MS Research Roundup: October 22, 2014
A Better CRISPR Mouse; Number Needed to Treat; Ann Romney to Raise $50M for Neuroscience Research
MS Research Roundup collects items of interest to multiple sclerosis researchers from around the Web. Send us your tips: email@example.com.
Better CRISPR Mouse
A powerful new way to edit DNA known as CRISPR just got a little easier. “Researchers at the Broad Institute of MIT and Harvard have created a line of mice whose cells naturally express the Cas9 protein used in CRISPR gene editing,” Bio-IT World staff summed up. “Until now, researchers using CRISPR have created complexes that include both Cas9 and guide RNA, delivering these to cells in order to switch off or modify specific genes.” The mouse was created in collaboration with CRISPR pioneer Feng Zhang, Ph.D., at the McGovern Institute for Brain Research at MIT. Details about the new mouse appeared September 25 in the journal Cell. Not only could the researchers more easily perturb multiple genes in vivo, but they could also experiment with immune dendritic cells ex vivo in the few days they live after they are removed from the mouse, noted co-author Aviv Regev, Ph.D., of the Broad and MIT. Researchers at more than one dozen institutions are already using the new mouse. Two versions (here and here) have been deposited at the Jackson Laboratory. For more on how neuroscientists are using CRISPR technology, check out the recent two-part series by writer Jessica Shugart at Alzforum on this minirevolution. (Alzforum, Broad Institute, Bio-IT World, Genetic Engineering and Biotechnology News)
Number Needed to Treat
What is the likelihood that any given drug can help an individual patient? The results of many clinical trials—and of more rigorous systematic reviews of multiple trials—are described in terms of relative risk, such as the astonishing findings that daclizumab reduced the annualized relapse rate by nearly half compared to interferon, as presented at MSBoston2014. The unpublished phase 3 results suggest daclizumab may be as effective as the best MS drugs now approved but with lower risk. But how can that be translated from a study population to a person? Evidence-based healthcare experts have advocated using “number needed to treat” (NNT), which tells how many people need to be treated to achieve the therapeutic outcome. In this example, MSDF calculated, nearly six people would need to be treated with daclizumab to reduce the relapse rate in one person over 2 years. A site, TheNNT.com, aims to push the NNT and its companion, number needed to harm (NNH), into more common use with patients and their doctors. The 200 write-ups do not include MS, but NNT is a simple calculation: 1/absolute risk reduction. (University of Oxford Centre for Evidence-Based Medicine, Wired) (Tip from Ben Goldacre)
Scientists know well how researching one disease can lead to unexpected insights into another. That’s the idea behind the new Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital in Boston. The magic number is 50. Romney is heading a campaign to raise $50 million to help more than 50 million people by expanding and redesigning a neuroscience program to “accelerate treatment, prevention and cures for five of the world’s most complex neurologic diseases, including multiple sclerosis (MS), Alzheimer’s disease, Lou Gehrig's disease (ALS), Parkinson’s disease and brain tumors.” The plan calls for hiring 50 more scientists. Romney, who was diagnosed with MS in 1998, credits her doctor and center co-director Howard Weiner, M.D., with both her good health at age 65 and her fascination with the crossover research approach. In one example, researchers in the labs of Weiner and co-director Dennis Selkoe, M.D., took an antibody developed to monitor the effectiveness of MS treatment and found it could knock down regulatory T cells to help the immune system fight a brain tumor. The center will be overseen by a bipartisan board of political powerbrokers with an interest in MS and neuroscience, including husband Mitt Romney, former Massachusetts governor and the 2012 Republican presidential candidate. (Boston Globe, Brigham and Women’s Hospital, Los Angeles Times, Mediaite, NBCNews, WHDH TV 7NBC) (Tip from Carey Goldberg and Gabrielle Strobel)