Key clinical point: A novel penclomedine has shown promising safety and efficacy in some adolescent and young adult patients with cancers involving the central nervous system.

Major finding: Two patients who responded to the treatment, 4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN), were in their 59th month of survival at last follow-up.

Study details: A phase 1/2 study of 15 adolescents and young adults with central nervous system cancers who received DM-CHOC-PEN.

Disclosures: The study was supported by Louisiana state grants, the National Cancer Institute, the National Institute of General Medical Sciences, and the Small Business Innovation Research program. Dr. Morgan is chief executive officer of Dekk-Tec, Inc., which is developing DM-CHOC-PEN.