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:

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.

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.

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?

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.

PAIRing up for Research: The Importance of Saying Thank You

Last week, we were excited to see a great example of pairing up (the slogan for our Parkinson's Advocates in Research, or "PAIR" program) at Vanderbilt University Medical Center (VUMC). In an article entitled, "Events honor early patients of novel Parkinson's study," VUMC reports on their study of deep brain stimulation surgery as a treatment for early stage Parkinson's. DBS is approved by the FDA as a treatment for mid stage Parkinson's, but not for those with earlier stage PD.

We were impressed to see how VUMC thanked their volunteers, all people with Parkinson's, who made the study possible. As the article notes, their 35 volunteers did an extraordinary thing by agreeing to either undergo surgery early on in their Parkinson's or to receive standard medicines. Each person also dedicated five separate weeks of their time over the course of two years.

As with all clinical studies, we only learned new lessons about PD because of those volunteers.

Will You "PAIR Up" for Parkinson's Research? 
This story rang true with PDF because VUMC's event reflects values similar to those that drive our PAIR program.  At PDF, we believe that people with PD must be primary partners in research­ as participants, but also in other capacities,­ in order for us to speed new treatments.

The importance of saying thank you is particularly key, because of findings such as those from a 2007 CISCRP survey which say that:

• 79 percent percent of clinical study volunteers report that they never hear from the sponsor or research staff after a trial has concluded, and; 
• 83 percent of volunteers say they are never told the results of their trial

Through PAIR, we are committed to changing this statistic and to ensuring that someone says thank you.  We are doing this in part through our signature PAIR Up for PD Research forums each April that - similar to VUMC -­ publicly thank people with Parkinson's disease who have volunteered for research studies.

    Courtesy of PDF Research Advocate Carolyn Weaver, made for her fellow Research Advocates.

Most of these forums are led by one of PDF's 180 Research Advocates (all of whom have completed one of our in-person PAIR Learning Institutes) in collaboration with local researchers.

PDF Research Advocate Bob Hankin at an April 2012 PAIR Up for PD Research Forum in New York, NY.

The thank you at the forum is usually a simple public recognition along with a small token from PDF (see first and last photos).  But it's an important step in ensuring people with PD are at the front and center of research.

We applaud VUMC's efforts to communicate with their volunteers and say thank you.  

What do you think about their efforts? Will you PAIR Up?

Visit our website to learn more about getting involved with PAIR, through our upcoming training for people with Parkinson's and care partners in Oak Brook, IL or our new online course.

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).

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.