Righting the Clinical Research "Enterprise"

By Ronnie Todaro, M.P.H., Director of National Programs

When opening the Sunday Review section of the New York Times this past weekend,  I did a double-take as a topic of much discussion here at PDF was featured front and center - trumping commentary on the state of the economy, international relations and the latest political debate.

"Do Clinical Trials Work?," by Clifton Leaf serves as a primer on some of the key issues that prevail within the clinical research enterprise - the tension between scientific inquiry and patient priorities; the odds that study sponsors take when betting on the success of a potential therapy; and the disconnect between those who participate in a study and those who may actually benefit from the treatment being tested. It also serves as yet another wake-up call on why people who are living with a disease, be it cancer, Alzheimer's or Parkinson's, need to be formally included in research decision-making.

I can't help but wonder, would clinical research be in a different state if people who actually had the disease were sitting around the table, eye-to-eye with researchers, study sponsors and federal regulators? Would their presence and perspective change the priorities and processes of clinical research? Would it create the sense of urgency that occurs when the personal is married with the professional?

Of course, we here at PDF would  answer in the affirmative as would our 200 Research Advocates across the country.  The bottom line - we can right this "Enterprise" - but only if we recognize who should be at the helm - the patient.

The Challenges of Research Funding

From Eddie Pelto, Director of Development

Just a few weeks ago, the Parkinson’s Disease Foundation’s grants review committee – comprising scientists and patient advocates – took on the task of deciding which of the 200 research grant applications we received would be approved for funding. PDF had the resources to fund only 10 worthy projects.

Researchers working on innovative, viable projects continually face the challenge of limited available funding. And funders, such as government agencies and foundations like PDF, have to make difficult choices based on available resources.

How can PDF meet the challenge to find sufficient funds to support the most promising research in tough economic times?

One of the ways we can do this is with a different kind of challenge, one that helps everyone.  If you have recently visited PDF’s website, you know that during the month of April, Parkinson’s Awareness Month, we are raising funds to meet the Light of Day Foundation $100,000 Challenge.

Challenge grants are funds given to a non-profit organization upon completion of certain requirements, such as raising funds. The challenge refers to the results that must be achieved before money is released.

In PDF’s case, the Light of Day Foundation asks that we raise $100,000 from our supporters and if we do, they will match all donations we receive by Tuesday, April 30!

And the best part is that with a challenge like this, everybody wins.  You are not only doubling the impact of your gift to Parkinson’s research, you are helping to double the impact of the grant maker’s gift too!

We may not be able to fund all of the applications we receive. But every bit helps us in funding more promising research until we overcome the ultimate challenge: ending Parkinson’s.

Take a look at what Bob Benjamin, Founder of the Light of Day Foundation has to say about the challenge:

Genetic Testing and You

From James Beck, Ph.D., Director of Research Programs

The genetic testing company 23andMe recently announced that it had reached its goal of enrolling 10,000 people with Parkinson's into its genetic testing program. I personally think that is fabulous.

While genetic abnormalities that lead to Parkinson's disease are rare, finding these cases has been a boon to understanding PD for all. From the location and then discovery of the first PD gene by PDF’s first supported fellow, Roger Duvoisin, M.D., and his colleagues in 1996 to the more recent genetic discoveries of today, PDF steadfastly supports research into understanding how genetics and PD interact.

As we move forward, genetic testing is becoming more sophisticated and cheaper too as the cost drops faster than comparable advances made in computing technology. This is akin to buying the original IBM PC desktop one day and then next year being able to bring home the latest iPad. 

Low costs are making genetic testing more ubiquitous and that is causing some problems. For scientists, the problems are a bit academic—they are drowning in data. For the PD community, these problems hit closer to home. Genetics testing has the potential to bring forth knowledge that before was unknowable—the future. The question now is are we ready?

Deciding to voluntarily undergo genetic testing is a very personal decision; and, like many other endeavors, it is not always happy sailing. This is why, in response to the many questions and concerns PDF has received about genetic testing, PDF tasked our Medical Policy Committee to provide guidance for those interested in gene testing.  

Why does this matter? Let me tell you the story I heard about a person with PD who decided to buy a gene test kit  This person wanted to see if he had a genetic cause to his disease.  Not surprisingly, the answer was no; he did not have a mutation in the few PD genes that are examined on a consumer level. However, he got more than he bargained for and what he did find out was not good. In his DNA were two copies of the bad variant of the APOE gene. He now knew he was at a 10-fold increased risk of developing Alzheimer’s disease—information he did not want nor was he prepared to know.

