Find Out What Our Researchers Are Working on Right Now
The Parkinson’s Foundation has provided funding to more than 550 researchers since 2010. These scientists and researchers receive Foundation awards to improve our understanding of Parkinson’s disease (PD), which will ultimately lead us to better treatments and a cure. We recently sat down with nine of them to get the latest highlights of their ongoing PD research:
Stay up-to-date on the latest Parkinson’s research:
Q&A with Parkinson's Specialist and Nurse Practitioner
Stephanie De Santiago, DNP, AGNP-C, has a doctorate in nursing (DNP) and is an adult-gerontology nurse practitioner at Banner Sun Health Research Institute, where she specializes in movement disorders and cognitive neurology. She is also a faculty associate for the Doctor of Nursing Practice program at Arizona State University. In this Q&A, Stephanie highlights the Nurse Navigation Program, a program she created at a community wellness center where nurses conduct discussion sessions with members and their care partners to address Parkinson’s disease (PD) management strategies.
The Parkinson’s Foundation award funded the addition of a Nurse Navigation Program to a Parkinson-specific community wellness center. The 12-week program ― available to wellness center members and their care partners ― was conducted by nurse navigators who held weekly 45-minute individual sessions to discuss critical PD management topics.
Each week, the 24 participants discussed topics such as motor and non-motor symptoms, medication management, hospital preparedness, fall prevention and more. Participants were encouraged to talk about their questions and concerns. The entire plan will soon be published in a scholarly journal.
What self-management tips can you recommend for someone with Parkinson’s?
Education. An important aspect of improving self-management of any disease. The more you understand the disease and what to expect, the more confident you will feel in your own ability to manage new symptoms or challenges.
Social support. Finding the support from others in a similar situation is a key factor in self-management of any chronic, progressive disease. Have a support system in place to problem-solve with and open up to without fear of judgement.
Exercise. People often feel powerless over PD which can decrease self-management ability. Daily exercise is not only great for the body, but it can also restore a sense of control over PD that may have been lost upon diagnosis.
What role do nurses play in community wellness for people with Parkinson’s?
Nurses play an important role in creating and sustaining an interdisciplinary approach to wellness for people with PD. Often there is little communication between members of the healthcare team and visits with movement disorder neurologist. Having a nurse readily available in the community wellness setting can be a great added resource for addressing any issues that come up between neurologist visits.
Nurses can trouble-shoot issues or communicate the situation to the appropriate healthcare team members, taking the burden off the already overwhelmed person with PD or care partner. Additionally, nurses can support patients and families by giving them valuable education and guiding them through the course of the disease. Ultimately, this can instill in people a sense of control over PD.
How did being an Edmond J. Safra Visiting Nurse Faculty Scholar influence this project?
My experience as an Edmond J. Safra Visiting Nurse Faculty Scholar was pivotal to the development and execution of this project. My Safra mentor, Edie Simpson, helped me formulate the premise of this project.
Preparing for the project, I learned about PD management, which helped me create nurse navigation program topics. Lastly, during the clinical experience I gained through the Safra program, I was able to work directly with patients and I met many great movement disorder specialists who I collaborated with not only during the project, but also now, professionally, as a nurse practitioner.
What changes did you see in participants after they completed the nurse navigation program?
Our results showed that after a year of progression of Parkinson’s resulting in an expected decline in quality of life, nurse navigation was able to improve self-efficacy for managing PD. This means that having a nurse navigator as an additional resource for people with PD can improve their confidence in their own ability to manage their PD despite the known progressive nature of the disease.
What is the “Self-efficacy for Managing Chronic Disease 6-item Scale”?
It is a simple scale for researchers to evaluate how much confidence a person has in their own ability to manage a chronic disease, such as Parkinson’s. It is useful to track changes over time. Other wellness centers and programs that evaluate self-efficacy changes can utilize this scale.
What Parkinson’s Foundation resources do you, your team or patients utilize?
Parkinson’s Foundation resources were crucial for this nurse navigation program. We used the Foundation fact sheets and handouts that are available online at Parkinson.org/Library for all of the patient education involved in the program. These are a great reference for people with PD because they are easy to read and understand and they cover almost any topic relating to PD.
Another extremely beneficial resource is the Aware in Care kit. These kits help keep people with PD prepare for possible hospitalization. Everyone with a PD diagnosis needs to have an Aware in Care kit that is appropriately packed in order to advocate for themselves in the hospital setting.
