The Robert Packard Center for ALS Research at Johns Hopkins
Scientists with the Packard Center for ALS Research at Johns Hopkins continue to make vital discoveries about ALS—to shed light on its cause, to explain the course of the disease at a molecular level, and to find and study the genes that either cause it outright or make people susceptible to it. All this, of course, drives Packard’s single-minded goal: Find therapies to beat the disease.
Our scientists, for example, first explained one of the main reasons for cell death in ALS. That find formed the basis for a decade of drug discovery leading to Riluzole, the only FDA-approved drug for ALS.
Scientific work at Packard is not about throwing darts at a board, hoping for a bulls-eye, but rather about taking deeply thoughtful, tailored approaches based on the cumulative understanding of ALS from the world’s leading scientists. This past year, thanks to the generous funding from MDA’s Wings Over Wall Street®, more than 30 outstanding researchers collaborated in our performance-driven model.
In addition, the projects our Center scientists share with other organizations widen the chances of discovery and shorten the path to therapy. Our strong ties with P2ALS, for example, a tight group of top researchers from Packard and Project A.L.S., has advanced gene-silencing as a therapy for familial ALS. The collaboration is also on the verge of making a plentiful supply of human ALS cells a reality. Having actual human ALS cells or tissues to study has been a holy grail of ALS research. They can reveal errors in the true disease; they can be our closest models for drug-testing.
Research projects funded by MDA's Wings Over Wall Street®
2002 2003 2004 2005 2006 2007 2008 2009 <--- click on year to view .pdf file.
Q: What would you do with more money?
A: As you can see from this past year’s work, we’ve opened new, more focused paths for research. Why is there an outpouring of glial cells in ALS? What does TDP-43 do once it’s outside the cell’s nucleus that makes it so toxic? Can that molecule’s tie with aging explain why ALS is a disease of later life? Could we sidetrack that? Can the drug we have in mind fix sick mitochondria? What happens then?
Our collaborations turn out a constant supply of potential targets for therapy and ideas for the research to refine those targets. Funding, however, is the fuel. Almost every week, I’m asked to approve a new, and more often than not, exciting research collaboration with our scientists that might lead to new treatments or offer hope of a cure. It’s hard to turn them down.
We can’t stress enough that the Center’s worldwide collaborative science would be close to impossible without philanthropic support. Additional research dollars received from MDA’s Wings Over Wall Street® will allow us to fund the completely new or next-step studies that need to be done.
We deeply, genuinely thank you for your help.
Jeffrey D. Rothstein, M.D., Ph.D., Director, Packard Center for ALS Research at Johns Hopkins
Piera Pasinelli, PhD, Science Director, Packard Center for ALS Research at Johns Hopkins
