
==== Front
Neurooncol Adv
Neurooncol Adv
noa
Neuro-Oncology Advances
2632-2498
Oxford University Press US

10.1093/noajnl/vdae090.110
vdae090.110
Final Category: Research Methods and Trial Design Considerations
AcademicSubjects/MED00300
AcademicSubjects/MED00310
RMTD-03 USE OF A BRAIN-PENETRATING ANTHRACYCLINE IN ANTHRACYCLINE-SENSITIVE BRAIN METASTASES: THE PROMISE OF BERUBICIN
Silberman Sandra CNS Pharmaceuticals, Houston/TX, USA

8 2024
02 8 2024
02 8 2024
6 Suppl 1 2024 SNO/ASCO CNS Metastases Conference i33i34
© The Author(s) 2024. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.
2024
https://creativecommons.org/licenses/by-nc/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (https://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com

Abstract

BACKGROUND

Brain metastases are a common complication of cancer and also type of brain tumor: 10%-26% of patients develop brain metastases. Chemotherapy for these tumors is usually ineffective due to an intact blood-brain barrier (BBB). Anthracycline-sensitive cancers that metastasize to the brain do not receive anthracyclines which do not penetrate the brain. Triple-negative breast cancer (TNBC), which can be treated with doxorubicin (DOX) systemically has an incidence of brain metastases of approximately 25–46%; although lymphomas usually require an anthracycline as part of their chemotherapy, when they appear as a primary disease in the brain anthracyclines cannot be used. Berubicin is an analog of doxorubicin that appears to cross the BBB with significant central nervous system (CNS) uptake. A pivotal trial for patients with recurrent glioblastoma (GBM) after first-line therapy has finished enrollment with 252 patients in the US and Europe with evidence of efficacy and no impact on cardiac function.

METHODS

CNS Pharmaceuticals plans to evaluate Berubicin in patients with Primary CNS Lymphoma (PCNSL) who have never received an anthracycline and in TNBC with documented brain metastases. Patients will be enrolled if they have shown progression in the brain and have not undergone primary treatment. Outcome measurements will include efficacy in terms of overall response rates (ORR), progression-free survival (PFS), and overall survival (OS) as well as a continuing evaluation of Berubicin’s safety and tolerability.

CONCLUSIONS

The use of anthracyclines for the treatment of malignancy has encompassed 50 years of showing significant activity in certain diseases, however, use has been limited by both the risk of cardiotoxicity as well as the inability to penetrate the BBB, where these tumors tend to metastasize. This study offers the promise of using an anthracycline that can cross the BBB effectively for sensitive tumors and Berubicin becoming an important part of their therapy
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