Fortunately, most scientific studies involving gene testing do not reveal the results to those who participate. Providing a blood sample for PD research remains a very easy way for everyone to become participants in the research process, moving us all closer to finally tackling this disease. 

As it stands today, nearly 90 percent of the people with PD do not have a clearly attributable genetic cause to their disease. Since consumer testing only looks at a handful of the known PD genes, the odds of using a consumer test to unveil a personal PD-genetic link are rare. And for those who do, that knowledge will not affect their current medical care. The bottom line with genetic testing is to look before leaping. Take the time to understand what you are buying and deciding what that knowledge is worth to you.

• PDF Direct-to-Consumer Statement
• PDF Genetics PD Expert Briefing - "What's New in Genetics and PD?"

How Much Does Parkinson’s Disease Cost? New IHS Report Indicates Burden on US Families

This week, a report conducted by IHS-Global was published in the journal Movement Disorders, providing the most comprehensive, economic analysis to date of the direct and indirect costs of Parkinson’s disease to individuals and society in the United States.  (In full disclosure, it was underwritten by the Pharmaceutical Research and Manufacturers of America).

While certain premises within the report – most notably, the estimate of the overall prevalence of Parkinson’s in the United States, which is probably on the low side  – may be uncertain, most of the numbers are well thought through and carefully applied to the known data.

Implications for Research Funding
The Parkinson’s community – through a statement prepared by Amy Comstock Rick, CEO of the Washington-based Parkinson’s Action Network, and co-signed by the other leading Parkinson’s organizations, including the Parkinson’s Disease Foundation– has correctly noted that the main policy implication of the study is that we as a society should be prepared to invest more money on the “front end” of the process – that is, in research aimed at understanding, easing and ultimately eliminating Parkinson’s disease – to save the medical, maintenance and lost-earnings costs of the on the “back end.”

It is striking to reflect, as Amy’s statement makes clear, that the estimated total of research funds invested directly on Parkinson’s-disease related research is a mere one percent of the annual economic “burden” of the disease, as estimated by IHS.  It needs to be more.

A Key Issue: Nursing Home Care
The report also raises the issue of high cost of health care for people with Parkinson’s.  Since much of this is for areas that are not covered by most Medicare and most private insurance plans, the burden of these costs falls primarily on individuals with Parkinson’s and their families.  One of the most striking statistics in the IHS report is the burden of nursing home care, estimated by the authors as a whopping $4.6 billion dollars a year – almost three times the cost of hospital care for the same population. (It is worth noting that if study indeed underestimates how many people live with Parkinson’s in the US, these costs are in fact higher.) Nursing home care, like many other areas of care needed by people who live with a chronic disease like Parkinson’s, is generally poorly covered, and the literature abounds with stories of families who find themselves forced to go on Medicaid to take get support for a family member who is afflicted.  Simply put, a humane society needs to do a better job in providing for the needs of people afflicted by chronic disease.

We congratulate the authors on their contribution and hope it will enliven the national conversation about public support of research and care for Parkinson’s and other chronic diseases.

A Positive Spin: Creativity and PD

We all appreciate the headlines that help us understand PD symptoms and side effects. But we also find it refreshing to see last week's selection of stories covering ways to take charge of PD with creativity.

Victoria Tane featured in the 2013 Creativity & Parkinson's Calendar

The Science Behind the Spin
Last Thursday, we saw a review published on, "The Awakening of Artistic Creativity and Parkinson's" by Rivka Inzelberg, M.D., in Behavior Neuroscience.  Dr. Inzelberg looked at more than 10 studies investigating, "the ability to produce innovative aesthetic works" in people living with Parkinson's.  She found that:

• People with PD who are not already artistic, may became so when treated with certain PD drugs,­ namely dopamine agonists and levodopa. 
• Creativity could be used by occupational therapists as a method to help people with PD.
• The relationship between PD treatments and creativity may help to scientists to understand artistic creativity generally.

The People Behind the Project
In second headline, we saw a real life example of creativity and PD. PDF's own Creativity and Parkinson's Project artist, Victoria Tane, was profiled in a Union Leader article you can read here.  Victoria was diagnosed with PD in 2010.  As an artist and jewelry maker since the 1980s, she has found her creativity a positive way to cope with PD. Victoria's jewelry, "Bits and Pieces ­- Six Geometric Bracelets," was chosen as the featured work of art for the month of February, in PDF's annual wall calendar. The calendar is a part of PDF's Creativity & Parkinson's Project which exists to explore, support and encourage the therapeutic value of creativity in Parkinson's.

