更新于：2024-05-01

Endodermal Sinus Tumor

内胚层窦瘤

更新于：2024-05-01

基本信息

别名

ENDODERMAL SINUS TUMOR、Embryonal carcinoma, infantile、Embryonal carcinoma, infantile type

+ [44]

简介

An unusual and aggressive tumor of germ-cell origin that reproduces the extraembryonic structures of the early embryo. It is the most common malignant germ cell tumor found in children. It is characterized by a labyrinthine glandular pattern of flat epithelial cells and rounded papillary processes with a central capillary (Schiller-Duval body). The tumor is rarely bilateral. Before the use of combination chemotherapy, the tumor was almost invariably fatal. (From DeVita Jr et al., Cancer: Principles & Practice of Oncology, 3d ed, p1189)

关联

项与内胚层窦瘤相关的临床试验

NCT06198296 / Not yet recruiting临床1期IIT

Immunotherapy For Adults With GPC3-Positive Solid Tumors Using Interleukin-15 And -21 Armored Glypican-3-Specific Chimeric Antigen Receptor Expressing Autologous T Cells

The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells.
Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise but have not been strong enough to cure most patients.
In order to get them to kill cancers more effectively, in the laboratory, the study team inserted a new gene called a chimeric antigen receptor (CAR) into T cells that makes them recognize cancer cells and kill them. When inserted, this new CAR T cell can specifically recognize a protein found on solid tumors, called glypican-3 (GPC3). To make this GPC3-CAR more effective, the study team also added two genes called IL15 and IL21 that help CAR T cells grow better and stay in the blood longer so that they may kill tumors better. When the study team did this in the laboratory, they found that this mixture of GPC3-CAR,IL15 and IL21 killed tumor cells better when compared with CAR T cells that did not have IL15 plus IL21 in the laboratory. This study will use those cells, which are called 21.15.GPC3-CAR T cells, to treat patients with solid tumors that have GPC3 on their surface.
The study team also wanted to make sure that they could stop the 21.15.GPC3-CAR T cells from growing in the blood should there be any bad side effects. In order to do so, they inserted a gene called iCasp9 into the FAST-CAR T cells. This allows us the elimination of 21.15.GPC3-CAR T cells in the blood when the gene comes into contact with a medication called AP1903. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. This drug will only be used to kill the T cells if necessary due to side effects .
The study team has treated patients with T cells that include GPC3. Patients have also been treated with IL-21 and with IL-15. Patients have not been treated with a combination of T cells that contain GPC3, IL-21 and IL-15. To summarize, this study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors that express GPC3 on their surface.
The 21.15.GPC3-CAR T cells are an investigational product not yet approved by the Food and Drug Administration.

开始日期2026-01-01

申办/合作机构

Baylor College of Medicine

[+1]

NCT04715191 / Not yet recruiting临床1期IIT

Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressing Autologous T Cells as an Immunotherapy for Children With Solid Tumors (CARE)

Patients may be considered if the cancer has come back, has not gone away after standard treatment or the patient cannot receive standard treatment. This research study uses special immune system cells called CARE T cells, a new experimental treatment.
The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells. Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise, but have not been strong enough to cure most patients.
Investigators have found from previous research that they can put a new gene (a tiny part of what makes-up DNA and carries a person's traits) into T cells that will make them recognize cancer cells and kill them. In the lab, investigators made several genes called a chimeric antigen receptor (CAR), from an antibody called GPC3. The antibody GPC3 recognizes a protein found solid tumors including pediatric liver cancers. This CAR is called GPC3-CAR. To make this CAR more effective, investigators also added two genes that includes IL15 and IL21, which are protein that helps CAR T cells grow better and stay in the blood longer so that they may kill tumors better. The mixture of GPC3-CAR and IL15 plus IL21 killed tumor cells better in the laboratory when compared with CAR T cells that did not have IL15 plus IL21 .This study will test T cells that investigators made (called genetic engineering) with GPC3-CAR and the IL15 plus IL21 (CARE T cells) in patients with GPC3-positive solid tumors.
T cells made to carry a gene called iCasp9 can be killed when they encounter a specific drug called AP1903. The investigators will insert the iCasp9 and IL15 plus IL21 together into the T cells using a virus that has been made for this study. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. The investigators will use this drug to kill the T cells if necessary due to side effects.
This study will test T cells genetically engineered with a GPC3-CAR and IL15 plus IL21 (CARE T cells) in patients with GPC3-positive solid tumors.
The CARE T cells are an investigational product not approved by the Food and Drug Administration.
The purpose of this study is to find the biggest dose of CARE T cells that is safe, to see how long they last in the body, to learn what the side effects are and to see if the CARE T cells will help people with GPC3-positive solid tumors.

