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The Sherer Report

Posted by  Todd Sherer, PhD, April 02, 2013

The Sherer Report

This Parkinsonís Awareness Month, The Michael J. Fox Foundation (MJFF) recognizes the vast progress weíve made in Parkinsonís research in the 12 years since our inception ó and thanks you for being an instrumental part of our work.

Today we have moved tangibly closer to real results for patients. And I am optimistic that the momentum weíve built over the past year in particular will continue to propel us forward to the goal we all want to reach ó new treatments, and a cure for Parkinsonís.

In 2000, this was not the case. The field was at a crossroads: Scientists had a limited understanding of the biology behind Parkinsonís disease (PD); symptomatic treatments almost exclusively focused on dopamine-based drugs; and no centralized force existed to coordinate PD drug development in a field that was quickly losing interest.

But while weíve made a lot of progress, in many ways weíre just now getting to the hardest part. The good news is, with your help, weíre ready to tackle new challenges head-on.

Here are a few examples of how far weíve come, and what lies ahead: Researchers continued to make significant strides in 2012 toward better understanding the biological processes at work in PD ó essential knowledge to developing treatments that could slow, or even stop, the disease from progressing in patientsí brains and bodies. Last fall, we had the opportunity to convene a workshop to address a budding hypothesis in the field ó that the protein alpha-synuclein, which behaves abnormally in the brains of all people with PD, spreads from cell to cell. This evidence is providing us with new insight into the onset of Parkinsonís disease, and its progression. In fact, the first-ever alpha-synuclein-based drug candidate, which entered clinical testing last year, works by addressing this cell-to-cell transmission of alpha-synuclein. The research is sponsored by Austrian biotech AFFiRiS with MJFF funding, and results are expected by year-end 2013.

More and more scientists believe that better Parkinsonís treatments will bypass the traditional focus on† the dopamine system. Recent clinical trial results show that the pipeline for novel, non-dopamine-based therapies to treat the symptoms of PD, and dyskinesia, is strong. Novel drug candidates targeting brain chemicals such as glutamate, serotonin and adenosine all yielded encouraging returns in the clinic. But the road ahead is marked by substantial hurdles, as pharmaceutical companies such as Addex Therapeutics work to push their drug candidates through stringent testing that will cost millions of dollars. Follow-on funding, even when results are good, is never a sure thing.

The good news is, I can say with confidence that in my 15 years in PD research, Iíve never before seen the kind of enthusiasm from industry to delve into the Parkinsonís space that I see today. Our Foundation is working urgently to bring together all the key players to make sure novel ideas get traction, and then lay out the next steps to get these ideas into the clinic.

In 2012, we entered into a first-of-its-kind collaboration with Sanofi to develop an existing Alzheimerís drug candidate for Parkinsonís-related cognitive decline. Iím happy to report that a clinical study launched in early 2013. Also last fall, Vanderbilt University partnered with Bristol-Myers Squibb to develop a glutamate-based drug that represents an entirely new class of treatment for the symptoms of Parkinsonís disease. These kinds of relationships, merging scientific innovation with industry resources, will ultimately be critical to bring new treatments to market.

But as you know, drug development is expensive and risky. Decades are invested, and billions of dollars spent, on treatments that all too often languish in ďthe Valley of DeathĒ ó that resource and expertise gap where an idea born in a university or biotech loses steam, and fails to secure the necessary support to move forward toward the clinic. None of the successes Iíve discussed in this report are immune to this challenge. Ongoing progress depends on strategic investment and collaboration, certainly among scientists, but especially from the Parkinsonís community.

Here, too, I see good news: The PD communityís desire to do their part and get involved is greater than ever. For instance, through the growing Team Fox network thousands are coming together to forge new bonds and take action locally. Thousands more are speeding research by completing profiles on Fox Trial Finder (foxtrialfinder.org), where they are matched with the clinical trials that need them, and which will be expanding into Western Europe this year. As you read on our blog, thanks to hundreds of volunteers around the globe, we are close to meeting the original recruitment goal for PPMI.* Additional opportunities exist to be a part of this landmark biomarkers study through the newly launched pre-motor cohort of PPMI, which is searching for individuals who do not have Parkinsonís but do have smell deficits, a sleep disorder called RBD, or a mutation in a gene called LRRK2. People who have lived with the disease for several years are also taking part in critical research to identify new biomarkers through BioFIND, a discovery effort that launched last fall.

Whether you contribute by participating in research, attending a Team Fox event in your community, or making a gift to the Foundation, you are invaluable to our progress. We count on you for so much, and Iím truly grateful for your dedication. The answer is in all of us, working together. This Parkinsonís Awareness Month and all year long, thank you for all you do.

Share with Todd what you think about these reports, and what you'd like to see in future editions. Email theshererreport@michaeljfox.org.†

*At press time (March 15, 2013), PPMI had not yet completed recruitment.

This first appeared in the Spring 2013 issue of The Fox Focus on Parkinson's. Read the full issue.

TAGS: Parkinson's Progression Markers Initiative, Alpha-Synuclein, Newsletters, The Sherer Report, Team Fox, LRRK2, Fox Trial Finder, Foundation News, Todd Sherer






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