Cervical Cancer: Epidemiology and Prevention Research Advances Mahboobeh Safaeian, Ph.D. safaeianm@mail.nih.gov National Cancer Institute HPV & Cervical Cancer Research Discovery of HPV Natural history of HPV and pathogenesis of cervical cancer defined Method for assembly of HPV VLP defined 1983 Late 80’s-90’s Cervarix first licensed in the EU for adolescent girls/young women Phase III clinical trials in humans 1992 HPV vaccine trials in animals and early phase trial in humans 1990’s-early 2000’s FDA licenses Gardasil for girls/young women 2000’s 2004 2006 2007 FDA extends Gardasil licensure to males & for prevention of anal infections/ lesions 2009+ Summary of Today’s Talk • Epidemiology of cervical cancer • Prevention • Immunogenicity of HPV Cervical Cancer epidemiology • Worldwide ~500,000 new cases per year • Worldwide ~250,000 deaths per year • 2nd/3rd most common cancer in women • Incidence and survival rates vary by geographical region Age-standardized incidence and mortality rates of cervical cancer, 2002 Mortality rate per 100,000 women 4.0 11.2 Incidence rate per 100,000 women 10.3 19.1 0 5 10 15 20 Developed Countries Less Developed Countries GLOBOCAN 2002 25 The Primary Risk Factor for Cervical Cancer: Human Papillomavirus (HPV) HPV • Small DNA virus (~8000bp) • More than 100 types identified based on the genetic sequence of the outer capsid protein L1 • 40 types infect the mucosal epithelium • ~15 carcinogenic HPV types HPV - epidemiology • Most common viral sexually transmitted infection • High infectiousness • Peak prevalence 15-25 years • The HPVs can infect men and women at multiple sites, including: – MEN: oral cavity, penis, scrotum, urethra, anus – WOMEN: oral cavity, cervix, vagina, vulva, anus • Infections generally transient (1-2 years) • Manifestation of infection depends on the site of infection and the genotype of HPV. – Carcinogenic neoplasia and cancer of cervix, oropharynx, and anus; less likely to cause cancer at other sites, ex: HPV16 and 18 – Non-carcinogenic anogenital warts, ex: HPV6 and 11 Working model of cervical carcinogenesis normal cervix HPV infection persistence high grade neoplasia invasion Working model of cervical carcinogenesis normal cervix HPV infection Younger age Increasing # sex partners “Male factor”* persistence high grade neoplasia invasion Working model of cervical carcinogenesis normal cervix HPV infection Younger age Increasing # sex partners “Male factor”* persistence HPV type (HPV-16) Host immune response Exogenous factors eg. Smoking? high grade neoplasia invasion Working model of cervical carcinogenesis normal cervix HPV infection Younger age Increasing # sex partners “Male factor”* persistence HPV type (HPV-16) Host immune response Exogenous factors eg. Smoking? high grade neoplasia Increasing age HPV type (HPV-16) Smoking Multiparity? OC use? Host immune responses invasion Secondary Prevention: Interrupting disease progression Working model of cervical carcinogenesis normal cervix HPV infection persistence high grade neoplasia invasion Screening for HPV infection and precursor lesions Frequent pap smear cytology HPV DNA testing (age 30+) Treatment of precursor lesions by excisional therapy is > 95% effective The Papanicolaou (Pap) test has significantly reduced the incidence of cervical cancer in populations where screening has been well implemented. Country Coverage Mortality reduction Iceland National 80% Finland Wide 50% Switzerland Wide 34% Denmark 40% 25% Norway 5% 10% Anderson et al. Organization and results of the cervical cytology screening program in British Columbia, 1955-85. Br Med J 1988; 296(6627): 975-8 Coverage: 85% Incidence of cervical cancer: fell 78% Mortality from cervical cancer: 72% If Pap Smears successfully prevented cervical cancer, why not simply use them to screen everyone else? A. Three-visit cycle: 1. Cytology; 2. Colposcopy; 3. Treatment B. Cytology is an insensitive test (negative test = poor reassurance); repeat iterations (e.g., regular or annual Pap smears) are necessary. C. Cytology is a poorly reproducible (subjective) test and difficult to maintain performance D. Colposcopy is not as good as you think it is. E. Bottom Line: U.S. program costs billions of $ annually F. Tests with better test characteristics, less visits per cycle, fewer cycles per lifetime HPV Testing for Screening ALTS Trial HPV vs Repeat Cytology for Triage of ASCUS (N=2,324) Sensitivity % Referral (CIN2+) HC2 95.9% 56.1% ThinPrep (ASCUS+) 85.0% 58.6% Solomon et al, JNCI, 2001 Comparative efficacy of visual inspection with acetic acid, HPV testing and conventional cytology in cervical cancer screening: a randomized intervention trial in Osmanabed District, Maharashtra State, India Cervical cancer incidence rates among screen negative women by study group (2000-2007) New cancer cases # of Women Incidence Rate (per 100,000) HPV 8 24,380 3.7 Cytology 22 23,762 15.5 VIA 25 23,032 16.0 Osmanabed District, Maharashtra State, India, Sankaranarayanan et al., NEJM, 2009 Current US Recommendations: Women 30 Years and Older Cytology Negative Oncogenic HPV Testing Oncogenic HPV - Repeat test in 3 years Oncogenic HPV+ Repeat test in 6-12 months Concerns about Screening Using HPV Tests • Will screening be used incorrectly, e.g., among young women? • Will the tests include the right types and proper thresholds for positivity? Primary Prevention: Prophylactic vaccination Current Prophylactic Vaccines are Based on L1 Papillomavirus-Like Particles • Empty shells composed of the L1 major virion protein 25 HPV VLP Vaccines Manufacturer Trade name HPV types Theoretic coverage Merck GlaxoSmithKline Gardasil® Cervarix™ L1 HPV6 20 ug L1 HPV11 40 ug L1 HPV16 40 ug L1 HPV18 20 ug 70% of CxCa 90% GW L1 HPV16 20 ug L1 HPV18 20 ug 70% of CxCa Expression system Aluminum salt (Alum): 225 ug Yeast ASO4: (Alum: 500 ug + MPL: 50 ug) Hi-5 Baculovirus Dosing Schedule 0, 2, 6 months 0, 1, 6 months Adjuvant Who Can Receive the Vaccine and For What Purpose? Licensure (FDA) Gardasil licensure Females ages 9-26 for the prevention of cervical, vulvar, vaginal, and anal disease Males ages 9-26 for the prevention of genital warts and anal disease Cervarix licensure Recommendations for Use (ACIP-CDC) Females ages 10-25 for the prevention of cervical disease Advisory Committee on Immunization Practices-CDC recommendations Females: Recommended for ages 11-12; as early as 9 yrs & catch up vaccination through age 26 Males: Gardasil recommended for boys 11-12; catch-up to 21 yrs HPV Vaccine Efficacy* Endpoint HPV 16/18-related CIN2/3 or AIS Efficacy 100% HPV 6/11/16/18 related CIN 95% HPV 6/11/16/18 related genital warts 99% *Among 16-26 year old females. CIN – cervical intraepithelial neoplasia; AIS – adenocarcinoma in situ HPV Vaccine Doesn’t Treat Established Infections Control Arm (298/220) HPV Arm (248/177) 49.8 48.8 % Clearance 50 40 31.3 33.4 30 VE = -2.0% 20 10 VE = 2.5% 0 6-Mo 12-Mo Time 29 Hildesheim et al., JAMA 2007 HPV Vaccine Adverse Reactions • Local reactions 84% (pain, swelling) • Fever 10%* • No serious adverse reactions reported *similar to reports in placebo recipients (9%) Does the vaccine protect against other HPV types? (cross-protection) Summary of the vaccine efficacy reported for selected HPV types not in the vaccine formulation: FUTURE1 Gardasil 6-month persistence PATRICIA2 Cervarix 12-month persistence CVT3 Cervarix 12-month persistence Related type HPV31 HPV52 HPV58 HPV33 33.6%* 2.3% 12.8% 22.5% 80.5%* -7.8% -17.7% 41.0%* 45.7%* -20.0% -6.3% 37.3% HPV45 20.1% 60.0%* 52.0% Outcome Evaluated Vaccine HPV type HPV16 HPV18 *Statistically significant VE 32 1: NEJM 2007 2: Lancet 2009 3: Cancer Discovery 2011 Does the vaccine protect against HPV infections at other anatomic sites? HPV causes cancer at several anatomic sites Figure compliments of Dr. Doug Lowy Protection against other cancers • Vaginal/vulvar- 100% VE • Anal cancer- from CVT ~85% in naïve population • Penile- high protection against penile precancer • Oral- no data yet Summary of Vaccine Findings in Young Women • High level prevention of precancerous cervical lesions when administered to females without infection by vaccine-type HPVs – Not therapeutic • Generally safe and well-tolerated – None of the deaths considered vaccine related – Cannot cause infection or cancer (b/c only contains 1 protein from each targeted type) • Additional results – High efficacy for prevention of HPV-related extra-genital lesions Summary of questions that still need addressed • How long is the duration of protection (past 10 yrs)? • Will the vaccine work in special populations (i.e.: HIV-positive)? • How should the vaccine be integrated into existing screening programs? • How should the vaccine be introduced in countries without screening programs? Cervical VE among women who received <3 doses of the HPV vaccine Background • 3-dose HPV vaccine regimen provides robust protection against cervical HPV infection