Monday, August 19, 2013

Old Drugs – New Cures?

From James Beck, Ph.D., Vice President of Scientific Affairs

Using old drugs as new cures seems like a surefire winner. It may be. However, after attending a recent meeting outside London hosted by the Cure Parkinson’s Trust, a small yet impactful British charity, it is clear that this path is neither clear nor easy. A committee of experts at the meeting evaluated and prioritized dozens of existing compounds – many are drugs used to treat other diseases – based upon their potential to stop Parkinson’s disease.

From left, James Beck, Tom Isaacs, and Steven DeWitte at
Cure Parkinson's Trust meeting at Cumberland Lodge.

Repurposing drugs for a new use like this has the potential to shave significant time from the drug development process for Parkinson’s. But existing drugs come with existing problems. And these problems cannot always be overcome. For instance, a recent science article covered by PDF suggested that a chemotherapeutic agent, nilotinib, might be useful for treating PD. However, it is not clear what the proper dose should be (the article suggested a low one would be effective). Moreover, with potential side effects that include sudden death, the committee felt it was not ethical to consider placing people living with PD in potential danger.

Still, there are drugs that appear to be safe and well tolerated, like statins used to lower cholesterol and metformin used for type II diabetes to name a few. These drugs are not chosen at random: By surveying thousands of people, epidemiology studies have been able to link drugs people take for other conditions (like these) with a lower risk of developing PD. There are also data from laboratory studies that suggest how these types of drugs may help. Even here, though, the path forward is not so clear. The mechanism of action, that is how might some of these drugs actually affect PD in people, is not known. This is not always a stumbling block; but when combined with drugs that are likely to have modest effects and low odds of success, it can be a problem, especially with limited resources.

Recognizing these issues, the Trust’s prioritization committee took a hard look at each potential drug candidate and recommended a handful as most promising. These few culled from a list of two dozen compounds will now advance towards evaluation in clinical trials. Hopefully, that phase will begin soon.

Wednesday, July 31, 2013

BigBrain - What Does It Mean?

From James Beck, Ph.D., Vice President of Scientific Affairs

This blog is part two in a series of three about the BigBrain.

Several weeks ago saw the announcement of a description of a new and highly detailed atlas of the brain, called BigBrain.  PDF wrote about how one person, making the decision to donate their brain, has made a significant contribution to science.  Indeed, that is true.  But what does this really mean for the future of neuroscience … and Parkinson’s research?
  1. BigBrain is like the Google Earth of the brain, allowing researchers to not only see the big picture of brain anatomy but also allowing them to zoom in with incredible resolution to practically see individual cells.
  2. Although BigBrain is like Google Earth, it currently does not have any labels.  That is, if you do not already know what you are looking at, BigBrain will not be able to help you.  (Is that New York City or Jersey City that I see?) Not all scientists are experts in neuroanatomy and so not everyone looking at BigBrain can delineate every brain structure.  Besides, annotating BigBrain will only need to happen once.  As this is done over time, researchers of all stripes will be able to ask interesting questions.
  3. BigBrain is helpful, but it's just that: one person’s brain … Just like the first genome sequenced in the Human Genome Project was just one person’s DNA.  Half the battle here has been establishing the procedures to actually create a BigBrain.  As more courageous individuals donate their tissue, scientists will be able to generate more BigBrains in order to capture all the individual variation that is in each of our heads.
In sum, BigBrain is what they call “enabling technology.”  On its own it does not do much, but when combined with other technologies it is a powerful tool that can move science forward.  Thinking what the personal computer or the Internet have done recently, it will be exciting to see how this will change neuroscience.

(A view from the BigBrain atlas. The substantia nigra, where dopamine neurons are lost in PD, are the dark bands located below the two dark circles in the center.)

Tuesday, July 16, 2013

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.

Friday, June 21, 2013

Going 3D: The BigBrain Is A Gift That Keeps on Giving

This blog is part one in a series of three about the BigBrain.

Do you remember the moment of putting on 3D glasses in a movie theater? There was awe and excitement as stories and images that were flat came to life. There was similar excitement yesterday when researchers reported in Science that they have created BigBrain, a high resolution 3D digital image of the brain.

To understand the excitement, it’s important to understand that like a 3D movie, this image isn’t simply a flat picture of the brain. It is incredibly in-depth. Have you ever had an MRI? Well this is hundreds of thousands times more detailed.  Some have compared BigBrain to Google Earth, which shows much of the globe, piece by piece. Scientists think this up-close-and-personal view will help us understand how the brain works.

