leading to triggering of the

apoptosis machinery

Further studies are required to better understand the

importance of the role AFP in modulating the level of

IAPs in tumor cells Elucidation of the role of AFP

in tumor cell-specific regulation of XIAP function in

apoptosis may have important implications for cancer

treatment and prevention AFP and AFP-derived

pep-tides can potentially be used to overcome drug

resist-ance caused by the differential mechanism of apoptosis

dysfunction in cancer cells

Experimental procedures

Purification of a-fetoprotein

Embryonic AFP was isolated from human cord serum

using ion-exchange, affinity and gel-filtration

chromatogra-phy as described previously [27] The purity and

homogen-eity of the protein were assessed by SDS⁄ PAGE and

western blotting with AFP-specific polyclonal antibodies

as described elsewhere [28] Recombinant human AFP

(rhAFP) was purified from the culture medium of

recom-binant Saccharomyces cerevisiae as described previously [43]

using affinity and gel chromatography

Cells

HepG2 cells originating from the American Type Culture

Collection were grown in Dulbecco’s modified Eagle’s

med-ium (ICN Biomedicals, Inc., Costa Mesa, CA) with addition

of l-glutamine, 10% heat-inactivated fetal bovine serum,

penicillin (100 unitsÆmL)1), streptomycin (0.1 mgÆmL)1) in a

humidified 5% CO2 atmosphere at 37C For a passage,

cells were incubated in 0.25% trypsin solution, then washed

and plated out

Preparation of cell-free cytosolic extracts

Cell-free cytosolic extracts were generated from human

hepatocarcinoma HepG2 as described previously [60] with

minor modifications [28] Cells (4· 108) were collected and

washed three times with 50 mL NaCl⁄ Pi and once with

5 mL hypotonic cell extraction buffer (CEB; containing

20 mm Hepes, pH 7.2, 10 mm KCl, 2 mm MgCl2, 1 mm

dithiothreitol, 5 mm EGTA, 25 lgÆmL)1leupeptin, 5 lgÆmL)1

pepstatin, 40 mm b-glycerophosphate, 1 mm

phenyl-methylsulfonyl fluoride) The cell pellet was then

resuspend-ed in an equal volume of CEB, allowresuspend-ed to swell for 20 min

on ice and then disrupted by passing through a needle The

homogenate was centrifuged at 5000 g for 10 min at 4C

to remove whole cells and nuclei Thereafter the

superna-tant was centrifuged at 15 000 g for 20 min at 4C The

procedure was repeated twice Cytosolic extracts were

assessed for protein content by Bradford assay and stored

in aliquots at)70 C

Analysis of caspase activity Caspase assays were performed with active recombinant caspase-3 (Alexis Corp., San Diego, CA), recombinant full-length rhXIAP (R&D Systems, Minneapolis, MN), purified AFP, and HSA (Sigma-Aldrich, St Louis, MO) All other reagents were from Sigma, unless stated otherwise RhXIAP (200 nm) was incubated with AFP (400 nm) or HSA (400 nm) in IAP buffer (50 mm Hepes, pH 7.5,

100 mm NaCl, 1 mm EDTA, 5 mm dithiothreitol, 0.1% Chaps, 10% sucrose) for 15 min at room temperature Thereafter the active recombinant caspase 3 (3 nm) was added to the reaction mixture, and incubation continued for a further 15 min under the same conditions For the control, caspase 3 was incubated with each of the following compounds separately: AFP (400 nm), HSA (400 nm) or XIAP (200 nm) The kinetics of caspase activity was moni-tored by cleavage of the fluorogeneic substrate [50 lm Ac-Asp-Glu-Val-Asp-7-amino-4-methyl coumarin (Ac-DEVD-AMC), Sigma] at 5-min intervals for 30 min To assess the effects of AFP, HSA and XIAP on caspase activity in cellu-lar extracts in vitro, rhXIAP (250 nm) was incubated with cytosolic extract (40 lg) activated by addition of 5 lm of bovine heart cytochrome c and 1 mm dATP in the presence

of AFP (400 nm) or HSA (400 nm) in 15 lL of a reaction buffer (10 mm Hepes, pH 7.2, 25 mm NaCl, 2 mm MgCl2,

5 mm dithiothreitol, 5 mm EDTA, 0.1 mm phenylmethyl-sulfonyl fluoride) for 1 h at 30C, and the reaction mixtures were analyzed for Ac-DEVD-AMC cleavage Caspase 3 activity was determined by adding 5 lL of cell extracts to 16 lL of substrate reaction buffer (20 mm Hepes, pH 7.2, 100 mm NaCl, 10 mm dithiothreitol, 1 mm EDTA, 0.1% Chaps 10% sucrose, 50 lm Ac-DEVD-AMC) for 40 min at 30C The reaction was stopped by the addi-tion 200 lL of cold NaCl⁄ Pi, and AMC liberation was measured using Victor-1420 Multilabel counter (Wallac, Finland) at excitation 355 nm and emission 460 nm All samples were analyzed in duplicate and the experiments were repeated three times For each sample, caspase activity was expressed in relative fluorescent units (RFU), showing the amount of cleaved substrate normalized for protein content