and disease – costly and difficult to complete, especially in lowresource settings • ~20% of women received <3 doses – due to pregnancy and colposcopy referral • Objective: compare efficacy of <3 doses to the standard regimen Dose- stratified VE after 4-yrs follow-up # of Doses Arm Control # with % with # persistent* persistent Women HPV16/18 HPV16/18 infection infection 3010 133 HPV16/18 VE (95%CI) 4.4% 3 80.9% (71.1% to 87.7%) HPV Kreimer AR et al JNCI 2957 25 0.9% *12 month+ persistence Dose- stratified VE after 4-yrs follow-up # of Doses Arm # Women # with persistent HPV16/18 infection Control 3010 133 % with persistent HPV16/18 infection 4.4% 3 HPV16/18 VE (95%CI) 80.9% (71.1% to 87.7%) HPV 2957 25 0.9% Control 380 17 4.5% 2 84.1% (50.2% to 96.3%) HPV 422 3 Kreimer AR et al Provisionally accepted JNCI 0.7% Dose- stratified VE after 4-yrs follow-up # of Doses Arm # Women # with persistent HPV16/18 infection Control 3010 133 % with persistent HPV16/18 infection 4.4% 3 HPV16/18 VE (95%CI) 80.9% (71.1% to 87.7%) HPV 2957 25 0.9% Control 380 17 4.5% 2 84.1% (50.2% to 96.3%) HPV 422 3 0.7% Control 188 10 5.3% 1 100% (66.5% to 100%) HPV 196 0 0.0% p trend= 0.2 Kreimer AR et al Provisionally accepted JNCI Lack of evidence for bias in immune levels by # of doses received HPV16 antibody titers one-month post-vaccination Doses received N Median Range 1 30 355 59 - 43388 2 140 425 4 – 56575 3 120 456 51 - 72760 Kruskal-Wallis 0.45 Summary • Sufficient evidence to modify existing 3-dose schedules? – Not randomized – Small number of events – Durability of protection for <3 doses • Proof of principle: <3 doses may provide a high degree of protection against cervical HPV-16/18 – Public health: could prevent similar amounts of disease with fewer doses of vaccine Immunogenicity of bivalent HPV vaccine among partially vaccinated young adolescent girls in Uganda Rationale • HPV vaccine produces high levels of antibodies – Shown to be protective of HPV16/18 infections and associated lesions – Neutralizing antibodies thought to be responsible for the protection • From CVT, vaccine efficacy among adult women who received either one or two doses of HPV vaccine was similar to women who received all three vaccines doses. • None of the HPV vaccine efficacy randomized clinical trials were conducted in Africa, where majority of cervical cancer cases occur. Rationale • Difficult to know whether partial vaccination will provide the same benefit of protection from HPV infection as full vaccination – Use immunogenicity data to address • Prior infection with HPV provides protection – Modest levels of antibodies produced following natural infection are sufficient for protection from subsequent infection with the same HPV type. Hypothesis • Given what we know from the vaccine and natural history studies, we hypothesize that the anti-HPV antibodies of girls who received 1 or 2 does of the vaccine even though decreased will be non-inferior compared to those who received 3 doses – Significantly higher than levels reported after natural infection required for protection Primary Objectives • To investigate whether the HPV16/18 immune responses elicited by the bivalent vaccine for girls who received one dose and those who received two doses are similar to girls who received all three doses of the bivalent HPV vaccine. Collaborators • PATH – D. Scott LaMontagne, PhD • Uganda – Emmanuel Mugisha, MPH, PhD – Edward Kumakech, BSN, MSc, MPH • NCI – Mahboobeh Safaeian, PhD – Ligia Pinto, PhD Immunogenicity of HPV VLP Prophylactic Vaccines: Where We Are and Where We Are Going 52 Studies at the HPV Immunology Lab at NCI HPV VLP Vaccine Cellular Immune Responses Humoral Immune Responses Neutralizing Abs Affinity Systemic Local B cell memory T cell proliferative responses Th1; Th2; inflammatory cytokines Cross-reactivity ? Correlates/Mechanisms ? Long-Term Protection Cross Protection Host Gene Expression Profiles Assays used to measure HPV Immunogenicity Polyclonal (P) vs. Monoclonal (M) P Neutralizing No Remark GSK Competitive Luminex immunoassay (cLIA) M Yes Merck Secreted alkaline phosphatase neutralization assay (SEAP-NA) P Yes Name ELISA Throughput, volume requirement High throughput, low sample volume High throughput, multiplexing, high sample volume Gold standard; Low throughput, Laborious high sample volume HPV16/18 Immunogenicity 55 Vaccine Immunogenicity - CVT HPV16 & HPV18 ELISA GMT: by vaccination arm Antibody Levels (GMT) EU/mL 