“Neurologists are a CRAZY bunch! Crazy enough to believe that if they can find a cure for ONE neurodegenerative disease…..they can find a cure for ALL!” ( Perception)
Well, I guess I always knew I was a little off my rocker…..so glad to be part of the dreamers…..it was the love of neuroscience and neurology that propelled me to study medicine and become a neurologist at a time when going into NEUROLOGY was EVEN LESS popular than it is now…..
The last neurology breakthrough had not occurred for at least 30 years previously and the BRAIN was EVEN more of an enigma than it is today. Neurology was tongue and cheeked referred to as “diagnose and adios!” by my classmates. I was often poked fun of by my friends because of this – I was considered a bit of a fool for going into such an obscure field when there had been no matches in neurology in the previous year and there were none in the year following my class…..
But, once a dreamer always a dreamer……
While completing my undergraduate degree at Penn, I had had a glimpse of what it could be ….the great power of the mind, the resilience of the human spirit, and the capacity with which individuals with severe traumatic brain injuries and devastating neurologic abnormalities were able to cope and persevere…..I knew they deserved better and I would do my best to help in what ever way I could advance that knowledge and understanding of the brain…. During those years, I often volunteered as control subject for various neurological and psychological studies…little did I know then that some day I, too, would become a Parkinson’s patient in need of the miracles of science to help my life become better and depend on those volunteers like myself to find answers and hopefully someday a cure!
As a resident, I volunteered in a few other studies that were testing specific medications for migraines ….and nothing is more rewarding than to see those studies completed and drugs come to market and even though for instance in one study I was given a higher dose which was not approved because not statistically significantly better than a lower dose it helped me in my practice years later. For instance, if someone was prescribed that medication and took a larger dose because headache would not respond …..the fact I had been on it for months without problems and others I knew had been on it without problems I was not so alarmed …although warned dose not FDA approved at such doses!
Interestingly, as a medical student I designed a visual study to test a particular hypothesis in schizophrenics……and now 20 years later a very similar design is being tested for other dopamine systems like Parkinson’s. Parkinson Plus and Huntington’s Disease and the preliminary findings will be displayed in a poster at WCP 2013 in MONTREAL- very exciting since I am also one of the test subjects in this….. perhaps this will be one way we might be able to early diagnose PD or differentiate early on between the various dopamine illnesses! So, Stay tuned!!!! Data will be presented by Dr. Mya Schiess and her colleagues from UT Houston Medical Center.
So, as you can see ever since my undergraduate years I have participated in many research projects as a subject and have conducted many of my own….both in the basic science arena but primarily as a physician and now as a patient as well helping to understand the role of sleep and sleep disorders in PD as well as other studies like one mentioned above.
I can not underscore the significance that research both in the basic science and in the clinical arena represent for present and future generations of patients afflicted with devastating neurodegenerative diseases like Parkinson’s and Alzheimer’s to name a couple.
Thanks to the countless volunteers over the years who have given of themselves unselfishly along with the work of visionary neuroscientists we were able to have a boom in knowledge and medical treatment that began in the 90’s known as the DECADE of the BRAIN. But, still we find that even though we have come so far our knowledge is still far inferior and lacking in many ways and the cure to Parkinson’s and many other devastating neurodegenerative diseases yet alludes us…..but I have complete confidence that some day WE WILL UNLOCK the MYSTERIES within ……
But, in order to do this we all need to work together in unison for a common goal- the overall well being of Human kind and ease of suffering of all involved in such devastating neurodegenerative diseases.
So, how can you help?
……by playing a part in Parkinson’s research.
You can participate in research as a patient or as a control…….
You maybe asking yourself the following questions:
I want to participate in a trial?
But, I am not sure if I can or if I qualify or which one to choose since there are so many? How do I know what trial is best for me?
First of all we have to remember that by definition a clinical TRIAL is an EXPERIMENT in which the outcome (answer) is not known!!!!
Therefore, if your sole purpose for participation is your individual well being you may be in for a world of heart break and disappointment!
Research should be approached as way of “paying it forward’!
Your sacrifice will be rewarded in the health of others to come and it is also a way of giving thanks to those that came before us and have sacrificed so much so that we may have the treatments we currently enjoy……
There are different trials and experiments with different outcomes and it would serve you to be well versed in order to make best informed decision FOR YOURSELF giving YOUR PRESENT CIRCUMSTANCES!!! ( This is crucial to remember because circumstances change and disease burden also changes usually increasing over time making some trials more difficult to enter into or be excluded from or included).
Always take into account ALL of your circumstances social, financial, medical before making a commitment or a decision that could potentially negatively impact your life
(because results are not known unless doing a phase 4 study trial in which results are already known for most part but need more specific information).
Here are the different types of trials:
first time trying new drug that has been extensively studied in animals or laboratory and hold promise. These are small 15-100 patients
Establish side effects/ tolerability
Observing patient response
Observe drug effect on patients disease
Establishing efficacy of drug compared to placebo – usually small
Confirmation of safety and efficacy-very large studies> 1,000
Post marketing to gather further information , optimal use, treatment-risks and benefits
There are several types of trials:
Quality of life trials
Compassionate use trials
The way to maximize success and a positive outcome for you is to consider these things before you engage in any trial: Look at what you are trying to get out of it ….is it purely to advance science or are you also hoping to gain something….if you are hoping to gain something …are the expectations realistic? Are they in sink with the goals of the study? Never participate to please your doctor…that will only lead to heart break when results don’t yield what you hope for….
because for us physicians and clinicians even when a study is negative we gleam and are able to learn lots from what went wrong….but even though it may not help YOU in your present situation….BUT MOST DEFINETELY HELP THOSE in FUTURE GENERATIONS!!!!
consider your physical limitations and capabilities and also that of those who care for you…..Also, keep in mind to ALWAYS ask the researcher if the participation in this particular trial will preclude you from getting other surgeries or treatments!!! MOST IMPORTANT- YOUR WELL BEING comes FIRST!
But, Always look upon your participation in a trial as a rare privilege and honor to change the course of science and human nature!
If you follow these simple guidelines you should not only get benefit, and contribute to science BUT also have FUN!!!
Don’t forget research is a TWO-WAY street OPEN communication is VITAL…..let your clinician know your concerns and also what interest and issues you have to better fit a study that suits your needs…..
With your help and participation and the growing scientific knowledge inspired by you, I believe that WE can FIND a CURE for Parkinson’s Disease…..Until then you can join me in my quest and be content in being called dreamers….for it is the dreamers with an exorbitant imagination that have the POWER to change the world! Even if we are but a CRAZY BUNCH!
Join us at PDF’s Parkinson’s Advocate in Research (PAIR) program to bring about better treatments and quality of life to all of us who live with Parkinson’s Disease.
Dr. M. De Leon is a retired movement disorder specialist, PPAC member and research advocate for PDF; Texas state assistant director for PAN (Parkinson’s Action Network). You can learn more about her work at http://www.facebook.com/defeatparkinsons101 you can also learn more about Parkinson’s disease at www.pdf.org or at www.wemove.org
for more information about research trials available for Parkinsons go to http://www.foxtrialfinder.org
http://grants.nih.gov/grants/guide/notice-files/NOT-NS-13-035.html a place where you may go to put your input as to what is needed in research to find cure for PD