开始日期2024-03-03

申办/合作机构

Baylor College of Medicine

[+1]

NCT05564026 / RecruitingN/AIIT

Pediatric Germ Cell Tumors: Outcomes, Genomics and Epigenetics

A Non-Therapeutic Study that aims to establish a cohort of GCT survivors to understand short term and long-term adverse effects of treatment and to conduct molecular analyses to improve risk stratification.

开始日期2023-04-12

申办/合作机构

The Children's Oncology Group Foundation, Inc.

100 项与内胚层窦瘤相关的临床结果

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0 项与内胚层窦瘤相关的专利（医药）

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项与内胚层窦瘤相关的文献（医药）

2024-02-20·International journal of surgical pathology

Retroperitoneal Sarcomatoid Yolk Sac Tumor in a Chemotherapy-Naive Patient With Testicular Postpubertal Type Teratoma: A Rare Case Report With Emphasis on Molecular Features.

Article

作者: Bo Zhang ; Payal Kapur ; Prasad R Koduru ; Liwei Jia

Sarcomatoid yolk sac tumor is a very rare histologic type of testicular germ cell tumor and is mainly reported in testicular germ cell tumor patients who receive chemotherapy. Herein, we report an extremely rare concurrent retroperitoneal sarcomatoid yolk sac tumor in a man with a testicular postpuberal teratoma before he received chemotherapy. A 37-year-old man initially presented with a persistent abdominal pain. Subsequent imaging studies revealed a 9.6-cm retroperitoneal mass, and 2 testicular masses (3.1 cm and 0.9 cm in greatest dimension, respectively). His serum tumor markers were within normal ranges. His radical orchiectomy demonstrated a postpubertal type teratoma with an adjacent scarring nodule. Later, his retroperitoneal tumor showed spindle tumor cells embedded in predominantly myxoid and focally fibrous stroma with diffuse and strong immunoreactivity for keratin AE1/AE3, SALL4 and glypican 3. No tumor necrosis or brisk mitotic figures were observed. A diagnosis of sarcomatoid yolk sac tumor was rendered. Fluorescence in situ hybridization analysis of his retroperitoneal sarcomatoid yolk sac tumor revealed polysomy 12 and MYC amplification, whereas no evidence of isochromosome 12p [i(12p)], and DNA sequencing showed 6 mutations per megabase (muts/Mb), and the somatic alterations included ARAF amplification and ATR I774Yfs*5. Considering its rarity, sarcomatoid yolk sac tumor may pose diagnostic challenges. Therefore, relevant clinicoradiologic information and ancillary work up, including immunohistochemistry and molecular studies, may be helpful for the accurate classification. Our tumor further raises awareness of this rare event, expands the spectrum of its clinical presentation, and explores the molecular features.

2024-02-12·BMC urology

46, XX disorder of sexual development associated with mixed germ cell tumor of the prostate: a rare case report.

Article

作者: Changrong Wang ; Jiangli Du ; Xueping Xiang ; Yuyong Wang ; Jingjing Xiang ; Qiaoping Xu

BACKGROUND:

Extragonadal germ cell tumors originating from the prostate are exceptionally rare. To the best of our knowledge, there have been no reported cases of mixed germ cell tumors in individuals with 46 XX disorder of sex development. In this study, we conducted a comprehensive analysis using whole genome sequencing to investigate the clinicopathological and molecular genetic characteristics of a submitted case, with the objective of elucidating its underlying pathogenesis.

CASE PRESENTATION:

A 40-year-old male patient was diagnosed with a combination of 46, XX disorder of sex development and a primary prostate mixed germ cell tumor with yolk sac tumor and teratoma components. Whole-genome sequencing revealed that the tumor cells had a high somatic mutational load. Analysis of genomic structural variations and copy number variants confirmed the patient's karyotype as 46, XX (SRY +). Additionally, the patient exhibited short stature, small bilateral testes, slightly enlarged breasts, elevated serum alpha-fetoprotein concentrations, elevated follicle-stimulating hormone and luteinizing hormone levels, and low testosterone levels.

DISCUSSION:

A case of 46, XX disorder of sex development, along with a primary prostatic mixed germ cell tumor, was diagnosed. This diagnosis has contributed to advancing our understanding of the genetic and phenotypic profile of the disease and may provide some insights for its treatment.