The One Day Vote
As part of our annual T-Shirt Design Contest, PDF unveiled our five finalist designs. All were created by people living with Parkinson's or loved ones. The winning design will be featured on a t-shirt worn by thousands during April, Parkinson's Awareness Month.

What Next?
If these stories resonate with you:

• Vote in the Parkinson's Awareness Month T-Shirt Design Contest here.
• Browse the gallery to get inspired by the creative works of others.
• Get creative. Talk to an occupational therapist about creativity or consider taking an art class.
• Submit your works­, paintings, illustrations, pottery, and more,­ to the Creativity and PD gallery.
• Order a free copy of the 2013 Creativity & PD calendar by emailing info@pdf.org or clicking here.

We hope you enjoyed these stories as much as we did. As Victoria Tane says, "Being an artist is part of the respite, the refuge and the reason that I am able to deal with Parkinson's disease in a pretty positive way."

We think that sums it up pretty well.

What Makes Us Distinctive? The PDF Way: A Letter from the Executive Director, Winter 2013

What makes PDF distinctive? 

As you may remember from September's post, I am now answering such questions in my introductory letter for the Parkinson's Disease Foundation's quarterly newsletters, and posting them on the PDF blog, so we can begin a conversation with you.

In PDF's recently published Winter 2013 issue, I discussed what makes us distinctive, what we call The PDF Way. How does it play out in our programs?

The PDF Way 

In our research portfolio, The PDF Way means supporting the creative ideas brought to us by scientific teams and individuals — peer-reviewed and time-tested. We provide long-term support to research teams at major universities; we award short-term research grants to individual investigators; and we help to solve specific research challenges through the staging of meetings among experts. (For a profile of one such scientist, see this issue's "Spotlight on Research".)

In our educational initiatives, The PDF Way makes creative use of technology to bring authoritative, relevant information to our target audiences. This includes running PD ExpertBriefings, a series of educational webinars for people with Parkinson's and their families, now in its fourth year; and providing online professional education for nurses, physical therapists and other "first responders" to the needs of people with Parkinson's. (For a list of upcoming PD ExpertBriefings, see page 7 or browse our website here.)

In our advocacy programs, The PDF Way means harnessing the energies of individuals and families with Parkinson's. The most striking example of this is the signature program we call Parkinson's Advocates in Research (PAIR), in which we deploy lay advocates to play leadership roles in research. (For examples of how these advocates work, see photographs on page 10, also featured here.)

In all of these activities, we make certain pledges to our community.

• To our donors, we promise accountability and efficiency. (For the fifth consecutive year, we boast both the four-star (highest) rating of Charity Navigator, the respected charity watchdog group, and the premier seal of approval of the Better Business Bureau.)
• To people with Parkinson's, we promise a place at the very center of our operations — whether through our People with Parkinson's Advisory Council (PPAC), through PAIR (see above), or in the way we consult the community in the design and execution of each and every program and service.
• And to our colleague organizations, we pledge collaboration — both to provide better service to people who are affected by PD, and to minimize duplication of effort.

Your Part in The PDF Way

An important part of The PDF Way is you. So in 2013, we invite you to be a part of it - whether by providing feedback on the PDF blog, joining a PD ExpertBriefing, or becoming part of Parkinson's Advocates in Research.

How would you like to be involved? What should PDF keep in mind in building our programs in 2013?

Together, we can expedite PDF's mission to improve the lives and futures of people touched by Parkinson's.

The Good, the Bad and the Ugly: Science Reporting in Parkinson’s

Will eating berries lessen your risk of Parkinson’s? What about coffee? What’s the real story on coconut oil? Is deep brain stimulation a miracle surgery?

All of us, whether or not we live Parkinson’s, are faced each day with a barrage of headlines about what might help or harm our health.

After such headlines appear, PDF often receives an influx of inquiries asking:

• Does this new drug/therapy/compound help Parkinson’s?
• Is this new drug a cure? 
• When will this new breakthrough be available?

The Good News
Scientists all over the world are conducting research into Parkinson’s. They are publishing their work in scientific journals and discussing it at meetings, and the media is reporting on it. This means that people touched by Parkinson’s disease have the chance to access the latest information about the disease online. According to this weekend's edition of the New York Times, which references studies by the Pew Research Center, people who aren’t connected to the Internet may actually be missing out on valuable health-related information!

