Balancing Safety and Urgency
During the last two decades of living with this challenging disease, as a physician and patient, I have often been asked about various medical issues as they relate to Parkinson’s disease. This includes the latest research findings. When faced with the question about the progress of Parkinson’s research and our proximity to a cure, my answer used to invariably be “They’re working on it.”. Although there is a worldwide community of clinicians and researchers devoting a great deal of time and attention to finding better treatments and a cure for what is currently incurable, in retrospect, it was a very ill-informed answer. As patients, we must recognize that “they” cannot further their work without “us”, that we are an integral part of this process.
As patients we face the challenges of this progressive degenerative neurologic disease on a daily basis. It affects all aspects of life – physical, emotional, mental, social – it is pervasive and unforgiving. We live with the knowledge that currently there is no cure. And so, we strive to maintain our quality of life, frustrated at times that the gold standard treatment for our disease has been around for almost 60 years and although effective, has its limitations. We need better treatments and ultimately a cure.
Likewise having been involved in the research process at different points of my career, I know that despite a researcher’s sincere desire to find the answers we seek, there are constraints of the governing and review process that slow down their work. Their hands are often tied and sometimes the best and most promising ideas never make it through the pipeline due to procedural constraints and funding issues.
Once in the pipeline, approximately half of the drug development time is spent in clinical trials. But researchers cannot conduct these vital studies without our participation and close to 85% of all clinical trials are delayed due to recruitment difficulties and a shocking 30% fail to recruit a single subject. Any delay a study faces due to difficulty in finding participants, leads to a huge waste of resources, money and most importantly time - time that those of us that must wake up each day facing the burden of Parkinson’s would rather spend fully functional and productive.
The difficulties in clinical trial recruitment are an unfortunate reality and I have long been a vocal advocate for clinical trial education which I believe is key to increasing informed and continued research participation. There are many benefits to participating in research. Research often doesn’t even involve testing a new treatment and even if it is an interventional trial, they are generally safe. The drug development process is heavily regulated and monitored by the country’s health authority with our safety in mind. By the time a drug is tested in humans, researchers have already had to do a tremendous amount of pre-trial work to show scientific evidence that their intervention will likely work and is safe.
But there are of course, risks. The harsh reality of those risks came to light just recently. We lost five people in our community during a study directed towards the pursuit of a better treatment for all of us. Although careful review of the events is ongoing, this type of news alerts us to the risks that participants in any clinical trial take, a risk we consent to, a remote possibility, but a possibility nevertheless.
It begets a fundamental ethical question: Is the advancement of medical research that may provide relief to the millions of people globally that are living with Parkinson’s disease more important than the safety of the individual? These shouldn’t be mutually exclusive.
We want to relinquish Parkinson’s disease to the history books. We are no longer willing to wait and have become increasingly frustrated with a system that we feel is too slow, too timid, too wrapped up in process and not cognizant of the urgency that the Parkinson’s community feels. We demand for the process of review and approval to be streamlined, for the timeline to be shortened. But the drug development process has been established for a reason. It is heavily regulated and monitored with our safety in mind. How do we speed up a practice that is designed to safeguard our safety? How do we safely accelerate a process that is inherently risky?
Therein lies the conundrum. How do we determine the balance between safety and urgency? Unquestionably the search must continue, science must continue to advance. But do we have a role in improving the safety of this science? It is in our best interests to ensure that our voices as a community are heard, that we have input into the clinical trial process. Although patient involvement is recommended in trial design, there needs to be more robust participation and input. Researchers first and foremost must know our concerns so that the outcome measures of any research that is undertaken, are relevant. That trials and their inherent risks are directed towards the most pressing needs of our community. Once established, patients need to be part of the protocol development, to ensure the protocol being used meets the needs of participants and that possible adverse outcomes are defined with patient experience in mind. And of utmost importance as patients, is to ensure that the information given to potential participants is fully complete, written clearly and is understandable by those who may not have any scientific background. Informed consent must be truly informed. And after completion, we must advocate for transparency and demand the reporting of all adverse events in published journal studies.
We must also advocate for continued support for the development of protocols that will ideally reduce the numbers of participants that are required. Supporting genotyping research for example so that more focused patient populations can be studied which in turn may reduce the number of trial participants or at least focus on that part of the population that will receive the most benefit.
Like most aspects of our world, technology is sure to have an impact in the field of medical research. For example, in silico clinical trials - individualized computer simulations that are used in the development of a medical treatment - will hopefully become reality for use in neurological research. As would hopefully tissue chips, tiny bioengineered systems that mimic the much larger complex organs and tissues of the human body. But our current understanding of human biology and physiology and the current state of technology does not yet allow the creation of virtual patients that duplicate the complexity and individual variability of humans. And beyond that challenge will be changing the system, investment in and the adoption of a new way of doing things. But there is still hope that as technology advances, there will be a time when the drug development process will not require human experimentation. We must work towards a time where no one is put at risk.
I personally plan on continuing my participation in research asking pertinent questions and supporting those studies that are in alignment with my level of acceptable risk. As I remember those five individuals that recently lost their lives I am truly filled with sorrow, but also a sense of deep gratitude. Gratitude for them and the thousands of volunteers that have come before me, sacrificed far more than I ever have, to advance the science. And I look to the thousands in the future that will continue the science either through trial participation, fundraising or advocacy and support, knowing that eventually there will be better treatments and a cure based on their dedication. For all those that further the progress that may someday alter the trajectory of my life, I am eternally grateful.
Soania Mathur, MD presented at the Third World Parkinson Congress in Montreal, Canada and the Fourth World Parkinson Congress in Portland, Oregon. Dr. Mathur is an active speaker for Parkinson Canada at patient-directed conferences and also serves as a resource for education projects. She works with The Michael J. Fox foundation for Parkinson’s Research and serves on their Patient Council. She is a member of The Brian Grant Foundation Advisory Board that helps to create educational programming. She is the founder of Designing A Cure Inc. (www.designingacure.com) which was initially created to raise funds directed towards research and awareness of Parkinson's disease and now serves as a platform to educate and inspire those living with this disease to take charge of their lives, to live well with Parkinson's.
Ideas and opinions expressed in this post reflect that of the author(s) solely. They do not reflect the opinions or positions of the World Parkinson Coalition®