A View from the Cutting Edge: News from Our 10th ALS Scientific SymposiumWhen our scientists gathered this year, everybody went back home with an anniversary present: The foundation for discovery is snapping into place. 
It was a gift. This year’s Packard Center annual symposium at the end of March was pretty much exactly what Center founders had hoped they’d see—short of a cure—when they set up the organization 10 years ago. Some 150 Packard scientists and collaborators—heads of academic institutes and laboratories, their postdocs, industry researchers and clinicians—gathered from the U.S., Canada and Europe to report on this year’s grant-supported ALS research. All in all, their work marks a clear move closer to the disease’s cause and, we believe, nearer to therapy. As at last year’s event, two days of the Baltimore meetings were for reporting Packard’s basic ALS science, while a third day featured updates on ALS clinical trials by scientists with the Packard-friendly Northeast ALS Consortium (NEALS). Set Up to Share
The presentations stand on sound science—more to come on that. But what likely set this meeting apart from others on ALS is sharing—and the extent of it. Packard investigators must share their data before publishing. Not having to wait until something’s in print to discuss it should accelerate progress. And it does just that. “This meeting captured the group’s best elements—open sharing, collaboration and progress,” Packard Director Jeff Rothstein wrote afterward. “I cannot think of any ALS meeting I’ve attended, Packard or otherwise, where people shared so much new, unpublished data.” “I must agree,” says Science Director Piera Pasinelli. “It truly represented the open spirit that we prize.” Click here for a quick tally of this year’s more concrete basic ALS research results. Look for an update on clinical trials in the June ALSAlert. From the BasicsSo what sparked the most comment in the symposium’s first two days? In short: genes and the animal models built from them, uncovering new molecular pathways, new stem cell uses and new therapeutic approaches. The growing number of reports of the significance of two new ALS genes, TDP-43 and FUS, translated this year into a host of Packard animal models. Models are crucial because TDP-43 and FUS offer striking alternatives to SOD1, the single, familial ALS gene that’s been some 15 years under study—and one where the path from gene to disease still isn’t clear. What’s exciting is that these new genes may be easier to track. Their toxicity likely comes from a more basic process, one that may occur earlier on in cells, rather than farther down in the biological path. That, the researchers hope, could not only expose a target but find one that trips ALS earlier in its course. Or it could make heightening a cell’s natural protective tactics more effective. Packard scientists also heard early results of a new, ultra high-speed way to screen for yet other ALS genes—both familial and those that increase sporadic risk. At least three new pathways with possible therapeutic targets were reported. One path, for example, contains a molecule that’s central to the process where nerves uncouple from muscle in ALS. Stem Cells Still HotExploring stem cells as therapy for ALS or as a source of regenerating the injured nervous system is still very much a focus of Packard scientists’ research. But for that work to take off, questions about the nature of stem cells and their behavior in the human nervous system need answers. It’s no surprise, then, that Packard researchers are using stem cells as tools as well as a possible therapy. (Check stem cells to see more studies.) A Last WordIt’s a common drawback with orphan diseases, and one reason ALS research hasn’t gone faster is because there hasn’t been the funding/interest to define the disease, to find out exactly what changes and when at a basic cellular and molecular level. Moreover, until recently, critical tools to do that didn’t exist. Center advisors, however, have always recognized those needs, and research grants for tools and pathology studies have never taken a back seat. In this symposium the value of that is clear. “We were all impressed with the amount of new data and the accomplishments,” says Packard Scientific Director, Piera Pasinelli. “It was a fine year.” ---
Check next month’s ALSAlert for our update from the clinical trials portion of this year’s symposium. | 
