By far, the greatest challenge facing the Parkinson’s community today is the lack of disease modifying therapies to slow or stop the progression of the disease.
Parkinson’s disease is one of the most complicated diseases we know of. No two people with Parkinson’s have the same symptoms, and there is no one-size-fits-all approach to treating the disease. People affected are often forced to stop working and manage their disease full time. If Parkinson’s itself isn’t disrupting enough, often the medications used to treat the disease cause a host of side effects that impact the quality of life and sometimes stop being effective all together. People with Parkinson’s and their caregivers will tell you that this disease simply takes over every aspect of their lives.
So how do the up to 1.5 million Americans living with Parkinson’s find hope? Through the progress researchers make toward finding ways to stop or slow the progression of Parkinson’s. Whether its movement toward understanding the genetics of Parkinson’s, the environmental effects, such as traumatic brain injury, or the quest for a biomarker, we will not make strides toward a cure without continued support of federally funded research at the National Institutes of Health (NIH) and the Department of Defense (DoD).
One of the most promising developments to come out of the research world recently is a new set of research priorities established by National Institute for Neurological Disorders and Stroke (NINDS) and the Parkinson’s community. These 31 priorities were set with the Parkinson’s Action Network’s guidance, but it will be up to everyone in the Parkinson’s community – patients, caregivers, clinicians, researchers, and nonprofits – to ensure that these priorities are funded and accomplished. Focusing resources on these recommendations will allow the research community to achieve the greatest impact in addressing unmet medical need in treatments for and causes of Parkinson’s.
In addition to the $135 million NIH dollars that went toward Parkinson’s research in 2013, the DoD houses the only Parkinson’s specific research program and has invested more than $370 million into that program since its inception in 1997. Any research breakthrough in prevention, diagnosis, and treatment of Parkinson’s disease through the DoD program may be immediately applied to the up to 1.5 million Americans living with the disease.
This commitment to research funding and established set of priorities help make private sector innovation possible, which is why PhRMA’s latest report is further evidence that Parkinson’s research is headed in the right direction. With the addition of 37 news drugs currently in clinical trials or under review by the Food and Drug Administration, and many more in development abroad, we are making strides toward finding more drugs and therapies that will make a difference in the lives of people living with Parkinson’s. But we shouldn’t rest easy until a disease modifying drug is discovered. Support from our elected leaders, federal government, and the private sector is absolutely critical toward that goal, and regardless which sector your voice comes from, it can make a difference.
I encourage you visit www.ParkinsonsAction.org to learn more about the importance of federally funded research and how you can get involved.
Amy Comstock Rick is CEO of Parkinson’s Action Network (PAN). PAN’s national advocacy network educates the public and lawmakers about the effects of Parkinson's disease and the need for federal research funding and policy support for the 500,000 – 1.5 million Americans living with the disease.
Rick has testified before several congressional committees and subcommittees and in 2011 was honored as an Innovator by the Genetic Alliance for her work on federal embryonic stem cell research policy.
As CEO of PAN, Ms. Rick currently serves as an officer or board member with several national coalitions and councils. She serves on the National Neurological Disorders and Stroke Advisory Council, the principal advisory body to the National Institute of Neurological Disorders and Stroke (NINDS) at NIH. She serves as Treasurer for the American Brain Coalition (ABC), a national non-profit organization comprised of some of the United States’ leading professional neurological, psychological, and psychiatric associations and patient organizations that, together, seek to advance the understanding of the functions of the brain, and to reduce the burden of brain disorders through public advocacy. She serves on the Board of Directors for the National Health Council, a national non-profit that brings together all segments of the health community to provide a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers. Ms. Rick also sits on the Board of Directors for Research!America, the nation's largest not-for-profit public education and advocacy alliance committed to making research to improve health a higher national priority. She also served as president and board member for the Coalition for the Advancement of Medical Research, a national leading bipartisan pro-cures coalition comprised of 100 nationally recognized patient organizations, universities, scientific societies, and foundations that successfully accomplished its mission and dissolved in early 2013.
