MS Research Roundup: April 18, 2014
Progress Toward New MS Clinical Trial Outcomes; Adaptive Design Evolves; A Call for Fundamental Neuroscience Research; MS Miracle Cures
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Seeking Clinically Meaningful Outcomes
It’s hard to test new therapies to slow or reverse MS if you can’t measure how well they work. An effort to develop more sensitive and reliable measures of disability reports progress . The MS Outcome Assessments Consortium (MSOAC) has gathered existing clinical trial data from 5000 people with MS who had been enrolled in industry and academic studies, with an additional 11,000 in progress. They have also developed new standards to help compare the data across studies. They will be analyzing the data from completed clinical trials to learn how well the MS Functional Composite and other disability measures work and if new measures are needed to predict clinically meaningful outcomes to the satisfaction of regulatory authorities, such as patient-reported outcomes. Eventually, the potential new outcomes tool will be made freely available. MSOAC is a multiple-stakeholder consortium that involves the U.S. Food and Drug Administration under its Critical Path Initiative, which aims to speed development and reduce cost of new therapies. (National MS Society)
Adaptive Design for Best Treatment Dose
Researchers are scrambling for biomarkers that can reliably predict clinical outcomes and can enable smaller and shorter clinical trials for promising therapeutics, especially in progressive MS. Another way to improve clinical trials is using adaptive designs to identify doses and to stratify patients who are sensitive to the treatment effects, such as in individualized cancer therapies. “Across the drug industry, around 20% of clinical trials now involve some element of adaptive design, in which researchers make preplanned adjustments to protocols at interim checkpoints,” Nature Medicine reports in a news story about a new consortium that is developing a software platform for the design, planning, and analysis of dose-finding trials. “This announcement is a major step forward,” said Ken Getz of the Tufts Center for the Study of Drug Development in Boston. (Nature Medicine)
Worrisome Drop in Basic Brain Research
The engine of discovery in science seems to be running on fumes, Science magazine reported in a news story. The concern comes from a funding analysis by the National Institute of Neurological Disorders and Stroke (NINDS). Between 1997 and 2012, applied research dollars rose (from 13% to 29% of total budget), while basic research dropped (from 87% to 71%), reported NINDS Director Story Landis in what appears to be her inaugural blog. “When we divided basic research into basic/basic and basic/disease-focused subcategories, we observed a striking decline in the funding of basic/basic research, which decreased from 52% to 27%,” Landis wrote. Basic/disease-focused has been rising slightly. Landis and her colleagues also found a decrease in basic/basic applications, despite evidence that they do as well as or better than others in peer review. (Science magazine, NINDS Blogs)
Hope Can Kill When It Comes to MS Miracle Cures
“Faced with the uncertainty of a condition that can manifest itself in many different ways, progress at variable speeds, and have multiple unpredictable endpoints, it’s not surprising that we are so prone to grasp at any suggestion of a cure, however improbable it may sound, and however scant the evidence,” wrote guest columnist Margo Milne in The Guardian’s Brain Flapping blog. Milne described herself as disabled by progressive MS. Using the example of experimental angioplasty to clear the blockage known as chronic cerebrospinal venous insufficiency, she compassionately cautioned against remortgaging the house for the latest treatment touted on the Internet before clinical trials show efficacy and safety. Judging by the comments section, not everyone was convinced that anecdotal stories of improvement could easily be the result of the normal ups and downs of MS. (The Guardian)
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