2023-10-27 06:23:31
(Vienna, October 27, 2023) A study led by the Medical University of Vienna shows a sustained survival benefit of so-called antiangiogenic therapy in the event of a recurrence of the most common malignant brain tumor in children and adolescents. This form of therapy starves the cancer by primarily interfering with the cancer environment. To date, there has been no curative treatment option available for these patients. 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 are responsible for around 20 percent of all brain tumors in children. In around a quarter of cases, the tumor recurs despite surgery, radiation therapy and chemotherapy. To date, there have been no curative treatment options for this rare tumor when it recurs. When a medulloblastoma recurs, it has almost always spread to the brain and therefore no neurosurgical total resection (complete removal) is possible. The tumor is resistant to conventional chemotherapy drugs and irradiation in the brain with a tumor-effective dose is only possible once. The new therapy starves the cancer by primarily intervening in the cancer environment. To date, patients with this disease have had no chance of a curative treatment.
Since 2006, the University Clinic for Pediatrics and Adolescents, part of the Comprehensive Cancer Center and the Comprehensive Center for Pediatrics of MedUni Vienna and Vienna General Hospital, has administered metronomic antiangiogenic drug therapy to these patients. “Antiangiogenic therapy prevents the tumor from forming blood vessels that it needs for further growth. This therapeutic approach in relapse is attractive in that it is not the tumor itself that is primarily attacked, but rather the cancer environment, the so-called ‘microenvironment’, and the tumor is starved, so to speak,” says first author of the MEMMAT study Andreas Peyrl from the University Clinic for Pediatrics and Medicine Adolescent medicine.
A quarter of patients show long-term survival of more than five years
In this context, metronomic means taking the medication daily in a low dosage so that these medications can be given without interruption over a long period of time. The drugs used are either low-dose chemotherapy drugs, but also drugs from other areas that are used, for example, in rheumatology or to lower blood lipids. Oral and intravenous therapy is reinforced by intraventricular chemotherapy drugs, which are injected directly into the cerebrospinal fluid. Although patients require regular hospital visits, the MEMMAT combination regimen examined is an overall well-tolerated and outpatient treatment.
The study, which was initiated and financed at MedUni Vienna and has now been published in JAMA Oncology, includes a total of 40 patients from 2014 to 2021 in Austria, the Czech Republic, Spain, France, Denmark, Sweden, Norway and the USA 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 a few cases of longer survival in this group of patients. “The results of the study are very encouraging; we can now offer those affected a promising therapy for the first time,” says Andreas Peyrl. In Austria, this rare form of the disease affects around three children per year. The next goal now is to further evaluate this medication in a randomized trial in Europe and the USA.
Publikation: JAMA Oncology
Sustained Survival Benefit in Recurrent Medulloblastoma by a Metronomic Antiangiogenic Regimen. A Nonrandomized 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.
two: 10.1001/jamaoncol.2023.4437
1698388646
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