The Bad and the Ugly
Not all sources report responsibly to you. Whether a source uses a sensationalistic headline, or leaves out important facts, they don’t always tell the real story.  For example, a story might report on deep brain stimulation as a helpful treatment, without communicating that the surgery does not stop disease progression. Another might report that smoking is associated with a lesser risk of developing Parkinson's, without telling you that picking up the habit isn't a good idea because more research is needed to understand why.

What Does It Mean?
So if you’re reading these headlines at home, how do you discern between fact and fiction? 

At PDF, part of our job is making sure you have quality information.  One initiative we developed last year was to include a "What Does it Mean?" component to the 50 or so scientific studies we cover each year. At the end of each report we ask “What Does it Mean?” and with the help of medical reviewers, science writers and research staff, we give you our best answer.

Sometimes we tell you that a drug could be available in the next five years. Other times, we let you know that a study was unsuccessful but is valuable for our understanding of Parkinson’s overall. Either way, we try to tell you the truth about how the science might impact your life with PD.

Other Strategies

Here are a few other strategies for assessing health information you find online:

• Ask Your Doctor: If you are seeing a Parkinson’s specialist, they should be very aware of the latest research and be able to explain how it applies to you.
• Evaluate Your Source: Where are you finding your information? Pay attention to who is publishing the information and when they published it. A few years ago, PDF published this article in our newsletter, with tips for evaluating whether a certain website is trustworthy. The Internet has changed, but these tips still apply.
• Call our HelpLine: PDF’s HelpLine is available at (800) 457-6676 or email at info@pdf.org from Monday to Friday, 9:00 AM to 5:00 PM to answer your specific questions about PD or specific news items you read.
• Watch Our PD ExpertBriefings: PDF’s online seminars, including this year’s live series and more than 30 recorded seminars, are led by some of the most trusted experts in the field. Need information on exercise? Find it here. Want to know about experimental medications? We have that too.

What About You?

What's your impression of Parkinson's science in the media? Do you find reporting responsible? Are you confused by the headlines or do you find them clear?

What can PDF to bring you the best information about Parkinson’s disease and the latest scientific findings?

P.S. Click here to see PDF's latest science headlines and scroll down in each one to see our answer to "what does it mean?"

Understanding the Progression of Parkinson's

Can you predict the progression of Parkinson's? ... or change its course?

Last Tuesday, during PDF's PD ExpertBriefing, "Understanding the Progression of Parkinson's," presenter Dr. Ron Pfeiffer had a few suggestions:

• Dr. Pfeiffer provided facts about PD progression - about the percentage of people with PD who develop certain symptoms or leave their jobs after a certain number of years - but also emphasized that these are general facts.  Each person's journey with Parkinson's is very different.
• He noted that there are no treatments proven to reverse PD, but he also emphasized there are actions people can take, such as exercising and joining support groups, that may improve life with PD. (In fact, there is extensive research into exercise right now.)

He also stressed that the picture of Parkinson's he painted during his presentation ... is how the progression Parkinson's looks now.  It's not necessarily the picture of PD in five, 10 or 15 years.  So how can we change it as quickly as possible?  Here's are some suggestions from PDF:

1. Advocate for PD Research: There may not be a cure for Parkinson’s, but you can be a part of the solution.  Join more than 150 PDF Research Advocates who are speeding the development of new treatments through the Parkinson’s Advocates in Research program.  In the Midwest?  We'll announce dates for our in-person three-day training in your region later this month and invite you to apply.  Don't have the time to commit to being a PDF Research Advocate or don't see a training nearby you right now? Take our four-part online course (available in mid-July) to advance your knowledge, and work with our Research Advocates in speeding new treatments. 
2. Inform Others About Parkinson’s: Parkinson's disease is not well understood.  Spread the word in your community and bring the latest educational information to families touched by Parkinson’s.  Browse PDF’s Awareness Toolkit to find tips for raising awareness that work for you, whether writing a letter to the editor or setting up a display in your library.
3. Fundraise for Research: PDF just announced $5.3 million in funding for Parkinson's research. We were only able to do so because of your support.   Support a PD organization, or join PDF Champions, the inspiring individuals who are raising funds for PDF’s programs, to improve the lives and futures of people touched by Parkinson’s.  Whether you run a lemonade stand that raises $20 or a golf tournaments that raises $50,000, you help to move the cause forward.

These are our ideas. What do you do individually to take charge of your Parkinson's?  What ideas do you have for the community to make progress now?

