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Int. J. Mol. Sci. 2016, 17(7), 1074; doi:10.3390/ijms17071074

Molecular Targeted Therapies for the Treatment of Leptomeningeal Carcinomatosis: Current Evidence and Future Directions

Department of Internal Medicine, Seoul National University Hospital, Seoul 03080, Korea
Cancer Research Institute, Seoul National University College of Medicine, Seoul 03080, Korea
Department of Neurosurgery, Seoul National University Hospital, Seoul 03080, Korea
Author to whom correspondence should be addressed.
Academic Editor: Dario Marchetti
Received: 19 May 2016 / Revised: 26 June 2016 / Accepted: 28 June 2016 / Published: 5 July 2016
(This article belongs to the Special Issue Brain Metastasis 2016)
View Full-Text   |   Download PDF [223 KB, uploaded 5 July 2016]   |  


Leptomeningeal carcinomatosis (LMC) is the multifocal seeding of cerebrospinal fluid and leptomeninges by malignant cells. The incidence of LMC is approximately 5% in patients with malignant tumors overall and the rate is increasing due to increasing survival time of cancer patients. Eradication of the disease is not yet possible, so the treatment goals of LMC are to improve neurologic symptoms and to prolong survival. A standard treatment for LMC has not been established due to low incidences of LMC, the rapidly progressing nature of the disease, heterogeneous populations with LMC, and a lack of randomized clinical trial results. Treatment options for LMC include intrathecal chemotherapy, systemic chemotherapy, and radiation therapy, but the prognoses remain poor with a median survival of <3 months. Recently, molecular targeted agents have been applied in the clinic and have shown groundbreaking results in specific patient groups epidermal growth factor receptor (EGFR)-targeted therapy or an anaplastic lymphoma kinase (ALK) inhibitor in lung cancer, human epidermal growth factor receptor 2 (HER2)-directed therapy in breast cancer, and CD20-targeted therapy in B cell lymphoma). Moreover, there are results indicating that the use of these agents under proper dose and administration routes can be effective for managing LMC. In this article, we review molecular targeted agents for managing LMC. View Full-Text
Keywords: leptomeningeal carcinomatosis; lung cancer; breast cancer; molecular targeted agent leptomeningeal carcinomatosis; lung cancer; breast cancer; molecular targeted agent

This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Lee, D.-W.; Lee, K.-H.; Kim, J.W.; Keam, B. Molecular Targeted Therapies for the Treatment of Leptomeningeal Carcinomatosis: Current Evidence and Future Directions. Int. J. Mol. Sci. 2016, 17, 1074.

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