Phlegm-busting Drug Ambroxol Shows Promise in Parkinson’s
One of the major genetic risk factors believed to contribute to the development of Parkinson’s disease (PD) is having a mutation in the gene called GBA1 (glucocerebrosidase). Unable to do its job correctly, this damaged gene leads to the build-up of unhealthy, misfolded clumps of alpha-synuclein in the brain. These clumps, called Lewy bodies, impact dopamine production and are the hallmark of PD. What if there was a way to prevent the build-up of Lewy bodies in the first place?
A 2020 study published in JAMA Neurology, titled, “Ambroxol for the Treatment of Patients with Parkinson Disease with and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial” (Mullin et al., 2020), investigated whether an over-the-counter cough syrup, called Ambroxol, may be the key. The cough syrup, specifically, an expectorant, is used to break up phlegm.
What is a phase 2a study?
A study conducted in a smaller group size that tests the effectiveness and safety of a drug being tested. Also known as a proof of concept study. In this case, the 2a study sought to find out if Ambroxol biologically impacts people with Parkinson’s.
Shown in previous studies to improve GBA1 function in neurons, this 2a study (known as a proof-of-concept study) set to find out if Ambroxol could cross the blood/brain barrier, and function as a molecular chaperone (in other words, physically help the GBA1 protein function properly).
This 186-day clinical trial of 17 people with PD ― with and without the GBA1 mutation ― involved participants taking progressively increasing doses of Ambroxol in the form of an oral tablet. Baseline measurements included physical and neurological examination, an electrocardiogram, blood sampling and spinal fluid examination obtained by lumbar puncture. Three additional in-person clinical visits were held on day 11, day 93, and day 186. Of note, at baseline, Ambroxol was undetectable in both the blood serum and spinal fluid. All study participants continued their normal L-dopa therapy throughout the trial.
Results
In study participants both with and without the gene mutation:
Ambroxol successfully crossed the blood-brain barrier.
Ambroxol was safe and well-tolerated at the administered dose.
Ambroxol successfully bound to the mutated genes’ protein, which physically helped the protein function properly.
Healthy levels of alpha-synuclein increased in the spinal fluid.
What Does This Mean?
This study showed that Ambroxol is safe to use as a treatment in people with Parkinson’s. Ambroxol may slow the progression of Parkinson’s disease. How? Taking Ambroxol as a medication can prevent the negative effects of the GBA mutation ― including possibly reducing the formation Lewy bodies at the source. Ambroxol shows promise, and warrants further investigation ― including conducting larger, placebo-controlled trials.
Of note, while Ambroxol has been used as a safe and effective over the counter expectorant for adults and children in more than 50 countries for 30-plus years, the administered dose in this trial was approximately 10 times the specified dosage. Additionally, Ambroxol is currently not approved for prescription or over the counter use by the U.S. Food and Drug Administration (FDA) for any indication, at this time.
Learn More
The Parkinson’s Foundation believes in empowering the Parkinson’s community through education. Learn more about the Parkinson’s and the GBA mutation in the below Parkinson’s Foundation resources or by calling our free Helpline at 1-800-4PD-INFO (473-4636).
Malerba, M., & Ragnoli, B. (2008). Ambroxol in the 21st century: pharmacological and clinical update. Expert Opin Drug Metab Toxicol, 4(8), 1119-1129. doi:10.1517/17425255.4.8.1119
McNeill, A., Magalhaes, J., Shen, C., Chau, K. Y., Hughes, D., Mehta, A., . . . Schapira, A. H. (2014). Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells. Brain, 137(Pt 5), 1481-1495. doi:10.1093/brain/awu020
Mullin, S., Smith, L., Lee, K., D'Souza, G., Woodgate, P., Elflein, J., . . . Schapira, A. H. V. (2020). Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial. JAMA Neurol. doi:10.1001/jamaneurol.2019.4611
Sanchez-Martinez, A., Beavan, M., Gegg, M. E., Chau, K. Y., Whitworth, A. J., & Schapira, A. H. (2016). Parkinson disease-linked GBA mutation effects reversed by molecular chaperones in human cell and fly models. Sci Rep, 6, 31380. doi:10.1038/srep31380
As the Parkinson’s Foundation celebrates the distribution of our 100,000th Aware in Care kit, we check in with our Aware in Care Ambassador, Myra Hirschhorn, to learn about her work educating health professionals and people with Parkinson’s about the Aware in Care kit and the importance of advocating for oneself through education in order to achieve safety across hospital care.