Protecting the People Who Test New Parkinson’s Treatments

We all want better treatments for Parkinson’s.  Tuesday’s announcement that one of PDF’s Research Centers - Rush University Medical Center, in Chicago, IL - received full accreditation from The Association for the Accreditation of Human Research Protection Programs, Inc. (AAHRPP) is another step in the right direction.

Fewer than 200 institutions around the country are fully or partially accredited by AAHRPP.

Before any new treatment, for PD or another condition, is approved it must first be tested in people – in clinical research studies – to ensure it is safe and effective.  The AAHRPP accreditation means that Rush is following the highest standards when it comes to protecting those people. 

As we discuss at length at trainings for PDF’s Parkinson’s Advocates in Research program, testing an experimental treatment always involves some risk – the reason a treatment is not yet available to everyone is that we don’t yet fully understand how it works. But we need to minimize those risks as much as we can and protect the rights of participants. 

AAHRP’s work is part of that progress. It tests research centers on a number of criteria to make sure the institutions are up to par.  To do its part, PDF has been involved in several initiatives, including collaboratively working on a Research Participants Bill of Rights, several pieces of which are featured in our educational booklet here.

If you would like to learn more about research studies and how you can ensure people with Parkinson’s are involved, learn more about our PAIR program or join our ongoing online course,which runs every Thursday in June (the recordings will be posted in July).

The Importance of Brain Banks for Neurological Research

Would you consider donating your brain for science?

A few years ago, Diana Barnwell wrote an article, entitled, My Last Gift.  She chronicled her decision to donate her brain … and the complicated issues that arose when making her decision. 

Studying actual human brains – donated by individuals upon their death, as a contribution to science – is a vital way to understanding neurological diseases such as Parkinson’s.  After all, the brain is the only mysterious organ that we can’t study during a person’s lifetime.

This is why it was so devastating to scientists around the world, particularly those at the Harvard-affiliated McLean Hospital, when the institutions’ brain bank suffered a freezer dysfunction last week.  The institution lost 150 brains that had been donated for research.

They say this constitutes one-third of the world’s largest collection of autism brain samples.  The brain bank also housed brains designated for Parkinson’s research.

How does brain donation work?  It varies at institutions around the country – from their requirements for donating to whom they consider eligible.  For instance, some institutions require a donor to be seen several times a year at their center, while others require just one visit.  Most times, much like any kind of organ donation, the decision to donate needs to be made well ahead of time.

For a listing of Parkinson’s brain banks in the US, browse PDF’s website, which includes our own PDF-funded brain bank at Columbia University Medical Center.

Back in 2010, Diana said her decision to donate was, “a magnificent legacy!”  Do you agree?

Easing Dyskinesia: PDF-funded Research from 2007 Leads to Testing of Experimental Drug

Earlier this week, PsychoGenics Inc., announced that an experimental drug for Parkinson’s, eltoprazine, seemed to reduce dyskinesia in people with Parkinson’s in early studies.  Dyskinesias are the twisting and writhing movements that occur as PD progresses – a common side effect of the medication levodopa (Sinemet®).   

Back in 2007, it was PDF-funded researcher Manolo Carta Ph.D., along with Anders Björklund, M.D., who performed the pre-clinical research that led to the identification of this drug.  At the time, while many researchers were looking at dopamine neurons as the culprit behind dyskinesias (through their interaction with levodopa), Dr. Carta’s proposal suggested something different – serotonin neurons.  His proposal led to a one-year research fellowship funded by PDF.

After completing his year of research with PDF funding during which he laid the foundation for this approach, he and his colleagues were able to investigate an experimental compounds that might help with dyskinesias.  With the support of other funders, they have now been able to study the effects of eltoprazine in people in this early stage trial – to see whether it is a viable drug treatment.

The study results announced earlier this week indicate positive news about eltoprazine’s potential to ease dyskinesia and possibly some non-motor symptoms of PD.

However, the drug will have to undergo rigorous testing – in additional people with Parkinson’s in phase II and phase III clinical trials – before we know if it’s safe and effective.  While we don’t yet know the fate of this drug, the results help us learn more about dyskinesias so we can find a solution in the future.

PDF believes it’s important to fund ideas like this so that scientists can have both the freedom to explore novel ideas for Parkinson’s and the time they need to gather data that can prove the promise of their ideas.  In Dr. Carta’s work, this proved to be the case. 

We believe this philosophy – funding creativity early on – will help researchers prove their case to other funders that can help them to further develop their ideas ... ultimately into new treatments and a cure for Parkinson’s. 

