From PPAC: Parkinson's Isn't Newsworthy ... Until It Personally Touches You

By Peggy Willocks, member, PDF People with Parkinson's Advisory Council

April is Parkinson's Awareness Month. And it won't be newsworthy that I came all the way from Tennessee to New York to participate in this past Saturday's Parkinson’s Unity Walk, where about 10,000 people with Parkinson's, friends and family "walked" through Central Park as a symbolic gesture to raise funding for research.

As a member of the Parkinson's Disease Foundation's (PDF) Advisory Council, it isn't newsworthy that I am also here as a voice of the patient in helping to have better and faster approval of therapies for the debilitating illness. PDF adopts the promise of a cure by creatively incorporating the opinion of those who actually live with the disease into the research program. After all, who wants to find a cure more than the patient? We met with PDF, the oldest established Parkinson's non-profit organization, for the final two days of last week.

But it should be newsworthy that Lizzie Graham was there from across the pond. Graham is Director of Fundraising and Global Communications of the European Parkinson's Disease Association (EPDA) from the UK. Maybe with the two group's collaboration we will make some headway.

What does it take to be newsworthy? Finding a cure would definitely be great news. The gold standard treatment is a pill discovered over 40 years ago. We won't find a cure this year. One reason is the cutting of funding from the NIH budget due to sequestration. Another is the lack of participants in clinical trials for Parkinson's.

It isn't newsworthy that this year marks my 20th year with the disease. But it may be newsworthy that this is my 13th year of having been one of six people in the world who allowed transplantation of retinal cells into my brain from a donor eye in hopes of producing dopamine, the chemical my brain lacks. However, the trial was halted several years later due to not meeting its endpoints.

Parkinson's is more than a tremor or immobility. Many are plagued with non-motor symptoms such as depression, psychosis and dementia, creating an economic burden of over $14 billion. Living with Parkinson's isn't a death sentence; it is a life sentence.

I hope I live to see the cure - now that would be newsworthy.

Peggy Willocks of Johnson City, TN, is a member of the PDF People with Parkinson's Advisory Council. Learn more about her work in the Parkinson's community by reading her biography on PDF's website here.

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:

The Doctor Is "In"... Your Living Room

Yesterday kicked off an exciting and intensive two-day gathering of researchers, health care providers, administrators, patient organizations, people with Parkinson’s and care partners on Long Island, NY. We gathered for the 2013 Merinoff Symposium, “Leveraging Telemedicine to Deliver the Highest Quality of Care to All Parkinson’s Patients,” hosted by the Feinstein Institute for Medical Research at North Shore–LIJ Health System and co-hosted by PDF, other national Parkinson’s organizations and telemedicine organizations.

Merinoff Symposium on Thursday, April 11

We are charged with exploring how telemedicine can enhance care for those living with PD, and creating a call to action for the future.

What is telemedicine? Also referred to as “virtual” office visits, it means being able to see your doctor or other health care professionals remotely, using your computer. You both would be able to see and talk to each other using video.

The common perception is that telemedicine is a futuristic tool used only when people live in remote rural areas, miles away from a movement disorder specialist, neurologist or primary care physician. What was made crystal clear by the end of day yesterday is that telemedicine, or what may be more accurately described as technology-enhanced care, has broader potential to transform care and services for all people PD, regardless of where they live. This transformation will result in providing people living with PD the care and services need, when they need it.

This point was driven home by the symposium’s panel of people with Parkinson’s and care partners. This panel was organized by PDF, moderated by our Executive Director, Robin Elliott and included PDF Research Advocates Jay and Marilyn Phillips. The panel was spot-on in identifying the range of issues that can be addressed by telemedicine - lack of access to core services and resources, challenges to managing the multiple medical visits across specialty areas, limitations of three-month or six-month physician visit intervals in providing an accurate picture of the challenges of living with the disease and the inability to participate in a clinical study that is too far away.

The panel was also essential in helping symposium attendees gain a deeper understanding of how partnering with patients can accelerate the adoption of telemedicine and reap the benefits at a faster rate than what would occur without this partnership. Panelist Steve DeMello discussed the need for patients to be engaged in “creating the science” and urged the audience to move away from a model where providers, “watch me do things and then tell me things.”

