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ATRT Publications

Recent Publications


Mar 16, 2024

CAR T cells redirected to B7-H3 for pediatric solid tumors: Current status and future perspectives

This article reviews studies that look at the potential of using B7-H3 CAR T cells to treat pediatric solid tumors. There are currently clinical trials underway to study the safety and effectiveness of this approach.

BrainChild-04 is a clinical trial open to ATRT patients at Seattle Children's Hospital.


Mar 14, 2024

ESTRO-SIOPE guideline: Clinical management of radiotherapy in atypical teratoid/rhabdoid tumors (AT/RTs)

A new guideline published in Radiotherapy and Oncology addresses the complex issue of radiotherapy for Atypical Teratoid Rhabdoid Tumors (ATRT) in young children. This summary outlines recommendations for treatment planning, radiation techniques, and considerations for minimizing risks to the developing brain.


Apr 18, 2023

Focal versus craniospinal radiation for disseminated atypical teratoid/rhabdoid tumor following favorable response to systemic therapy

While radiation therapy (RT) is an important tool in treatment for ATRT, it comes with risks for severe cognitive impacts among young patients. Given that most ATRT patients are diagnosed before the age of three, it is especially important to understand how these impacts can be minimized while still obtaining the increased survival benefits that RT can provide. The aim of this study was to see if RT dose and delivery fields (focal versus craniospinal) impacted patient outcomes.


Feb 9, 2023

Recent Progress and Novel Approaches to Treating Atypical Teratoid Rhabdoid Tumor

This article provides a thorough overview of the latest published information on ATRT research – summaries of results in recently completed clinical trials for newly diagnosed as well as relapsed ATRT, information about ongoing clinical trials, and a look at novel agents being tested in animal models of ATRT before possible inclusion in human clinical trials.


Apr 1, 2022

Clinical Predictors of Survival for Patients with Atypical Teratoid Rhabdoid Tumors

This study used data from the National Cancer Database (NCDB), one of only two patient registries that track patient outcome data for ATRT. The analysis patients who underwent bone marrow/stem cell transplantation in addition to chemo and radiation saw an even greater increase in progression-free survival.


Mar 18, 2021

Relevance of Molecular Groups in Children with Newly Diagnosed Atypical Teratoid Rhabdoid Tumor: Results from Prospective St. Jude Multi-Institutional Trials

An article published online in Clinical Cancer Research offers some insight into differing outcomes associated with the three molecular subtypes of ATRT. The study included 74 newly diagnosed ATRT patients, from infants to adolescents, who were treated in two clinical trials at multiple institutions using protocols established through St. Jude Children’s Research Hospital. Infants with ATRT-TYR had the best overall survival. ATRT-SHH was associated with metastatic disease and poorer outcomes regardless of age.  Among children – regardless of molecular subtype – those whose ATRT had not spread beyond the tumor site benefited from post-surgical chemotherapy and radiation therapy of the brain and spine.


Feb 3, 2021

Current recommendations for clinical surveillance and genetic testing in rhabdoid tumor predisposition: a report from the SIOPE Host Genome Working Group

Atypical Teratoid Rhabdoid Tumor can occur in some children who have rare genetic conditions known as rhabdoid tumor predisposition syndrome making young children vulnerable to an increased risk of developing Rhabdoid tumors. Tumor surveillance protocols for these rare families have not been established. The European Society for Pediatric Oncology (SIOPe) Host Genome working group invited pediatric oncologists and geneticists to contribute to an expert meeting that culminated in a consensus statement to guide clinicians and patient families.


Dec 17, 2020

Mithramycin Induces Promoter Reprogramming and Differentiation of Rhabdoid Tumor

This article in the journal EMBO Molecular Medicine examines the potential of a long-known antibiotic for use against rhabdoid tumors like ATRT. While Mithramycin showed promise due to its effectiveness against tumor cells, its toxicity limited its use. The study investigates a drug known as EC8042, a less toxic analogue of mithramycin, and explores optimal dosing for future clinical trials.


