The invention relates to the use of inhibitors of the enzyme mTOR kinase (mammalian target of rapamycin) in the treatment of neuro-oncol. diseases, in particular tuberous sclerosis, neurodegenerative diseases, in particular Alzheimer's disease, and neuroinflammatory diseases, in particular multiple sclerosis and primary progressive aphasia, via intrathecal, or preferably intraventricular, administration of said inhibitors. Thus, from the evidence presented in the disclosure, it was evident that it is possible to reaching locally, within the cranial theca, a concn. of inhibitor greater than that reached in the same region through systemic administration; from there it is concluded that the inhibitor present here is capable of inhibiting the mTOR signalling pathway; it is also seen that this concn. is capable of performing an immunosuppressive action locally, while remaining in the blood at unassayable levels and without affecting the normal activity of mTOR in peripheral organs.
Intrathecal administration of mTOR inhibitors for the therapy of neurodegenerative, neuroinflammatory and neurooncologic diseases
GIOVAGNOLI, Stefano;MAGINI, Alessandro;EMILIANI, Carla
2013
Abstract
The invention relates to the use of inhibitors of the enzyme mTOR kinase (mammalian target of rapamycin) in the treatment of neuro-oncol. diseases, in particular tuberous sclerosis, neurodegenerative diseases, in particular Alzheimer's disease, and neuroinflammatory diseases, in particular multiple sclerosis and primary progressive aphasia, via intrathecal, or preferably intraventricular, administration of said inhibitors. Thus, from the evidence presented in the disclosure, it was evident that it is possible to reaching locally, within the cranial theca, a concn. of inhibitor greater than that reached in the same region through systemic administration; from there it is concluded that the inhibitor present here is capable of inhibiting the mTOR signalling pathway; it is also seen that this concn. is capable of performing an immunosuppressive action locally, while remaining in the blood at unassayable levels and without affecting the normal activity of mTOR in peripheral organs.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.