FDA Approves Kynmobi Sublingual Film to Treat Off Episodes in Parkinson’s

Kynmobi and FDA approval

The U.S. Food and Drug Administration has approved Sunovion’s Kynmobi (apomorphine hydrochloride) as an on-demand sublingual treatment for off episodes, or times when medication wears off, in people with Parkinson’s disease.

The medication, formerly known as APL-130277, is an apomorphine film that is placed under-the-tongue (sublingual administration) when patients start experiencing a worsening of their symptoms. It can be taken up to five times a day, at doses ranging from 10 mg to 30 mg.

Sunovion expects it to become available to patients in the U.S. by September.

“Today’s approval of Kynmobi advances treatment options for people with Parkinson’s disease who experience OFF episodes and the associated disruption of everyday activities,” Antony Loebel, MD, president and CEO at Sunovion, said in a press release.

“We are pleased to offer the Parkinson’s disease community a novel treatment option that we believe offers a convenient way for patients to rapidly improve impaired movements and better control their motor symptoms when they need it,” Loebel added.

Levodopa is considered the gold standard for Parkinson’s treatment. But several years after starting the medication, most patients begin experiencing fluctuations in their motor symptoms caused by a faster wearing off of the treatment’s effects. These “off” episodes can happen at any time of the day, and most patients experience more than one episode each day.

Kynmobi’s active ingredient, apomorphine, can cross the blood-brain barrier, a semipermeable membrane that protects the brain from the external environment, and mimic the effects of dopamine in the brain. As such, it can counteract the loss of dopamine-producing neurons in the brain, a hallmark of Parkinson’s.

Apokyn, by US World Meds, is an approved apomorphine injection treatment for Parkinson’s patients experiencing off episodes. Its efficacy in easing motor symptoms is established, but it can pose significant challenges to patients, such as the need for an under-the-skin injection, an initial dose titration that should be supervised in a clinic, and common side effects such as nausea and injection site complications. These challenges are thought to have limited its use.

Kynmobi is expected to provide an easier mode of administration and to be eliminate more slowly from the body, helping to ease the feeling of nausea caused by abrupt reductions in apomorphine blood levels.

“Several years after a person is diagnosed with Parkinson’s disease they may notice problems such as having trouble getting out of bed in the morning or having difficulty getting out of a chair, or that they feel frozen while trying to walk as the effect of their maintenance medication diminishes,” said Stewart Factor, professor of neurology at Emory University School of Medicine.

“The approval of Kynmobi affords health care providers with a needed option that can be added to their patients’ medication regimen to adequately address OFF episodes as their Parkinson’s disease progresses,” Factor added.

Kynmobi’s approval was based on data from a Phase 3 study (NCT02469090), in which the oral medication was compared to placebo as an on-demand treatment of motor symptoms during off periods.

The trial included 109 patients who had at least two hours of total off periods per day, including well-defined morning off episodes, despite being responsive to levodopa treatment. In an initial open-label phase, all enrolled were given increasing doses of Kynmobi (10−35 mg) until an optimal dose was identified.

Patients were then randomly assigned to either Kynmobi or placebo, taken to treat up to five off episodes throughout a day, for 12 weeks. All continued to receive their stable anti-parkinsonian medications.

The trial’s main goal was improvements in motor symptoms — defined as changes on the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III — from before dosing to 30 minutes after dosing at week 12.

Results showed that patients using Kynmobi experienced an 11.1 point reduction in their MDS-UPDRS scores at week 12, while those on placebo showed reductions of 3.5 points. This 7.6-point difference between both groups was significant, and clinical improvements were seen as early as 15 minutes after dosing, and persisted for up to 90 minutes.

Kynmobi also helped more patients (31%) achieve full control of their motor symptoms — a full “on” response — within 30 minutes at week 12, compared with 14% of those given a placebo.

The treatment was generally well-tolerated, with most treatment-related side effects being mild to moderate, and reversible after its use was stopped. The most common were nausea, sleepiness, and dizziness. One person with known cardiac risk factors who was treated with Kynmobi died due to heart failure.

“We know from our research and discussions with the Parkinson’s community that OFF episodes can significantly disrupt a patient’s daily life,” said Todd Sherer, PhD, CEO, The Michael J. Fox Foundation for Parkinson’s Research. “The Foundation supported early clinical development of sublingual apomorphine, and this approval brings an important new treatment option for people with PD [Parkinson’s disease] who experience OFF [periods].”