But perhaps the most amazing part of the BigBrain story, is the one of how it came to be. BigBrain was reconstructed from a real donated brain, from a 65-year old woman who had passed away. She gave her brain to science, and now we may all benefit for years to come.

For it’s true that the brain truly is the final frontier. Did you know that even with BigBrain, brain donation remains an urgent need for understanding PD?

We can study other organs while a person is alive, but we cannot easily access and view a brain. The only way to study to do so to date has been to study brains donated to science. Brain donations enable scientists to learn more, for example by comparing a person’s medical records and PD symptoms, to their brain tissue after death.

That said, brain donation often brings up many questions and concerns for people with PD and their families. A few years ago, PDF Research Advocate Diana Barnwell wrote a beautiful story, "My Last Gift", about her decision to donate her brain to research, which may provide food for thought.

What’s your view of brain donation, and of this gift that made BigBrain possible?

At PDF, we always say that we can’t do our part without you. And in looking at BigBrain as an example, it’s true. In fact, this brain is only the first to be mapped. Scientists report planning to map other donor brains, including a male brain and others whose donors lived with certain diseases or conditions.

This emphasizes that it's your participation in research – including via brain donation – that brings us closer to solving Parkinson’s. So we say thank you to the anonymous donor that made BigBrain possible, and thank you to you.

Learn about brain donation by reading Diana's story, "My Last Gift," here, or by browsing PDF's list of US brain banks here.

Wednesday, June 19, 2013

Of Patents and Parkinson's

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

Should or could a human gene be patented? On June 13th, the Supreme Court of the United States delivered their unanimous ruling regarding what has been called the Myraid Genetics case.  The plaintiffs in this case sought to invalidate Myriad’s patent on two genes that when mutated can lead to a significant increased risk of breast and ovarian cancers.  Because of the patent for these two genes, Myriad, a clinical diagnostic testing company, was the only entity that was permitted to perform the clinical tests that can both inform women if they carried mutations in these genes and if they are at an elevated cancer risk.

In their ruling, the Supreme Court concluded that naturally occurring genes, like the ones in the patent held by Myraid, are a product of nature and therefore are not eligible for patent protection.

The emphasis of the Court is on naturally occurring genes and that is important because the Court made clear that almost any modification to that naturally occurring gene would be eligible for patent protection—provided it met all the regular standards of a patent.  The example the Court provided was for the ‘cDNA’ sequence of a gene.

When a Gene is Not Natural
So what is cDNA anyway?

It may helpful to think of the genes found in most animals as magazine stories. These stories are constantly interrupted by advertisements, forcing them to span dozens of pages instead of just a few.  These interruptions to the gene story are called introns and, just a like a real magazine story, they get skipped by the body as it reads the gene when making a protein in a process called transcription.  In the laboratory, this “ad-free” or more precisely “intron-free” version of the gene can be converted back to DNA (the cDNA) for later use in a biotech setting.  Because this edited version of the gene did not appear in nature, it is eligible for patent protection.

What Does This Mean?
In general, this decision will not really affect much in the normal operation of science and business.  Whenever a gene is patented, the ad-free cDNA version is almost always included as part of that patent.  Importantly, it is this cDNA version that is really of the utmost value to a company because the naturally occurring sequence is often so large and unwieldy it is not practical to use—this is because the cDNA form can easily be just one-fifth the size of the naturally occurring gene. That is a lots of ads that were cut!

For companies that have a product that relies almost exclusively on the naturally occurring sequence, say for a diagnostic test, this ruling will be more disruptive.  In fact, several competitors have just announced that they will offer the same cancer gene test for less than what Myriad charged.

Ok, so what does this mean for Parkinson’s disease?
Again, not much really.  There are several companies that have been trying to develop gene therapies for treating Parkinson’s.  While the patents these companies may hold on the naturally occurring gene sequence is likely not enforceable anymore, the shortened version of the gene still has a valid patent and that is what counts—it is this shortened form that is actually used in the experimental therapy.

While the Court’s ruling that naturally occurring genes are not patentable is certainly historic, I think the initial effects are relatively limited, especially for Parkinson’s.  Time will tell how this ruling will be applied to other areas of intellectual property law.

Tuesday, June 18, 2013

Expression vs. Insensitivity: A Message to Kanye West from a Person with Parkinson’s

From Tom Palizzi
Chair, PDF People with Parkinson's Advisory Council

Kanye West is without question a richly talented and multifaceted artist. With utmost respect for freedom of expression and the inherent controversial nature of art in general, there is, however, a fine line between expression and insensitivity.

