MS Research Roundup: 27 February 2016
FDA Fast-Tracks PPMS Drug Review; Molecular Target Runs Out of Air; Happy Accidents in the Lab; New Twist on Patient-Powered Research
MS Research Roundup collects items of interest to multiple sclerosis researchers from around the Web. Send us your tips: email@example.com.
FDA Fast-Tracks Review of Potential Progressive MS Drug
The first-ever drug to show any effect in a large clinical trial of progressive MS has just received “breakthrough therapy” status from the U.S. Food and Drug Administration. The designation means the FDA will make every attempt to speed the review process of approving the experimental drug, ocrelizumab (Ocrevus, Genentech/Roche), for primary progressive MS (PPMS), a chronic disabling form of the disease that does not begin with relapses. In most people, MS begins with the inflammatory relapsing-remitting form (RRMS) and later moves into the chronic disabling neurodegenerative stage known as secondary progressive (SPMS). “The catch, experts say, is that people with this form of the disease should keep their expectations low, because even the new drug that’s now in the last stages of development doesn’t appear to have a big clinical impact,” journalist David Nather wrote in STAT. —CCM (Genentech/Roche, Reuters, STAT)
A Case Not Yet Proven
Sometimes, researchers come across a protein that seems like the perfect candidate for a drug target for MS. Such was the case for hypoxia-inducible factor-1α, or HIF-1α, a protein that controls multiple genes related to neuroinflammation. But Katerina Akassoglou, PhD, at the University of California, San Francisco, has shown that HIF-1α probably does not play as major a role in MS pathogenesis as researchers had thought. Evidence suggested that HIF-1α might contribute to inflammatory demyelination: in the early stages of MS, patients display elevated levels of HIF-1α and hypoxia-like tissue changes like those caused by HIF-1α activity. To investigate the role of HIF-1α, Akassoglou used the experimental autoimmune encephalomyelitis (EAE) mouse model of MS. Although the mice had high levels of HIF-1α in astrocytes and myeloid cells, boosting or depleting HIF-1α from those cells had no effect on disease progression. Other work suggests that the protein’s role may be highly cell-type specific. The negative results were published last year in eNeuro, an open-access journal published by the Society for Neuroscience (SfN). Akassoglou summarized her findings in a late December post on Neuronline, SfN’s members-only discussion forum. —SS (Neuronline)
And the Winner Is …
It’s not as famous as the Oscars, but the Golden Mole Award for Accidental Brilliance may be a new MSDF favorite. The contest is sponsored by National Public Radio. We’re rooting for #6 on the short list of 12 finalists (of 300 entries). As summed up by Adam Cole of NPR, “Melissa Brown was at Northwestern University studying mice with multiple sclerosis. Like most MS researchers, Brown exclusively used female mice — the males just don't get sick. She was embarking on a set of experiments using mice with a genetic mutation that hobbles their immune system. Normal female mice get very sick. Female mice with the mutation seem to do better. Brown wanted to find out why. But when the results came in, some of them were the opposite of what was expected. Some of the normal mice were healthy. Some of the mice with the mutation were getting sick. A closer inspection of the test subjects revealed why. The mice with the surprising results were male! A graduate student on the project, Margaret Caulfield, was new to the lab and hadn't yet learned to identify the nearly imperceptible genitals of male mouse pups. The mix-up turned out to be a lucky one. It helped Brown, Caulfield and graduate student Abby Russi pinpoint a special group of cells that protect male mice from MS — innate lymphoid cells. The discovery opens new doors for MS researchers.” —CCM (NPR’s science desk, Skunk Bear)
Patient-Turned-Scientist Seeks Cure
Here’s a new twist on patient-powered research. “Lots of patients have become advocates to raise money to research a disease that affects their family. And some researchers have redirected their expertise to help loved ones who fall ill. But [Sonia] Vallabh’s and [Eric] Minikel’s story is quite unusual: Both traded other fields for careers in science.” So writes Karen Weintraub in a story for STAT, a new life sciences publication from the publisher of the Boston Globe. A rare prion brain disease killed Vallabh’s mother at age 51, and she carries the same risky genetic mutation. After quitting their jobs and switching careers, the husband-and-wife team are now both PhD students working in the same lab at the Broad Institute Cambridge, Massachusetts. They just published a new paper they hope will lead to greater understanding and treatment of prion disease. Co-authors include a large bioinformatics team to analyze vast amounts of shared data, from the Exome Aggregation Consortium and the 23andMe database. —CCM (New Yorker, Science Translational Medicine, STAT)
Edited by Linda Felaco.