What are your thoughts? Do you or a loved one need a drug for dyskinesia?

Please share your thoughts below, and as always, call our HelpLine at (800) 457-6676 with any questions you or a loved one may have about Parkinson's.

What's On Your Wishlist? Seeking Your Feedback for our Educational Programs

What is the one topic you wish PDF's educational programs would cover about Parkinson's disease?

... medications?

... nutrition?

.... exercise?

We hope you tell us by taking our very short (promise - it's just four questions) survey.

You may already know that PDF regularly hosts online seminars, or PD ExpertBriefings, along with a host of other educational programs.  Available live (both online and by phone) and recorded (both online and as DVDs), they are some of our most popular programs.

But we want to make sure we're covering the topics that are relevant to your life with Parkinson's.  So we're asking you to rank several topics and then tell us about any others we left out.  Please, if you have just a few minutes today, make your voice heard.


You tell us what you need our programs to cover. --> We'll do our best to deliver.

Take Survey Now

(PS: To get a sense of past and future topics, browse our past online seminars and sign up for those upcoming online seminars.)

PDF Champions Friday!

PDF CHAMPIONS FRIDAY

Surprise… Happy 60th Birthday to Gary Chard from Wilmington, DE!  There is no better way to celebrate your birthday than with a surprise party thrown by family and friends - - while fundraising for PDF at the same time!

At the beginning of the year, Gary’s wife Beth Ann came to PDF with the fantastic idea of turning Gary’s special ‘secret’ bash into a PDF Champions fundraising event http://www.pdf.org/en/pdf_champion. Gary was diagnosed with Parkinson’s disease in 2008 at the age of 56. Together Gary and Beth Ann have been extremely involved in the Parkinson’s community. Gary presents educational topics about Parkinson’s to support groups in NJ, PA, MD and DE and Beth Ann has become a true care partner and a PDF Research Advocate http://www.pdf.org/en/research_advocate.

I am so pleased to announce that Gary’s party raised over $2,500 for Parkinson’s research! Applause all around!

Remember: The Cure Begins with YOU!

Notes from a Day Spent Listening to Scientists Judging PDF Research Applications

Last Friday, March 16, was the occasion for reviewing applications for PDF’s International Research Grants program and Research Fellowships programs. Before us were some 30 proposals from some of the best young (and not-so-young) scientific investigators in the world.

As I sat there in the meeting as an observer, listening to the members of our scientific review committee as they made their comments and pronounced their judgments, I found myself scribbling notes on what seemed to be the principles on which they were basing their decisions. What they were saying, it seemed to me, said a lot about how we go about choosing the projects we will fund. Here are some of the ideas I picked up:

Relevance to Parkinson’s Disease. Every proposal that we fund – without exception -- must show promise in its potential to advance our understanding of PD, or charting the path to its cure.

New Ideas, New Investigators. To maximize the leverage of the program, successful applications will be one of two kinds. One is the innovative pilot project that shows promise for leading to a larger-scale endeavor that will be eligible for funding from the NIH (drawing on PDF’s “leverage” function). The other is the Parkinson’s-related application from an exemplary scientist whose past contributions have been largely in areas other than Parkinson’s – and who could be inspired by means of the PDF grant to turn his or her attention to PD.

Demonstrating Results. The renewal of an earlier award to the same scientist depends on the investigator’s ability to show “significant progress” since the first grant – that is, you don’t get a second grant if you can’t show that you used the first grant well.

Establishing Credibility. To make it through the grant review process, good ideas aren’t enough; the applicant needs to be able to demonstrate – both in his/her personal accomplishments and in the reputation of the lab in which the work will be done – a stellar track record in producing innovative and useful science.

Including Advocates in the Research Process. The meeting included three members of Parkinson’s Advocates in Research (PAIR), a PDF program in which lay leaders in the PD community are prepared to take on a variety of roles advocating for clinical research. An example of their contribution came early in the day, when one of these advocates raised a question about the validity of animal models in Parkinson’s research, which generated a spirited exchange among the scientist members of the committee.

I hope you are as impressed as I am with this little vignette of PDF’s research culture!

I conclude with a quotation from a memo on the mission of the program from Dr. James Beck, who is our Director of Research Programs at PDF and staffs the grants review committee. In thanking the scientists in the room, he said, in effect: “The two groups that will benefit most from your decisions today are not even here. One is the world of talented young scientists whose work will be made possible through your efforts. The other is the community of almost one million in the U.S. who live with Parkinson’s. In behalf of these two communities, PDF thanks you!”

Amen, James.