1. Why did you become an Aware in Care Ambassador?
The Aware in Care kit is such a great solution to what has been a tremendous concern to folks living with Parkinson’s disease (PD). I am pleased to be a part of bringing this excellent resource to folks who can benefit from having the kit ready and available to them, should it be needed for a planned or emergency hospital visit. Hats off to the Edmond J Safra Philanthropic Foundation and the Parkinson’s Foundation for developing this!
2. How has your role as an Aware in Care Ambassador helped you connect with larger organizations to educate the community about the hospitalization kit?
The Lee Silverman Voice Therapy Global (LSVT Global) holds a two-day program for the purpose of educating physical therapists, occupational therapists and speech-language pathologists on valuable information and techniques to assist folks with Parkinson’s. On the second day, folks with PD attend to learn about the program. Several years ago, I began sharing Parkinson’s information at these programs held near my home in South Jersey and included details on the Aware in Care kit as a way to engage with both health professionals and people with PD while educating them on this incredible resource.
Recently, representatives of both the Aware in Care program and LSVT directors agreed to have Aware in Care Ambassadors attend the LSVT Global Seminars throughout the country to share the information in a similar manner. In addition, the kit information cards will be added to the welcome bags given to therapists and people with Parkinson’s. With participating health professionals better informed about the Aware in Care campaign, our hope is that they will return to their clinics better equipped to provide safer hospital care!
3. Can you explain the doctor letters you use as Ambassadors and how they can be used by others with PD?
The new doctor’s letter shares important information about PD to further inform the medical staff during a planned hospitalization. I hope to share this with movement disorder specialists in my area as they may want to provide them to their patients to distribute. I will also share the letter with those in my support group and my email distribution list. I will encourage patients to have it signed by their movement disorder specialist and place it in their Aware in Care kits to be used if/when they are hospitalized. Our goal is for the letter to complement the Aware in Care kit to facilitate safe visits to the hospital.
4. What advice would you give someone who is hesitant to share the kit with hospital staff?
Understandably, you may feel uncomfortable with the need to inform the nurses and staff members about Parkinson’s, but it is important to remember that by doing so, you are making the best situation possible for yourself or for your loved one. Certainly, we want you or your loved one to improve rather than have a setback due to a lack of knowledge on the part of the hospital staff.
My advice is to review the information in the Aware in Care kit with a particular emphasis on the nurse fact sheet, the medications with the dosing times and the special considerations. Be open to any questions from the staff member.
5. What is your favorite component of the kit?
Each part really is so helpful and all materials together provide the pertinent information that is needed. If I had to choose, I feel that the Nurse Fact Sheet pad because it provides valuable points to be aware of in addition to the vital information regarding medications. There are plenty of copies to share with all the medical staff assisting the person with Parkinson’s so that each person can have an individual copy, which is key!
Unfortunately, I have heard too many issues that have come up with folks being provided drugs like Haldol and the horrible reaction that follows it. I hope those days will be behind us now as we take the step to advocate for ourselves and educate those who are caring for us!
At the Parkinson's Foundation, one of the ways we make life better for people living with Parkinson’s disease (PD) is through research. Every day, your generous donations help us fund cutting-edge PD research initiatives and support scientists working on the causes and treatments for Parkinson’s that could one day lead to a breakthrough.
In this Neuro Talk, our Chief Scientific Officer, James Beck, PhD, walks us through the research we’re supporting right now and how your support goes a long way at the Parkinson's Foundation.
Neuro Talk: Myths and Realities of Parkinson's Disease
Does Parkinson’s disease (PD) only affect movement? Can doctors predict its progression? Can stem cells cure Parkinson’s? In this Neuro Talk, our Chief Scientific Officer, James Beck, PhD, debunks seven common myths about Parkinson's disease.
Quarantine Playlist: Our Most Popular Podcast Episodes
As coronavirus continues to impact our daily life, we are committed to providing you with the latest updates and ways you can stay engaged with the Parkinson’s community. While you practice social and physical distancing, exercise your brain with our podcast playlist.
