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Preclinical Tests May Lead to New Approach to Treat CNS Lymphoma

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Key Points

  • Pomalidomide was shown to have excellent blood–brain-barrier penetration, a requirement in the treatment of brain tumors and significant preclinical therapeutic activity against central nervous system (CNS) lymphoma in murine models.
  • Treatment with pomalidomide was also shown to have a significant impact on the tumor microenvironment in CNS lymphoma.

A drug recently approved for use in multiple myeloma is now being tested for its ability to fight central nervous system (CNS) lymphoma, a deadly cancer of the immune system that can affect the brain, spinal cord and fluid, and eyes. The clinical trial, now open at the three campuses of Mayo Clinic in Florida, Minnesota, and Arizona, follows successful testing of the drug, pomalidomide (Pomalyst), in mouse models of CNS lymphoma. Results of the preclinical testing were published in PLOS ONE.

Pomalidomide is an immunomodulatory agent that was approved for use in multiple myeloma in February of this year.

Promising Results in Preclinical Trial

"We believe pomalidomide could be beneficial in patients with this cancer because it does two things that most anticancer drugs do not do," said Han Tun, MD, an oncologist at Mayo Clinic in Florida. "The drug has excellent brain penetration, which is a requirement in treatment of brain tumors. The other interesting thing is that it is not only active directly against lymphoma cells but also alters the tumor microenvironment."

Dr. Tun is the senior investigator of the PLOS ONE study and the principal investigator for the clinical trial, which is accruing patients.

"Our preclinical study suggests pomalidomide is very promising. Treatment with pomalidomide in mouse models for CNS lymphoma significantly improved the survival and suppressed the tumor growth," he said "The phase I clinical trial was developed based on these preclinical results."

Dr. Tun received a grant from Celgene to support this research.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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