Endocrine Abstracts (2002) 3 S29

Repairing and protecting neurones, a dual goal for cell based therapy to the brain

M Peschanski

INSERM U 421, Faculte de Medecine, Creteil, 94010 cedex, France.

For more than a dozen years, a major combined biological and clinical research endeavour has been dedicated to the set up of new therapeutics based upon cell and gene therapy for neurodegenerative diseases. This research essentially takes into account two determinant characteristics of all these diseases, that can be briefly summarised as follows: 1. a neurodegenerative disease is due to the loss of one or a small number of specific populations of neurones, allowing in some cases for focal intra-cerebral intervention; 2. this neuronal loss is always progressive, according however to a schedule which is quite different from one disease to another, therefore providing a potential 'therapeutic time window' for protective intervention. Without a discrete knowledge of the physiopathology of the diseases, and of their specific molecular and cellular mechanisms, one can envision basically two different, and complementary therapeutic modalities for these diseases. First, one may consider substituting to the degenerated neurones, homologous cells that are capable of replacing them anatomically and functionally. This so-called 'substitutive' therapeutics is essentially based, at this moment, upon the use of neural cells obtained from human foetuses following elective abortions. Second, proteins have been identified that are able to protect neurones against various experimental aggressions in animals and are, therefore, good candidates to rescue neurones affected, though not yet degenerated, during disease progression. This so-called 'conservative' therapeutics is essentially attempted, at this moment, by cell or viral based transfer of a gene of interest into the brain. In this talk, I will present data concerning both pre-clinical and clinical studies that have been carried out over the past decade for Parkinson's and Huntington's diseases, as well as discuss new opportunities that are potentially offered by current research on various types of stem cells.

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