{"id":127,"date":"2007-07-01T14:52:30","date_gmt":"2007-07-01T14:52:30","guid":{"rendered":"https:\/\/blogs.nature.com\/actionpotential\/2007\/07\/clinical-trials-and-tribulations.html"},"modified":"2007-07-01T14:52:30","modified_gmt":"2007-07-01T14:52:30","slug":"clinical_trials_and_tribulatio","status":"publish","type":"post","link":"https:\/\/blogs.nature.com\/actionpotential\/2007\/07\/clinical_trials_and_tribulatio.html","title":{"rendered":"Clinical trials and tribulations"},"content":{"rendered":"

It’s double-blind or nothing when it comes to phase III<\/span> clinical trials. Although placebo groups are absolutely vital to the clinical validity of medical treatments, a recent article in The Lancet<\/em> has me thinking about the ethics of treating desperate patients with saline.<\/p>\n

The subthalamic nucleus is overactive in people with Parkinson’s disease, presumably because it loses GABAergic input from the globus pallidus. Subthalamic nucleus lesions improve Parkinson’s disease symptoms. Kaplitt et al.<\/em><\/a> generated a gene therapy agent that would silence but not destroy subthalamic nuclei neurons. They inserted glutamic acid decarboxylase (GAD<\/span>), the enzyme responsible for GABA<\/span> production, into a viral vector and injected it into subthalamic nuclei of people with Parkinson’s disease. In their phase I trial in 12 patients, the authors showed that their therapy was safe and virtually side-effect-free. Unlike most phase I trials, the authors also showed that their treatment was effective: intra-thalamic injections of the GAD<\/span> agent reduced Parkinson’s symptoms.<\/p>\n

That’s great news for a possible new treatment for Parkinson’s disease. But it also means that at some point soon, people with advanced Parkinson’s symptoms will volunteer for brain surgery and a 50% chance at treatment. Some would argue that even placebos have their upside<\/a>, and I certainly understand their importance. I also understand that the patients who volunteer for these studies are often at the end of their medical ropes and are willing to roll the dice. I just feel for the folks who wake up 6 months after surgery and realize that their symptoms haven’t improved.<\/p>\n","protected":false},"excerpt":{"rendered":"

It\u2019s double-blind or nothing when it comes to phase III clinical trials. Although placebo groups are absolutely vital to the clinical validity of medical treatments, a recent article in The Lancet has me thinking about the ethics of treating desperate patients with saline.  Read more<\/a> Continue reading →<\/span><\/a><\/p>\n","protected":false},"author":88,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-127","post","type-post","status-publish","format-standard","hentry","category-uncategorized"],"_links":{"self":[{"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/posts\/127","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/users\/88"}],"replies":[{"embeddable":true,"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/comments?post=127"}],"version-history":[{"count":0,"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/posts\/127\/revisions"}],"wp:attachment":[{"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/media?parent=127"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/categories?post=127"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/blogs.nature.com\/actionpotential\/wp-json\/wp\/v2\/tags?post=127"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}