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Neo Inquires About the Healing Relationship

relationships, what ifs, shut in, toilet paper, serenity, laughter

Neo, the neocortex of Dr. C’s brain mentioned in previous columns, exclaims, “That was amazing, Dr. C, to watch Mrs. Dr. C apply the healing relationship, and poof, you’re all better!” Neo was referring to last week’s column, in which Dr. C emerged from a long period of darkness in his life.

Dr. C grimaces at Neo’s remark. It might sound that simple, but the process is much more involved. “Remember,” he says, “she has been with me for a long time. That is the special knowledge that Mrs. Dr. C has. As James Taylor wrote in his song ‘Something in the Way She Moves,’ ‘She has the power to go where no one else can find me.’ She is a witness to the best of Dr. C.”

“You have mentioned the healing relationship in 10 columns,” Neo points out. “I’ve read all that, but I still don’t understand how it works.”

Dr. C settles into a comfortable position, stretches, and then explains to Neo the development of the concept. Sounding very professorial, he begins, “From the beginning of human civilization, healers, shamans, have been an important part of a sustainable social collective. Healers are often chosen by the tribe because of a gift for sensing the inner person. They often undergo years of training, rites of passage, vision quests, or other training provided by an elder shaman.

“Some of these initiates pass from the ordinary to the extraordinary, and some do not. Those who do learn the intricate dance that is the healing process become skilled artisans within the healing relationship. Only a few will attain such skills.”

During the soliloquy, Mrs. Dr. C opens the door and enters, laden with groceries. Dr. C offers to help, and she asks, “What have you two been up to while I was out?”

Dr. C mentions Neo’s amazement at the healing relationship. “It is his contention that all the stress was eliminated and problems solved with your intervention and use of the healing relationship when I was engulfed in darkness.”

He continues, “The descriptors in the column provide a look at various parts of the healing relationship. This is to help clear the confusion about its nature. But the healing relationship is more than the sum of its parts. There is a holistic experience connected to the phenomenon, one that cannot be known through a process that reduces it to a collection of features or steps in a process. Within this healing relationship is the possibility of experiencing a sacred well-being moment.

“The possibility of sacred well-being is available to everyone, trained or untrained, anytime and anywhere. Training with the healing relationship increases the probability it will occur, but it is not a guarantee. Training also helps with meaning-making during follow-up,” he adds.

Mrs. Dr. C responds, “What the ‘good doctor’ forgets is that his brush with the darkness impacted us both. We both had to go through that dark, briar-filled forest of emotions, despair, and fear.

“I think there are two more important points to make. One, I have never wanted to become a ‘caretaker’ instead of a ‘wife.’ Caretaker implies to me a distancing from the relationship of spouse. Second, I do not view the healing relationship as a cure. It does not change your symptoms of Parkinson’s or vision loss. It does not make them go away or stop the progression. But it provides something else.”

She thinks for a moment, gazes out the window at the birds circling the feeders, and then says, “It gives me strength. It is a frightening thing for me to feel that there might be nothing I can do. I feel like I am helping to heal a wound, much like scar tissue over an injury. The Parkinson’s and vision loss still happened, the pain is still there, and the scars will always be present. Neither of us can change that. Sometimes I remind him that we just need to follow the map provided.”

Dr. C agrees, “It’s not a cure but rather a path to better living with what exists, even if it’s a chronic illness. The map comes with the sacred experience, and if I do not follow it then all I can expect is failure. Using the healing relationship comes down to believing it is possible, and then having a sacred healing experience with a healer followed by learning everything about it.”

Mrs. Dr. C points at the shopping bags. “It would also help if you could put away some groceries.” She gives Dr. C and Neo a friendly wink.

***

Note: Parkinson’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Parkinson’s News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Parkinson’s disease.

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Finding, and Becoming, a Personal Trainer Specializing in Parkinson’s

personal trainer, guilt, Q&A

I received my Parkinson’s diagnosis five years ago. I fully intended to attack it head-on, starting with exercise. Parkinson’s is not for wimps. Dancing was an important part of my plan but I needed more.

The first step of my plan was to find a personal trainer. I went on a quest to find an experienced trainer with an understanding of Parkinson’s. Additionally, I needed someone who understood me. My research led me to a local community center that employed trainers who listed Parkinson’s disease as a specialty. Great. The first planning session was scheduled for that week.

