P2ALS is a product of the scientific times, say Packard members Nicholas Maragakis and Jeff Rothstein. Just-out technology has let it happen.
P2ALS: Pooling Ammunition in the ALS WarA new $15 million effort pulls together intellectual fire power from top U.S. labs for high-yield ALS research. The focus is tight. The dedication is sure. Last month, a new partnership among some of the best U.S. scientific minds in the field shifted the status quo in ALS research. Some $15 million in seed money from two anonymous donors came with a requirement for clear milestones and deadlines—an industry-like approach to investigation—and launched P2ALS. The enterprise is run by two “P” organizations, the Packard Center and Project A.L.S., who are pooling key researchers for the first time. “It’s an unprecedented opportunity for synergy in ALS research,” says Packard Director Jeff Rothstein. Project A.L.S. Research Director, Valerie Estess likens it to rival companies working together. “The project,” says Estess, “will fuel the field with strategies and tools for treating the disease.” Both research-based organizations will stay separate entities. But they’ll join forces where overlap exists in work they sponsor. They’ll also collaborate where one organization’s scientists have expertise or tools so advanced that sharing truly raises the potential for a jump in therapy-based research. P2ALS’s planners anticipate a ripple effect will advance ALS science and efforts toward therapy worldwide. Why does this come now? Part of the reason is a breakthrough in stem cell technology to produce iPS cells—for induced pluripotent stem cells. Project A.L.S. scientists are actively molding iPS cells into tools to study Lou Gehrig’s disease. Unlike other stem cells, these can be fashioned from adult human skin cells. And that work will dovetail, for example, with Packard Center studies already exploring stem cell implants as a way to slow the disease. Also, because iPS cells can likely be programmed to become any kind of cell touched by ALS, there’s a real possibility of having the first true human models of all or selected parts of the disease. The timing is also right because of a new shift in ALS pathology studies. A host of recent studies—many from Packard scientists—show that ALS distorts normal biochemical conversations between motor neurons and the neighboring glial cells in the spinal cord. New studies are revealing the importance of this sort of “cross talk” for healthy spinal cords. A better understanding could open an entirely new direction for therapy. And last, two surges have come in ALS genetics studies, largely from Project A.L.S. and Packard scientists. One is in finding new genes for ALS. That’s matched by an equal and timely advance in being able to silence genes effectively. P2ALS, then, concentrates its energy on three broad aims: 1) understand glial and motor neuron signaling 2) uncover genes that cause or increase the risk of ALS while we advance gene silencing therapy and 3) use the most advanced stem cell techniques to develop human-based models that will speed therapy. The superscript in the P2 part of this new partnership isn’t just for style. By pooling the science, we really are multiplying our efforts to cure ALS. | 