The Parkinson’s disease (PD) community often hears that exercise is crucial to living a healthy live with PD, but why does fitness make such an impact? Bas Bloem, MD, breaks down the science behind its benefits to those with PD and how to get started.
In our first podcast episode, Michael Okun, MD, discusses the early signs of Parkinson’s and practical steps to take after the life-changing diagnosis.
Finding the right PD medication balance is often an unpredictable and sometimes frustrating. Irene Malaty, MD, discusses “off” time, what causes it and how it can be managed.
What we put into our bodies is always important, but especially when it comes to people with PD. Can symptoms be managed through what we eat? Can our diet improve the effects of our medications?
Medical marijuana is a hot topic in the PD community for its possible effect on easing PD symptoms such as pain, anxiety, sleep and nausea. Danny Bega, MD, sheds light on the subject.
Driving and Parkinson’s can be a difficult conversation. Lissa Kapust, creator of DriveWise, speaks about driving with Parkinson’s, assessments and making the complicated decision.
Parkinson’s is more than movement symptoms. Non-movement symptoms often go under-reported because many find them too embarrassing to discuss. Tanya Gurevich, MD, discusses sexual health and common solutions to autonomic problems.
We are devoted to the health and wellbeing of the PD community. For our latest information on the coronavirus and Parkinson’s visit Parkinson.org/Coronavirus.
Parkinson’s disease (PD) is now the world’s fastest growing brain disorder, even faster than Alzheimer’s. Ten million people live with Parkinson's world-wide. Over the past 25 years, the number of people with Parkinson’s has more than doubled. At this current rate, the number will double again in the coming generation. In the U.S., the number of Americans with the condition has increased by 35% in the last 10 years alone. We must act to stop Parkinson’s disease.
Four authors (three PD specialists and a neuroscientist) wrote a book called Ending Parkinson’s Disease that highlights the rise of the disease, the factors contributing to the increase and what steps we can take together to help end the disease.
Our prescription for action includes a “PACT” that details steps we can do to:
1. Prevent the disease
Several environmental factors, including air pollution, heavy metals, certain pesticides, and industrial chemicals like trichloroethylene are linked to PD. One pesticide called paraquat more than doubles the risk of developing Parkinson’s disease, kills the weeds that RoundUp cannot and has been banned by 32 countries, including China. Yet, use in the U.S. has doubled in the past decade. Despite a petition signed by more than 100,000 members of the Parkinson’s community, the U.S. Environmental Protection Agency has failed to act. We need to ban this pesticide and take other actions to minimize the risk of Parkinson’s disease from the foods we eat, the water we drink and the air we breathe.
2. Advocate for better policies and resources
In addition to better environmental policies, to end Parkinson’s disease will require additional resources. The National Institute of Health (NIH) funds $3 billion per year to enhance our understanding of HIV. This research has likely prevented millions, including many of us from ever developing the disease and led to numerous treatments that now makes a HIV a chronic, rather than rapidly fatal condition. By contrast the NIH funds less than $200 million per year for Parkinson’s disease. We need to change that.
3. Care for all affected
Many individuals with Parkinson’s do not see a neurologist or specialist for PD. Those who do not are more likely to fracture their hip or be placed in a skilled nursing facility. Expert care models, like ParkinsonNet developed by Bas Bloem, MD, and his colleagues, telemedicine and Centers of Excellence can provide better care to almost anyone anywhere. We need to embrace such models and ensure that Medicare, supported by taxpayers like you, does the same.
4. Treat the disease with novel therapies
The most effective medication for Parkinson’s remains a 50-year-old drug, levodopa. While effective, it does not address the underlying disease and has its own complications. To develop a new generation of therapies aimed at the underlying pathology of the disease, we need better, objective measures of the disease and to support efforts to develop gene-targeted therapies. The PD GENEration study and other efforts can help individuals better understand their genetic risk of the disease. Additional surgical interventions can also advance our treatment of those already affected by the disease.
All of these will require the collective action of all us. The same collective action that changed the course of polio through a March of Dimes and the course of HIV through a Quilt that covered the National Mall. We hope that the book will help catalyze such action and we look forward to your thoughts and suggestions.
The coronavirus pandemic quickly changed our day to day lives and has given rise to an increase in healthcare appointments being administered through telemedicine (the distribution of health-related services and information using technology).