Grant Review at PDF

Today is a big day for many at PDF — it is our annual grant review. Of course, those most affected by what goes on in the big conference room will not be there. There are two groups who depend on tomorrow's outcome: the scientists who have given us their best ideas for our scientific advisors to judge and the people who live every day with Parkinson's and are counting on these great ideas to make a difference in their disease and their lives. I am confident that the team of scientists helping — members of our scientific advisory board and other experts from around the country — will select only the best science today.

All in all, nearly 25 reviewers have pitched in their time to help whittle down the 200 letters of intent to 63 full grant proposals to, now, only the top 20 or so grants. Because of time, it is these top 20 applications that will actually get discussed.

And for the second time, PDF is doing what no other PD organization in America is doing—making certain those people living with PD have a literal seat at the table and participate in the decision making process. These people, PDF's Research Advocates, part of the Parkinson's Advocates in Research program, insure what we will fund is not just the best for science's sake but for the sake of people too. Something in which we, as an organization, take great pride and you as a community should too.

Tomorrow would be difficult to organize without the obvious help of my colleagues at PDF. But equally important is the help from everyone who will not be joining me in the room — you, the Parkinson’s Community. Your generous support of allows us to do what we do every day: support research and ideas that will improve the lives and futures of people touched by Parkinson’s. Our research program would simply not be without you. Thank you.

What Can Alzheimer’s Tell us About Parkinson’s?

From James Beck, Ph.D., Director of Research Programs

Understanding how Parkinson’s disease starts and how it progresses remain two of the key unanswered questions about the disease. This is why a recent story in the New York Times caught my eye.

The article described how two independent groups of scientists found evidence that Alzheimer’s disease may spread from brain cell to brain cell much like a virus. These scientists engineered mice to make a human form of a protein called tau in only one small part of the brain. Clumps of the tau protein are a hallmark of Alzheimer’s disease. Like the rubble of demolished buildings, these clumps are all that are left of brains cells that have slowly died because of Alzheimer’s.

Proteins and Clumps

Proteins in the body are folded into special shapes … almost like pieces of origami. Some proteins, if they are not folded correctly to begin with or become “crumpled” due to some cellular problem, will stick together in clumps. Similar to how clumps of the protein tau are a hallmark of Alzheimer’s, clumps of a protein called alpha-synuclein are the hallmark by which Parkinson’s disease is diagnosed. These alpha-synuclein clumps are called Lewy bodies.

Parkinson’s and the Contagion Theory

Parkinson’s researchers have long noted what appears to be the spread of Lewy bodies from one brain region to another. Researchers observed possible clinical evidence that Parkinson’s could “spread” from one cell to another, when fetal cells were transplanted into the brains of people with Parkinson’s disease. The transplants subsequently developed Lewy bodies. That is, something about a Parkinson’s brain made genetically unrelated cells that were only a few years old develop the signs of Parkinson’s disease.

What came out of these observations was the controversial idea that Parkinson’s, once started, might be transmissible from brain cell to brain cell.

Several recent scientific papers are beginning to lend credence to this idea:

• Last year, Dr. Patrik Brundin’s research team in Lund, Sweden published a paper demonstrating that mis-folded alpha-synuclein (the basis of Lewy bodies) can spread from cell to cell in culture and could also spread in the brain of a mouse.
• More recently, Dr. Virginia Lee's team from the University of Pennsylvania published similar findings this past November.
• At a recent New York Academy of Sciences meeting on Parkinson’s disease, unpublished data was presented that also demonstrated how a single injection of alpha-synuclein clumps into a mouse brain could lead to the widespread formation of Lewy bodies in many different parts of the brain.

What Does It Mean?

Well it brings hope that a Parkinson’s therapy might be found in one of two ways: using special antibodies that target alpha-synuclein or a vaccine which primes the immune system to also target alpha-synuclein.

Either approach may not only halt the spread of Parkinson’s disease but maybe could also prevent it.

Your Feedback

What do you think about the recent Alzheimer’s study and how it relates to Parkinson’s? As always, please share your comments and I will do my best to respond.

Notes from AAN 2011, Part II: Which Doctors Treat Parkinson's?

From James Beck, Ph.D., Director of Research Programs

As mentioned last week, this year the American Academy of Neurology held its annual convention in Honolulu, Hawaii, attracting nearly 7,000 neurologists and neuroscientists.

I wanted to provide you with an update of interesting and new developments that were presented at the meeting last week, in particular, three studies which discussed whom people with living with Parkinson's disease see for their care.