Prior to joining PAN, Amy was the U.S. Senate-confirmed Director of the U.S. Office of Government Ethics from 2000-2003, and Associate Counsel to the President in the White House Counsel’s Office from 1998-2000. She began her federal service in 1988 as an attorney at the U.S. Department of Education, and left in 1998 as Assistant General Counsel for Ethics. She received a Bachelor of Arts degree from Bard College and a Juris Doctor degree from the University of Michigan.
Parkinson’s patients need a cure. The millions living with the disease today, the many more who will age into Parkinson’s risk and their loved ones need a therapy that could prevent, stop or halt progression of the disease. No current treatments impact the disease process itself; they only treat the symptoms. In addition, the symptomatic treatments at our disposable leave much to be desired. Some symptoms — such as cognitive decline — have no therapeutic option. Medication for the motor symptoms wears off before the next time of dosage, its efficacy diminishes with long-term use and it brings on side effects including debilitating dyskinesia. Deep brain stimulation is not an option for all patients. The greatest issue facing Parkinson’s patients is that there is no way to stop their disease.
The most valuable tool we’re after to accelerate research toward a cure is a biomarker. In 2004, Michael J. Fox spoke at the PhRMA Annual Meeting and told attendees, “We’re excited because we know that a reliable biomarker is not only important for diagnosis, but essential to your work in developing drugs that slow or stop the disease.” Over the past decade that need has amplified as disease-modifying therapies have advanced further in the development pipeline and our understanding of new targets and the genetics of Parkinson’s has grown. In 2010 a consortia of industry leaders and The Michael J. Fox Foundation launched the Parkinson’s Progression Markers Initiative (PPMI) to identify and validate a biomarker. We’ve built an infrastructure and a subject base that is already proving fruitful toward that goal and continues to grow in scale.
Part of the reason PPMI we think has been so successful is the collaborative aspect: buy-in from so many, varied players and an open access model of data sharing. Rather than working in siloes, hoarding research tools, and sequestering data and samples, we need to work together. We can move the needle forward faster by opening data access, sharing research tools and convening to problem solve and strategize. We all share the same goal — that cure that millions are waiting for — and we will get there, together.
Todd Sherer, PhD, is the Chief Executive Officer of The Michael J. Fox Foundation for Parkinson's Research (MJFF), reporting to the Board of Directors. Formally trained as a neuroscientist, he directs the organization's research strategy and is responsible for the organization's overall scientific and fundraising direction to speed treatment breakthroughs and a cure for Parkinson's disease.
Dr. Sherer has been a key architect of the Foundation's strategy to define high-priority research areas for Parkinson's disease - therapeutic targets and approaches that are closest or most critical to practical relevance in patients' daily lives - in order to leverage donor-raised capital to push projects in these areas toward the clinic. He has played a major role in the Foundation's efforts to increase the pharmaceutical industry's investment in Parkinson's disease drug development and engage the patient community to encourage and expand participation in clinical research. Today he is one of the world's foremost experts on the science and business of Parkinson's drug development, speaking frequently on these topics at conferences, to the media and to members of the Parkinson's community.
Dr. Sherer's work with the Foundation began in 2003, when, as a postdoctoral fellow at Emory University in Atlanta, he was awarded MJFF funding to investigate the role of environmental factors in Parkinson's disease. He joined the Foundation's staff full time as Associate Director, Research Programs, in April 2004. He was promoted to Vice President, Research Programs, in June 2006 and Chief Program Officer in November 2010, finally assuming the role of Chief Executive Officer in May 2011.
Dr. Sherer is a member of the Board of Directors of the Parkinson's Action Network and participates in the Institute of Medicine of the National Academies Forum on Neuroscience and Nervous System Disorders. He is a collaborating scientist for the Coalition Against Major Diseases (CAMD) and a member of the CINAPS Advisory Committee at the National Institute for Neurodegenerative Disease and Stroke, National Institutes of Health (NIH). Dr. Sherer also serves on the National Center for Advancing Translational Sciences (NCATS) Council and the Cures Acceleration Network Review Board at the NIH. Additionally, Dr. Sherer was selected to serve as a council member on FasterCures' TRAIN (The Research Acceleration and Innovation Network) program.