Direct protein–protein binding assay

To determine possible interactions between AFP, caspase 3, caspase 9, and XIAP, we used a direct coprecipitation assay with purified proteins Human recombinant XIAP (350 ng), His-tagged human recombinant caspase 9 (50 ng), anf act-ive His-tagged rat recombinant caspase 3 (300 ng) were mixed with 0.5 mgÆmL)1 AFP or 0.5 mgÆmL)1 HSA in

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15 lL buffer A (20 mm Hepes, pH 7.4, 100 mm NaCl,

0.5 mm EDTA, 0.5 mm dithiothreitol, 0,1% Chaps, 10%

sucrose) and incubated for 2 h at 4C Thereafter, 15 lL

of Ni-Sepharose beads (Qiagen, Valencia, CA) in 80 lL of

buffer B (20 mm Na2HPO4, pH 7.2, 0.2 m NaCl) were

added to the reaction mixture and incubation was

contin-ued for the next 1 h The beads were separated from

supernatants by centrifugation and both fractions were

collected Protein–bead complexes were washed four times

and boiled in 20 lL of reducing 2· Laemmli sample

buf-fer Samples, protein–beads and supernatants were analyzed

by SDS⁄ PAGE ⁄ immunoblotting in 12.5% polyacrylamide

gel in with b-mercaptoetanol To study direct AFP–XIAP

protein interaction, human recombinant XIAP (1.5 lg) and

human recombinant AFP (2 lg) were incubated for 1 h at

4C in 30 lL of buffer C (20 mm Hepes, pH 7.2, 140 mm

KCl, 5 mm MgCl2, 2 mm dithiothreitol, 1 mm EDTA,

0.1% OVA) Thereafter, protein mixtures (5 lL) were

sub-jected to 8% nondenaturing continuous polyacrylamide gel

(Tris pH 8.7) and separated by Native electrophoresis [61]

Immunoprecipitation

Cytosolic extracts obtained from HepG2 cells were

nor-malized for protein content (500 lg of total protein in

100 lL of buffer C) and activated by addition of 5 lm

cytochrome c and 1 mm dATP for 30 min at 30C in the

presence of AFP (6 lg) The reaction mixtures were

cooled and incubated with 3 lg of the following

antibod-ies: polyclonal rabbit anti-AFP [28], normal rabbit IgG

(Sigma), or with rabbit anti-(caspase 3) (Santa Cruz, Santa

Cruz, CA) for 2 h with a gentle mixing at 4C

Thereaf-ter, 40 lL of protein A–Sepharose bead slurry (Amersham

Pharmacia Biotech) were added to the each sample

Sam-ples were incubated overnight in a rotating shaker at

4C The beads were pelleted by centrifugation and after

intensive washing, were syringe dried The bound proteins

were eluted by boiling in 25 lL of 2· sample buffer

Sam-ples in aliquots of 10 lL were loaded onto the 12.5%

SDS polyacrylamide gel and subjected to SDS⁄ PAGE ⁄

immunoblotting

Immunoblotting analysis

Samples after SDS⁄ PAGE or native PAGE were

electro-blotted onto a poly(vinylidene difluoride) membranes

(Amersham Pharmacia Biotech) using a semidry transfer

apparatus (Bio-Rad Laboratories, Hercules, CA)

Follow-ing blockFollow-ing, the membranes were incubated with primary

rabbit anti-AFP IgG [28] or goat anti-XIAP IgG (R&D

Systems, Minneapolis, MN), or anti-(caspase 3) IgG (Santa

Cruz) and then incubated with donkey anti-(rabbit IgG)

(Amersham Pharmacia Biotech) or anti-(goat IgG) (Imtek,

Russia) each conjugated to horseradish peroxidase The

blots were visualized using ECL or ECL Plus method

(Amersham Pharmacia Biotech) according to manufacturer instructions

Theoretical calculation of protein structure

In order to predict the tertiary structure of the AFP mole-cule [44] the molecular modeling software package sybyl was used (Tripos Associates, Inc., St Louis, MO) A model

of the dimeric structure of the AFP was reconstructed by using of the atomic coordinates obtained by X-ray crystal-lography for HSA [48] and plotted using molscript v 2.1 [62] The atomic coordinates of HSA (code 1AO6) were obtained from the Protein Data Bank [63] The primary structure alignment of AFP and HSA was constructed using multalin [64] The model of AFP was minimized to

an energy gradient < 0.050 kcalÆmol)1ÆA˚)1using the Tripos force field and a combination of Simplex [65] and Powell algorithms [66] Coordinates of the NMR structures of the BIR2 [49] and BIR3 [50] domains were achieved from the Protein Data Bank files: 1C9Q and 1F9X, respectively

Acknowledgements

This work was supported in part by the International Science & Technology Center, ISTC (grant #1878); by the Academy of Finland (Grant # 107762), the Neobi-ology program of the TechnNeobi-ology Development Center for Finland (TEKES), and the Sigrid Juse´lius Founda-tion

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2 Hickman JA (2002) Apoptosis and tumorigenesis Curr Opin Genet Dev 12, 67–72

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4 Oliver L & Vallette FM (2005) The role of caspases in cell death and differentiation Drug Resist Updates 8, 163–170

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