Jun 14, 2020

Genomic and immunologic characterization of INI1-deficient pediatric cancers

Checkpoint inhibitor drugs offer new hope for many types of cancer by unleashing the body’s immune system to attack cancer cells. To date, however, these drugs have not shown much success against pediatric cancers. A new study from researchers at the Dana Farber Institute and Boston Children’s Hospital may challenge that finding when it comes to patients with ATRT. A clinical trial based on this study’s results is already underway.


Apr 30, 2020

Locoregionally administered B7-H3-targeted CAR T cells for treatment of atypical teratoid/rhabdoid tumors.

In a recently published letter to the journal Nature Medicine, researchers proposed that it might be possible to interrupt that lethal relationship through an immunotherapy approach called CAR-T. Normal immune system defenders called t-cells are gathered from a patient’s blood and reprogrammed in the laboratory to seek out and neutralize a target, in this case B7-H3. The researchers theorize that neutralizing B7-H3 would unmask ATRT cells so that the body’s normal defenses could attack them.


Feb 27, 2020

Efficacy of High-Dose Chemotherapy and Three-Dimensional Conformal Radiation for Atypical Teratoid/Rhabdoid Tumor: A Report From the Children's Oncology Group Trial ACNS0333

A treatment protocol involving surgery followed by high-dose chemotherapy and radiation therapy yielded dramatically improved survival compared to previously reported therapies.  Among 65 patients evaluated on the regimen called ACNS0333, 35% survived without relapse or new malignancies for four years after enrollment.  Patients over three years of age fared even better, with 48% event-free survival (EFS) at four years.  By comparison, two earlier studies of children with a spectrum of brain tumors who were treated with dose-intensified chemo showed 6.4% of the AT/RT patients survived without such events for a period of two years.


Dec 31, 2019

Molecular subgrouping of Atypical Teratoid / Rhabdoid Tumors (ATRT) – a reinvestigation and current consensus

To date, the only common genetic feature identified in ATRT has been the inactivation of a gene called SMARCB1, usually through the complete or partial deletion of chromosome 22. The authors of this study looked at all ATRT research findings to date, searching for other commonalities. Their goal was to identify and describe subgroups of ATRT for further study to hopefully uncover targets for new treatments, and to allow a more thorough understanding of how patients in each subgroup respond to existing therapies.

Older Publications

Nov 3, 2019

Survival Benefit with Gross Total Resection and Adjuvant Radiotherapy in Childhood Atypical Teratoid/Rhabdoid Tumors: Results of a Single-Center Cohort of 27 Cases

This study from a group of neurosurgery clinics in Turkey looked at 27 patients diagnosed with AT/RT between January 2000 and December 2017.  Total resection of the tumor was possible in nearly half (13) of the patients, though three died of postoperative complications. The remaining patients received chemotherapy and/or radiation therapy. Among the many variables compared in this group (sex, age at diagnosis, location of tumor, gross total resection, chemotherapy, radiotherapy), only the administration of radiotherapy was consistently associated with longer overall survival.

Apr 12, 2019

Role of early and aggressive post-operative radiation therapy in improving outcome for pediatric central nervous system atypical teratoid/rhabdoid tumor

Researchers followed 28 pediatric AT/RT patients through surgery and subsequent radiation therapy for an average of four years. The patients’ data showed that early and aggressive radiation therapy after surgery is critical for successful disease control. Delaying radiation therapy until the AT/RT progressed led to unfavorable outcomes in the group.

Mar 5, 2019

Human Pluripotent Stem Cell-Derived Tumor Model Uncovers the Embryonic Stem Cell Signature as a Key Driver in Atypical Teratoid/Rhabdoid Tumor

In their successful efforts to develop a better model of human AT/RT in mice, researchers also identified a promising therapeutic target for AT/RT as well as other pediatric cancers. This potential target merits further study.