10000 1000 HPV Vaccinated 100 Naturally Infected Not infected 10 1 0 1 6 7 12 24 36 48 Safaeian et al. in preparation I. Natural History Background • HPV vaccine evokes high levels of antibodies that are protective for HPV16/18 infections and associated lesions • Neutralizing antibodies are demonstrated to be sufficient for protection in animals Do antibodies produced following HPV infection protect against subsequent HPV infections? Impact of antibodies on future infection - Methods Costa Rica HPV16/18 Vaccine Trial (CVT): Control Arm Control Arm: N=3736 Exclusions: N=923 HPV16 DNA- N=2813 HPV16 Sero+ (N=699; 25%) HPV16 Sero- (N=2114; 75%) Impact of antibodies on future infection - Methods • Exposure: anti-HPV16 (or 18) serostatus at entry among HPV16 (or 18) DNA-negatives at enrollment – Tertiles • Outcome: Rates of newly detected HPV16 (or 18) infections during follow-up • Direct (polyclonal) ELISA used to measure seropositivity Multivariate Rate Ratio Elevated antibody levels associated with decreased risk of infection compared with seronegatives for both HPV16 (50%) and HPV18 (60%) 10 Seronegative: Reference Seropositive: Low tertile Seropositive: Medium Tertile Seropositive: High Tertile Sero- 1 0.5 0 HPV16 HPV18 Safaeian et al., JNCI, 2010 Implications • High levels of vaccine evoked antibody Median HPV 16 AB in the high tertile group 133 (85-255) Median HPV16 AB among vaccinated women At 7 months At 12 months 3631 (1823-5520) At 48 months 2304 (1381-4308) 1212 (623-2449) • Duration of current vaccine protection may be long. II. Vaccination Cross-protection Summary of the ATP vaccine efficacy reported for selected HPV types not in the vaccine formulation FUTURE1 Gardasil 6-month persistence PATRICIA2 Cervarix 12-month persistence CVT3 Cervarix 12-month persistence Related type HPV31 HPV58 HPV33 33.6%* 12.8% 22.5% 80.5%* -17.7% 41.0%* 45.7%* -6.3% 37.3% HPV45 20.1% 60.0%* 52.0% Outcome Evaluated Vaccine HPV type HPV16 HPV18 *Statistically significant VE 64 1: NEJM 2007 2: Lancet 2009 3: Cancer Discovery 2011 Which marker to follow-up? • 48 women – Randomly selected, 3 doses of the vaccine received • Sera from enrollment, 1m and 12m visits • SEAP (an assay to measure neutralizing potential) with: – HPV16, HPV18 (positive control) – HPV31, HPV45 (vaccine efficacy observed) – HPV52, HPV58 (no vaccine efficacy) – BPV (negative control) Cross-Protection Pilot Study * * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross-Protection Pilot Study * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross-Protection Pilot Study * * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross-Protection Pilot Study * * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross protection findings • Characterized and optimized the SEAP for several phylogenetically related HPV types • Neutralizing antibodies may be responsible Cross-protection Nested Case-control Study • HPV vaccinated arm only • Compare immune responses of: – Cases: HPV vaccinated women with an incident infection with HPV types 31 (n=97), 45 (n=55), and 58 (n=100) – Controls: HPV vaccinated women without infection with these types (n=120) • Serum from multiple time-points tested and levels compared between cases and controls. Acknowledgements • Proyecto Guanacaste, Costa Rica – Rolando Herrero – Ana Cecilia Rodriguez – Paula Gonzalez – Carolina Porras – Silvia Jimenez – Jorge Morales • DDL – Wim Quint – Leen-Jan van Doorn • • • • • HPV Immunology Lab Ligia Pinto Troy Kemp David Pang Marcus Willliams • US NCI – Allan Hildesheim – Sholom Wacholder – Mark Schiffman – Diane Solomon – Mark Sherman – John Schiller – Doug Lowy – Sarah Coseo – Wenny Lin – Arpita Ghosh • GSK Sylviane Poncelete Francis Dessy • PPD • Mark Esser Special thanks to the women of Guanacaste who participated in this study. E EXTRA SLIDES Results from Randomized Clinical Trials of HPV L1 VLP Vaccines Both quadrivalent (HPV-16,-18, -6, -11) and bivalent (HPV-16, 18) vaccine trials showed: - High levels of immunogenicity >99% seroconversion rates in 9-26 years old Antibody titers considerably higher than observed in natural infection Antibodies increase with each dose and decline over time after the 3rd dose Heterogeneity observed between individuals; this heterogeneity not well understood 74 Antibody Responses to the Vaccine Sustained Through 6.4 years B, Romanowski for the GlaxoSmithKline Vaccine HPV-007 Study Group; 75Lancet 2009 Serum Neutralizing Antibody Levels After