First, the trainer reviewed my medical history with me. I was newly diagnosed, so my emotions were in overdrive and I was extremely guarded. Answering the questions that were not related to Parkinson’s was easy but then …

Trainer: “Do you have the shakes?”

Me: “Do you mean, do I tremor?”

Trainer: “No. I just call it the shakes.”

Really? The shakes? I was speechless, which is not an everyday occurrence. It was a small room so a quick escape was not feasible, but my mind left the building after question #1. The entire ordeal was only 20 minutes, but it left a lasting impression on me … the 45-year-old mom newly diagnosed with young-onset Parkinson’s.

The seed was planted. Could I be a personal trainer?

That very moment put me on the path to where I am today. I researched different programs and enlisted the help of a friend. Eventually, our Parkinson’s fitness program was up and running. We knew basic terms, including “tremor.” Initially, our program was PWRMoves! We added Rock Steady Boxing a year ago.

I never forgot that trainer who asked me if I had the “shakes.” (I guess I should thank him.) He planted the seed of my desire to earn a personal trainer certification. However, one thing held me back — the final exam. It intimidated me. Learning the material was not problematic, but the thought of going somewhere and taking a test terrified me. Ultimately, anxiety would hit and it would be game over. The right program was out there. I just needed to find it.

After keeping my toes in the water for years, I took the plunge and enrolled in an online course with ISSA. It was a perfect fit for me. The course fit my schedule and allowed me to work at my own pace. I also purchased a hardcopy of the training book; not an ebook but a real book with pages. It was exactly what I needed: a combination of the technology of an online course and the old-school approach of a hardcopy book.

Now I am the personal trainer and I still have a lot to learn. However, I passed Parkinson’s 101 for personal trainers — you tremor when your muscles are fatigued. You do not “have the shakes.” Understanding something so simple can make a world of difference for everyone.

***

Note: Parkinson’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Parkinson’s News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Parkinson’s disease.

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New Therapy Using Patients’ Own Cells May Halt Parkinson’s Progression, Case Study Suggests

transplanting cells

A new therapeutic approach in which patient-derived dopamine-producing neurons are transplanted into the brain may halt Parkinson’s disease progression, a case report suggests.

The approach uses patient-derived induced pluripotent stem cells (iPSCs),  which are cells collected from the skin or blood that researchers can reprogram in a lab dish to revert them back to a stem cell-like state that has the capacity to then differentiate into almost any cell type.

“Because the cells come from the patient, they are readily available and can be reprogrammed in such a way that they are not rejected on implantation. This represents a milestone in ‘personalized medicine’ for Parkinson’s,” Kwang-Soo Kim, PhD, said in a news story. Kim is co-senior author of the study and director of the Molecular Neurobiology Laboratory at McLean Hospital in Massachusetts.

Two-year data following the first of two interventions suggest the therapy resulted in at least a stabilization of the patient’s motor function and an improved quality of life. However, clinical studies with longer follow-up periods are needed to confirm the therapeutic potential of this approach in Parkinson’s patients, the researchers noted.

The case study, “Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease,” was published in the New England Journal of Medicine.

Parkinson’s is characterized by the gradual loss of dopamine-producing (dopaminergic) neurons in the substantia nigra, a region of the brain responsible for movement control. The death of dopaminergic neuron results in lower dopamine levels, affecting the regulation of muscle movement and coordination.

While the potential use of tissue transplants to replace the lost dopaminergic neurons in Parkinson’s patients has been studied since the 1980s, the creation of iPSCs offered the hope to transplant precursors of dopaminergic neurons into a patient’s brain.

In 2018, a team of researchers in Japan reported the implantation of precursors of dopaminergic neurons into the brain of a Parkinson’s patient. Six other patients were expected to receive this experimental therapy that used iPSCs developed from skin cells of an anonymous donor. Researchers plan to collect all safety and effectiveness data by the end of this year.

When implanting cells derived from other individuals, patients need to receive immunosuppressive therapies (for an undetermined period of time) to prevent the development of immune responses against the implanted cells. However, the use of a patient’s own cells would make the need for immunosuppression unnecessary.