This theme of patient engagement is near and dear to the heart of those of us here at PDF, where we have been advocating and pioneering ways for people with PD and care partners to work in partnership with the research community to bring about treatments at a faster pace. Our Parkinson’s Advocates in Research program has trained 200 Research Advocates around the country through our Learning Institutes. These Advocates, along with many others with PD, are ready to join forces with health care providers, researchers, patient organizations, administrators and policy makers to create a new way of doing business when it come to living with PD in the present and determining the future.

It is time to embrace and act on the invitation of panelist Jay Phillips when he said, “we are here and we are ready to work with you!” It is time that we recognize the value and necessity of this outstretched hand and how essential the offer of this partnership is to the success, not only of telemedicine, but to health care and research as a whole.

Thank You for Advancing PD Research

Have you ever been part of a Parkinson’s research study? This Parkinson’s Awareness Month, PDF says thank you for helping to bring about better treatments at a faster pace.

After all, the only way that new treatments and a cure for Parkinson’s can be found is by ordinary people taking the extraordinary step of volunteering in studies.

Two of our Research Advocates, Patti Meese in Arizona and Diane Cook in Colorado (see photos below), have been taking the lead in organizing forums on Parkinson’s research in their communities. Part of PDF’s signature PAIR Up for Parkinson’s Research series, the forums:

• provide information about local Parkinson’s research
• explain how you and your loved ones can get involved and, 
• most importantly thank those who have participated in studies.

Many other Research Advocates across the country are also thanking study participants at support group meetings and Parkinson’s conferences big and small.

It’s central to PDF that people with Parkinson’s and care partners are included in every step of research and are given credit for their critical involvement.  We can’t say it too much: without your participation, there can be no new treatments or research toward a cure. Thanks for your commitment to moving Parkinson’s research forward!

If you live near Phoenix, join Patti at the Arizona PAIR Up for Parkinson’s Research forum.

It starts at 9:30 AM MST on Saturday, April 13. http://www.pdf.org/en/event_calendar/event/743.

If you live near Denver, join Diane at the PAIR Up for PD Research forum.

It starts at 8:30 AM MDT on Saturday, April 20. http://www.pdf.org/en/event_calendar/event/748.




Artwork courtesy of Penny Teem, participant in the Parkinson's Quilt.

Parkinson’s and the President: How Does the $100 Million BRAIN Initiative Announcement Impact our Community?

Dr. Francis Collins, Director of the National Institutes of Health (NIH), introduced him as the "Scientist in Chief."  And he introduced himself as "the kid who had trouble with high-school physics."  But there was no mistaking the passion for his subject of the man who stood in front of us in the East Room of the White House this morning, announcing a major federal initiative in brain science.

It was President Obama at his rhetorical best, mixing easy banter with a deeply serious expression of his commitment to the brain research initiative that he described as the "next great American project."

When he described the pathos of someone watching "a beloved family member slip behind the mask of Parkinson's disease," you could practically feel a shudder of recognition among the normally restrained scientists, government officials and advocacy group leaders like myself who packed the room.  Perhaps "Empathizer in Chief" is more to the point; the guy really seems to mean it.

So what does this mean for Parkinson's?  We won't know for sure at least until the President delivers his budget message to Congress next Wednesday, and possibly not even then.  But the overall strategy of the BRAIN Initiative is clear: to use the resources of the federal government and private sectors to find out more than we know now about how the brain works – specifically, how the various parts connect with one another, and in what patterns.

All of this could have exciting implications for finding the solution to Parkinson's disease.

As Dr. Eric Kandel, the Columbia University scientist who won the Nobel Prize for his work on memory, said to me at a reception following the event, "this is an historic moment!"

We'll see.  There are several big questions to be answered, beginning with money:

• The President is calling for initial spending of $100 million in the year that begins October 1, 2013, but it is far from clear how much of this will be new money and how much will be existing funds repackaged for the new initiative.
• Then there's the matter of who spends it and how between the NIH, the Defense Advanced Research Projects Agency and the National Science Foundation.  And there will be others.
• Lastly, how will patient advocates be involved? PDF certainly supports this initiative’s collaborative nature, utilizing federal research agencies and private scientific organizations.  We urge the President to expand this commitment to include patient advocates as we have done through the Parkinson’s Advocates in Research program.  After all, the people affected each day by Parkinson's disease are critical to our goals of finding better treatments at a faster pace.