During his career as a bench researcher, Dr. Sherer published over 30 peer-reviewed articles in scientific journals. He earned his PhD in Neuroscience from the University of Virginia and holds a BS in Psychology from Duke University in Durham, North Carolina.
Parkinson’s disease, a chronic, progressive neurological condition that affects 1.5 million in the United States and costs the American economy more than $14 billion annually, is one of the most mysterious challenges facing the biopharmaceutical industry today. The symptoms of the disease – tremors, difficulty speaking and swallowing and instability – are well-known and evident. But to develop treatments to help those already with the disease and the 60,000 additional individuals who develop the condition every year, remains a challenge.
Our latest Medicines in Development Report on Parkinson’s Disease is both a window into the work being done to combat this progressive disease and a reminder of the magnitude of the task that still lies before us. While the 37 Parkinson’s treatments in the pipeline offer hope for affected individuals and their loved ones, the fact remains that there is still a significant knowledge gap that stands between what researchers know now and developing a potential cure.
The development of new treatments for Parkinson’s has been slowed in large part because the root cause of the disease is still largely an unknown. Until we know more about what causes dopamine-producing cells to die off, developing medicines to stop that process from happening will continue to be difficult.
Early diagnosis of Parkinson’s disease will be important as new treatments are developed to stop or reverse the disease. According to some estimates, individuals with Parkinson’s lose as much as 80 percent of their dopamine-producing cells before symptoms appear. Early diagnosis is crucial to allow help slow Parkinson’s advance; identifying and treating patients before symptoms appear will help minimize the loss of dopamine cells in the brain and help maintain motor skills. One promising early-detection tool would use brain imaging to measure dopamine levels in the brain to give health care providers and early warning on Parkinson’s development and a head start on treating the disease.
Advances in treating diseases like Parkinson’s require developing a better understanding of the root cause of the condition. Fortunately, with the medications currently in the biopharmaceutical pipeline and the clinical tests, researchers are coming closer to unlocking the secrets of Parkinson’s every day and improving the lives of millions who are affected by the disease in one way or another.
Gabriela Lavezzari, Ph.D., M.B.A. joined PhRMA in July 2012 as Assistant Vice President, Scientific Affairs. In this role, Dr. Lavezzari is the primary staff lead for a variety of strategic initiatives aimed at establishing PhRMA as a valuable source of scientific expertise in innovative biopharmaceutical research and development within the Scientific & Regulatory Affairs (S&RA) division of PhRMA. Dr. Lavezzari brings to PhRMA over ten years of combined research experience in the government and industry, with multi-disciplinary expertise in Personalized Medicine and Regulatory Science.
Prior to joining PhRMA, Dr. Lavezzari served as Director Extramural Development at the Medco Research Institute, a subsidiary of Medco Health Solutions, where she led clinical utility and cost-effectiveness research to create value-based reimbursement decisions for a variety of diagnostics products across different therapeutic areas. Prior to Medco, Dr. Lavezzari spent two years at Theranostics Health, a proteomic-based diagnostics company where she led the laboratory operations and the oncology product development. Prior to Theranostics, Dr. Lavezzari worked at Social Scientific Systems where she provided scientific support to and managed multiple Adult Clinical Trial Group (HIV/AIDS), laboratory science, laboratory technical and specialty laboratory committees, subcommittees and working groups.
In addition to her experience in the industry, Dr. Lavezzari spent almost five years in research at the National Institutes of Health (NIH), National Institute of Neurological Disorder and Stroke (NINDS) and at Georgetown University, where she completed her post-doctoral training in Neuroscience.
Dr. Lavezzari received her PhD in Biological Sciences from University of Milano (Italy), and has received her MBA from the New York Institute of Technology (NYIT, NY).