Now, a team of researchers at the McLean Hospital and Massachusetts General Hospital (MGH) reported the case of a 69-year-old man treated with a similar approach using the patient’s own iPSCs.

The man had a 10-year history of progressive Parkinson’s disease with no signs of dyskinesia (abnormal involuntary movements that characterize advanced Parkinson’s). He was treated with extended release carbidopalevodopa tablets, Neupro patches (by UCB), and Azilect (by Teva Pharmaceuticals).

He reported poor control of his symptoms, with three hours of “off”-periods — when the medications’ effects wear off and symptoms worsen before a new dose can be taken. Higher levodopa doses caused him lightheadedness associated with a drop in blood pressure when changing to a standing position (orthostatic hypotension).

The researchers used the man’s skin cells to create iPSCs and develop them into precursors of dopaminergic neurons, which were tested extensively, including a mouse model used in human-derived transplant studies.

Using these data, Kim applied to the U.S. Food and Drug Administration (FDA) for a single-patient, investigational new drug application and also received approval from the hospital board to implant the cells into the patient’s brain.

The man underwent two surgeries, in 2017 and 2018 (separated by six months) at the Weill Cornell Medical Center in New York, and at MGH.

At each surgery, four million cells were delivered into the putamen, a large brain structure involved in movement control that is filled with dopamine receptors and receives signals from the substantia nigra. The first intervention targeted the putamen on the left hemisphere of the brain, while the second targeted the one on the right hemisphere.

The cells were delivered using a new minimally invasive neurosurgical implantation procedure developed by Jeffrey Schweitzer, MD, PhD, the study’s lead author, a Parkinson’s specialized neurosurgeon, in collaboration with other neurosurgeons at MGH and Weill Cornell. Schweitzer is director of the Neurosurgical Neurodegenerative Cell Therapy program at MGH.

The patient’s motor function was assessed through the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III and quality of life with the 39-item Parkinson’s Questionnaire.

Two years after the first intervention, imaging tests showed that the transplanted cells were alive and working correctly as dopaminergic neurons, highlighting the technical success of this personalized cell-replacement approach.

There were no reports of side effects or immune reactions against the cells (without the need for immunosuppressive therapy), or signs that the cells caused any unwanted growth or tumors.

Notably, there was at least a stabilization in the man’s motor function, with MDS-UPDRS scores varying over time, but never reaching the initial values, and he reported improvements in his day-to-day activities and quality of life.

The man reported less than one hour of “off” period per day and the levodopa equivalent daily dose was lowered by 6%, “a reduction of uncertain clinical importance,” the researchers wrote.

“This strategy highlights the emerging power of using one’s own cells to try and reverse a condition — Parkinson’s disease — that has been very challenging to treat. I am very pleased by the extensive collaboration across multiple institutions, scientists, physicians, and surgeons that came together to make this a possibility,” said Bob Carter, MD, PhD, another co-senior author of the study and MGH’s chief of Neurosurgery.

Despite these apparently positive results, the researchers emphasized this is just a first step in this therapy’s development.

“These results reflect the experience of one individual patient and a formal clinical trial will be required to determine if the therapy is effective,” said Schweitzer.

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MJFF Suggests Ways to Help Advance Clinical Trials During Pandemic

online research tools

With numerous clinical trials interrupted due to the COVID-19 pandemic, The Michael J. Fox Foundation (MJFF) is highlighting online ways that Parkinson’s (PD) patients and others can help to advance research.

People are necessarily spending more time at home, but study participation remains crucial to treatment development. Even before the outbreak, volunteer shortages delayed 85 percent of clinical trials. Thirty percent were unable to even get started.

“It can be an empowering and impactful opportunity for people with and without Parkinson’s disease to help accelerate breakthroughs in treatment,” the MJFF states on its webpage about research participation. “You have the power to help researchers understand how Parkinson’s starts and progresses.”

One research opportunity is the organization’s Fox Trial Finder, an online tool that matches individuals with studies for which they may be eligible. Even if the trial is on hold, patients and others can still express interest. Study coordinators will follow up once recruitment restarts.

Trials can be found through a guided search, or, based on location and keyword, by independent searches. Those interested may also register to receive email alerts about local studies that are or will be recruiting.

Another way to get involved is by joining the organization’s Fox Insight, a digital platform and clinical study aimed at building a large cohort of patients and age-matched control volunteers to help focus treatment priorities, inform their development, and optimize trial design.

