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Packard Center for ALS Research at Johns Hopkins

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ALS Alert Newsletter | February

Packard Scientists Share Research Results

P2ALS meeting in New York City brings together leading ALS researchers.

P2ALS researchers, including Packard scientists, shared their latest research in a February meeting in New York City.

On February 9 and 10, ALS researchers from P2ALS and several different pharmaceutical companies gathered in New York as part of the semi-annual meetings of P2ALS. P2ALS was founded in 2010 as a joint effort between The Robert Packard Center for ALS Research and Project A.L.S. with the goal of finding new treatments for ALS. This collaboration established a highly cooperative network of scientists that share unpublished findings to facilitate rapid testing and verification of complex research questions posed by ALS. Initially, P2ALS focused on genetics, and motor neuron and glial biology-- basic science that allowed the identification of pathogenic pathways in ALS and potential therapeutic targets.

Now that this collaboration is beginning its third year, P2ALS has begun to aggressively shift focus, moving from identifying potential targets to validating and verifying these targets, with an eye on preclinical development. To this end, P2ALS is actively engaging the pharmaceutical industry to facilitate development of novel therapies for ALS.

At this most recent meeting held in New York City, the headquarters of Project A.L.S., scientists from around the world shared their most recent research, which covered advances in motor neuron and astrocyte biology, the genetics of familial and sporadic ALS, including new avenues of study opened up by the discovery of the C9ORF72 gene, and recent progress in the use of stem cells in the study and treatment of ALS.

After the researchers presented their results, they answered questions and addressed concerns from other scientists in the room. This allowed the presenters to consider new experiments and optimize their study design.

Although the results presented at this meeting cannot be shared with the public, the audience enthusiastically received both the cutting edge science presented and the ensuing discussions. Several representatives from the pharmaceutical industry presented or moderated sessions at the meeting.

John Gerdes with Piera Pasinelli
John Gerdes, a Packard and a P2ALS researcher from University of Montana confers with Packard science director Piera Pasinelli.

This meeting created a platform for the P2ALS scientists to actively engage and forge collaborative projects with drug discovery professionals from the pharmaceutical industry. Several new collaborative projects are under discussion as a result of the meeting, with follow-up discussions to ensue over the coming weeks. All pharmaceutical companies represented at the meeting were enthusiastic in their willingness to engage with P2ALS, as well as look for ways to begin or continue working with P2ALS to help the consortium continue to fight its battle against ALS.

“This has been an intensive and highly productive open collaboration among the 18 participating laboratories. Through a lot of the initial research in the past two years, P2ALS has identified promising targets for therapy,” said Piera Pasinelli, Science Director at the Packard Center. “What we need to do now is to use the same team-based focused approach to work together with pharmas to rapidly validate and develop new drugs.”

"The enthusiastic response from the pharmaceutical representatives to foster new collaborations and the stated intent to continue engagement with the consortium speak to the success of the meeting," said Manish Raisinghani, P2ALS Scientific Alliance Manager.

Many Packard scientists presented their results at the P2ALS meeting, including Jeff Rothstein, Director of the Packard Center and of the Johns Hopkins Brain Science Institute, Nicholas Maragakis, a neuroscientist at the Johns Hopkins University School of Medicine, and Bryan Traynor, a researcher at the National Institutes of Health in Bethesda, Maryland.

“It was nice to see how the model of open data sharing and collaboration came to fruition in this past meeting, catching the interest of pharmaceutical groups as well as others in the ALS community,” said Pasinelli. The next P2ALS meeting will be held in Baltimore in September 2012.

Carrie Arnold

Join the Fiesta!

The 2012 Fiesta 5K is filling up fast! Start your team today. The Fiesta 5K is the signature event of the Robert Packard Center and all funds raised at the event will benefit Packard’s research efforts.

Signing up is easy. Visit You can start a team, join a team, register to run on your own or volunteer! Don’t miss the fun!

May 5, 2012 at Power Plant Live in downtown Baltimore!

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Join the Fiesta!
The 2012 Fiesta 5K is filling up fast! Start your team today. The Fiesta 5K is the signature event of the Robert Packard Center and all funds raised at the event will benefit Packard’s research efforts.

The hope is in the science.
Please consider a tax-deductible donation to the Robert Packard Center for ALS Research at Johns Hopkins. Your donation will help further our scientific research.


Johns Hopkins School of Medicine

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