People who live with Parkinson’s (PwP) today are faced with the inevitability of the neurodegenerative nature of their condition – a slow loss of control over life as time progresses. All ambitions and expectations for the future become compromised, and the resulting sense of loss becomes increasingly burdensome. The principal medicine for this is encapsulated in one word – hope. Unfortunately, hope is not a word often used or associated with science – hope is uncertain and science relies on evidence of certainty.
To translate hope into scientific terms, there must be something tangible and real. From the moment of diagnosis, the sand in the hourglass starts to run and, as time passes, the urgency required to maintain hope intensifies.
In plain English, for most people with Parkinson’s the only way the hopes for new treatments can be converted into belief of new treatments, and then onto realisation, is through the conduct of clinical trials. It stands to reason, therefore, that PwP’s hopes are almost totally vested in the conduct, management and success of clinical trials.
Barriers to advancing clinical trials
The inordinate amount of time, money and regulatory hurdles in bringing any new drug to market in any condition are well documented, but there are some specific problems in Parkinson’s. One of the issues facing industry when assessing whether to develop a new Parkinson’s treatment is measurement. Parkinson’s is different in every PwP and there can be huge fluctuations in symptoms within very short time spans. The lack of an objective, continuous and accurate method of assessing PwP means that the results and statistical analysis of clinical trials become difficult to interpret and, quite often, are inconclusive. This, coupled with costs and the length of time needed to show clinically significant changes in the trial participants’ symptoms, too often renders Parkinson’s too risky a business case for pharmaceutical companies.
Nevertheless, there are now several new scientific avenues which are being explored as targets for the development of therapies with good prospects of delaying, stopping or even reversing the condition. But with the costs of conducting robust and meaningful studies of any one of these being so prohibitively high, there is still huge reluctance for industry to enter the Parkinson’s arena for reasons of commercial viability alone. What is needed, therefore, is a means of de-risking investment by means of a scientific screening process.
A Way Forward
In 2012 The Cure Parkinson’s Trust (CPT) instigated its Linked Clinical Trials initiative (LCT). The objective was to apply new scientific and clinical knowledge gained about the condition and to find ways of addressing the barriers which stand in the way of translating new science into new treatments.
Linked Clinical Trials
In 2012 and 2013 CPT compiled comprehensive dossiers of over a hundred prospective therapeutic targets for Parkinson’s, all of which have the necessary safety profile to be administered to people living with the condition now. These dossiers were scrutinised and prioritised by a specially selected group of nine of the world’s most renowned Parkinson’s specialists (‘The Committee’) at meetings held in Grand Rapids, Michigan and Cumberland Lodge, Windsor respectively. A shortlist was drawn up of the drugs with the most likely prospects of slowing, stopping and/or reversing the progression of the disease.
The idea behind this initiative is to accelerate recent breakthroughs in Parkinson’s, and to reduce the costs and time incurred in ‘standard’ trials. This is achieved by focusing on specific data from the trial which is sufficiently robust to determine whether further investigation and financial investment are merited in subsequent large-scale studies. Also, the structure of LCT is such that it allows direct comparison between prospective therapies by harmonising trial designs and outcomes.
In recognition of the importance of this project, CPT intends to pool resources with other funding bodies and expertise to accelerate the progress of this exciting and cutting-edge clinical initiative with a sense of real urgency.
Tom Isaacs was diagnosed with Parkinson's at the young age of 27 and since then has done everything he can to raise funds, heighten awareness and find a cure.
Having completed his highly successful 1,250 mile sponsored walk in 1999, Tom left his job as Director of a London property company in April 2002 to undertake his Coastin' challenge. By April 2003, Tom had walked 4,500 miles around the British coastline, climbed the highest mountains in England, Scotland and Wales and run the Flora London Marathon, raising over £350,000. In 2005 he was elected UK Charity Personality of the Year and a year later he co-founded The Cure Parkinson’s Trust, an organisation of which he is President and which has committed over US$9 million to research around the world.
