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A glimmer of hope in the struggle against neurodegenerative diseases: the virtues of proteins that can save dying neurons

Neurodegenerative diseases such as Alzheimer, Parkinson, Huntington and Amyotrophic Lateral Sclerosis (ALS) have at least two common features. On one hand they are both characterized by the degeneration of a specific type of neuronal cell in the brain. On the other hand, there is no long-lasting treatment which can arrest their disastrous effects. The group of Professor Ann Kato has recently demonstrated the essential role of a family of proteins, the IAP’s (Inhibitors of Apoptosis) in rescuing motor neurons, that is the target cells in ALS or Charcot’s disease. These results may represent a step in the search for a reliable therapy for the disease and the work is being published in the month of February in the scientific journal "Nature Cell Biology".

Diseases characterized by neurodegeneration affect individuals over 50 years of age and they attack one particular class of neurons in the brain or spinal cord. The research interest of Professor Ann Kato and her team is principally focalized on ALS in which there is a progressive paralysis caused by the destruction of motor neurons which exist in both the brain and spinal cord. These cells are responsible for the movements of the upper and lower limbs and their elimination causes muscular atrophy and finally death.

During the last 3 years, the objectives of the experiments of Professor Kato consist in trying to understand why these neurons die and to study the intracellular and molecular pathways which can prevent their degeneration. It was during the course of their experiments to attempt to prolong the survival of the cells that the group of Professor Kato found that a newly discovered family of proteins called Inhibitors of Apoptosis Proteins (IAP’s) were capable of preventing the death of motor neurons. The results of these experiments show that if these particular proteins can be increased inside motor neurons, then it is possible to prevent their degeneration. This discovery opens the way for promising new therapeutic perspectives in both gene therapy for diseases of neuronal degeneration and also in the field of classical pharmacology which concerns the search for new small molecular weight substances which can increase the production of IAP family members or substances which can play the same role as the IAP’s in the brain and spinal cord.

We hope that this discovery will not only encourage but also accelerate clinical and genetic research in this disease but also in other neurodegenerative diseases such as Alzheimer’s and Parkinson’s. This work represents another small step in the continuing search for efficient therapeutic agents.

Reference of the article: Nature Cell Biology, February 2002, vol. 4, and pages175-179

Pour tout renseignement complémentaire, n'hésitez pas à contacter:
prof. Ann C. Kato, Ph. D., phone 022 702 54 50
or Charles-Antoine Courcoux, attaché de presse, phone 022 379 77 96