This long-term study, which includes patients, families, and caregivers, collects de-identified self-reported data about health experiences. Through an associated genetic sub-study, powered by consumer genetics company 23andMe, eligible individuals can help researchers gain a holistic view of Parkinson’s.

“Technology now allows thousands to contribute vast volumes of data on their lived experience of disease, from symptoms and quality of life to treatment satisfaction and research participation preferences,” MJFF states about Fox Insight, which opened in 2017.

Go here to register and for an informational video.

Elsewhere, the University of Rochester’s neurology department is offering a 20-minute survey that aims to help scientists learn more about Parkinson’s symptoms and everyday issues of importance to patients. The anonymous responses will be used to guide additional studies and to help develop a disease-specific patient-reported outcome measure for clinical trials. The survey may be completed online, on paper, or over the phone.

Bastyr University, in California and Washington state, is leading an online study about complementary and alternative medical care in Parkinson’s. It aims to help scientists learn more about diet, lifestyle and Parkinson’s progression, and the long-term effect of complementary and integrative care on health and life quality.

Patients are asked to complete two online questionnaires every six months for five years. Survey topics cover diagnosis, disease status, sense of balance, daytime sleepiness, walking, dressing, eating, falling, speech, handwriting, pain, vision, sense of smell, comprehension and cognition, sexual dysfunction, dyskinesia, and posture.

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Do you have dizziness that improves when you sit down? Learn about a possible cause.Sponsored Post

If you’re experiencing dizziness, lightheadedness, or fatigue upon standing, these symptoms may be caused by a condition called neurogenic orthostatic hypotension, or “nOH”

What is nOH?

Neurogenic orthostatic hypotension (nOH) causes a drop in blood pressure after standing or changing positions. In patients with diseases like Parkinson’s, OH may be caused by damage to the nervous system (“neurogenic”). Due to this damage, the body may not release enough of a chemical called norepinephrine, which normally helps keep blood pressure from dropping too low. Without enough norepinephrine, blood pressure remains low after standing. This can lead to feelings of dizziness, lightheadedness, fatigue, blurred vision, and other symptoms.

People often think the symptoms of nOH are due to their underlying nervous system disorder, such as Parkinson’s, and assume the symptoms are something they must learn to live with. However, nOH is a separate condition with symptoms that can also be managed.

What else do I need to know about nOH?

While nOH can result in sudden feelings of dizziness or lightheadedness at any time, these symptoms tend to occur more often in the morning. They may interfere with your ability to complete your usual daily activities. It’s important to understand that nOH symptoms may also result in serious consequences, such as falls.

If you’re experiencing symptoms like these upon standing or changing positions, or feel like you may black out or faint, it may be time to talk to your doctor. Before you do, take a few minutes to complete this online symptom checker to help prepare for your discussion.

Be honest about your symptoms. Describe how you feel when you stand up or change positions.

Talking with your doctor about nOH?

Here are some tips and resources to help you prepare for your next visit and for talking to your doctor.

Track your symptoms. Use a notebook, tracker, or your phone to keep track of symptoms so you can discuss them with your doctor.

Talk about what’s important to you. Talk to your doctor about the impact your symptoms are having on your life (especially if they are limiting or interfering with your daily activities).

  • It’s important to discuss how your symptoms are affecting you so that you and your doctor can develop a management plan together

At your doctor appointment, ask for blood pressure readings in different positions (lying down if possible, or sitting, then standing).

  • This is very important for diagnosing nOH and determining your management options

Questions to ask your doctor.

  • When meeting with your doctor, it may be helpful to have a list of questions to ask to help you better understand nOH and its management

There are different approaches to treating the symptoms of nOH

Living with nOH can be challenging, but your doctor may suggest some lifestyle changes that may help manage your symptoms. Always talk to your doctor before making any changes to your nOH management plan or your daily routine.

To hear about the experience of other patients and their care partners, check out the videos below. You can learn more about nOH and download additional resources at nOHmatters.com.

©2020 Lundbeck. nOH Matters is a registered trademark. All rights reserved. UBR-D-100789

The preceding article is content provided by our sponsor Lundbeck. The views and opinions expressed in the content above are not the views and opinions of Parkinson’s News Today or its parent company, BioNews Services, LLC.