Tom was a Board Member of the European Parkinson’s Disease Association from 2005 until 2010. Tom acted as the patient representative on the Steering Committee and chaired the Patient Advocacy Committee for the World Parkinson Congress in 2010 and 2013. He made a total of 15 speeches at the two events. He is also a leading contributor to the SENSE-PARK project which is a European funded initiative establishing more personalised, objective measuring devices for people with Parkinson’s and those who treat them. He is also a co-founder of Parkinson’s Movement.
In 2012 Tom was awarded the prestigious, Dr Rana International Parkinson’s Community Service Award. He was also one of the torchbearers in the run up to the 2012 Olympic Games.
Tom has written a book “Shake Well Before Use” about his walk and his experiences with Parkinson’s which he conveys with passion, optimism and humour. He speaks regularly about his condition and the ability of people with Parkinson’s to inject urgency into progressing the delivery of new therapies to the clinic.
I believe one of the biggest challenges we face with Parkinson's is the management of our therapies. The medical world tends to rely heavily on prescription drugs and surgical procedures such as Deep Brain Stimulation (DBS) to help us control our symptoms.
I do not feel qualified to write about DBS surgery as I will not consider this treatment. This was a decision made after much thought, research, and discussions with family and friends. I am sure others will speak on this topic.
While I am incredibly grateful for medications which make it possible for me to continue walking and functioning as normally as possible, they also create other issues. Drugs do not have side effects, they have effects, and we need to pay attention to all the effects of the drugs we are taking. In Parkinson's disease, this can be very challenging as so often we are taking 6 or 7 different drugs in our attempt to manage our health. Questions need to be asked: Are we being prescribed another drug because of our Parkinson's or is it given to us to help us cope with the effects of another drug? Are all these drugs necessary? Are there other ways to treat the symptoms with exercise, diet, or lifestyle changes?
It is often said, it takes a cocktail of drugs to treat Parkinson's. This cocktail can include a dopamine agonist, Selegiline (as a neuroprotective), Comtan (makes Sinemet more effective), Provigil (prevent daytime sleepiness), a drug to help sleep at night, and an anti-anxiety drug as well as Sinemet. It can be almost miraculous in helping people with Parkinson's to live a normal life or it can be a time bomb resulting in physical and behavioral effects such as obsessive/compulsive disorder (compulsive gambling, eating, shopping, sex), disinhibition, hyper sexuality, delusional behavior, increased heart rate, and weight gain, among other things.
The most frightening part of this is how the effects can creep up slowly over time to the point where we don't realize the medications are causing these effects until they have damaged our relationships with friends, family, and coworkers, sometimes irrevocably.
So what can we do?
I would like to see a team approach to Parkinson's disease with neurologists, physical therapists, nutritionists, and psychologists working together with patients and their partners or advocate of their choice. Drugs need to be prescribed and monitored very carefully and alternatives to drugs should be considered before simply automatically prescribing one more medication. Experts agree that exercise can improve symptoms of Parkinson's disease and may even delay its progression. I would like to see more studies on the effect of diet on Parkinson's and also studies on the use of Cannabis with Parkinson's.
More than anything, I would like to see a change in attitude towards the management of Parkinson's and other chronic disorders. Can we find a balance between medication and lifestyle changes? Can we be less dependent on pharmaceutical drugs to make us “normal”?
How normal do I have to be, anyway?
To learn more about Terri, visit her blog at www.studiofoxhoven.com/parkjournal
Terri Reinhart was diagnosed with Parkinson's disease at the age of 49. She is a retired kindergarten teacher and a freelance artist and writer. She lives in Colorado with her very patient husband, Chris, their daughter, Emma, and an assortment of chickens, geese, rabbits, as well as Lexus, a service puppy in training.
Like most Parkinson’s organisations, the European Parkinson’s Disease Association (EPDA) believes there are many hurdles to overcome if people with Parkinson’s (PwPs) lives are to improve across Europe and the globe.
But, perhaps unusually, the EPDA’s priorities are not focused on research for new treatments or even a cure. Instead, the main challenges from our organisation’s point of view revolve around the fact that the majority of PwPs are not currently able to lead a dignified life that they are in control of.
