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Archive - Feb 9, 2010


Experimental Drug Targets Cognitive Defects in Huntington’s Disease

The experimental drug dimebon (latrepirdine), being developed for patients with Alzheimer's disease and long used as a hay fever treatment in the former Soviet Union, appears well tolerated and may improve cognition (thinking, learning, memory skills) among individuals with Huntington's disease, according to results of an early-stage clinical trial. "This is the first clinical trial that has focused on what is perhaps the most disabling aspect of the disease," said University of Rochester Medical Center neurologist Dr. Karl Kieburtz, the lead author of the study. "While more investigation needs to be done, these results are encouraging and show, for the first time, a statistically significant benefit in terms of improved cognitive function in patients with Huntington's disease." The dominantly inherited genetic disease, which killed famous American folk singer Woody Guthrie, is a progressive neurodegenerative disorder that impacts movement, behavior, and cognition, and generally results in death within 20 years of the disease's onset. The disease steadily erodes a person's memory and his/her ability to think and learn. Over time, this cognitive impairment contributes to the loss of the ability to work and perform the activities of daily life. There are no treatments currently available that effectively alter the course of the disease or improve cognition. The only approved therapy for Huntington's disease, tetrabenazine, treats only motor symptoms and does not alter the course of the disease or prevent cognitive decline. Abnormalities in mitochondria have been implicated in the development of Huntington's disease and dimebon stabilizes and improves mitochondrial function.

Existing Drug May Be Useful in Treating 37 Million with River Blindness

Researchers at The Scripps Research Institute, together with colleagues, have reported that a drug (closantel) currently used as the standard treatment for sheep and cattle infected with liver fluke may be useful in treating river blindness, a tropical disease that is the world's second leading infectious cause of blindness for humans (the leading cause is trachoma, caused by infection with Chlamydia trachomatis). Dr. Kim Janda, an author of the new report, said that there is an urgency to fighting the infection that leads to river blindness, which is also known as onchocerciasis. Despite several eradication efforts, the disease affects more than 37 million people in Africa, Central and South America, and Yemen. "Victims of onchocerciasis suffer severe skin lesions, musculoskeletal pain, and various stages of blindness," said Dr. Janda, adding that patients also experience decreased body mass index, decreased work productivity, and social stigmatization. The new research shows that clostanel has the potential to inhibit the molting process of the parasite (Onchocerca volvulus) that causes river blindness. "We think this finding holds terrific potential for the treatment of river blindness, one of 13 recognized neglected tropical diseases," said Dr. Christian Gloeckner, the first author of the study. River blindness is caused by thread-like filarial nematode worms, O. volvulus, which are transmitted among humans through the bite of a black fly. The worms then multiply and spread throughout the body. When the worms die, they cause a strong immune system response that can destroy surrounding tissue, including that of the eye. Currently, the only drug available for mass treatment of river blindness is ivermectin, and it now appears that resistance to that drug is emerging.