Cervarix & Gardasil Vaccination Direct Comparison Reverse Cumulative Distribution Curves After 3 Doses (HPV DNA/Sero-Negative at Entry) 18-26 years 76 (Einstein M. et al. Hum Vaccine 2009) Assays used to measure HPV Immunogenicity Polyclonal (P) vs. Monoclonal (M) P Neutralizing No Remark GSK Competitive Luminex immunoassay (cLIA) M Yes Merck Secreted alkaline phosphatase neutralization assay (SEAP-NA) P Yes Name ELISA Throughput, volume requirement High throughput, low sample volume High throughput, multiplexing, high sample volume Gold standard; Low throughput, Laborious high sample volume HPV Vaccine Studies at the HPV Lab Defining Markers of Immune Responses to Vaccination (Ultimate goal is to correlate against long-term vaccine success/failure) Humoral Immunity: HPV Neutralizing antibodies induced by vaccination in blood and at the cervix Validation and optimization of assays to identify ideal assay and sample type Cross-Neutralizing antibodies associated with protection against crossrelated HPV types Antibody Avidity and B cell memory responses 78 HPV Vaccine Humoral Immunity: Systemic and Mucosal Anti-HPV16 and Anti-HPV18 Antibody Responses from Vaccinated Women 1. Which is the ideal method for monitoring antibody responses in clinical trials? 2. Are systemic levels of antibodies reflective of cervical levels ? (Kemp et al. 2008. Vaccine) 79 Main Methods Used in Clinical Trials for Measurement of Antibodies - HPV IgG Levels - ELISA (direct and V5/J4 inhibition assay) - Luminex Bead Assay (cLIA) - Neutralization Potential - SEAP (neutralization assay) 80 ELISA correlated very well with the SEAP-NA Serum Cervical Secretion A. C. 105 N= 100 Spearman= 0.91 p< .0001 105 Anti-HPV16 Titer (1/Dilution) Anti-HPV16 Titer (1/Dilution) 106 104 103 102 101 100 N= 42 Spearman= 0.84 p< .0001 104 103 HPV-16 102 101 100 100 101 102 103 104 105 106 100 101 Anti-HPV16 VLP IgG (EU/ml) 104 D. 106 105 N= 42 Spearman= 0.89 p< .0001 105 N= 100 Spearman= 0.85 p< .0001 Anti-HPV18 Titer (1/Dilution) Anti-HPV18 Titer (1/Dilution) 103 Anti-HPV16 VLP IgG (EU/ml) B. SEAP-NA 102 104 103 102 101 100 104 103 HPV-18 102 101 100 100 101 102 103 104 Anti-HPV18 VLP IgG (EU/ml) ELISA 105 106 10-1 100 101 102 103 Anti-HPV18 VLP IgG (EU/ml) 81 (Kemp et al. 2008. Vaccine) 104 Serum antibody titers are generally levels ELISA reflective of cervical SEAP-NA (10-20 fold lower) A. 106 N= 42 Spearman= 0.73 p< .0001 105 Anti-HPV16 Titer (1/Dilution) Anti-HPV16 VLP IgG (EU/ml) 106 C. 104 103 102 101 100 N= 42 ELISA Spearman= 0.74 p< .0001 105 104 103 HPV-16 102 101 100 100 101 102 103 104 105 101 102 Anti-HPV16 VLP IgG (EU/ml) D. 106 105 N= 42 Spearman= 0.64 p< .0001 106 N= 42 Spearman= 0.75 p< .0001 Anti-HPV18 Titer (1/Dilution) Anti-HPV18 VLP IgG (EU/ml) 104 Anti-HPV16 Titer (1/Dilution) B. Serum 103 104 103 102 101 100 105 104 103 HPV-18 102 101 100 10-1 100 101 102 103 Anti-HPV18 VLP IgG (EU/ml) 104 105 101 102 103 Anti-HPV18 Titer (1/Dilution) 82 (Kemp et al. 2008. Vaccine) Cervix 104 Cross-protection • 48 women – Randomly selected, 3 doses of the vaccine received • Sera from enrollment, 1m and 12m visits • SEAP with: – HPV16, HPV18 (positive control) – HPV31, HPV45 (vaccine efficacy observed) – HPV52, HPV58 (no vaccine efficacy) – BPV (negative control) HPV Neutralizing Titers (SEAP) Cross-Protection Pilot Study 10000 Pre-Vax * 1 dose All doses * 1000 100 * * 10 1 HPV16 HPV18 HPV31 HPV45 HPV52 HPV58 *p<0.001 BPV HPV Type Kemp…Safaeian et al., Vaccine, 2011 HPV Neutralizing Titers (SEAP) Cross-Protection Pilot Study Pre-Vax 10000 * 1 dose All doses * 1000 * * 100 * 10 1 HPV16 HPV18 HPV31 HPV45 HPV52 HPV58 *p<0.001 BPV HPV Type Kemp…Safaeian et al., Vaccine, 2011 HPV Neutralizing Titers (SEAP) Cross-Protection Pilot Study Pre-Vax 10000 * 1 dose All doses * 1000 * 100 * * * 10 1 HPV16 HPV18 HPV31 HPV45 HPV52 HPV58 *p<0.001 BPV HPV Type Kemp…Safaeian et al., Vaccine, 2011 HPV Neutralizing Titers (SEAP) Cross-Protection Pilot Study Pre-Vax 10000 * 1 dose All doses * 1000 * 100 * * * 10 1 HPV16 HPV18 HPV31 HPV45 HPV52 HPV58 *p<0.001 BPV HPV Type Kemp…Safaeian et al., Vaccine, 2011 Cross protection findings • Characterized and optimized the SEAP for several phylogenetically related HPV types • Neutralizing antibodies may be responsible HPV Vaccine Humoral Immunity: Analysis of New Functional Markers Antibody Avidity Memory B cell responses (Dauner & Pan, submitted) 89 Expanding our understanding of the humoral immune response • Functional aspects of B cell responses to HPV vaccines have not been examined previously – Avidity plays an important role in protection against infections – Memory B cells are important for long-term protection against viral infection • We are interested in understanding: – The effect of vaccine on affinity and B-cell memory – The correlation between affinity/B-cell memory measures and total antibody levels generated by vaccination • Ultimate goal: Evaluate role of antibody avidity and memory B-cells on long-term protection against vaccine types and cross-related types. 