One of our priorities, therefore, is to positively influence the key Parkinson’s stakeholders to challenge existing mindsets, shift attitudes and remove the hurdles that prevent PwPs from receiving early and appropriate treatment as well as individualised care.
Here’s a little context that explains our position. The EPDA’s Charter for People with Parkinson’s was launched with the support of the World Health Organisation in 1997. It stated simply that people with Parkinson's have the right to:
In short, PwPs need time, tailored care, understanding and respect. But all the evidence shows that 17 years later (and counting), this is still far from a reality for most PwPs in Europe.
Equal opportunities for PwPs are lacking – in terms of treatment, clinical management and proper, well-informed diagnoses. Too many patients are not able to access Parkinson’s specialists and/or multidisciplinary teams. Quite simply, PwPs are not receiving the right treatment at the right time. (See www.epda.eu.com/move-for-change for more about this.)
Another serious and under-recognised challenge is the need for our society as a whole to accept that it’s not just dealing with single diseases anymore but groups of diseases. The longer we live, the more diseases we will acquire. A rather dismal message, perhaps, but once you’re over 65 the fact is you’re likely to have at least three or four diseases moving through your body.
In 2011, the European Brain Council revealed that the annual cost of the brain diseases across Europe was practically €800 billion. To make matter worse, to get the existing medications to PwPs and other patients through the European regulatory process currently takes nearly 13 years. This is something we’ve all got to work together on – to try to get those medicines from the bench of the scientists to the people living with these illnesses much faster without in any way jeopardising safety and efficacy.
But there are many ways we can all help to improve PwPs’ quality of life. For example, the EPDA has recently embarked on the ground breaking European Parkinson’s Strategic Health Initiative (SHI). Entitled ‘My PD Journey’, the SHI seeks to unite all stakeholders involved in the management of the disease in order to develop comprehensive and tailored strategies that will ultimately result in the right treatment and care at the right time for each individual. It has the potential to improve the lives of many thousands of people.
We believe this initiative is just one of many that can offer proper information and deliver adequate solutions to PwPs and their families. We need to make European – and the world’s – healthcare systems more patient-centric… and fast.
For more information about the SHI, contact John@epda.eu.com
The European Parkinson’s Disease Association (EPDA) is the only European Parkinson’s disease umbrella organisation. Founded in 1992, we are a non-political, non-religious and non-profit organisation. Representing 45 member organisations, we advocate for the rights and needs of more than 1.2 million people with Parkinson’s and their families across Europe.
We exist because the facts relating to Parkinson’s in Europe are startling. The disease is the second most common neurodegenerative disease (after Alzheimer’s) and the number of people living with the disease is forecast to double by 2030 as the population ages. The current economic impact of the disease is enormous – the annual European cost is estimated at €13.9 billion – yet Parkinson’s is just one of many neurodegenerative disorders, which will surpass cancer as the most common group of medical conditions by 2040.
Because Parkinson’s is a chronic illness, the issues that face persons with Parkinson’s disease vary with the stage of the disease. One challenge for research is how best to improve quality of life across the span of this disease. This past fall and winter the NINDS and the Parkinson’s community wrestled with this challenge in developing research recommendations for Parkinson’s disease (PD recommendations for research). Two main themes emerged. First was the emphasis placed on developing better treatments for the non-motor manifestations such as declining cognition, dyskinesias, and impaired balance, autonomic and gait dysfunction. These troublesome symptoms usually occur in the later stages of the illness and persist despite the beneficial treatments for tremor and bradykinesia, ie., medications, brain stimulation, and exercise. Research avenues include pharmacologic or electrical modification of brain circuits to enable the brain to compensate for the pathologic changes that have already occurred. The other challenge relates to the second main theme – to develop a means to slow or stop the spread of pathologic changes in the brain that underlies the progression of symptoms.