2024-02-11·European journal of surgical oncology : the journal of the European Society of Surgical Oncology and the British Association of Surgical Oncology

Survival difference among adult and pediatric mediastinal yolk sac tumors cases: A meta-analysis of case reports.

Review

作者: Anas Dabsha ; Ismail A M H Elkharbotly ; Mohammad Yaghmour ; Amr Badr ; Fady Badie ; Sherif Khairallah ; Yomna M Esmail ; Mohamed Hossny ; Amr Rizk ; Amr El-Demiry ; Galal Ghaly ; Shaikha Al-Thani ; Michelle Demetres ; Abdelrahman Mohamed ; Jonathan Villena-Vargas ; Mona Kamal ; Mohamed Rahouma

BACKGROUND:

Mediastinal Yolk sac tumors (YST) are rare and highly malignant extragonadal germ cell tumors with rapid growth and early metastases. We sought to conduct a meta-analysis of published case reports/case series to compare differences in survival, demographics, and treatment modalities between adult and pediatric patients with YST.

METHODS:

Ovid Embase, Cochrane, and Ovid Medline databases were searched for primary mediastinal pure YST cases. The primary outcome was overall survival (OS). Log-rank and Cox regression were used. This study is registered on PROSPERO (CRD42022367586).

RESULTS:

Among 846 studies, 87 met our inclusion criteria including 130 patients (Adults: 90 and Pediatrics: 40). About 41.5% of the patients were from the United States. The median age was 23.0 (Q1-Q3: 17.0-30.0), 88.5% were males, and (32.3%) were Asian. Stage II represented almost 40%. AFP was elevated in 96.9%. Respiratory distress was the presenting symptom in 65.4%. Chemotherapy, radiotherapy, and surgery were utilized in 84.6, 23.1, and 64.7% respectively. Median OS was 24 months (Adults: 23 months, Pediatrics: 25 months, P = 0.89). 3- and 5-year OS were 34.4% and 22.9% in adults and 41.5% and 41.5% in pediatrics, respectively. On multivariate analysis, anterior location of tumors, receipt of chemotherapy, and undergoing surgery were associated with better OS.

CONCLUSION:

Primary mediastinal YSTs are rare, but lethal neoplasms. Our meta-analysis showed that mediastinal YSTs mimic other non-seminomatous mediastinal GCTs in terms of clinical characteristics and available treatment options. Early diagnosis, neoadjuvant chemotherapy, and surgical resection are the key points for effective management and improved outcomes.