Parkinson’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Self-care Skills Program Improves Daily Life for Patients and Caregivers, Study Finds

life quality and self-care skills

A program that teaches Parkinson’s patients and their caregivers techniques to manage day-to-day activities over the course of this disease can help improve both life quality and effective self-care, a study from Sweden suggests.

The study, “Self-Management Education for Persons with Parkinson’s Disease and Their Care Partners: A Quasi-Experimental Case-Control Study in Clinical Practice,” was published in Parkinson’s Disease.

A long-term progressive disease, Parkinson’s requires that patients and the people caring for them develop skills and coping mechanisms in order to best deal with its physical and emotional changes over time.

The Swedish National Parkinson School (NPS) opened to help teach self-care skills to these people. The seven-week program builds on the same principles as cognitive behavioral therapy.

“The NPS promotes awareness of thoughts, feelings, and actions in relation to the impact of disease on daily life and introduces techniques of self-monitoring in order to provide the knowledge and tools needed to enhance the ability to live and handle life with the disease,” the researchers wrote. “It does not primarily focus on the disease itself, but on how to live a good life in the presence of disease.”

The program consists of seven, two-hour sessions, where people with Parkinson’s and their care partners come together in a small group with a qualified instructor — a health care professional who is trained to deliver the program and has experience working with Parkinson’s.

Each session, which ends with a 15-minute relaxation exercise, focuses on a specific topic, including “self-monitoring,” “stress,” “anxiety and depression,” “communication,” “enriching activities,” and “my future life with Parkinson’s disease.”

These topics are “then applied to the participants’ own life situation through practical exercises and homework assignments,” the study notes.

Researchers evaluated NPS in terms of how it affected quality of life, looking at data collected on 48 patients and 30 of their caregivers, after going through the seven-week program. For comparison, they also assessed 44 patients and 25 caregivers who underwent seven weeks of standard care for Parkinson’s. Possible confounding factors — such as familial deaths or births — were similar in both groups.

Participants were given a battery of questionnaires before and after their respective seven-week intervention.

Among patients, those who completed the NPS showed significant improvements on two measures of health-related quality of life: average scores on the Parkinson’s Disease Questionnaire 8 (PDQ-8; higher scores indicate poorer quality) decreased from 28.1 to 23.4. Average scores on the EQ-5D, where higher scores indicate better quality, increased from 0.87 to 0.88. Other measures showed similar trends, but differences were not statistically significant.

In contrast, among patients who received standard of care, scores tended to indicate a worsening quality of life after seven weeks. Decreasing scores (indicating poorer quality) were statistically significant for three subdomains of the 11-item Life Satisfaction Checklist (LiSat-11): satisfaction with life as a whole, leisure, and contacts.

Prior to the intervention, some scores among standard care patients were also significantly higher (implying better quality) than those of the NPS group — namely, the LiSat11 satisfaction with life, as well as some domains of the Health Education Impact Questionnaire. Afterward, these differences were again no longer significant.

No statistically significant changes in questionnaire scores among caregivers who completed the NPS were seen; scores tended to be stable or to increase slightly. For caregivers in the standard of care group, scores tended to be stable or worsen, and there were statistically significant drops in scores for life satisfaction as a whole on the LiSat-11 checklist.

NPS participants also answered a questionnaire evaluating the program itself. Overall, both patients and their caregivers reported high satisfaction.

“For example,” the researchers wrote, “both PwPD [people with Parkinson’s disease] and care partners agreed that participating in the NPS was worthwhile, that its content was relevant, and that their understanding of PD had improved.”

Results are “encouraging” and show “the programme can improve health and the skills required to handle disease and manage symptoms in everyday life and can strengthen the mindset of persons affected by PD of being in charge and not allowing disease to control life,” the researchers concluded.

“The NPS should therefore be offered as an integrated part of a holistic person-centred standard care, and resources should be allocated for the provision of the programme,” they added.

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Predictive Models Could Help Diagnose Early-stage Parkinson’s, Study Finds

predictive models

Predictive models can help diagnose Parkinson’s disease in early stages, and could be used to distinguish Parkinson’s from conditions that present similarly, a new study suggests. The findings indicate that losing the sense of smell is particularly significant for predicting Parkinson’s.

The study, “Non-motor Clinical and Biomarker Predictors Enable High Cross-Validated Accuracy Detection of Early PD but Lesser Cross-Validated Accuracy Detection of Scans Without Evidence of Dopaminergic Deficit,” was published in Frontiers in Neurology.