90 Avidity ELISA • Affinity is the measure of the strength of a single interaction between molecules • Avidity is the total strength due to multiple interactions – Applies to antibodies because they are bivalent • Technique – ELISA based methods using a chaotropic agents disrupt proteinprotein interactions (between antibody and antigen) 91 Avidity Levels Increase With the Number of Doses Vaccine Received Sample Collection Month 0 Month 1 Month 0 Month 1 Month 6 Month 2 Month 7 92 Month 12 Avidity Levels and Correlations with Antibody Titers Following Vaccination Time Avidity Index* (Median) IQR Correlation with ELISA (Spearman r) p value Month 1 0.96 0.75-1.25 0.08 0.56 Month 12 2.78 2.45-2.93 0.45 0.001 *Data reported as the Molar concentration that reduces bound antibody levels by 50% (Dauner et al. Submitted) 93 Antibody Avidity Studies • The avidity assay is highly reproducible. • Avidity levels increase with increased number of vaccination doses. • Avidity levels do not correlate well with antibody titers. • Further evaluation of avidity is planned to better understand its kinetics and potential role in longterm protection. 94 4 Critical Obstacles to Reducing Cervical Cancer Incidence 1. Vaccination schedules need to be simplified and adaptable for different populations. 2. One-day screening and treatment based on affordable efficacious technologies must be instituted. 3. Cryotherapy must be improved to increase the likelihood of full ablation of persistent infections and precancerous lesions. 4. The two prevention strategies, vaccination and screening, must studied together to understand the integration of the two and the impact of vaccination on screening. HPV Screening 52 clusters 1200-3900 mujeres asignadas al azar HPV Cytology VIA Control 34,126 32,058 34,074 mujeres 31,488 79.7% screened 79.7% screened 78.6% screened 7.0% 13.9% positive 10.3% positive 89.1% colposcopy positive 87.9% colposcopy Osmanabed District, Maharashtra State, India, Sankaranarayanan et al., NEJM, 2009 98.7% colposcopy Study Groups • 200 girls aged 12-20 years (as of August 31, 2011) who received only one dose of HPV vaccine in the period October 1, 2008October 31, 2009, as a part of the PATH-UNEPI HPV vaccine demonstration project • 200 girls aged 12-20 years (as of August 31, 2011) who received only two doses of HPV vaccine in the period October 1, 2008October 31, 2009, as a part of the PATH-UNEPI HPV vaccine demonstration project • 200 girls aged 12-20 years (as of August 31, 2011) who received all three doses of HPV vaccine from October 1, 2008-October 31, 2009, as a part of the PATH-UNEPI HPV vaccine demonstration project. In the right settings, cervical cancer can be prevented with a moderately sensitive test • Forgiving disease: long pre-clinical detectable phase • Almost all precancer can be treated when detected early • With regular screening, an insensitive test will detect all but the most rapidly developing cases! HPV Testing Group Cytologic Testing Group Rate (N) Rate (N) VIA Group Rate (N) Control Group Rate (N) Incidence of 47.4 (127) all cervical cancer 60.7 (152) 58.7 (157) 47.6 (118) Incidence of 14.5 (39) stage II or higher cervical cancer 23.2 (58) 32.2 (86) 33.1 (82) Cancer death 21.5 (54) 20.9 (56) 25.8 (64) 12.7 (34) Sankaranarayanan et al. NEJM. 2009 B. Competitive Luminex Immunoassay (cLIA) A. HPV16 ELISA C. Pseudovirion Neutralization Assay PE H16.V5 HRP HRP HRP HRP SEAP SEAP VLP VLP 293TT SEAP B. Competitive Luminex Immunoassay (cLIA) A. HPV16 ELISA C. Pseudovirion Neutralization Assay PE H16.V5 HRP HRP HRP HRP SEAP SEAP VLP VLP 293TT SEAP Figure X. HPV-16 VLP specific-IgG ELISA, competitive Luminex ImmunoAssay (cLIA) and pseudovirion neutralization assays. Illustration is a schematic representation of the 3 assays that were used to measure HPV16 antibodies from the placebo arm of the HPV 16/18 vaccine trial performed in Costa