Immunotherapy for Rare Solid Tumors and Sarcomas
İrfan KARAHANa , Seda KAHRAMANb , Bülent YALÇINb
aAnkara Bilkent City Hospital, Clinic of Medical Oncology, Ankara, Türkiye
bAnkara Yıldırım Beyazıt University Faculty of Medicine, Ankara Bilkent City Hospital, Department of Medical Oncology, Ankara, Türkiye
Karahan İ, Kahraman S, Yalçın B. Immunotherapy for rare solid tumors and sarcomas. In: Şendur MAN, ed. Current Immunotherapy Landscape for Solid Tumors. 1st ed. Ankara: Türkiye Klinikleri; 2024. p.130-5.
ABSTRACT
Management of rare tumors and sarcomas is a challenging issue due to their relatively low frequency and lack of adequate randomised trials, as well as their dismal prognosis. Over the past decade, research has focused on immunomodulatory strategies to enhance treatment responses. Immunomodulatory treatment can be classified as immune-checkpoint inhibitors, macrophage-based therapies, CART cell therapies and neoantigen-based therapies. In this section, these therapies, their results and future prospects will be discussed.
Keywords: Immunotherapy; rare tumors; sarcomas; immune check point inhibitors
Referanslar
- Vivekanandhan S, Bahr D, Kothari A, Ashary MA, Baksh M, Gabriel E. Immunotherapies in rare cancers. Mol Cancer. 2023;22(1):23. [Crossref] [PubMed] [PMC]
- Mittra A, Takebe N, Florou V, Chen AP, Naqash AR. The emerging landscape of immune checkpoint inhibitor based clinical trials in adults with advanced rare tumors. Hum Vaccin Immunother. 2021;17(7):1935-39. [Crossref] [PubMed] [PMC]
- Petrelli F, Consoli F, Ghidini A, Perego G, Luciani A, Mercurio P, et al. Efficacy of Immune Checkpoint Inhibitors in Rare Tumours: A Systematic Review. Front Immunol. 2021;12:720748. [Crossref] [PubMed] [PMC]
- Kaufman HL, Russell J, Hamid O, Bhatia S, Terheyden P, D'Angelo SP, et al. Avelumab in patients with chemotherapy-refractory metastatic Merkel cell carcinoma: a multicentre, single-group, open-label, phase 2 trial. Lancet Oncol. 2016;17(10):1374-85. [Crossref] [PubMed]
- Nghiem P, Bhatia S, Lipson EJ, Sharfman WH, Kudchadkar RR, Brohl AS, et al. Durable Tumor Regression and Overall Survival in Patients With Advanced Merkel Cell Carcinoma Receiving Pembrolizumab as First-Line Therapy. J Clin Oncol. 2019;37(9):693-702. [Crossref] [PubMed] [PMC]
- Migden MR, Rischin D, Schmults CD, Guminski A, Hauschild A, Lewis KD, et al. PD-1 Blockade with Cemiplimab in Advanced Cutaneous Squamous-Cell Carcinoma. N Engl J Med. 2018;379(4):341-51. [Crossref] [PubMed]
- D'Angelo SP, Larkin J, Sosman JA, Lebbé C, Brady B, Neyns B, et al. Efficacy and Safety of Nivolumab Alone or in Combination With Ipilimumab in Patients With Mucosal Melanoma: A Pooled Analysis. J Clin Oncol. 2017;35(2):226-35. [Crossref] [PubMed] [PMC]
- Johnson DB, Bao R, Ancell KK, Daniels AB, Wallace D, Sosman JA, et al. Response to Anti-PD-1 in Uveal Melanoma Without High-Volume Liver Metastasis. J Natl Compr Canc Netw. 2019;17(2):114-7. [Crossref] [PubMed] [PMC]
- Naing A, Meric-Bernstam F, Stephen B, Karp DD, Hajjar J, Rodon Ahnert J, et al. Phase 2 study of pembrolizumab in patients with advanced rare cancers. J Immunother Cancer. 2020;8(1):e000347. Erratum in: J Immunother Cancer. 2020;8(1). [Crossref] [PubMed] [PMC]
- Klein O, Senko C, Carlino MS, Markman B, Jackett L, Gao B, et al. Combination immunotherapy with ipilimumab and nivolumab in patients with advanced adrenocortical carcinoma: a subgroup analysis of CA209-538. Oncoimmunology. 2021;10(1):1908771. [Crossref] [PubMed] [PMC]
