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Current 

Molecular Medicine

Volume 3, Number 3, 2003

 

Contents

 

HIV Vaccines

Executive Editor: Melissa Pope

 

HIV Vaccines: A Global Perspective Pp.183-193

J. Esparza and S. Osmanov

[Abstract]

 

Cellular Immunity for Prevention and Clearance of HIV Infection Pp.195-208

Spyros A. Kalams

[Abstract]

 

Defining the Protective Antibody Response for HIV-1 Pp.209-216

John R. Mascola

[Abstract]

 

The Role of Mucosal Immunity in Prevention of HIV Transmission Pp.217-228

Pamela A. Kozlowski and Marian R. Neutra

[Abstract]

 

Dendritic Cells as A Conduit to Improve HIV Vaccines Pp.229-242

Melissa Pope

[Abstract]

 

Subunit Protein Vaccines: Theoretical and Practical Considerations for HIV-1 Pp.243-263

Michael W. Cho

[Abstract]

 

Sites, Mechanism of Action and Lack of Reversibility of Primate Lentivirus Inactivation by Preferential Covalent Modification of Virion Internal Proteins Pp.265-272

Elena Chertova, Bruce J. Crise, David R. Morcock, Julian W. Bess, Jr., Louis E. Henderson and Jeffrey D. Lifson

[Abstract]

 

Live Recombinant Vectors for AIDS Vaccine Development Pp.273-284

Rebecca Voltan and Marjorie Robert-Guroff

[Abstract]

 

Cytokine, Chemokine, and Costimulatory Molecule Modulation to Enhance Efficacy of HIV Vaccines Pp.285-301

Jeffrey D. Ahlers, Igor M. Belyakov and Jay A. Berzofsky

[Abstract]

 

HIV Vaccines in Infants and Children: Past Trials, Present Plans and Future Perspectives Pp.303-312

Jeffrey T. Safrit

[Abstract]

 

Abstracts

 

[Back to top] HIV Vaccines: A Global Perspective

J. Esparza and S. Osmanov

 

Twenty years after its recognition, HIV/AIDS has become the most important infectious disease globally and the leading cause of death in Africa. A preventive vaccine represents the best long-term hope for its control. The development of such a vaccine, however, has encountered a number of scientific challenges, including the lack of information on immune correlates of protection, the limitations in our understanding of the relevance of primate protection experiments in relation to vaccine-induced protection in humans, and the significance of genetic and immunologic variability of HIV strains for potential vaccine efficacy. Despite these uncertainties, the first phase I trial of an HIV vaccine was conducted in the United States in 1987. Since then more than 30 candidate vaccines have been tested in over 70 phase I/II clinical trials in both industrialized and developing countries. The first HIV vaccine trial in a developing country was conducted in 1993, six years after the first trial in the United States. Since then eighteen phase I/II trials and one phase III trial have been or are being conducted in developing countries, and additional phase II and III trials are planned to start in 2003. Most of these initial trials have been conducted in Thailand, but more recently trials have been initiated in Africa, Latin America and the Caribbean. Over the past years, the HIV vaccine development effort has followed three major overlapping paradigms. The first “wave” of candidate vaccines aimed at inducing neutralizing antibodies. The second wave focused on stimulation of CD8+ T-cell responses. The current “wave” of HIV vaccine research is aimed at optimizing both humoral and cell-mediated immune responses. The first generation of candidate vaccines (based on the HIV envelope protein) entered phase III efficacy evaluation in 1998, and definitive results from these trials will become available in 2003. Plans to ensure wide access to future HIV vaccines must be developed well in advance.

 

[Back to top] Cellular Immunity for Prevention and Clearance of HIV Infection

Spyros A. Kalams

 

Despite the major strides that have been made in HIV therapy with the advent of potent antiretroviral drugs, these medications are quite expensive and are still not readily available for the vast majority of infected individuals worldwide. Even when available, the long-term toxicities associated with anti-retroviral medications and the frequent emergence of drug-resistance mutations can complicate therapy, making the formulation of effective vaccines imperative. This chapter will review the current state of understanding regarding cell-mediated immune responses that are associated with control of HIV replication. This knowledge has generated sound hypotheses regarding the prospects for augmenting cell-mediated immunity through immune-based therapies. With regard to prophylactic vaccines, it is presently unclear which vaccine-induced immune responses will protect against infection. While much progress has been made in formulating vaccine constructs designed to elicit cell-mediated immune responses, sterilizing immunity is unlikely to be achieved with the current vaccines. However, the ability to control viremia and prevent disease progression in animal infection models looks promising. The ability to measure immune responses has also advanced markedly over the past few years and will allow investigators to more accurately measure the immunogenicity of vaccine constructs, and correlate the magnitude and breadth of these responses with protection.