Rica. A. HPV 16 ELISA measures both non-neutralizing and neutralizing serum IgG that binds to baculovirus expressed L1 VLP 16 coated on ELISA plates. Human IgG bound to the VLPs is detected with a horse-radish peroxidase (HRP) conjugated xxx anti-human IgG. Antibody levels are reporeted in ELISA units/mL. B. HPV 16 cLIA measures serum IgM, IgA and IgG that compete with a phycoerythrin-labeled (PE) neutralizing monoclonal antibody, H16.V5, for binding to a neutralizing epitope on L1 VLP 16 conjugated to a Luminex microsphere. Antibody levels are reported in milli-Merck Units/mL (mMU/mL). C. Pseudovirion neutralization assay measures serum IgM, IgA and IgG that can neutralize SEAP expressing L1+L2 pseudovirions from binding to and infecting 293TT cells. SEAP expression is measured 72 hrs after infection and antibody titers are reported reflect the reciprocal of the dilution that results in a 50% reduction in SEAP activity compared to control wells. Antibody levels are reported in titer units (TU). HPV Immunoepidemiology Studies Natural History Vaccination Studies at the HPV Immunology Lab HPV VLP Vaccine Cellular Immune Responses Humoral Immune Responses Neutralizing Abs Affinity Systemic Local B cell memory T cell proliferative Th1; Th2; responses inflammatory cytokines Cross-reactivity ? Correlates/Mechanisms ? Long-Term Protection Cross Protection Host Gene Expression Profiles HPV16/18 Immunogenicity 106 Vaccine Immunogenicity - CVT HPV16 & HPV18 ELISA GMT: by vaccination arm Antibody Levels (GMT) EU/mL 10000 1000 HPV Vaccinated 100 Naturally Infected Not infected 10 1 0 1 6 7 12 24 36 48 Safaeian et al. in preparation I. Natural History Background • HPV vaccine evokes high levels of antibodies that are protective for HPV16/18 infections and associated lesions • Neutralizing antibodies are demonstrated to be sufficient for protection in animals Do antibodies produced following HPV infection protect against subsequent HPV infections? Impact of antibodies on future infection - Methods Costa Rica HPV16/18 Vaccine Trial (CVT): Control Arm Control Arm: N=3736 Exclusions: N=923 HPV16 DNA- N=2813 HPV16 Sero+ (N=699; 25%) HPV16 Sero- (N=2114; 75%) Impact of antibodies on future infection - Methods • Exposure: anti-HPV16 (or 18) serostatus at entry among HPV16 (or 18) DNA-negatives at enrollment – Tertiles • Outcome: Rates of newly detected HPV16 (or 18) infections during follow-up • Direct (polyclonal) ELISA used to measure seropositivity Multivariate Rate Ratio Elevated antibody levels associated with decreased risk of infection compared with seronegatives for both HPV16 (50%) and HPV18 (60%) 10 Seronegative: Reference Seropositive: Low tertile Seropositive: Medium Tertile Seropositive: High Tertile Sero- 1 0.5 0 HPV16 HPV18 Safaeian et al., JNCI, 2010 Implications • High levels of vaccine evoked antibody Median HPV 16 AB in the high tertile group 133 (85-255) Median HPV16 AB among vaccinated women At 7 months At 12 months 3631 (1823-5520) At 48 months 2304 (1381-4308) 1212 (623-2449) • Duration of current vaccine protection may be long. II. Vaccination Cross-protection Vaccine Efficacy Against Related HighRisk HPV Types Vaccine HPV type HPV16 HPV18 Meerck GSK CVT Gardasil Cervarix Cervarix Related type 6-month persistence 12-month persistence 12-month persistence HPV31 33.6% 80.5% 45.7% HPV33 22.5% 41.0% 37.3% HPV52 2.3% −7.8% −20.0% HPV58 2.8% −17.7% −6.3% HPV45 20.1% 60.0% 52.0% Which marker to follow-up? • 48 women – Randomly selected, 3 doses of the vaccine received • Sera from enrollment, 1m and 12m visits • SEAP (an assay to measure neutralizing potential) with: – HPV16, HPV18 (positive control) – HPV31, HPV45 (vaccine efficacy observed) – HPV52, HPV58 (no vaccine efficacy) – BPV (negative control) Cross-Protection Pilot Study * * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross-Protection Pilot Study * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross-Protection Pilot Study * * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross-Protection Pilot Study * * * * *p<0.001 Kemp…Safaeian et al., Vaccine, 2011 Cross protection findings • Characterized and optimized the SEAP for several phylogenetically related HPV types • Neutralizing antibodies may be responsible Cross-protection Nested Case-control Study • HPV vaccinated arm only • Compare immune responses of: – Cases: HPV vaccinated women with an incident infection with HPV types 31 (n=97), 45 (n=55), and 58 (n=100) – Controls: HPV