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Atypical Teratoid Rhabdoid Tumor (ATRT)

Atypical teratoid rhabdoid tumor (ATRT) is a highly aggressive pediatric brain tumor that primarily affects young children. It is a rare disease with limited therapy options. Our company is at the forefront of drug and therapy development services for ATRT, aiming to provide pharmaceutical companies with efficient and professional solutions.

Overview of ATRT

Atypical teratoid rhabdoid tumor (ATRT) is a highly malignant brain tumor predominantly found in children under the age of three. ATRT is an aggressive tumor, often presenting with rapid progression and metastasis. It can occur anywhere in the central nervous system, including the spinal cord and brain. The incidence of ATRT in children with CNS tumors is approximately 1% to 2%. Due to its rarity and aggressive nature, ATRT poses significant challenges in terms of diagnosis and therapeutics.

Molecular Alterations of ATRT

The most common and defining genetic alteration is the loss of function of the SMARCB1 gene, also known as SNF5 or INI1. SMARCB1 is a tumor suppressor gene that plays a critical role in regulating cell growth and division. Its loss of function disrupts normal cellular processes and contributes to the development of ATRT. Other genetic alterations, such as alterations in TP53 and alterations in the MYC gene, have also been observed in some cases of ATRT.

Enhancer landscape in ATRT and specifcity to molecular subtypesFig. 1 Enhancer landscape in ATRT and specifcity to molecular subtypes. (Nesvick, C. L., et al., 2020)

Targeted Therapies of ATRT

Epigenetic Regulators

Epigenetic regulators, such as EZH2 and histone deacetylases (HDACs), have been found to play a critical role in ATRT. Increased EZH2 activity, particularly in the ATRT-SHH subtype, has been identified as a potential target for therapy. Inhibition of EZH2 with specific inhibitors, such as UNC1999, has shown selective toxicity to ATRT-SHH cell lines.

Receptor Tyrosine Kinases

ATRT-MYC and ATRT-TYR subtypes have been found to have a critical dependence on receptor tyrosine kinase pathways, particularly PDGFR. Inhibition of receptor tyrosine kinases, such as dasatinib and nilotinib, has shown selective toxicity to ATRT-MYC cell lines. These inhibitors hold promise as targeted therapies for ATRT-MYC and ATRT-TYR tumors.

Immune Checkpoints

Immunotherapy, particularly T cell-based therapies, has shown promise in treating ATRT. Blockade of PD-L1, an immune checkpoint protein, has resulted in reduced tumor growth and prolonged survival in preclinical models of ATRT-MYC. Additionally, targeting B7-H3, a protein expressed by ATRT cells, with chimeric antigen receptor (CAR) T cells has led to tumor regression in preclinical models.

Target/Mode of Action/Class
Classic Chemotherapy and DNA Damaging Agents Alkylating agents Antimetabolite Guanosine analogs Vinca alkaloid
Platinum compounds Intercalating agents Topoisomerase inhibitors
Kinase Inhibitors AKT ALK, TGFbeta Aurora A EGFR-HER2
Cell Cycle Targets CDK2 inhibitors CDK4/6 inhibitors
Epigenetic Targeting Compounds Bromo/BET BRD9 Demethylating agent EZH2
G9a lysine methyltransferase Histone deacetylase inhibitors (HDACi)
Pathway/Lineage Specific Compounds BMP Notch WNT inhibitor Antibody
Ornithine decarboxylase Oncolytic virus
Other compounds ALDH inhibitor LOX inhibitor Diferuoylmethane Flavonoid
PPARg agonist Exosome release inhibitor MDM2, MDM4, MDMX

Our Services

At our company, we are dedicated to advancing the field of ATRT diagnostics and therapy development. We offer a range of services to support the identification and development of novel therapies for ATRT.

Therapy Development Platforms

Animal Models of ATRT

Animal models play a crucial role in preclinical research and the evaluation of potential therapies for ATRT. Our company specializes in the development of robust and relevant animal models that accurately mimic the characteristics of ATRT.

Patient-derived Xenograft Models
Patient-derived xenograft (PDX) models provide a distinctive avenue for investigating the properties of AT/RT tumors within an in vivo environment. At our company, we possess expertise in the development of PDX models specifically tailored for AT/RT research. Through the transplantation of patient-derived AT/RT tumor tissues into immunodeficient mice, we can establish models that faithfully recapitulate the genetic and histopathological characteristics of the original tumor.
Genetic Engineering Models
Our company specializes in developing GEMMs specifically tailored for AT/RT research. B We were able to perform conditional knockout of Snf5 and p53 genes in glial fibrillary acidic protein (GFAP)-expressing cells. The resulting mice developed CNS AT/RT with histological and gene expression similarities to the human counterpart. 
Optional Species Mouse, Rat, Zebrafish, Others

In addition, we also provide other customized animal models to meet diverse needs. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Nesvick, Cody L., et al. "Atypical teratoid rhabdoid tumor: molecular insights and translation to novel therapeutics." Journal of neuro-oncology 150 (2020): 47-56.
  • Hoffman, Lindsey M., et al. "Advancing biology-based therapeutic approaches for atypical teratoid rhabdoid tumors." Neuro-oncology 22.7 (2020): 944-954.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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