“There are advantages to telemedicine, and right now considering the COVID-19 pandemic, telemedicine is an excellent way to connect to your physician and avoid the spread of the virus social distancing.”
Telemedicine appointments can be completed using a laptop, computer, tablet or phone with a camera, often through an app or program using an internet connection. Telemedicine appointment benefits include easy access to healthcare professionals, and no transportation hassle or waiting rooms. Below are nine tips to help you prepare for your next telemedicine appointment:
Preparing for your telemedicine appointment:
Get ready. Prepare as you would for an in-person visit. Make sure you have your medication list handy and write your list of questions you would like to ask.
Medication check. Check to see if you have enough pills at home or if you need refills. Remember, try to have a 90-day supply on-hand during quarantine.
Communication program check. Download the technology/application your physician is using for the call ahead of time. You will most likely use a program such as, Epic, Zoom, FaceTime, Skype or others. Your provider will let you know which program to download.
Device preparation. Make sure you set up in a location with a good internet connection. Charge your device before the appointment starts or try to keep it plugged in.
Be ready to move. Try to position yourself near a space where you can perform typical tasks your doctor or specialist may ask you to complete. For instance, a movement disorders specialist or physical therapist may ask you to walk. It may be helpful to have a family member ready to help during the appointment.
Final touches. Have a paper and pen nearby, along with any items your healthcare specialist may have asked you to bring to your appointment (for instance, a thermometer).
Video Tips:
Limit any clutter between you and the camera
Avoid backlighting (like windows behind you)
Limit background noise that may come from a TV, radio, pets or affect your device’s microphone (a fan overhead, loud AC unit, open window)
Speak in your normal voice
Do not place papers or objects near the microphone. If you are not using your cell phone, place it away from your device.
Ask your questions. Take notes and add them to your medical file.
Provide updates. Your pharmacy, insurance or contact information may have changed. Let the doctor’s office know before your appointment ends.
Are my Parkinson’s-related telemedicine appointments covered under Medicare?
The Centers for Medicare and Medicaid Services (CMS) provides coverage for various telehealth appointments. In response to the coronavirus pandemic, CMS expanded coverage to more hospitals', physicians’ and other healthcare organizations’ Medicare coverage of telehealth visits ― including emergency department visits, physical therapy and occupational therapy appointments. View the full list on the CMS.gov here.
PD Health @ Home features new weekly Parkinson’s disease (PD) wellness and education virtual events designed for the PD community. We have new, exciting upcoming events scheduled that help promote mindfulness, highlight COVID-19 issues, exercise classes and more. Remember to join us and catch up on previous virtual events now.
These are the top 10 most popular PD Health @ Home episodes YOU found to be the most helpful:
1. Emotional Stress, Mindfulness Meditation and Mindful Movement
In this Mindfulness Monday, we introduce you to mindfulness and discuss the role emotional stress plays on the brain. Event recorded April 6.
3. Expert Briefing: PD and Medication: What’s New?
In this Expert Briefings Tuesday event, Fernando Pagan, MD, highlights the latest PD medications and what is next in Parkinson’s treatments. Event recorded April 14.
In this Expert Briefing Tuesday, we highlighted the changes that can occur in thinking and memory for people with Parkinson’s and their care partners and the steps you can take to reduce the effects these symptoms might have. Event recorded April 7.
5. Ask the Expert: How Can I Benefit from Telemedicine?
In this Wellness Wednesday, Roy Alcalay, MD, explains what to expect from a telehealth appointment, the benefits of telemedicine and provides helpful tips for your next appointment.
In this Wellness Wednesday, Dr. Okun reviews hospital safety for people with Parkinson’s who may be experiencing COVID-19 symptoms and answers questions from our Facebook community. Event recorded April 8.
8. Overcome Exercise Apathy with Live Fitness Demos
In this Take Time Thursday, we help you break some exercise apathy habits while at home and show you some exercises that get you out of your chair. Event recorded April 23.
In this Fitness Friday, Physical Therapist Joellyn Fox leads you through our PD-tailored at-home fitness class. This session will help you improve your posture through seated stretching and core exercises. Event released April 10.
In this Fitness Friday, Joellyn Fox will guide you through exercises that can help improve arm strength, highlighting correct form, chair and standing exercises. Event released April 24.