#1-The first was a health economics presented by Marcy Tarrants, Ph.D., of Teva Neuroscience and her colleagues (presentation P01.087).

This group examined the records of newly-filled prescriptions for people diagnosed with Parkinson's and found that, overall:

• the prescribing doctor was a neurologist (both general and movement disorder specialists) in 46 percent of the cases;
• while the prescribing doctor was an internist or primary care physician in another 41 percent of cases.
  

However, when one examines whether those prescriptions are for new treatments (think newly diagnosed or just starting a medication) or for existing treatments (those who have been living with the disease), the numbers shifted:

• only 40 percent of the new treatment prescriptions were from neurologists;
• versus 59 percent of existing treatment prescriptions being made by neurologists.
  

#2- A similar study by Benjamin George, a medical student at the University of Rochester, and his colleagues who worked under the mentorship of Ray Dorsey, M.D., M.BA., now at Johns Hopkins University (presentation P01.088) found in a small study that:

• only 30 percent of those individuals with Parkinson's living in nursing homes see a neurologist;

• the rest see primary care physician or internist 50 percent of the time;

• and 20 percent see no physician at all.

#3-The results of the first two studies tie into the outcomes reported from a related report by Karen Zheng and colleagues working under the mentorship of Melissa Nirenberg, M.D., Ph.D., from the PDF Research Center at Weill Cornell Medical Center. (see photo; presentation P01.081). In a chart review of 120 people with Parkinson's, this group examined the underlying cause of those 25 percent who experienced acute worsening of their Parkinson's motor symptoms, or exacerbations. They found two main causes of exacerbations: infections and medication problems: about 25 percent suffered an infection, often a urinary tract infection, and 35 percent had problems with pharmacy errors or taking their medications on time (evenly split). While many of the exacerbations were reversible, about a third had recurrent problems and about a fifth never improved.



The lesson here is that prompt medical attention by someone trained to recognize the complications of Parkinson's by, you guessed it, a movement disorder specialist is important to achieving the best outcomes to these exacerbations.

Conclusions
These numbers in the first two studies, and the conclusions of the third, reiterate a concern that PDF has for PD community: not enough people are seeing the medical specialists who can best treat their disease.

PDF recommends everyone with PD see a movement disorder specialist or, if one is not in your area, a neurologist who can perhaps work with a specialist that you travel to see once or twice a year.

Do you need a recommendation? Call PDF to find the specialist nearest to you.

Please share your thoughts on these studies and check for further reports!

A Season of Giving

From Eddie Pelto, Director of Development

Each December, many of us prepare for holiday festivities with family and friends. Sometimes this includes shopping for gifts – it is, after all, the traditional ‘season of giving.’

In keeping with that sentiment, it is also at this time of year that millions of people choose to make their charitable contributions. At PDF, year-end giving accounts for 20 percent of our overall annual revenue. As we have mentioned previously, much of this support comes from individual supporters around the country.

We rely on the generosity of new and returning supporters during the holiday season to make our plans for the coming year. This year alone we were able to give away $5.5 million dollars to researchers in the US and around the world who are searching for a cure for Parkinson’s disease. We could not have done that without the thousands of individual gifts we receive from you.

So at this time of year, we express thanks to those of you who made our programs possible. We also hope that you will remember PDF at this time of year.

If you want your donation to count for 2010 tax-returns, you must act before December 31st. Some last-minute actions you can take to invest today:

• Charge the donation by credit card. Even though you don't pay the credit card bill until next year, the donation by December 31, 2010, is good for 2010 – you can make your donation securely online at PDF’s website http://support.pdf.org or by simply calling us at (800) 457-6676.
• Mail a check by Friday, December 31. As long as you put it in the mail, the donation is treated as made in 2010 even though the charity doesn't receive the check or cash it until 2011.
• Make a gift of stock. If you wish to make a gift of securities please have your broker contact us at (800) 457-6676 to let us know of your contribution – it’s a fast and simple way to make a big difference.
• Don’t forget matching gifts. Does your employer match contributions? Be sure to check with your human resources department and fill out the simple paperwork which could as much as double or triple your gift to PDF. You can also search for your company on PDF's website.

We consider your gift an investment in the programs that will bring us closer to the goal we all share: to understand and find the cure to Parkinson's disease and related movement disorders; and, for as long as this search continues, to ensure that those individuals and families who live with Parkinson's are able to achieve and maintain the best possible quality of life.

That is truly a gift that keeps on giving.

Happy Holidays!