A meeting of minds: Overcoming roadblocks in the development of therapies for neurodegenerative disorders

Neurological and psychiatric disorders continue to intrigue and perplex the clinical and scientific world. Neurodegenerative conditions follow a relentless course, causing increasing disability as they progress, and ultimately, early demise. Despite intensive basic and clinical research, progress has been painfully slow, with not a single treatment developed to slow or limit the progression of any of the many neurodegenerative disorders. This includes some of the most common diseases such as Alzheimer's disease (AD) and Parkinson's disease (PD), as well as other equally debilitating conditions such as amyotrophic lateral sclerosis (ALS). So why has progress been so slow?There is no denying that neurodegenerative disorders are complex, but the discovery of several genes causing rare familial forms should have provided vital clues to jumpstart our understanding of neuronal dysfunction and disease pathogenesis, and hence to develop therapies. Each new discovery of a gene linked to neurodegenerative disease is hailed as a major breakthrough » Each new discovery of a gene linked to neurodegenerative disease is hailed as a major breakthrough. « and widely anticipated to lead to improved treatment. However, subsequent advances have generally been disappointingly slow. For example, mutations in genes such as the amyloid precursor protein, causing AD (Goate et al, 1991); Cu/Zn superoxide dismutase (SOD1) in ALS (Rosen et al, 1993); and Huntington in Huntington's disease (HD) (Huntington's disease collaborative research group, 1993) were uncovered more than 15 years ago. Since then, despite several thousands of research articles, these advances are yet to be translated into therapies of any proven benefit to patients.What is holding up progress? » What is holding up progress? «The reasons are multifold. Many of the disease‐causing genes so far unearthed are largely uncharacterized; their functions are obscure. The laboratories making these discoveries have often lacked the expertise to uncover the functions …