Patients thrill to reports of a promising antisense drug against Huntington disease, but no one is sure yet whether it works. Dardengo's father, shown holding her son Joel in a 1989 photo, died of Huntington disease. Michelle Dardengo walks her dog near her home in Coquitlam, Canada. The dark shadow of Huntington disease fell squarely over Michelle Dardengo's life on the day in 1986 that her 52-year-old father was found floating in the river in Tahsis, the remote Vancouver Island mill town where she grew up. Richard Varney had left his wedding ring, watch, and wallet on the bathroom counter; ridden his bike to a bridge that spans the rocky river; and jumped. The 4.5-meter drop broke his pelvis. The town doctor happened to be fishing below and pulled Varney out as he floated downstream, saving his life. The once funny man who read the Encyclopedia Britannica for pleasure; the good dancer who loved ABBA, the Three Tenors, and AC/DC; the affable volunteer firefighter--that man was disappearing. He was being replaced by an erratic, raging misanthrope wedded to 40-ounce bottles of Bacardi whose legs would not stay still when he reclined in his La-Z-Boy.
Neurological disorders affect millions of patients worldwide. The inaccessibility of the brain to physical examination and the complexity of clinical evaluation for such conditions represent major challenges. Subjective symptoms such as chronic pain, for example, can be difficult to confirm, while cognitive impairment can result from a range of different pathologies. In the absence of clear biological markers indicating a particular pathology, it can be difficult to provide a rapid, definitive diagnosis. These challenges are particularly pertinent in neurodegenerative diseases, as few effective treatments for slowing or stopping such conditions are currently available.
WASHINGTON – A single injection of a fragment of a life-extending protein hormone could improve cognition in those with neurodegenerative illnesses, according to new research published Tuesday. The klotho protein was found to enhance cognitive and physical performance in aging or impaired mice, said a study carried out by scientists at the University of California, San Francisco and published in the journal Cell Reports. "With our new aging demographic, cognitive dysfunction and lack of mobility are now emerging as our biggest biomedical challenges, and there are no truly effective medical therapies for these debilitating problems," said lead author Dena Dubal. "Our findings suggest that treatment with a klotho fragment enhances brain function across the lifespan and could represent a new therapeutic strategy to boost brain resilience against neurodegenerative diseases like Alzheimer's and Parkinson's disease." But researchers said clinical studies would be necessary to determine the safety and effectiveness of injecting klotho in humans.