 

[Back to top] Defining the Protective Antibody Response for HIV-1

John R. Mascola

 

The development of an effective HIV-1 vaccine would be greatly facilitated by knowledge regarding the type and quantity of antibodies that are protective. Since definitive immune correlates are established only after a vaccine has been shown to be effective in humans, animal models are often used to guide vaccine development. Experimental lentivirus infection of non-human primates has shown that neutralizing antibodies can protect against infection. Most specifically, studies of passive antibody transfer in the chimeric SIV/HIV-1 immunodeficiency virus (SHIV) model have provided quantitative data on the level of protective antibody required. While direct extrapolation to humans is difficult, these data likely provide important insights into the protection afforded by antibodies against HIV-1. When used alone, high levels of neutralizing antibodies are required to completely block infection. However, even modest levels of antibody can provide partial protection and affect disease course. Understanding the exact level of protective antibody becomes even more complex in the setting of active immunization and coexisting cellular immunity. Despite this uncertainty, recent findings from lentiviral animal models strongly suggest that neutralizing antibodies will contribute to protection against HIV-1. Based on these data, a major goal of HIV-1 vaccine strategies is the induction of neutralizing antibodies against circulating primary HIV-1 strains.

 

[Back to top] The Role of Mucosal Immunity in Prevention of HIV Transmission

Pamela A. Kozlowski and Marian R. Neutra

 

Vaccines designed to prevent mucosal transmission of HIV should establish multiple immune effectors in vaccine recipients, including antibodies which are capable of blocking HIV entry at mucosal epithelial barriers and of preventing initial infection of target cells in the mucosa. Immunological analyses of HIV-resistant humans and data obtained in nonhuman primate vaccine studies indicate that both secretory and serum antibodies may play an important role in protection against mucosal transmission of HIV or SIV, whereas cytotoxic T cells are required for clearance of mucosal infection and prevention of systemic spread. This review summarizes the roles of IgA and IgG antibodies in preventing mucosal infection by other viral and bacterial pathogens, and then discusses the various mechanisms by which antibodies might contribute to protection against HIV at mucosal surfaces. These include prevention of mucosal contact, blocking attachment of virus or infected cells to epithelial cells, interception of virus during transepithelial transport, neutralization of virus in the mucosa, and elimination of locally infected cells through antibody-dependent cell-mediated cytotoxic reactions. The regional nature of mucosal immune responses is reviewed in light of its relevance to HIV vaccine development. We conclude that mucosal immunization should be considered a component of vaccine strategies against HIV.

 

[Back to top] Dendritic Cells as A Conduit to Improve HIV Vaccines

Melissa Pope

 

Many potential HIV vaccine strategies are being explored in both animal model and human settings. The success of any vaccine relies on relevant antigenic determinants being presented to the immune system for the activation of broad and long-lasting immunity. Effective immunity against HIV infection will likely require both the cellular and humoral arms of the immune system, where HIVspecific killer cells eradicate infected targets and neutralizing antibody responses contribute by preventing the initial infection of host cells. As the most potent antigen presenting cell of the immune system, the dendritic cell (DC) orchestrates the activation of adaptive immune responses as well as contributing to the early innate responses to a pathogen, which may also aid in the initial control of infection. It follows therefore, that the efficiency of a vaccine antigen would be greatly enhanced if targeted to the appropriate DCs to ensure optimal presentation to and subsequently activation of the immune system. This review will discuss (i) the current status of DC biology, covering distinct DC subsets and stages of activation and how these influence the types of immune responses that are induced, (ii) how DCs can be exploited to improve the efficacy of HIV vaccine strategies currently under investigation, (iii) what has been learned from in vivo model systems using DCs, and (iv) future considerations to advance HIV vaccinology.

 

[Back to top] Subunit Protein Vaccines: Theoretical and Practical Considerations for HIV-1

Michael W. Cho

 

With the spread of AIDS still rampant in many parts of the world, there is a global urgency to develop a vaccine against HIV-1. Without a doubt, developing an effective vaccine against the virus has been a monumental scientific challenge. Although advances in molecular biology and biotechnology over the years have enabled us to generate “designer antigens,” our ability to transform them into successful vaccine candidates has been limiting. This review will be divided into three sections: First, the theoretical benefits and limitations of subunit protein vaccine strategy will be presented. Secondly, recent progress in our understanding of immune responses against AIDS vaccine candidates that incorporate recombinant proteins or peptides will be reviewed, mainly those that are designed to elicit humoral immune responses. Finally, some of the factors that must be considered in designing and evaluating future vaccine candidates will be discussed.

 

[Back to top] Sites, Mechanism of Action and Lack of Reversibility of Primate Lentivirus Inactivation by Preferential Covalent Modification of Virion Internal Proteins

Elena Chertova, Bruce J. Crise, David R. Morcock, Julian W. Bess, Jr., Louis E. Henderson and Jeffrey D. Lifson

 