Parkinson’s disease is now thought to begin decades before motor symptoms occur. New symptoms sequentially plague patients as the pathologic changes spread from one part of the nervous system to another, and when the brain is severely affected the previously beneficial treatments lose their effectiveness. The scientific community seems rightly focused on discovering how to prevent progression of the disease with the concomitant spread of the underlying pathology. Recent evidence suggests this could be the consequence of propagation and transmission of synuclein aggregates from cell to cell. To test treatments in patients we will need to develop measures (biomarkers, brain imaging reagents) to track the spread of synuclein pathology in people. In the earliest stages, before symptoms of PD emerge, synuclein aggregates may found in the autonomic and enteric nerves and/or olfactory system. It’s exciting to consider that a treatment to prevent spread of synuclein aggregation, combined with a means of diagnosing PD before symptoms even occur, could eradicate the disease entirely. Preventing spread from brainstem to cortex might preserve cognition.
The NINDS currently supports a range of clinical, translational and basic research programs to accelerate the development of new therapies. One highlight is the Parkinson’s Disease Biomarkers Program (PDBP), focused on the discovery of biomarker candidates for early detection and measurement of disease progression. Another is the PD induced pluripotent stem cell (IPSC) consortium which has generated IPSCs and dopamine neurons from skin biopsies of affected individuals with known mutations (NINDS IPSCs for PD research). Additional NINDS program information is available on the NINDS PD Research Web.
Challenges are many but the science is continuously drawing us closer to understanding this complex condition. With luck and resources clever investigators seem on the edge of real breakthroughs. How close we are is difficult to predict, but we have a roadmap to get us there.
The mission of the National Institute of Neurological Disorders and Stroke (NINDS) is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.
To support this mission, NINDS:
The greatest issue facing Parkinson’s patients today is the lack of any therapy that could slow the disease progression. In its early and mid stages, Parkinson’s primarily affects the dopamine system and, as we have seen in studies of placebo response, the dopamine system responds to hope. The impact of a therapy that can slow the disease would go beyond just the benefit of neuroprotection, it would help people with Parkinson’s to see a brighter future. Research has shown that the placebo response in randomized trials is affected by the probability of the subject receiving active medication: the higher the probability that a study participant will receive the study drug, the higher the placebo response in the control group. Hope is a powerful therapy for Parkinson’s, and, if coupled with a drug or intervention that truly slows the disease, we could see dramatic benefits.
The most important way to accelerate R&D for developing disease-modifying therapies would be to establish a definitive measure of the disease, and the most effective way to measure the disease would be to measure the changes in neurons that result from the disease. This will most likely require new imaging techniques such as a novel radioligand or high-resolution MRI modalities. Once we can measure the disease, than approaches to directly attack the pathology such as by attacking the protein alpha synuclein can be tested for efficacy directly.
Parkinson’s is a disease of loss—the loss of crucial neurons in the brain—and research is far from developing techniques to replace that lost tissue. However, if we could stop the disease so that symptomatic therapies could keep working as well as they do in early disease, we could change the course of Parkinson’s and deliver a better life for people with Parkinson’s.
Dr. Schmidt joined the National Parkinson Foundation (NPF) as Chief Information Officer and Vice President, Research and Professional Programs in June 2009 where he is responsible for the Parkinson’s Outcomes Project, a longitudinal study of Parkinson’s disease to identify best practices in care to achieve optimal patient-reported and clinically measured outcomes. With over 13,000 clinical evaluations of over 7,000 patients, the Parkinson’s Outcomes Project is the largest clinical study of Parkinson’s disease ever conducted and includes the largest set of patient-reported outcome measures ever collected in a prospective study.
Prior to NPF, Schmidt was an investment banker in Norwalk, CT., president a software company supporting chronic disease management, and COO of an on-line education joint venture of Oxford, Stanford, and Yale universities. He is an active member of several trade groups and is widely published in both scientific and trade journals on issues around medicine, health information technology and finance.
Dr. Schmidt earned his bachelor’s degree at Harvard University and was awarded an M.S. and Ph.D. from Cornell University, Sibley School of Mechanical Engineering where he studied gait and balance and total joint replacement. He completed a fellowship at the Hospital for Special Surgery in New York.