- Nowak AK, Lesterhuis WJ, Kok PS, Brown C, Hughes BG, Karikios DJ, et al. Durvalumab with first-line chemotherapy in previously untreated malignant pleural mesothelioma (DREAM): a multicentre, single-arm, phase 2 trial with a safety run-in. Lancet Oncol. 2020;21(9):1213-23. [Crossref] [PubMed]
- Forde PM, Anagnostou V, Sun Z, Dahlberg SE, Kindler HL, Niknafs N, et al. Durvalumab with platinum-pemetrexed for unresectable pleural mesothelioma: survival, genomic and immunologic analyses from the phase 2 PrE0505 trial. Nat Med. 2021;27(11):1910-20. [Crossref] [PubMed] [PMC]
- Yap TA, Nakagawa K, Fujimoto N, Kuribayashi K, Guren TK, Calabrò L, et al. Efficacy and safety of pembrolizumab in patients with advanced mesothelioma in the open-label, single-arm, phase 2 KEYNOTE-158 study. Lancet Respir Med. 2021;9(6):613-21. [Crossref] [PubMed]
- Peters S, Scherpereel A, Cornelissen R, Oulkhouir Y, Greillier L, Kaplan MA, et al. First-line nivolumab plus ipilimumab versus chemotherapy in patients with unresectable malignant pleural mesothelioma: 3-year outcomes from CheckMate 743. Ann Oncol. 2022;33(5):488-99. [Crossref] [PubMed]
- Raghav K, Liu S, Overman MJ, Willett AF, Knafl M, Fu SC, et al. Efficacy, Safety, and Biomarker Analysis of Combined PD-L1 (Atezolizumab) and VEGF (Bevacizumab) Blockade in Advanced Malignant Peritoneal Mesothelioma. Cancer Discov. 2021;11(11):2738-47. [Crossref] [PubMed] [PMC]
- Giaccone G, Kim C, Thompson J, McGuire C, Kallakury B, Chahine JJ, et al. Pembrolizumab in patients with thymic carcinoma: a single-arm, single-centre, phase 2 study. Lancet Oncol. 2018;19(3):347-55. [Crossref] [PubMed]
- Rodriguez CP, Wu QV, Voutsinas J, Fromm JR, Jiang X, Pillarisetty VG, et al. A Phase II Trial of Pembrolizumab and Vorinostat in Recurrent Metastatic Head and Neck Squamous Cell Carcinomas and Salivary Gland Cancer. Clin Cancer Res. 2020;26(4):837-45. [Crossref] [PubMed]
- Husain M, Chen L, Liebner D, Beane J, Rubinstein M, Pollock R, et al. Emerging Trends in Immunotherapy for Adult Sarcomas. Oncologist. 2023;28(5):e233-e41. [Crossref] [PubMed] [PMC]
- Kim T, Bui NQ. The Next Frontier in Sarcoma: Molecular Pathways and Associated Targeted Therapies. Cancers (Basel). 2023;15(6):1692. [Crossref] [PubMed] [PMC]
- Tawbi HA, Burgess M, Bolejack V, Van Tine BA, Schuetze SM, Hu J, et al. Pembrolizumab in advanced soft-tissue sarcoma and bone sarcoma (SARC028): a multicentre, two-cohort, single-arm, open-label, phase 2 trial. Lancet Oncol. 2017;18(11):1493-501. Erratum in: Lancet Oncol. 2017;18(12 ):e711. Erratum in: Lancet Oncol. 2018;19(1):e8. [Crossref] [PubMed]
- Ben-Ami E, Barysauskas CM, Solomon S, Tahlil K, Malley R, Hohos M, et al. Immunotherapy with single agent nivolumab for advanced leiomyosarcoma of the uterus: Results of a phase 2 study. Cancer. 2017;123(17):3285-90. [Crossref] [PubMed] [PMC]
- Shi Y, Cai Q, Jiang Y, Huang G, Bi M, Wang B, et al. Activity and Safety of Geptanolimab (GB226) for Patients with Unresectable, Recurrent, or Metastatic Alveolar Soft Part Sarcoma: A Phase II, Single-arm Study. Clin Cancer Res. 2020;26(24):6445-52. [Crossref] [PubMed]
- Chen AP, Sharon E, O'Sullivan-Coyne G, Moore N, Foster JC, Hu JS, et al. Atezolizumab for Advanced Alveolar Soft Part Sarcoma. N Engl J Med. 2023;389(10):911-21. [Crossref] [PubMed] [PMC]
- Delyon J, Biard L, Renaud M, Resche-Rigon M, Le Goff J, Dalle S, et al. PD-1 blockade with pembrolizumab in classic or endemic Kaposi's sarcoma: a multicentre, single-arm, phase 2 study. Lancet Oncol. 2022;23(4):491-500. [Crossref] [PubMed]
- D'Angelo SP, Mahoney MR, Van Tine BA, Atkins J, Milhem MM, Jahagirdar BN, et al. Nivolumab with or without ipilimumab treatment for metastatic sarcoma (Alliance A091401): two open-label, non-comparative, randomised, phase 2 trials. Lancet Oncol. 2018;19(3):416-26. [Crossref] [PubMed]
