Central Nervous System Lymphoma: Novel Therapies

Shannon P. Fortin Ensign, Diamone Gathers, Julia Erin Wiedmeier, Maciej M. Mrugala

Research output: Contribution to journalReview articlepeer-review


Primary central nervous system lymphomas (PCNSLs) are very rare neoplasms and continue to be challenging to treat. While high-dose methotrexate (HD-MTX)-based regimens are the currently accepted standard first-line therapy for newly diagnosed patients, the optimal induction therapies are still unknown. The role of consolidation therapies continues to evolve with a variety of chemotherapy regimens, including high-dose chemotherapy with stem cell rescue and reduced or deferred whole brain radiotherapy being used. Importantly, several recent advances have been made in the treatment of PCNSL. The incorporation of targeted therapy and immune therapy remain promising strategies. Several agents, successfully used in treatment of systemic lymphomas, have shown activity in PCNSL, frequently leading to durable responses in the relapsed/refractory patients. Many ongoing studies will likely lead to a better understanding of the roles of these treatments, especially as the first line and potentially also as maintenance. In addition, the use of molecular profiling to predict disease response to targeted agents and understand relapse patterns will become increasingly important. Clinical trials in PCNSL are critical yet frequently challenging to conduct given the rarity of the condition and lack of suitable subjects. Therefore, multi-institutional and international collaboration is of utmost importance to accelerate progress in understanding the biology and design better treatments for this disease. It is critical to consider patients of all demographics in the design and study of future treatment algorithms to have the largest impact on patient care and outcomes.

Original languageEnglish (US)
Pages (from-to)117-136
Number of pages20
JournalCurrent treatment options in oncology
Issue number1
StatePublished - Jan 2022


  • Molecular profiling
  • Novel therapies
  • Primary central nervous system lymphoma
  • Targeted therapy

ASJC Scopus subject areas

  • Oncology
  • Pharmacology (medical)


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