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New therapeutic approach for malignant brain tumors in children and adolescents

Department of Pediatrics and Adolescent Medicine

(Vienna, 27-10-2023) A study led by the Medical University of Vienna shows a sustained survival benefit of so-called anti-angiogenic therapy in recurrence of the most common malignant brain tumor in children and adolescents.

This form of therapy starves the cancer by interfering primarily with the cancer environment. Until now, no curative treatment option has been available for these patient:ins. The study has now been published in the renowned journal JAMA Oncology..

Medulloblastomas are the most common malignant brain tumors in children and adolescents and account for about 20 percent of all brain tumors in children. In about a quarter of cases, the tumor recurs despite surgery, radiation and chemotherapy. To date, there are no curative treatment options for this rare tumor in recurrence. In recurrence, medulloblastoma has almost always spread to the brain, making total neurosurgical resection (complete removal) impossible, the tumor is resistant to conventional chemotherapeutic agents, and radiation to the brain at a dose effective for the tumor is possible only once. The new therapy starves the cancer by interfering mainly with the cancer environment. Until now, patients with this disease had no chance of curative treatment.

The University Department of Pediatrics and Adolescent Medicine, part of the Comprehensive Cancer Center and the Comprehensive Center for Pediatrics at MedUni Vienna and Vienna General Hospital, has been administering metronomic anti-angiogenic drug therapy to these patients since 2006. "Antiangiogenic therapy prevents the tumor from forming blood vessels, which it needs to continue growing. This therapeutic approach to recurrences is attractive in that it is not primarily the tumor itself that is attacked, but the cancer environment, the so-called 'microenvironment,' and the tumor is starved, so to speak," says lead author of the MEMMAT study Andreas Peyrl of the University Department of Pediatrics and Adolescent Medicine.

A quarter of patients have long-term survival of more than five years
Metronomic in this context means that the drugs are taken daily in low doses so that they can be administered without interruption over a long period of time. The drugs used are either low-dose chemotherapeutic agents, but also drugs from other fields, such as those used in rheumatology or to lower blood lipids. Oral and intravenous therapy is supplemented by intraventricular chemotherapeutic agents injected directly into the cerebrospinal fluid (CSF). Although regular hospital visits are required for patients, the investigated MEMMAT combination regimen is an overall well-tolerated and outpatient treatment.

The study, initiated and funded at MedUni Vienna and now published in JAMA Oncology, and involving a total of 40 patients in Austria, the Czech Republic, Spain, France, Denmark, Sweden, Norway and the USA between 2014 and 2021, shows promising results in patients with previously irradiated medulloblastoma recurrence. A quarter of patients show long-term survival of more than five years. With previous therapies, there was only isolated prolonged survival in the patient:in group. "The results of the study are very encouraging, we can offer patients a promising therapy for the first time," says Andreas Peyrl. In Austria, about three children per year develop this rare form of the disease. The next goal is now to further evaluate this drug in a randomized study in Europe and the USA.

Publication: JAMA Oncology
Sustained survival benefit in recurrent medulloblastoma with metronomic antiangiogenic therapy. A non-randomized controlled trial.
Andreas Peyrl, Monika Chocholous, Magnus Sabel, Alvaro Lassaletta, Jaroslav Sterba, Pierre Leblond, Karsten Nysom, Ingrid Torsvik, Susan N. Chi Thomas Perwein, Neil Jones, Stefan Holm, Per Nyman, Helena Mörse, Anders Öberg, Liesa Weiler-Wichtl, Ulrike Leiss, Christine Haberler, Lisa Mayr, Karin Dieckmann, Marcel Kool, Johannes Gojo, Amedeo A. Azizi, Nicolas André, Mark Kieran, and Irene Slavc.
doi: 10.1001/jamaoncol.2023.4437