项与内胚层窦瘤相关的新闻（医药）

2023-10-18

·信达生物

【学术前沿】信迪利单抗发挥在儿童肿瘤免疫治疗价值，中山大学肿瘤防治中心最新研究成果在国际期刊发表

转载自：中山大学肿瘤防治中心儿童肿瘤科编者按目前，儿童肿瘤患者的标准治疗主要包括手术、化疗和放疗。令人遗憾的是，对于儿童实体瘤的预后仍然不容乐观，PD-1等新型免疫检查点抑制剂在儿童肿瘤的应用值得探索。国家一类创新药信迪利单抗率先针对儿童晚期及难治肿瘤进行了积极的尝试，探索儿童肿瘤免疫治疗的潜力。中山大学肿瘤防治中心张翼鷟教授团队在该方面的研究结果现已刊发国际权威期刊《Signal Transduction and Targeted Therapy》，研究显示，信迪利单抗不论是作为单一药物疗法还是与其他药物联合治疗的一部分，都为儿童肿瘤治疗提供了初步依据。近日，中山大学肿瘤防治中心张翼鷟教授团队在国际权威期刊《Signal Transduction and Targeted Therapy》（IF：39.3）在线发表了题为“信迪利单抗在儿童晚期或复发难治肿瘤中安全性及有效性的I期临床试验”的研究结果。儿童肿瘤患者的标准治疗主要包括手术、化疗和放疗。令人遗憾的是，对于儿童肿瘤患者中复发或晚期实体肿瘤的预后仍然不容乐观，免疫检查点抑制剂等新型治疗在儿童肿瘤的应用值得探索。KEYNOTE-051和ADVL1412研究PD1抗体在儿童肿瘤中的疗效，但在亚洲儿童人群中，PD-1抗体的前瞻性研究非常有限。Pembrolizumab和Nivolumab等药物因为费用高昂阻碍了它们在国内的广泛应用，导致儿童肿瘤领域国内PD-1抗体数据的巨大空白。本研究首次探索了国产PD-1抗体信迪利单抗治疗儿童晚期及难治复发的肿瘤的安全性和有效性。研究纳入了29名受试者，分为三个递增剂量的队列：1 mg/kg、3 mg/kg和10 mg/kg。主要的研究目标包括确定剂量限制性毒性并评估治疗的安全性和耐受性。次要研究终点包括客观反应率（ORR）、无进展生存期（PFS）以及总生存期（OS）。受试者的临床病理类型包括除了霍奇金淋巴瘤以外，也包括一些罕见的儿童肿瘤病理亚型，例如原发性纵隔大B细胞淋巴瘤， NK/T细胞淋巴瘤，恶性间皮瘤，间叶原性恶性肿瘤，间变室管膜瘤，上皮样肉瘤，胸腺瘤，卵黄囊瘤，非典型脉络丛乳头状瘤等。安全性方面，信迪利单抗耐受性表现良好，29名患者中有89.7%患者至少出现一种治疗相关不良事件。包括：贫血（34.5%）；疲劳（6.9%）；甲状腺功能减退（17.2%）；甲状腺功能亢进（13.8%）等。3名患者（10.3%）出现了三级以上的治疗相关不良反应，包括转移酶升高和肌炎，以及血小板计数降低。安全数据没有显示出剂量（1-10 mg/kg）相关的特征。未发生治疗相关的严重不良事件（SAEs）。疗效方面，霍奇金淋巴瘤患者中，总体ORR和DCR分别为60.0%和100%，20%患者最佳疗效为CR。在除霍奇金淋巴瘤以外的肿瘤中观察到2例CR（NK/T细胞淋巴瘤和间变室管膜瘤），3例PR（包括1例弥漫性大B细胞淋巴瘤、1例尤文肉瘤和1例胸腺瘤）3例SD (包括1例上皮样肉瘤、1例非典型脉络丛乳头状瘤和1例原发性纵隔大B细胞淋巴瘤）ORR 为29.4%，DCR 为47.1%。另外，对患儿的肿瘤标本进行外显子测序结果发现G 蛋白偶联受体 (GPCR) 是参与信号传递的最大的细胞膜相关分子基因家族，与治疗抵抗和免疫逃避有关，结果表明对免疫检查点抑制剂的无反应与 GPCR 途径的激活有关。使用WES对受试者的FFPE肿瘤和匹配的外周血样本进行基因组分析综上，研究结果显示信迪利单抗在儿童中的耐受性良好。虽然在这项研究中，信迪利单抗在大多数儿童实体瘤中并未表现出明显的单药疗效，但观察到了对霍奇金淋巴瘤的抗肿瘤活性。这一研究为未来进一步探讨PD-1/PD-L1抑制剂在儿童肿瘤治疗中的潜力，不论是作为单一药物疗法还是与其他药物联合治疗的一部分，提供了初步依据。中山大学肿瘤防治中心张翼鷟教授、路素英副教授为本研究的共同通讯作者。中山大学肿瘤防治中心阙旖医师、王娟主治医师，孙斐斐主治医师以及重庆医科大学附属儿童医院王珊教授是本研究的共同第一作者。文献：Yi Que, Juan Wang, Feifei Sun, Shan Wang, Jia Zhu, Junting Huang, Zhenzhen Zhao, Li Zhang, Juan Liu1, Jiaqian Xu,Zijun Zhen, Xiaofei Sun, Suying Lu1 and Yizhuo Zhang. Safety and clinical efficacy of sintilimab (anti-PD-1) in pediatric patients with advanced or recurrent malignancies in a phase I study. Signal Transduction and Targeted Therapy (2023) 8:392 . https://doi-org.libproxy1.nus.edu.sg/10.1038/s41392-023-01636-9.点击蓝字阅读原文