But the overall picture is encouraging -- even exciting. This the first time that the President, after years of slogging through the thickets of the nation's fiscal crisis looking for an exit, has made a major statement about medical research, comparing the challenge -- and the potential for its success -- with the moon shot of the 1960s, the development of the computer chip in the 1970s, and the exploration of the human genome of the 1990s.

The common thread through all of these triumphs was what he described as the American "genius for innovation," an outgrowth of a national character of "dreamers and risk-takers."

"How can we afford to do this?" he asked.  Then he answered himself: "How can we afford not to?"

To learn more see PDF's official statement on our website here.

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?"

Are You a Driver and a Partner? Notes on ASENT and Patient Collaboration

Two Saturdays ago, in a Washington DC hotel, PDF pulled off an interesting little coup in its long-term bid to bring people with Parkinson’s (and other neurological disorders) into the center of conversations about the process of clinical research and drug development.

 The occasion was the plenary session on the last day of the annual scientific conference of the American Society for Experimental Neurotherapeutics (ASENT), and the title of the session was: New Models for Collaboration: Patients as Drivers and Partners in Neurological Research. I have served on the Board of Directors of this very worthwhile organization for three years now, and they asked me to help put together an expert panel on this important topic.

Note that the title of the panel referred to patients and clinical research participants as Drivers, and Partners.  Not as Research Subjects, or Attentive Audience Members – important as these roles are in the right context – but as Drivers, and Partners.  In other words, as full players in the process, shouldering their way up against the other weighty and recognized players in clinical research such as the scientists, the government regulators, and the industry collaborators (e.g., drug companies and biotech firms).

Why is this so important?  The answer is that the needs, opinions and requirements of people who live with neurological disorders have an absolutely crucial role to play in the way clinical research in the United States is organized, conducted and evaluated.  What should we be measuring in a clinical trial (often described as “outcome measures”)?  How should participants in trials be treated – from the information that is shared with them to the reimbursement for necessary travel expenses that is provided to them?  And how can recruitment be accelerated, and retention secured, so as to assure that each trial is initiated and completed in the shortest possible time – for the benefit both of the participants’ well-being and the company’s pocketbook?  On these and related issues, patients/participants have important things to say, and important opinions and needs to be accommodated.

Members of the panel, who were assembled and directed by my colleague Veronica (Ronnie) Todaro, PDF’s Director of National Programs, were diverse, interesting and eminently well qualified for the task.  Two presentations in particular stood out for me: Dr. Petra Kaufman, M.D., M.Sc., Associate Director for Clinical Research at NIH/NINDS, who presented a brilliant and comprehensive overview of how patient organizations can be involved in recruitment and retention for clinical trials of new treatments in brain disorders; and Dr. Russell Katz, M.D., the long-time Director of the Division of Neurology Products at the US Food and Drug Administration, who listed the many ways in which patients can be involved in the process of drug approval.

There were three things that I found most exciting about the panel.

1. First – this was most evident in the presentations of Drs. Kaufman and Katz - it gave an encouraging and convincing picture of the many ways that patients can get involved in the clinical research process.
2. Second, the experience filled me with hope that the health care system is at last ready to consider how patients can be integrated into the clinical research process, to the benefit of all the major partners and to the lasting assurance of the people who live with Parkinson’s and other neurological disorders.  (One reflection of this was the healthy size of the audience of doctors and scientists that we attracted – on a Saturday morning, no less, at the very end of the meeting!).
3. And third, it gave me a great sense of pride to see how my own organization, the Parkinson’s Disease Foundation, was playing such an important role in this process -- not only in behalf of Parkinson’s community, but of all groups that are committed to solve brain disorders.  (A sparkling reflection of this was the masterly performance as moderator of Linda Morgan, a talented MBA pharmacist who is a leader of PDF’s national People with Parkinson's Advisory Council and one of the first advocates active with our Parkinson's Advocates in Research program).

ASENT will soon be making available the slide-decks of our speakers to a wider audience.  We will keep you posted on this blog when they do.

What are your suggestions and opinions? Are there additional ways in which patients can be usefully involved in the clinical research process?  Do you feel as if you have the opportunity to be a driver and a partner?