Gene therapy for the eradication of glioblastoma

Section: 

  KEYWORDS

  •  Glioblastoma
  •  EMX2 gene
  •  Tumor stem cells

 

 

 

 

Summary

Gene therapy by induction of EMX2 expression for the eradication of Glioblastoma (GBM), characterized by simultaneously targeting a remarkable number of distinct metabolic nodes, crucial for the malignancy of the same species in the most advanced stages of its progression ( patent application IT 102015000046666).

Technology

The proposed experimental therapeutic treatment, patented in SISSA and developed by prof. Mallamaci consists of a gene therapy for the eradication of Glioblastoma (GBM), characterized by simultaneously targeting a remarkable number of distinct metabolic nodes, crucial for the malignancy of the same species in the most advanced stages of its progression. This explains the high frequency of response observed in the experimental studies conducted so far. In addition, the therapeutic activity unfolds specifically in the tumor stem cells, facilitating their eradication. The treatment arises from the enhancement of the results of the study of normal mechanisms of regulation of cortico-cerebral astrogenesis conducted at the Laboratorio di Sviluppo della Corteccia Cerebrale of SISSA (http://lccd.sissa.it/). It is based on the controlled overexpression of the EMX2 gene, implicated in the normal regulation of cerebral histogenesis, capable of inducing the collapse of all the currently tested glioblastomas, in vitro and in vivo.

Advantages

The proposed experimental therapy, compared to the others available today, allows to act simultaneously on a remarkable number of distinct metabolic nodes, among which many are crucial for the malignancy of the tumor in the most advanced stages of its progression. As such, it portends:
- a high frequency of success with primary tumors of different molecular origins;
- a particular ability on the part of the treatment to counteract recurrences.

Reference market

Every year, the GBM afflicts 2000 new patients in Italy, 15,000 in the EU, over 200,000 in the whole world, with a survival time that does not exceed 15 months. From an economic point of view, the "global market" for the treatment of this family of tumors was quantified at US $ 659 million in 2014 and is estimated to triple by 2024 to reach US $ 3.3 billion (Source: GlobaData - 2014).

Technology readiness level

The therapy obtained very encouraging results in the in vitro experimentation already carried out and in the first in vivo experiments, on murine models (TRL 3/4).

Scientific referent

Prof. Antonello Mallamaci - Director of the Laboratorio di Sviluppo della Corteccia Cerebrale of SISSA