Together, we made life better for people in our Parkinson’s community in 2019. Your support allowed us to launch new, exciting initiatives that are changing lives, while funding critical research and local classes tailored to people with Parkinson’s disease (PD). With your support, we are reaching more people living with this disease in the U.S. and are closing the gap to help the 60,000 Americans diagnosed every year.

Thanks to YOU, we were able to accomplish the following in 2019:

1. Launched First-of-its-Kind Free Genetic Testing Initiative
   We launched PD GENEration: Mapping the Future of Parkinson’s Disease, a national initiative that offers free genetic testing for clinically relevant PD-related genes and free genetic counseling. We are excited to reach our goal of testing and providing genetic counseling for up to 15,000 people with PD.
    
2. Funded $12.2 Million to Further Parkinson’s Research
   This was an impactful year for Parkinson’s research. We established four new Research Centers that will receive a total of $8 million to launch PD-specific research studies. We also  simultaneously funded $4.2 million across 46 research grants that support the work of promising scientists in the PD field, cutting-edge clinical trials and fellowships.
    
3. Designed Program for People Newly Diagnosed with Parkinson’s
   With a focus to reach the 60,000 people who are newly diagnosed with PD each year in the U.S., Newly Diagnosed: Building a Better Life with Parkinson's Disease aims to close the gap between diagnosis and knowing where and how to find the right information and resources to live better with PD. Order or download the free Newly Diagnosed kit. 

4. Shared Research Findings at the 5th World Parkinson Congress
   In June, we enthusiastically joined our international community at the 5th World Parkinson Congress (WPC) in Kyoto, Japan, where our PD experts shared 10 research posters.
    
5. Expanded our Centers of Excellence Care Network
   Every year, we hope to bring expert care to more people with PD. In 2019, we expanded our Center of Excellence global network to include three new centers. Centers of Excellence are a medical center with a specialized team who provide expert Parkinson’s care. Find a Center of Excellence in your area.
    
6. Partnered with Michael J. Fox Foundation for Parkinson’s Research to Report New Economic Burden and Host Policy Forum
   In 2019, we collaborated with the Michael J. Fox Foundation (MJFF) to publish that the economic burden of Parkinson’s is nearly $52 billion every year. Together, we also hosted the 2019 Parkinson’s Policy Forum, bringing more than 150 advocates from across the U.S., along with leading experts in PD research to advocate for our community.
    

7. 

   Granted $1.5 Million to Local Communities for Parkinson’s Programs
   We proudly funded $1.5 million throughout 118 community-based grants that provide education and outreach programs, along with local research initiatives, that address unmet needs in the PD community.

8. Issued First Patient-Centered Research Agenda for Women with PD
   Recognizing long-standing gender disparities in Parkinson’s research and care, our Women and Parkinson’s Initiative created the first patient-centered action agenda to maximize quality of life for women with Parkinson’s.
    
9. Appointed Aware in Care Ambassadors
   In 2019, we appointed our first-ever  Aware in Care Ambassadors, a volunteer group to help distribute Aware in Care kits that serve to bolster best practices in treating patients with Parkinson’s disease to both patients and healthcare providers.
    
10. Designed New Online Nurse Course
    With the prevalence of PD expected to increase in the coming years, we wanted to provide more professional education opportunities for nurses. In 2019, we launched a new online course for nurses who deliver care throughout all stages of Parkinson’s. 

As much as we accomplished in 2019, we are committed to furthering our reach and impact in 2020 to help even more people live better with Parkinson’s. Your continued support is the only way we can make that happen. Thank you.

Parkinson’s Foundation Research Advocates were the first people with Parkinson’s disease (PD) to help guide the grant review process for the Department of Defense (DoD) Parkinson’s Research Program. The program supports research to understand, prevent, diagnose and treat Parkinson’s disease.

This year, Michael S. Fitts, MLIS, a Parkinson’s Foundation Research Advocate participated as a consumer reviewer and voting member to help determine how $16 million will be spent on Parkinson’s research in 2019. Consumer reviewers, like Michael, are asked to represent a collective view of people with PD by commenting on the impact of the research on issues like diagnosis, treatment and quality of life.

"Since I began my advocacy work within the PD community, I’ve been continuously blessed with opportunity after opportunity to touch the lives of diverse individuals from all walks of life,” said Michael Fitts. “Most recently I received a nomination from the Parkinson’s Foundation and was selected to serve on the Congressionally Directed Medical Research Programs’ (CDMRP), Parkinson’s Research Program (PRP) as a consumer advocate, as a full voting member."