免疫疗法临床结果

2023-10-17

·生物谷

中山大学张翼鷟团队发布首个国产PD-1免疫治疗儿童肿瘤临床数据

这项1期临床研究结果显示信迪利单抗在儿童中的耐受性良好。虽然在这项研究中，信迪利单抗在大多数儿童实体瘤中并未表现出明显的单药疗效，但观察到了对霍奇金淋巴瘤的抗肿瘤活性。中山大学肿瘤防治中心张翼鷟教授团队在 Signal Transduction and Targeted Therapy 期刊发表了题为：Safety and clinical efficacy of sintilimab (anti-PD-1) in pediatric patients with advanced or recurrent malignancies in a phase I study（信迪利单抗在儿童晚期或复发难治肿瘤中安全性及有效性的I期临床试验）的研究论文。该研究首次探索了国产PD-1抗体信迪利单抗（Sintilimab）治疗儿童晚期及难治复发的肿瘤的安全性和有效性。该研究纳入了29名受试者，分为三个递增剂量的队列：1 mg/kg、3 mg/kg和10 mg/kg。主要的研究目标包括确定剂量限制性毒性并评估治疗的安全性和耐受性。次要研究终点包括客观反应率（ORR）、无进展生存期（PFS）以及总生存期（OS）。受试者的临床病理类型包括除了霍奇金淋巴瘤以外，也包括一些罕见的儿童肿瘤病理亚型，例如原发性纵隔大B细胞淋巴瘤， NK/T细胞淋巴瘤，恶性间皮瘤,，间叶原性恶性肿瘤，间变室管膜瘤，上皮样肉瘤，胸腺瘤，卵黄囊瘤，非典型脉络丛乳头状瘤等。安全性方面，信迪利单抗耐受性表现良好，29名患者中有89.7%患者至少出现一种治疗相关不良事件。包括：贫血（34.5%）；疲劳（6.9%）；甲状腺功能减退（17.2%）；甲状腺功能亢进（13.8%）等。3名患者（10.3%）出现了三级治疗相关不良反应，包括转移酶升高和肌炎，以及血小板计数降低。安全数据没有显示出剂量（1-10 mg/kg）相关的特征。未发生治疗相关的严重不良事件（SAEs）。疗效方面，霍奇金淋巴瘤患者中，总体ORR和DCR分别为60.0%和100%，20%患者最佳疗效为CR。在除霍奇金淋巴瘤以外的肿瘤中观察到2例CR（NK/T细胞淋巴瘤和间变室管膜瘤），3例PR（包括1例弥漫性大B细胞淋巴瘤、1例尤文肉瘤和1例胸腺瘤），3例SD（包括1例上皮样肉瘤、1例非典型脉络丛乳头状瘤和1例原发性纵隔大B细胞淋巴瘤）ORR 为29.4%，DCR 为47.1%。另外，对患儿的肿瘤标本进行外显子测序结果发现G蛋白偶联受体（GPCR）是参与信号传递的最大的细胞膜相关分子基因家族，与治疗抵抗和免疫逃避有关，结果表明对免疫检查点抑制剂的无反应与GPCR通路的激活有关。使用全外显子测序对受试者的FFPE肿瘤和匹配的外周血样本进行基因组分析综上，这项1期临床研究结果显示信迪利单抗在儿童中的耐受性良好。虽然在这项研究中，信迪利单抗在大多数儿童实体瘤中并未表现出明显的单药疗效，但观察到了对霍奇金淋巴瘤的抗肿瘤活性。这一研究为未来进一步探讨PD-1/PD-L1抑制剂在儿童肿瘤治疗中的潜力，不论是作为单一药物疗法还是与其他药物联合治疗的一部分，提供了初步依据。

临床1期临床结果免疫疗法

2021-01-13

·BioSpace

Cytovia Therapeutics Partners with National Cancer Institute to Develop Novel Gene-Edited, iPSC-Derived GPC3 CAR NK Cells for the Treatment of Solid Tumors

