(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 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’s environment. To date, there have been no curative treatment options available for these patients. The study was published in the renowned journal JAMA Oncology.
Medulloblastomas are the most common malignant brain tumors in children and adolescents and account for about 20% of all brain tumors in children. In about a quarter of cases, the tumor returns despite surgery, radiotherapy and chemotherapy. To date, there have been no curative treatment options for this rare tumor when it recurs. When a medulloblastoma recurs, it almost always spreads to the brain, and therefore no total neurosurgical resection (complete removal) is possible. The tumor is resistant to conventional chemotherapy drugs and irradiation of the brain with an effective dose for the tumor is only possible once. The new therapy starves cancer by intervening primarily in the cancer’s environment. To date, patients with this disease have had no chance of curative treatment.
Since 2006, the University Clinic for Pediatrics and Adolescents, part of the Comprehensive Cancer Center and the Comprehensive Center for Pediatrics at MedUni Vienna and the Vienna General Hospital, has administered metronomic antiangiogenic drug therapy to these patients. “Antiangiogenic therapy prevents the tumor from forming the blood vessels necessary for further growth. This therapeutic approach to relapse is attractive because it is not primarily the tumor itself that is attacked, but rather the cancer’s environment, the so-called ‘microenvironment’, and the tumor is starving, so to speak,” says the first author of the MEMMAT study. Andreas Peyrl, from the University Clinic of Pediatrics and Adolescent Medicine.
A quarter of patients have long-term survival of more than five years
In this context, metronomic means taking the medicine daily in low dosage so that these medicines can be administered without interruption for a long period of time. The medicines used are low-dose chemotherapy drugs, but also medicines from other areas that are used, for example, in rheumatology or to reduce 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 outpatient and well-tolerated treatment.
The study, which was initiated and funded by MedUni Vienna and now 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. results in patients with recurrence of previously irradiated medulloblastoma. A quarter of patients have long-term survival of more than five years. With previous therapies, there have only been a few cases of improved 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 objective now is to better evaluate this medicine in a randomized trial in Europe and the USA.
Publication: JAMA Oncology
Sustained survival benefit in recurrent medulloblastoma by a metronomic antiangiogenic regimen. A non-randomized clinical 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
Slavic.
doi: 10.1001/jamaoncol.2023.4437