Sustained radiographic and clinical response in patient with bifrontal recurrent glioblastoma multiforme with intracerebral infusion of the recombinant targeted toxin TP-38: case study

Neuro Oncol. 2005 Jan;7(1):90-6. doi: 10.1215/S1152851703000589.


Glioblastoma multiforme remains refractory to conventional therapy, and novel therapeutic modalities are desperately needed. TP-38 is a recombinant chimeric protein containing a genetically engineered form of the cytotoxic Pseudomonas exotoxin fused to transforming growth factor (TGF)-alpha. TGF-alpha binds with high affinity to the epidermal growth factor receptor, which is uniformly overexpressed in malignant gliomas, often because of gene amplification. Prior to therapy with TP-38, the patient described here was completely refractory to multiple other therapies, with radiographic and pathologic evidence of tumor progression. After therapy, she improved clinically, was weaned off steroids and anti-convulsants, and experienced a progressive decrease in enhancing tumor volume. Despite multiple prior recurrences, she has not progressed for >43 months after TP-38 therapy. Small remaining areas of enhancement demonstrate no evidence of tumor histologically and are hypometabolic on positron emission tomography. This report describes a dramatic and sustained clinical and radiographic response in a patient with a bifrontal glioblastoma multiforme treated with intratumoral infusion of a novel targeted toxin, TP-38.

Publication types

  • Case Reports
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Brain Neoplasms / drug therapy*
  • Brain Neoplasms / pathology
  • Exotoxins / administration & dosage*
  • Female
  • Glioblastoma / drug therapy*
  • Glioblastoma / pathology
  • Humans
  • Injections, Intraventricular
  • Magnetic Resonance Imaging
  • Middle Aged
  • Pseudomonas aeruginosa
  • Recombinant Fusion Proteins / administration & dosage*
  • Transforming Growth Factor alpha / administration & dosage*
  • Treatment Outcome


  • Exotoxins
  • Recombinant Fusion Proteins
  • Transforming Growth Factor alpha