How can we find better treatments for Parkinson's disease (PD) at a faster pace? At the Parkinson’s Disease Foundation (PDF), we argue that one solution is ensuring that people with Parkinson’s are primary partners in research alongside scientists, industry and government. The innovation of patient engagement is something that the medical community is starting to recognize as a necessary component to patient-centered research and care delivery.
While patient engagement may not sound revolutionary, it represents a marked departure from traditional models of medical practice and research in which the doctor holds the knowledge and makes the decisions. Patient engagement means collaborative partnerships in which patients have a say in which treatments are on the market and which are best for his or her care.
Looking back at how the concept has evolved, we see that in the early stages, the buzz was all about personal engagement, about a person’s role in his or her own care, with his or her doctor and health care team.
But over time, our understanding of patient engagement is evolving into something broader — from ways in which informed patients can help improve their health status as an individual, to ways in which patients can improve care and accelerate therapies for the community as a whole.
At PDF, our commitment to fostering and advocating for increased patient involvement began ten years ago through the formation of the PDtrials coalition and website. These programs sought participation from people with Parkinson’s in research, primarily as informed participants in clinical studies. Much like other agencies, PDF's understanding of patient engagement is evolving over time — we now recognize that although study participation is critical, it is just one of the many ways that people living with Parkinson’s can improve clinical research so that better therapies are available as soon possible.
So in 2008, PDF formed Parkinson’s Advocates in Research (PAIR). This program provides ongoing training and support to people with Parkinson’s and care partners to create a foundation from which they can become involved in decision-making at each phase of the drug development process.
To date, the PAIR program has trained 230 people with Parkinson’s and care partners from 42 states to serve as Research Advocates who “pair up” with research institutions and scientists to bring about better treatments at a faster pace.
What is most exciting to us – after years of PDF Research Advocates calling on agencies, researchers and companies to say, in effect: we want to help you do your job better so that our lives improve – is that now the same entities are calling us to learn how Research Advocates can join them in their work.
We know that when advocates from other disease communities demanded a role in research it tipped the balance, and helped bring about new treatments. Parkinson’s disease should be no different.
Robin Elliott has led the Parkinson's Disease Foundation, Inc. (PDF) since October 1996. In the last 17 years, Mr. Elliott’s vision has ushered in a new age for PDF, fortifying PDF’s programs of research, education and advocacy for the Parkinson’s community. Under his leadership, the professional staff has grown from just four full-time employees to more than 20 and the PDF budget has more than tripled, rising from $2.7 million in 1996 to over $10 million in fiscal year 2013.
He has been active in fostering collaborations amongst Parkinson’s organizations, including negotiating a merger with the Chicago-based United Parkinson’s Foundation in 1998. He also played an instrumental role in the creation and organization of the World Parkinson Congress in 2006 and in the conception of the PDtrials campaign, an initiative of the major Parkinson's patient voluntary groups to accelerate the development of new treatments for the disease.
Active in development, communications and nonprofit management in New York City for more than 30 years, Mr. Elliott has served as vice president for development and external affairs at Teachers College, Columbia University (1988-95) and (with the same title) at Hunter College, The City University of New York (1982-88); as deputy to the Chancellor for University Relations at the City University of New York (1979-82); and as director of information and education at the Planned Parenthood Federation of America (1971-79).
Mr. Elliott currently serves as Chairman of the board for the Community Health Charities of New York, Treasurer of the board for the American Society for Experimental NeuroTherapeutics (ASENT), Chair of the board for the American Brain Coalition (ABC) and board member of the Empire State Stem Cell Board (ESSCB). He was formerly Chair of New Yorkers for the Advancement of Medical Research, a pro-stem-cell research coalition of disease advocacy groups, scientists and universities, and citizens’ groups.
Mr. Elliott grew up in southern England and received his formal education at Bradfield (a preparatory school; 1954-59); Magdalen College, Oxford University (B.A. in Philosophy, Politics and Economics, 1962); and Columbia University (M.A. in American Government and Politics, 1965).