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The Robert Packard Center for ALS Research at Johns Hopkins

Since its founding in 2000, the Robert Packard Center for ALS Research at Johns Hopkins has been at the forefront of amyotrophic lateral sclerosis (ALS) research. As the first international scientific operation dedicated solely to ending ALS, Packard brings researchers from across the globe together to push the limits of scientific knowledge and uncover new approaches to the treatment of ALS.

Funding and support from MDA's Wings Over Wall Street immediately impacts Packard scientists and accelerates the pace at which they can conduct research. This year, the Packard Center is preparing to celebrate its twentieth anniversary. We are proud and incredibly grateful to have worked in partnership with Wings throughout that time. Over the past 20 years Packard Center investigators have contributed to the discovery of all ten of the major disease pathways currently recognized to underline ALS including nine genes associated with the disease. Of the two drugs approved by the FDA to treat ALS, one resulted directly from the work of a Packard investigator. Together with Wings, Packard will not stop until we have an effective treatment that halts disease progression or prevents ALS onset altogether.

Research by numbers; highlights from 2000-2020

•             168 projects funded

•             60 new animal models created

•             9 new genes discovered

•             9 disease mechanisms identified

•             1 FDA approved drug

•             3 drugs in Phase 1 trial

•             3 drugs in the pipeline

From research to practice: The story of C9orf72

Discovered by Packard investigator Bryan Traynor in 2011, mutations in C9orf72 are the most common associated with familial ALS and frontotemporal dementia (FTD). In 2014, just three years after its discovery, a gene therapy (anti-sense oligonucleotide; ASO) for C9orf72 ALS was developed and showed promise in early studies performed by researchers, including Packard investigators Chris Donnelly and Jeff Rothstein. In January of this year, Ionis Pharmaceuticals and Biogen partnered on a phase 1 clinical trial with the ASO BIIB078. Progress of this type is incredibly encouraging and we could tell a similar story for other heritable forms of ALS including SOD1 (positive results were reported by Ionis Pharmaceuticals and Biogen following completion of a Phase 1 clinical trial last December). However, we still do not know what causes the majority of “sporadic” ALS cases.

Beyond 2020: taking ALS to the end

As Packard looks towards the future, our research priorities are clear. While we will continue to promote advancements in the study of familial ALS, we must also seek out and facilitate research projects that increase our understanding of sporadic ALS and our ability to design effective therapies.

Availing of recent developments in both biology and technology, we now have reliable methods to identify and compare clinical and molecular signatures from individual ALS patients. This advancement is particularly important for the study of sporadic ALS. Through the Packard Center’s Answer ALS Research Program, scientists and clinicians are working to define the molecular changes that drive ALS. Comprised of more than 20 clinical, research and technology centers, and with the participation of over 1000 ALS patients, the program is generating the most comprehensive collection of ALS patient data ever conceived. This data will serve as a foundation for clinical trials, help researchers and physicians categorize patients to administer better treatment options (personalized medicine), and allow scientists to define subtype biomarkers so that people can be diagnosed earlier and treatments can be monitored more effectively.

Research projects funded by MDA's Wings Over Wall Street®

2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 <--- click on year to view .pdf file.