Advances in the Diagnosis and Treatment of Leptomeningeal Disease

Akanksha Sharma*, Justin T. Low, Priya Kumthekar

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Purpose of Review: Leptomeningeal disease (LMD) is a rare, late complication of systemic cancer and is associated with significant neurological morbidity and high mortality. Here we provide an overview of this condition, summarizing key recent research findings and clinical practice trends in its diagnosis and treatment. We also review current clinical trials for LMD. Recent Findings: Improved molecular diagnostic tools are in development to enable more sensitive detection of LMD, including circulating tumor cells and circulating tumor DNA. The use of targeted and CNS-penetrant therapeutics has shown survival improvements with tyrosine kinase inhibitors, antibody–drug conjugates, and select chemotherapy. However, these studies have primarily been phase I/II and retrospective analyses. There remains a dearth of clinical trials that include LMD patients. Summary: The combination of patient-specific molecular information and novel therapeutic approaches holds significant promise for improving outcomes in patients with LMD.

Original languageEnglish (US)
Pages (from-to)413-425
Number of pages13
JournalCurrent neurology and neuroscience reports
Issue number7
StatePublished - Jul 2022


  • Antibody–drug conjugate
  • Circulating tumor DNA
  • Circulating tumor cells
  • Intrathecal treatment
  • Leptomeningeal disease
  • Tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Neuroscience(all)
  • Clinical Neurology


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