Pohled neurologa na buněčnou a genovou léčbuchorob nervového systému

Neurologist’s View on the Cellular and Gene Therapy for Nervous System Diseases

Lesional impairment of the brain and spinal cord is a serious and usually irreversible disorder. There is only a smallchance of functionally significant compensation of lesions. Neurons and glia aremarked by theminimal regeneration.Though plasticity and adaptability represent important mechanisms, which can contribute to the compensation ofstructural and functional CNS disorders, irreversible structural and functional deficits may develop. Transplantationof various cells incl. neurons from fetal brain or transplantation of genetically modified cells was therefore proposed.Clear verification of this method by animal tests and by clinically controlled trials is a necessary condition. Itsapplication is expected in atrophic-degenerative and vascular diseases of CNS, tumors, injuries, seizure, inflammatoryand demyelinating diseases in which the replacement of neurons, glia and enhancement of their regeneration playthe key role. Transplantation of embryonic stem cells, fetal neural cells, and haematopoietic stem cells introducedby stereotaxic, hematogenic or intrathecal procedure gets the attention. Bioethical problems and the possiblecontribution of this method based on experience with transplantation of fetal cells into basal ganglia of 3 patientswith Parkinson´s disease and haematopoietic stem cell transplantation in multiple sclerosis (n 1) are discussed.

Key words:
bioethics, cell and gene therapy, CNS tumors, Parkinson´s disease, multiple sclerosis.