CAMBRIDGE, Mass., Jan. 13, 2021 (GLOBE NEWSWIRE) -- Cytovia Therapeutics an emerging biopharmaceutical company focusing on Natural Killer cells in cancer, announced today that it has signed a licensing agreement with the National Cancer Institute (NCI), part of the National Institutes of Health, to apply its gene-edited iPSC-derived NK cell technology to develop GPC3 CAR NK cell therapeutics. Dr Mitchell Ho, PhD, Director of the Antibody Engineering Program and Deputy Chief of the Laboratory of Molecular Biology at the NCI Center for Cancer Research has developed novel antibodies and chimeric antigen receptors (CAR) binding to glypican-3 (GPC3) on liver cancer cells. Dr. Ho has published data on the humanized GPC3 antibody in scientific reports (nature research) in 2016 as well as on the GPC3 CAR in Gastroenterology in 2020. GPC3 is an oncofetal antigen involved in Wnt-dependent cell proliferation. It is highly expressed in Hepatocellular Carcinoma tumor cells as well as multiple other solid tumors, including ovarian cancer and lung cancer, but not expressed in adult normal tissues. Cytovia has also signed a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute. Under the CRADA, Cytovia will collaborate with Dr. Ho’s laboratory to develop and evaluate gene-edited iPSC-derived GPC3 CAR NK cells. Cytovia expects to initial new drug application (IND) for its GPC3 CAR NK cells in the first half of 2022. Dr. Daniel Teper, Chairman and CEO of Cytovia Therapeutics commented: “GPC3 is an exciting new validated target for Hepatocellular Carcinoma, an area of major unmet medical need, as well as other solid tumors. We look forward to collaborating with Dr. Ho, a pioneer in GPC3 research, to develop a novel gene-edited, iPSC-derived GPC3 CAR NK that will advance toward a cure for liver cancer.” Dr. Ho added: “Natural Killer cells play a major role in the immuno-surveillance of liver cancer. GPC3 is expressed in more than 70% of Hepatocellular Carcinoma cells but not on healthy cells. We look forward to investigating whether GPC3 CAR-NK therapy could provide a new safe and effective off-the-shelf option for patients with liver cancer.” ABOUT GENE-EDITED, IPSC-DERIVED NK CELLS Chimeric Antigen Receptors (CAR) are fusion proteins that combine an extracellular antigen recognition domain with an intracellular co-stimulatory signaling domain. Natural Killer (NK) cells are modified genetically to allow insertion of a CAR. CAR-NK cell therapy has demonstrated initial clinical relevance without the limitations of CAR-T, such as Cytokine Release Syndrome, neurotoxicity or Graft vs Host Disease (GVHD). In addition, CAR-NKs are naturally allogeneic, available off-the-shelf and may be able to be administered on an outpatient basis. Recent innovative developments with the induced pluripotent stem cell (iPSC)-derived CAR-NKs, an innovative technology, allow large quantities of homogeneous genetically modified CAR NK cells to be produced from a gene-edited iPSC master cell bank, and thus hold promise to expand access to cell therapy for many patients. ABOUT HCC Hepatocellular carcinoma (HCC) is the most common primary liver malignancy and a leading cause of death worldwide, with 800,000 new cases diagnosed globally every year. The incidence in Asia is amongst the highest in the world (75%) with 400,000 in China alone. In the US, it is estimated to reach upwards of 30,000 by the end of 2020 and continues to be on the rise. Despite advances in immunotherapy, with current treatment options including multi-kinase inhibitors (TKI) and checkpoint inhibitors, life expectancy for patients diagnosed with HCC remains very low. The disease is often diagnosed at an advanced stage, with a median survival of approximately 6 to 20 months following diagnosis, and a 5-year survival rate below 10% in the US. Fortunately, new options including cell therapy and bispecific antibodies offer promise towards a cure for liver cancer. ABOUT GPC3 Glypican-3 (GPC3) is a cell-surface heparan sulfate proteoglycan expressed in the liver and the kidney of fetuses but is hardly expressed in adults, except in the placenta. However, it is highly expressed in HCC, ovarian clear cell carcinoma, squamous cell carcinoma of the lung, melanoma, hepatoblastoma, nephroblastoma (Wilms tumor), yolk sac tumor, and some pediatric cancers. GPC3 promotes Wnt-dependent cell proliferation and has been strongly suggested that it is related to the malignant transformation. Therefore, GPC3 is a promising target for cancer immunotherapy and can serve as a biomarker for predicting tumor recurrence and treatment efficacy. About Cytovia Therapeutics Cytovia Therapeutics Inc is an emerging biotechnology company that aims to accelerate patient access to transformational immunotherapies, addressing several of the most challenging unmet medical needs in cancer. Cytovia focuses on Natural Killer (NK) cell biology and is leveraging multiple advanced patented technologies, including an induced pluripotent stem cell (iPSC) platform for CAR (Chimeric Antigen Receptors) NK cell therapy, next-generation precision gene-editing to enhance targeting of NK cells, and NK engager multi-functional antibodies. Our initial product portfolio focuses on both hematological malignancies such as multiple myeloma and solid tumors including hepatocellular carcinoma and glioblastoma. The company partners with the University of California San Francisco (UCSF), the New York Stem Cell Foundation (NYSCF), the Hebrew University of Jerusalem, INSERM, and CytoImmune Therapeutics. Learn more at and follow Cytovia Therapeutics on Social Media (Facebook, LinkedIn, Twitter, and Youtube). Contact for investor enquiries: Anna Baran-Djokovic Vice President, Investor Relations Anna@cytoviatx.com 1 (646) 355 1787

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