By exploiting the intrinsic chemistry of retroviruses, we have developed a novel method for generating whole inactivated virion vaccine immunogens with functional envelope glycoproteins. The method takes advantage of the fact that the internal proteins of retroviruses are adapted to the intracellular (reducing) environment, and have cysteine residues present in thiol-form (S-H), while the surface proteins of retroviruses (the envelope glycoproteins SU and TM) are adapted to the (oxidizing) environment of the extracellular milieu, and have their cysteines present as disulfides (S-S). Treatment of retroviral virions with appropriate mild oxidizing agents thus results in preferential covalent modification and functional inactivation of key S-H-containing internal viral proteins, such as the nucleocapsid (NC) protein, that are required for infectivity, while the envelope glycoproteins with their disulfide bonded cysteines remain unaffected. This treatment thus results in virions that do not retain detectable infectivity, but preserves the conformational and functional integrity of the envelope glycoproteins. We have extensively used the disulfide reagent 2,2’-dithiodipyridine (aldrithiol-2, AT-2) to inactivate HIV and SIV via this mechanism and such inactivated virions appear to be a promising vaccine immunogen based on macaque studies. We have biochemically characterized the targets and mechanisms of inactivation involved in AT-2 treatment of virions, and investigated the kinetics of inactivation. Although extremely unlikely under physiological conditions, reversibility of this type of inactivation is a theoretical concern. We have therefore conducted a series of in vitro experiments, in cell free systems and in cell culture, to evaluate this possibility. The results indicate that as judged by both biochemical and biological (infectivity) criteria, inactivation by AT-2 does not appear to be reversible under conditions likely to obtain in vivo.

 

[Back to top] Live Recombinant Vectors for AIDS Vaccine Development

Rebecca Voltan and Marjorie Robert-Guroff

 

Live recombinant vectors entered the AIDS vaccine field with the realization that live attenuated HIV vaccines posed too great a safety risk, and that subunit vaccines elicited antibodies which lacked the breadth or potency needed to induce sterilizing immunity. Vectored vaccines provided a means to bring the cellular arm of the immune system into play by mimicking natural viral infection. By delivering antigens within host cells, processing and presentation could occur for induction of cellular immune responses. This recombinant vector approach, either alone or combined with other strategies, has produced impressive results. Recombinants have been generated from DNA and RNA viruses and bacteria. With few exceptions, each vector poses some risk, yet each possesses unique features that make it attractive. In addition to safety, key considerations in vector selection have included previous success as a vaccine against the wild-type agent or other pathogens; ability to induce potent, persistent immune responses; ability to target mucosal inductive sites and antigen presenting cells; lack of integration into the host genome; presence of pre-existing immunity in people; ease of mucosal administration; cloning capacity; ease of engineering and production; and stability of the final product. Here we up-date the status of several live recombinant vectors that have shown good potential in pre-clinical studies. Some have progressed to human clinical trials, and others will shortly. The abundance of vectors, coupled with the complexity arising from use of combination regimens with other vaccine types and heterologous vectors, will necessitate selection of the most promising candidates for large-scale efficacy trials in people. The sooner comparative studies can be designed and implemented in which live recombinant vectors containing the same inserted genes are evaluated head-to-head, the closer we will be to an eventual vaccine.

 

[Back to top] Cytokine, Chemokine, and Costimulatory Molecule Modulation to Enhance Efficacy of HIV Vaccines

Jeffrey D. Ahlers, Igor M. Belyakov and Jay A. Berzofsky

 

Understanding key intervention points in developing immune responses may allow the rational inclusion of biological adjuvants into vaccines that could potentiate the immune response both quantitatively and qualitatively and enhance effective memory responses. Cytokine and chemokine combinations can potentially help target antigen to the appropriate antigen presenting cell and initiate maturation of these presenting cells, attract cells expressing different chemokine receptors, steer cellular immune responses toward Th1 and CD8 CTL, and enhance systemic and mucosal IgG and secretory IgA antibodies and determine their isotype balance. Animal protection studies suggest that synergistic combinations of cytokines and immunomodulating molecules may be required to protect from a viral challenge. For example, GM-CSF has been shown to be synergistic with IL-12 or CD40 ligand for induction of CTL and for antiviral protection, and the triple combination of GM-CSF, IL-12, and TNF alpha appears to induce the most effective protection in some mouse models. Chemokineantigen fusions have also been shown to enhance immunogenicity of the antigen. Combinations of costimulatory molecules have been found to be synergistic when incorporated in a vaccine. Combined use of newer more potent vaccine constructs, containing codon optimized epitopes, relevant CpG motifs, cytokines, costimulatory molecules and chemokines, used in heterologous prime-boost strategies with viral vector vaccines or recombinant proteins, might afford the most potent vaccine approaches yet developed. In this review we will discuss the application and delivery of cytokines, costimulatory molecules, and chemokines toward improving current vaccine strategies.

 

[Back to top] HIV Vaccines in Infants and Children: Past Trials, Present Plans and Future Perspectives

Jeffrey T. Safrit

 

This review will address the recent history in HIV vaccine trials in the pediatric population while giving due respect to the pediatric vaccine successes achieved over the past decades. Success and failure seen when utilizing the neonatal macaque model of SIV infection and the ramifications of these studies will be discussed. The short list of pediatric HIV vaccine trials currently in progress and those in early planning stages will be reviewed. Finally, future perspectives on the impact of a vaccine that could be used to potentially avert mother-to-child transmission of HIV and lead ultimately to the establishment of immunity throughout adolescence and beyond will be presented.