DoD studies involve research that, if successful, impacts not only veterans with Parkinson’s, but the entire PD community. The research also focuses on understanding the mechanisms of PD. Consumer advocates and scientists have worked together in this unique partnership since 1997.

Colonel Stephen J. Dalal, Director of the CDMRP expressed his appreciation for the consumer advocate’s perspective during these scientific review sessions. “Consumer advocates are an integral part of the CDMRP’s scientific review process,” Col. Dalal said. “They provide a key ingredient to the review process, the patient’s perspective, which is real and urgent. The collaboration of consumer advocates alongside the scientists’ subject matter expertise is truly a unique collaboration that is difficult to find in most medical research programs.”

Scientists applying propose to conduct innovative research to understand, prevent, diagnose and treat Parkinson’s disease ― for everyone with PD, including servicemembers and veterans. The Parkinson’s Research Program fills important gaps in the Parkinson’s research field by supporting groundbreaking, high-risk, high-gain research while encouraging out of the box thinking. Grant reviewers like Michael provide the critical perspective of someone living with PD when reviewing these applications.

Since 2015, Parkinson’s Foundation Research Advocates alongside scientists have been key decision makers in directing nearly $100 million of DoD research funding through the grant review process. Parkinson’s Foundation Research Advocates Paul Zimmet, A.C. Woolnough, Fred Woodlief, Samantha Erwin, Jay Phillips, Bel Broadley and Maria De Leon have all participated in DoD review. 

Interested in playing a key role in Parkinson’s research? Learn more about becoming a research advocate.

The past two weeks have offered more definitive information on Nilotinib (Tasigna), a drug already approved for treatment of leukemia, as a potential treatment for Parkinson’s disease (PD) symptoms. On December 16, JAMA Neurology published the results of a Phase II safety trial ― a study that tests the effectiveness of a drug or treatment in a larger group of people. Study findings revealed that Nilotinib had more adverse events than the placebo (a pill not containing an active drug) but was reasonably safe. An accompanying editorial and podcast discuss the narrowing path forward for this drug in Parkinson’s disease as the clinical effects were unfortunately not robust.

Northwestern University, a Parkinson’s Foundation Center of Excellence, and the Parkinson Study Group, in a study led by Tanya Simuni, MD, in the NILO-PD study (25 sites) reported that Nilotinib was safe and tolerable but did not “exert a clinically meaningful benefit or biological effect to benefit those with Parkinson's disease.”

Though there may be a potential future for cancer drugs called C-Abl inhibitors to be repurposed for Parkinson’s disease, we are recommending that people with Parkinson’s and families pass on this particular drug at this time. Though it is reasonably safe, it is still a drug used as a cancer treatment with more adverse effects than in the placebo group. The data on the medication’s effectiveness was weak at best and does not justify the risk for treatment. There was an accompanying editorial in JAMA Neurology and a podcast on the narrowing path forward for this and other similar drugs found here.

Study authors address the idea of “reasonably safe,” biomarkers, clinical outcomes and implications for future clinical trials. Though the Parkinson Study Group Trial co-sponsored by the Michael J. Fox Foundation results have not been published, the trial was stopped. We expect to see the full publication in the next few months.

Previously, Nilotinib was tested for safety in a phase I clinical trial ― a trial where researchers test a new drug or treatment in people for the first time ― on a small group of participants with Parkinson’s several years ago at Georgetown University Hospital, a Parkinson’s Foundation Center of Excellence. The original study was small and did not include a placebo.

While Nilotinib may not be the answer the PD community was looking for, the Parkinson’s Foundation firmly believes research breakthroughs can happen at any moment, which is why the Foundation continues to fund critical research, from clinical trials to new initiatives. 

Learn More

Find out more about Parkinson’s and Nilotinib in the below Parkinson’s Foundation resources or by calling our free Helpline at 1-800-4PD-INFO (473-4636).

• Clinical Trials

You can find out more about our National Medical Director, Dr. Michael S. Okun, by also visiting the Center of Excellence, University of Florida Health Norman Fixel Institute for Neurological Diseases. Dr. Okun is also the author of the Amazon #1 Parkinson's Best Seller 10 Secrets to a Happier Life and 10 Breakthrough Therapies for Parkinson's Disease.

Men are more likely to develop Parkinson’s disease (PD) than women, and the onset of PD in men happens at a younger age. However, women with PD have a higher mortality rate, and once they have Parkinson’s, progression is faster. Research suggests that women get the disease at later in life when compared to men, at least in part, due to the natural protection estrogen provides. There are studies that have demonstrated that hormone replacement therapy (HRT) can provide dopaminergic neuroprotection in both young and menopausal female mice.