"On Sight," the opening track on Mr. West's new album Yeezus, includes the lyrics:

“The monster about to come alive again/
 Soon as I pull up and park the Benz/ 
 We get this b*** shaking like Parkinson’s”

Many of us appreciate the lighter side of having a chronic and degenerative movement disorder, though as many interpret such statements as harsh and insensitive. While it appears Mr. West is familiar with one of the more recognizable symptoms of Parkinson's disease, there are several other, less obvious challenges people with Parkinson's face.

For more than 55 years, the Parkinson's Disease Foundation (PDF) has been helping people understand and cope with Parkinson's disease. In a unique and bold move, the foundation formed the People with Parkinson's Advisory Council (PPAC) to ensure the effectiveness of their actions.

As Chair and on behalf of PPAC, I would be delighted to help Mr. West better understand the truths and myths of "Parkinson's" and how it indiscriminately impacts the lives of roughly 1,000,000 Americans and their families. Notable people such as Michael J. Fox, Muhammad Ali and Ben Petrick, my peers and millions of others are testament to the enduring spirit of people with Parkinson's.

Mr. West, please take a moment and visit to learn more and, by all means, feel free to contact me or my colleagues with any questions.


Tom Palizzi
Chair, People with Parkinson's Advisory Council

Tuesday, April 30, 2013

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.

Friday, April 19, 2013

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:

Thursday, April 11, 2013

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.

Saturday, April 6, 2013

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.

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.

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

Thursday, April 4, 2013

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.

Vitamin D...Again?

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

A recent paper published in the American Journal of Clinical Nutrition and picked up by the popular press provided some provocative evidence that vitamin D may provide a short term benefit to some people living with Parkinson’s disease.  PDF has covered the science regarding vitamin D for some time, for example in articles featured here and here.

While the importance of vitamin D in people with PD is not new—most people with PD have too low a level—this paper now suggests that only some individuals may benefit from raising vitamin D levels.  The paper's authors hypothesize that those few individuals have a variation in a protein that binds to vitamin D. They may respond better than others when their vitamin D levels are raised, which may result in a slower progression in disease symptoms.  However, even the authors were notably cautious in their interpretation saying, "vitamin D supplementation may stabilize PD for a short period in patients [with the protein variation], although this effect may be nonspecific for PD."

That is, this study may help explain why vitamin D is not the cure-all for Parkinson’s as some less reputable sources may claim.  Whether this conclusion will stand the necessary scrutiny of continued research is unknown.

What I do know is that good nutrition is important for good health, especially if you have a chronic disease like Parkinson’s.  It is probably worthwhile to have your physician check your vitamin D levels at your next physical—I did.  Maybe like me, you may find out you have low levels of vitamin D. On my doctor’s advice, I now take a few vitamin D pills.  I am not expecting much, but I think it a good idea.  Like checking the tire pressure on your car: it is a little thing that will play a small role in hopefully making your journey through life a bit easier.

For more information on nutrition and PD, read PDF’s Fact Sheet on Nutrition or watch PDF’s PD ExpertBriefing on Nutrition.

Tuesday, March 26, 2013

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.

Tuesday, March 12, 2013

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?

Friday, February 22, 2013

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.

Tuesday, February 5, 2013

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:

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.

Tuesday, January 15, 2013

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.

Monday, January 7, 2013

Dr. Bill Weiner: Eminent Scientist, Independent Voice and True Friend

Last month, the Parkinson's community lost a dear member, one who dedicated his career to the treatment of the disease. William J. "Bill" Weiner, M.D.,  known to many at the University of Maryland where he served as Chairman of Neurology and the Director of the Maryland Parkinson's Disease and Movement Disorders Center, passed away on December 29, 2012.

Involved in treatment trials for Parkinson's disease since levodopa in the 1960s, Bill was truly one of a kind in his profession – a fine scientist and a dedicated clinician who was committed to thinking for himself and to expressing his views fearlessly just as they were, arrived at not through crowd-sourced conventional wisdom but through his own learned, astringent, original, independent analysis.  You always knew where you were with Bill, and that he would give it to you straight, with kindness and yet with humor, and from principle rather than from special interest – his own or any other.

We all are grieving with Bill's family, including his wife Lisa Shulman, a fellow Parkinson's scientist/clinician, and treasuring the memories of the philosopher and teacher we all knew.

Please feel welcome to share any of your memories below.

See 2006 Interview with Dr. Weiner in PDF News & Review

Photo credit: American Academy of Neurology.