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Editorial [Hot topic: Prion Diseases: Targets and Treatments of Symptoms and Disease Progression (Guest Editor: Brian Appleby)]

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This month's edition of CNS and Neurological Disorders-Drug Targets is dedicated to the treatment of prion diseases. Although rare, prion diseases are devastating to patients and families. Because of its rarity, the expertise in the field is sparse and misunderstandings are commonplace. Prion diseases are an important field in the study of science in general. They are the only known transmissible diseases that do not utilize nucleic acids and they have sporadic, genetic, and transmissible etiologies. Because of its unique features, the prion disease field has and will continue to contribute to general scientific and medical knowledge. From mathematics (e.g. nucleation) to psychiatry (e.g. stress diathesis), the field of prion diseases has much to offer. Unfortunately, despite our knowledge of the subject, it remains an invariably fatal illness with no current treatment or cure. The articles in this issue illustrate our current knowledge of the disease and its investigational treatments.

The veteran researcher, Paul Brown, commences the issue by giving a general overview of the disease and an historical account of its investigational treatments. Dr. Brown shares his wisdom on the evolution of our knowledge regarding the disease and gives useful insight into what can be expected in the future. In a pragmatic fashion, Dr. Brown also highlights the pitfalls of developing investigational treatments and proposes important points for prion disease researchers to ponder when developing treatment strategies.

Surachai Supattapone and colleagues present a review on the use of complex polyamines for the treatment of prion diseases. Although the majority of investigational treatment approaches have targeted the conversion of the native prion protein (PrPc) to the pathological prion protein (PrPres), the authors suggest a different therapeutic model. Complex polyamines serve two rules in combating prion disease: 1) disaggregation of amyloid proteins and 2) clearance of prions from cells. This approach posits the possibility of treating active disease as opposed to slowing its propagation.

An article by Constance Riemer and colleagues examines prion diseases from an immunological perspective. Addressing the reactive astrogliosis that is characteristic of prion diseases, Reimer and colleagues focus on the possible inflammatory reactions caused by PrPres accumulation. They propose anti-inflammatory treatments that target pro-inflammatory cytokines in the hope of delaying disease progression by mitigating the toxic effects of PrPres accumulation.
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Document Type: Research Article

Publication date: 2009-11-01

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  • CNS & Neurological Disorders - Drug Targets aims to cover all the latest and outstanding developments on the medicinal chemistry, pharmacology, molecular biology, genomics and biochemistry of contemporary molecular targets involved in neurological and central nervous system (CNS) disorders e.g. disease specific proteins, receptors, enzymes, genes. Each issue of the journal will contain a series of timely in-depth reviews written by leaders in the field covering a range of current topics on drug targets involved in neurological and CNS disorders. As the discovery, identification, characterization and validation of novel human drug targets for neurological and CNS drug discovery continues to grow; this journal will be essential reading for all pharmaceutical scientists involved in drug discovery and development.
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