Finding safe drugs to treat neurodegenerative diseases

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People diagnosed with Huntington’s disease, most in their mid-thirties and forties, face a devastating prognosis: complete mental, physical, and behavioral decline within two decades. “Mutant” protein clusters, long blamed for the progression of the genetic disease, have been the primary focus of therapies in development by pharmaceutical companies. But according to new research from Prof. Gerardo Lederkremer and Dr. Julia Leitman of Tel Aviv University’s Department of Cell Research and Immunology, in collaboration with Prof. Ulrich Hartl of the Max Planck Institute for Biochemistry, these drugs may not only be ineffective — they may pose a serious threat to patients.In two ground-breaking studies, published in the journals PLOS ONE and Nature Communications, Prof. Lederkremer and his team demonstrated that protein clusters are not the cause of toxicity in Huntington’s disease. On the contrary, these aggregates actually serve as a defense mechanism for “stressed” brain cells. Conducted on tissue cultures using cutting-edge microscopic technology, their studies identified a different causative agent — the “stress response” of affected brain cells.”The upsetting implication for therapy of this disease is that drugs being developed to interfere with the formation of protein aggregates may in fact be detrimental,” said Prof. Lederkremer. …


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