Could the female sex hormone, estrogen, be a therapeutic approach for delaying or reducing PD symptoms for men?

Recently published in the Journal of Neuroscience, a study titled, “Female Sex and Brain-Selective Estrogen Benefit α-Synuclein Tetramerization and the PD-like Motor Syndrome in 3K Transgenic Mice” (Rajsombath, Nam, Ericsson, & Nuber, 2019) investigated this possible therapeutic neuroprotective effect.

Using mice called 3K that show motor and neural changes associated with PD, researchers injected male mice under the skin with the hormone therapy DHED. What makes DHED so special is that it was designed to only activate estrogen in the brain. This matters because estrogen therapy has been associated with an increase in cancer in other parts of the human body.

The motor performance and brain health of the 3K male and female mice were compared along with whether DHED affects the progression of PD-like symptoms in males. The motor evaluations included their ability to clasp, climb down a pole, gait (walk) and balance on an accelerating rotarod, which is a lot like lumberjack logrolling. There were also highly sophisticated tests to determine possible changes in the build-up or clearing of protein clumping in the brain, along with the decline or increase in the health of dopamine neurons.

Results

Like the sex differences found in people with PD, 3K male mice developed PD-like symptoms faster than female mice. Furthermore, male mice treated with DHED had:

• Improved clasping abilities
• Improved downclimbing
• Improved gait
• Improved balance
• Better clearing of risky alpha-synuclein (protein clumps in the brain)
• Healthier dopamine neurons

What Does This Mean?

This study focused on the 3K male mice and how they responded to the estrogen therapy, DHED. When the male mice were treated with the DHED, they showed improvements in all the motor functions tested. They also showed significant improvements in the brain, including healthier dopamine neurons and lower amounts of alpha-synuclein at risk for clumping. Remember, clumped alpha-synuclein becomes Lewy Bodies ― a hallmark of PD.

It is also important to note that the successful development of the 3K model itself – which duplicates many differences in male and female PD at motor, cellular and molecular levels – is a significant step forward in closing the gender gap in PD research. Having a model that helps unravel how the pathology differently affects the two sexes informs new avenues of research that could lead to the development of tailored medications and interventions to meet the distinct needs of men and women with PD.

Learn More
The Parkinson’s Foundation believes in empowering the Parkinson’s community through education. Learn more about the Parkinson’s and Gender differences in the below Parkinson’s Foundation resources or by calling our free Helpline at 1-800-4PD-INFO (473-4636).

• Center of Excellence Series: University of Pennsylvania Movement Disorder Center Is a Leader in Women’s Parkinson’s Research
• Parkinson’s Foundation Identifies First Woman-Focused Parkinson’s Research Agenda
• Alpha-synuclein & DNA: The Ties that Bind

Over the next three years the Parkinson’s Foundation will invest more than $50 million to Parkinson’s disease (PD) research and clinical care. At the heart of our research initiatives are scientists and researchers who have received Foundation awards to improve our understanding of Parkinson’s, which will ultimately lead us to a cure.

While there is no known single cause for non-genetic (idiopathic) Parkinson’s disease, most cases are likely caused by interaction between a person’s genetics and their environment. Genetics cause about 10% to 15% of all Parkinson's. This is called familial PD. However, not everyone who carries PD gene mutations develop the disease.

Briana De Miranda, PhD, received a Postdoctoral Fellowship for Basic Scientists from the Parkinson’s Foundation to study the role environmental exposures play in the risk for both idiopathic and familial PD. Environmental factors that are linked to increased PD risk include pesticides, such as rotenone and paraquat, and the industrial solvent trichloroethylene (TCE).

These chemicals disrupt the functioning of the mitochondria, the “powerhouse” of the cell. In addition, Dr. De Miranda has found that rotenone, paraquat and TCE cause activation of the protein affected by one of the most commonly inherited PD genetic mutations, LRRK2.

Her goal is to investigate whether mutations in LRRK2 increase susceptibility to the damaging effects of these chemicals, even when a person is exposed to low levels. She will also assess whether inhibiting the LRRK2 protein protects against these effects.

To achieve this goal, she will study the interactions of these environmental toxicants with LRRK2 in brain nerve cells. She will also use an animal model to study LRRK2 activation following exposure to these toxicants.

Clinical trials are currently underway for LRRK2 inhibitors for use in inherited PD cases. Our hope is this research may provide evidence that these drugs may help prevent PD induced by environmental toxicant exposure and lead to expanded use of LRRK2 inhibitors to treat those with idiopathic PD who have been exposed to these chemicals.