vaccinated women without infection with these types (n=120) • Serum from multiple time-points will be tested ALTS ASCUS Conclusions • HPV triage (sensitivity 92% with 53% of women referred to colposcopy) – detects as much CIN 3 as immediate colposcopy – spares approx half of women the cost and anxiety of colposcopy • Repeat cytology (sensitivity 95% with 67 % of women referred to colposcopy) – safe option at a threshold of ASCUS – women must be compliant with follow-up visits – however, the trade off of sensitivity with specificity is not as favorable as with HPV testing. Geographic distribution of the world ASIR of cervical cancer, by country, estimated for 2008 (per 100 000 women-years). Arbyn M et al. Ann Oncol 2011;22:2675-2686 © The Author 2011. Published by Oxford University Press on behalf of the European Society for Medical Oncology. All rights reserved. For permissions, please email: journals.permissions@oup.com HPV NEGATIVE Younger age Increasing # sex partners HPV type (HPV-16) “Male factor”* Host immune response Exogenous factors eg. Smoking? HPV POSITIVE HPV type HPV PERSISTENCE CIN-3 Increasing age INVASIVE CANCER Increasing age HPV type (HPV-16), Multiple HPV types? Smoking Multiparity? OC use? Chlamydia? No use of condoms? HLA? Nutrients? *no circumcision, increasing number of sex partners, visits to prostitutes, no condom use OC: Oral contraceptives **data from case-control studies HLA: Human leukocyte antigen Vaccine, Vol 24 Supplement 3, 2006. © 2006 Elsevier Limited. All rights reserved. Chapter 05, Figure 7 OVERVIEW OF FACTORS MOST CONSISTENTLY REPORTED TO PLAY A ROLE AT DIFFERENT STAGES IN THE NATURAL HISTORY OF THE HPV AND CERVICAL NEOPLASIA CIN: Cervical intraepithelial neoplasia Phase III Trials--Main Results •Design: double blind, placebo controlled trials of >10,000 women •Main Finding: protection against cervical precancer caused by HPV16/18 among women naïve to these types during the vaccination period: VE ~100% •Tolerability: slightly more injection site pain than control vaccine •Immunogenicity: 99.5% seroconversion with titers 10-50 fold higher than natural infection •Safety: no evidence of elevated adverse events among vaccinees Burden of HPV in all cancers Site Attributable to HPV (%) Of which, HPV16/18 (%) Total Cancers Attributable to HPV % of all cancers Cervix 100 70 492,800 492,800 4.54 Penis 40 63 26,300 10,500 0.1 Vulva, vagina 40 80 40,000 16,000 0.15 Anus 90 92 30,400 27,300 0.25 Mouth 3 95 274,300 8200 0.08 Oropharynx 12 89 52,100 6200 0.06 10,862,500 561,100 5.17 All sites Adapted from Parkin and Bray, Vaccine 2006 30 20 10 5 1955 1960 1965 1970 1975 1980 1985 1990 1995 YEAR Denmark Finland Norway Parkin DM, et al. Cancer incidence in five continents, vol. I–VIII. Lyon: IARC CancerBase No. 7; 2005. Sweden Vaccine, Vol 24 Supplement 3, 2006. © 2006 Elsevier Limited. All rights reserved. Chapter 02, Figure 8 AGE-STANDARDIZED RATES PER 100,000 (WORLD) TIME TRENDS IN AGE-STANDARDIZED (WORLD) INCIDENCE RATES OF CERVICAL CANCER INCIDENCE IN FOUR NORDIC COUNTRIES Two Broad Classes of HPV Vaccines Prophylactic • • Antibody-mediated protection Rely on IR to structural proteins (L1 VLPs) Therapeutic • CMI-mediated protection • Rely on IR to proteins required for maintenance of infection & transformation (E2/E6/E7) 129 Methods • Cohort- between an ITT and ATP – Excluded those with zero follow-up time or HPV16 and18 DNA+ at enrollment • Endpoint- Event defined as newly detected HPV16 or 18 infection that persisted 1+ years • Statistical analysis – Dose-specific VE and 95%CI Efficacy Against Incident Infection by Other High Risk HPV Types High-risk HPV types 31 16 35 33 58 52 X X X 34 18 45 39 68 26 51 30 53 56 66 Dose- stratified VE after 4-yrs follow-up # of Doses Arm # Women # with persistent HPV16/18 infection Control 3010 133 % with persistent HPV16/18 infection 4.4% 3 HPV16/18 VE (95%CI) 80.9% (71.1% to 87.7%) HPV 2957 25 0.9% Control 380 17 4.5% 2 84.1% (50.2% to 96.3%) HPV 422 3 0.7% Control 188 10 5.3% 1 100% (66.5% to 100%) HPV 196 0 Kreimer AR et al Provisionally accepted JNCI 0.0% Dose- stratified VE after 4-yrs follow-up # of Doses Arm # Women # persistent HPV16/18 infection Control 3010 133 % with persistent HPV16/18 infection 4.4% 3 HPV16/18 VE (95%CI) 80.9% (71.1% to 87.7%) HPV 2957 25 0.9% Control 380 17 4.5% 2 84.1% (50.2% to 96.3%) HPV 422 3 0.7% Control 188 10 5.3% 1 100% (66.5% to 100%) HPV 196 0 Kreimer AR et al Provisionally accepted JNCI 0.0%