Feb 19, 2019

Safety and feasibility of outpatient autologous stem cell transplantation in pediatric patients with primary central nervous system tumors

This study from Children’s Hospital Colorado looked at pediatric patients with central nervous system (CNS) tumors who received stem cell transplants using their own cells. Some were transplanted as hospital inpatients; others received preparatory, high-dose chemotherapy and stem cell transfusions on an outpatient basis. Through more than two years of follow-up, outcomes were similar for both groups. This shows that outpatient autologous stem cell transplants are safe and effective for properly selected children with CNS tumors.

Feb 18, 2019

A Phase 2 Study of Alisertib in Children with Recurrent/Refractory Solid Tumors or Leukemia: Children’s Oncology Group Phase 1 and Pilot Consortium (ADVL0921)

A targeted therapy drug called alisertib was tested among 137 children with acute leukemias or relapsed/refractory solid tumors including AT/RT with certain mutations. Previously, the drug showed significant ability to trigger cell death in tumor samples in the laboratory. It also showed modest activity in a phase 2 trial among adults with ovarian cancer, certain lymphomas and acute myeloid leukemia. The 137 children in this trial could only tolerate a smaller dose of the drug, which generated responses in less than 5% of the cases.

Dec 27, 2018

Comprehensive Analysis of Chromatin States in Atypical Teratoid/Rhabdoid Tumor Identifies Diverging Roles for SWI/SNF and Polycomb in Gene Regulation

For over 20 years, scientists have known that AT/RT is characterized by the inactivation of a gene called SMARCB1, usually through the complete or partial deletion of chromosome 22. But little is known as to how this leads to tumor development. Understanding the molecular mechanisms behind this is urgently needed in order to develop effective treatment strategies for AT/RT patients. In this study, investigators used a sophisticated method called ChIP sequencing to compare eleven AT/RT samples from all three recently identified molecular subgroups to existing data on a variety of brain tumors, normal brain tissue and the embryonic stem cells that eventually develop into brain. The results provide a better understanding of AT/RT’s basic biology and pointed out regulatory mechanisms disrupted in AT/RT. With further study, these may lead to potential targets for therapy.

Dec 8, 2018

Overall Survival of Primary Intracranial Atypical Teratoid Rhabdoid Tumor Following Multimodal Treatment: A Pooled Analysis of Individual Patient Data

This 2018 paper involved 18 patients with AT/RT treated at a center in Beijing, China, as well as published data on another 483 such patients worldwide. The authors evaluated the relative effectiveness of treatments including surgical tumor resection, radiotherapy, conventional chemotherapy, intensified chemotherapy or some combination thereof. Their findings suggest the best overall survival rates (88% at five years) were among patients whose masses could be removed surgically without any visual residual tumor and who subsequently received radiotherapy as well as conventional and intensified chemotherapy.

Sep 14, 2017

Oncolytic Herpes Virus rRp450 Shows Efficacy in Orthotopic Xenograft Group 3/4 Medulloblastomas and Atypical Teratoid/Rhabdoid Tumors

Pediatric brain tumors like AT/RT or medulloblastomas may or may not be totally removed through surgery. And subsequent treatments like radiotherapy and intensive chemotherapy can have debilitating side effects and limited effectiveness. This study looks at the possible use of a weakened herpes virus to infect and kill these brain cancer cells, much like a viral approach already FDA approved for use in melanoma patients. The researchers delivered a cancer-fighting but weakened form of herpes virus to AT/RT or medulloblastoma cancer cells in the laboratory as well as in mouse models. Their promising findings warrant further study.

Jan 10, 2016

Atypical teratoid/rhabdoid tumors-current concepts, advances in biology, and potential future therapies

Recent advances in technologies for cancer research have led to identification of subgroups within the diagnosis of AT/RT and corresponding potential molecular targets for treatment.  Further study may help physicians identify whether these subgroups of patients respond differently to existing therapies. Meanwhile, there is still no agreement on a standard of therapy for AT/RT. This 2016 article by an international group of physicians provides a thorough overview of published data on state-of-the-art approaches for clinical management of children with AT/RT.  It also outlines the current thinking on potential new targets for treatments within identified subgroups of AT/RT patients, and summarizes results of both laboratory research and early-stage clinical trials of these targeted therapy models.

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