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Brain Cancer treatment details. Gene Therapy. Johns Hopkins, Baltimore, MD, United States

Survival: 3.4 months
Toxicity Grade: 4
Treatments: Gene Therapy
Country: United States
City/State/Province: Baltimore, MD
Hospital: Johns Hopkins
Journal: Link
Date: 1/2005

Patients: This phase II trial involved 21 patients. There were 15 men and 6 women and the average age was 49.7 years. Sixteen patients had malignant glioblastoma multiforme (GBM), one had anaplastic astrocytoma, and four had anaplastic oligodendrocytoma. All but three patients had received chemotherapy prior to treatment.

Treatment: The treatment consisted of a gene therapy agent: aprinocarsen. Aprinocarsen is an antisense oligonucleotide against protein kinase-C activity, which is needed for tumor growth.

Toxicity: The authors noted one patient's death as follows: "One case suggested that aprinocarsen could have contributed to the early neurological deterioration seen in patients treated on this research protocol...[this patient] subsequently died of his brain tumor." Grade 3-4 toxcities included elevated liver enzymes, thrombocytopenia, fatigue, nausea, and vomiting. In addition, increased intracranial pressure and blood-brain barrier permeability was observed.

Results: The median overall survival was 3.4 months. Compared to a control population, no significant response or therapeutic benefit to treatment was observed, and the trial was prematurely terminated.

Support: Aprinocarsen was initially developed as Isis 3521 by Isis Pharmaceuticals, Inc. (Carlsbad, Calif.), which supplied the drug for this trial. After completion of this study, this agent was licensed to Eli Lilly, and its name was changed to LY900003 (Affinitak).

Correspondence: Stuart A. Grossman, MD

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