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Drug for treatment of cerebral diseases

Brain tumorsGlioblastomaHedgehog pathwayMalignant gliomas


Method for rapidly and effectively distributing an antitumor compound, tested on human and murine brain tumor cells, as well as in animal models of glioblastoma, the most common and severe brain tumor in humans, through intranasal administration.

Technical features

This compound has the ability to block biological / biochemical processes that lead to uncontrolled tumor growth. In animal models of glioblastoma the compound was administered nasally in order to quickly reach the brain, through these studies it was possible to ascertain that the antitumor action of the compound is effective at much lower doses than those used in the classic routes of administration. Unlike standard administrations, it will be possible to use the minimum pharmacologically active quantity (intranasal administration) of the compound in a very localized way, thus avoiding any side effects due to a massive administration of the drug in regions of the body far from the site affected by the tumor, the brain. and ensuring direct absorption into the blood, thus avoiding metabolic transformations that can significantly reduce the plasma concentrations of the compound.

Possible Applications

  • Treatment and prevention of brain tumors
  • In brain tumors whose growth is particularly dependent on biological/biochemical processes related to the Hedgehog signaling pathway


  • Use of minimal pharmacologically active quantity of the compound
  • Localized method
  • Non-invasive
  • Direct absorption into the bloodHigh plasma stability of the drug