Parkinson's Foundation Postdoctoral Fellowships for Basic Scientists are two-year fellowships for young scientists, fresh from their PhD training, to study at major research institutions. Postdoctoral Fellowships for Clinical Neurologists are awarded to young clinicians who have completed their neurology residency and want research experience.

What's Next: Reporting Our Findings

Parkinson’s Foundation research awards fund Parkinson’s studies than can span up to three years. Scientists submit yearly progress reports to the Parkinson’s Foundation, and we report findings once the studies have concluded. Stay up to date with our latest research findings at Parkinson.org/Blog.

One of the only facts we know about Parkinson’s disease (PD) and medical marijuana is that more research is needed to understand the utility of marijuana or cannabis to treat Parkinson’s symptoms. In its latest research endeavor, the Parkinson’s Foundation has launched a survey to identify themes in cannabis and marijuana use and perceived benefits and risks to inform the design and priorities of future PD clinical trials.

The Parkinson’s community has looked to marijuana or cannabis to provide some relief from PD-related movement and non-movement symptoms. However, little is known about the effects of marijuana or cannabis for Parkinson’s. Unknown information includes benefit to symptoms, potential side effects and safety issues.

The Parkinson’s Foundation “Cannabis and Parkinson’s disease” survey will hear directly from people with Parkinson’s about their:

• Reasons for or against marijuana or cannabis use
• Methods and frequency of marijuana or cannabis use
• Benefits or risks of marijuana or cannabis use

“We exist to help the PD community, and right now they are interested in knowing if and how cannabis can be beneficial or if it can lead to a better quality of life. We want to help answer that question,” said James Beck, PhD, Parkinson’s Foundation Chief Scientific Officer. “When it comes to research, this is an unexplored area that has the potential to treat Parkinson’s symptoms.”

The Parkinson’s Foundation plans to administer the anonymous survey to both men and women with Parkinson’s, across all racial and ethnic groups. The Foundation hopes to reach 10,000 people with Parkinson’s. The survey has received Institutional Review Board (IRB) approval ― a high research standard approved by an independent oversight committee and governed by Federal Regulations. 

The survey will be administered in January and February 2020. The Foundation will publish survey results in early fall 2020.

How the Foundation Has Addressed Medical Marijuana and Parkinson’s

Few clinical studies have enrolled people with Parkinson’s to investigate the effects of medical marijuana on PD symptoms. As part of its research initiatives, the Foundation remains committed to addressing the needs and priorities of the Parkinson’s community.

In July 2019, Dr. Beck provided testimony to the U.S. Food & Drug Administration (FDA) regarding the agency’s proposed rule concerning scientific data and information about the safety, manufacturing, product quality, marketing, labeling and sale of products containing cannabis or cannabis-derived compounds. “Special warnings to consumers with neurologic disease such as PD, should be included in the labeling of cannabis-derived products,” said Dr. Beck during the FDA session. “Information is key for consumers to make decisions that are appropriate to their health needs.” 

The Foundation held its first-ever medical marijuana research conference in March 2019. At the conference PD experts and people with Parkinson’s discussed the evidence for use of medical cannabis in PD, including gaps in knowledge, potential health effects and safety concerns in an effort to establish a consensus to guide the patient community and future research efforts.

In 2016, the Foundation, in partnership with Danny Bega, MD, from Northwestern University and other researchers, published the attitudes about cannabis at 40 Centers of Excellence. This is the Foundation’s first study to provide data on the practices, beliefs and attitudes of expert PD physicians concerning cannabis use. While there is no general agreement on what the benefits might be for people with PD, the survey confirmed that cannabis is a popular subject within Parkinson’s Foundation centers as 95 percent of neurologists reported having been asked to prescribe cannabis.

Learn More

Check out the below Parkinson’s Foundation marijuana and Parkinson’s resources:

• Marijuana and Parkinson’s: What Do We Really Know?
• Expert Briefing webinar: Marijuana & PD: What Do We Really Know?
• Ask the Experts: The Challenges of Using Marijuana as a Parkinson’s Treatment, Part 1
• Podcast: Episode 26: Medical Marijuana: Going Green for PD?

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:

1. Join our email list at Parkinson.org
2. Follow us on Facebook, Twitter and Instagram.
3. Watch the latest Parkinson’s disease videos on our YouTube channel.

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.

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).

• GBA Mutation May Lead to Novel Therapeutics
• Genetic Testing

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. 

Learn more about the research you fund.

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.

Watch the latest Parkinson’s disease videos on our YouTube channel.