The drug pomalidomide, already approved for use in persons with multiple myeloma, is a promising therapeutic agent for central nervous system (CNS) lymphoma, say investigators.

An immunomodulatory agent, pomalidomide (Pomalyst®) won FDA approval earlier this year as a treatment for multiple myeloma. Now, oncologist Han W. Tun, MD, of Mayo Clinic in Jacksonville, Florida, and colleagues have tested the drug in mouse models of CNS lymphoma, a deadly cancer of the immune system that can affect the brain, the spinal cord and spinal fluid, and the eyes.

The team’s work has yielded encouraging results, as described in the journal PLOS ONE (2013;8[8]:e71754): The preclinical research showed that pomalidomide significantly improved survival and suppressed tumor growth, exhibiting significant therapeutic activity against CNS lymphoma.

Continue Reading

In a statement issued by Mayo Clinic, Tun explained that pomalidomide could be beneficial in patients with CNS lymphoma because the agent possesses two features most anticancer drugs do not:

Pomalidomide has excellent brain penetration, a requirement in the treatment of brain tumors.

Pomalidomide not only is active against lymphoma cells, it also alters the tumor microenvironment in several ways that work against the tumor. 

Tun, who was senior investigator on the mouse study, is principal investigator for the phase 1 clinical trial, which is now accruing patients. More information can be found at