- Wagner MJ, Othus M, Patel SP, Ryan C, Sangal A, Powers B, et al. Multicenter phase II trial (SWOG S1609, cohort 51) of ipilimumab and nivolumab in metastatic or unresectable angiosarcoma: a substudy of dual anti-CTLA-4 and anti-PD-1 blockade in rare tumors (DART). J Immunother Cancer. 2021;9(8):e002990. [Crossref] [PubMed] [PMC]
- Italiano A, Bessede A, Pulido M, Bompas E, Piperno-Neumann S, Chevreau C, et al. Pembrolizumab in soft-tissue sarcomas with tertiary lymphoid structures: a phase 2 PEMBROSARC trial cohort. Nat Med. 2022;28(6):1199-206. [Crossref] [PubMed]
- Toulmonde M, Penel N, Adam J, Chevreau C, Blay JY, Le Cesne A, et al. Use of PD-1 Targeting, Macrophage Infiltration, and IDO Pathway Activation in Sarcomas: A Phase 2 Clinical Trial. JAMA Oncol. 2018;4(1):93-7. [Crossref] [PubMed] [PMC]
- Pollack SM, Redman MW, Baker KK, Wagner MJ, Schroeder BA, Loggers ET, et al. Assessment of Doxorubicin and Pembrolizumab in Patients With Advanced Anthracycline-Naive Sarcoma: A Phase 1/2 Nonrandomized Clinical Trial. JAMA Oncol. 2020;6(11):1778-82. [Crossref] [PubMed] [PMC]
- Wilky BA, Trucco MM, Subhawong TK, Florou V, Park W, Kwon D, et al. Axitinib plus pembrolizumab in patients with advanced sarcomas including alveolar soft-part sarcoma: a single-centre, single-arm, phase 2 trial. Lancet Oncol. 2019;20(6):837-48. [Crossref] [PubMed]
- Li J, Zhao X, Chen L, Guo H, Lv F, Jia K, et al. Safety and pharmacokinetics of novel selective vascular endothelial growth factor receptor-2 inhibitor YN968D1 in patients with advanced malignancies. BMC Cancer. 2010;10:529. [Crossref] [PubMed] [PMC]
- D'Angelo SP, Shoushtari AN, Keohan ML, Dickson MA, Gounder MM, Chi P, et al. Combined KIT and CTLA-4 Blockade in Patients with Refractory GIST and Other Advanced Sarcomas: A Phase Ib Study of Dasatinib plus Ipilimumab. Clin Cancer Res. 2017;23(12):2972-80. [Crossref] [PubMed] [PMC]
- Blay JY, Chevret S, Le Cesne A, Brahmi M, Penel N, Cousin S, et al. Pembrolizumab in patients with rare and ultra-rare sarcomas (AcSé Pembrolizumab): analysis of a subgroup from a non-randomised, open-label, phase 2, basket trial. Lancet Oncol. 2023;24(8):892-902. Erratum in: Lancet Oncol. 2023;24(10):e405. [Crossref] [PubMed]
- Moyes KW, Lieberman NA, Kreuser SA, Chinn H, Winter C, Deutsch G, et al. Genetically Engineered Macrophages: A Potential Platform for Cancer Immunotherapy. Hum Gene Ther. 2017;28(2):200-15. [Crossref] [PubMed]
- Cassier PA, Italiano A, Gomez-Roca C, Le Tourneau C, Toulmonde M, D'Angelo SP, et al. Long-term clinical activity, safety and patient-reported quality of life for emactuzumab-treated patients with diffuse-type tenosynovial giant-cell tumour. Eur J Cancer. 2020;141:162-70. [Crossref] [PubMed]
- Rohaan MW, Borch TH, van den Berg JH, Met Ö, Kessels R, Geukes Foppen MH, et al. Tumor-Infiltrating Lymphocyte Therapy or Ipilimumab in Advanced Melanoma. N Engl J Med. 2022;387(23):2113-25. [Crossref] [PubMed]
- Kelly CM, Antonescu CR, Bowler T, Munhoz R, Chi P, Dickson MA, et al. Objective Response Rate Among Patients With Locally Advanced or Metastatic Sarcoma Treated With Talimogene Laherparepvec in Combination With Pembrolizumab: A Phase 2 Clinical Trial. JAMA Oncol. 2020;6(3):402-8. [Crossref] [PubMed] [PMC]
- Chawla SP, Van Tine BA, Pollack SM, Ganjoo KN, Elias AD, Riedel RF, et al. Phase II Randomized Study of CMB305 and Atezolizumab Compared With Atezolizumab Alone in Soft-Tissue Sarcomas Expressing NY-ESO-1. J Clin Oncol. 2022;40(12):1291-300. [Crossref] [PubMed]