It is widely agreed that non-motor manifestations of Parkinson’s disease  typically predate the onset of motor symptoms that are more specifically characteristic of the disease. Identifying Parkinson’s in these very early stages,  which is necessary for starting treatment as soon as possible, is a continuous clinical challenge.

A particular problem in this regard is differentiating between Parkinson’s and SWEDD (scans without evidence of dopamine deficit). SWEDD is a disease category in which a person has symptoms that are indicative of Parkinson’s, but they don’t have the changes in dopamine activity in the brain that are characteristic of Parkinson’s. The researchers who authored the new study described SWEDD as “a [Parkinson’s disease] lookalike.”

In the new study, researchers constructed five different predictive models with the dual goals of differentiating between people with early Parkinson’s and people without disease, and between people with early Parkinson’s and people with SWEDD.

Conceptually, these models were constructed by feeding clinical, biological, and demographic data into computers. Then, using specialized algorithms, the computer develops rules for distinguishing between the two relevant groups. The different models used are essentially just different underlying algorithms for this same purpose.

“Every feature used was first proven relevant in the literature. Of those, we allowed each model to pick out which predictors were most important,” study co-author Charles Leger, a PhD candidate at York University in Canada, said in a press release. “No model is guaranteed to provide the best fit. With five models, if you get the same feature that stands out, then you know that particular variable is very important in distinguishing disease. Neurologists could apply one or more of the models to their own data to … distinguish Parkinson’s pathology from pathology masquerading as Parkinson’s.”

The data used for the models was obtained from the Parkinson’s Progression Markers Initiative (PPMI) database, an observational, international clinical study to establish Parkinson’s biomarkers. The specific features analyzed in the models included loss of sense of smell, education, daytime sleepiness, and rapid eye movement (REM) sleep behavior disorder.

In total, data for 295 people with early PD, 43 with SWEDD, and 130 with no evidence of disease were analyzed. In order to be able to test their models, the researchers built them using only a subset of this data. The models were then tested on the remaining data.

The diagnostic accuracy of the models was assessed by calculating the area under the receiver operating characteristic curve (AUC). AUC is a statistical measurement of how well a given model can distinguish between two groups — in this case, early Parkinson’s vs. no disease and early Parkinson’s vs. SWEDD, in the two respective analyses. AUC values can range from 0 to 1; a value closer to 1 indicated better distinguishing accuracy.

All five models performed well at distinguishing early Parkinson’s from no disease; AUC values ranged from 0.86 to 0.928. Distinguishing early Parkinson’s from SWEDD was less definitive, with AUC values between 0.743 and 0.863.

“The discrepancy of model performance between early PD/control and early PD/SWEDD classification is, at least in part, due to the wide range of disorders encompassed by the SWEDD category,” the researchers wrote, noting that some in the field have called for a removal of the term or category SWEDD for this very reason.

Across the models, loss of sense of smell was the most important differentiator of early Parkinson’s in both analyses, with the second-most important being REM sleep behavior disorder. This indicates the importance of these symptoms in identifying early Parkinson’s disease.

Interestingly, daytime sleepiness, age, and education were important for distinguishing early Parkinson’s from SWEDD, but weren’t important for differentiating early Parkinson’s from no disease. Other variables, including levels of Parkinson’s-related biomarkers in the fluid surrounding the brain and spinal cord (cerebrospinal fluid, CSF), namely alpha-synuclein, were more important for distinguishing early Parkinson’s from no disease than from SWEDD. These findings, “warrant further investigation,” the researchers wrote.

Overall, this study supports the use of these models for detecting early Parkinson’s disease.

“These models could be very useful in differentiating patients who may present with Parkinson’s-like symptoms not related to Parkinson’s pathology from patients who actually have the disease,” said study co-author Joseph DeSouza, PhD, a professor at York University.

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Parkinson’s Foundation, VA Join to Help US Veterans Manage Disease

veterans and Parkinson's

The Parkinson’s Foundation and the U.S. Department of Veterans Affairs (VA) are collaborating on an effort to improve the health and life quality of veterans with Parkinson’s disease.

The two want to provide people who served in the U.S. Armed Forces with better resources and ways to manage Parkinson’s, a disease that affects nearly 1 million people in the country and about 110,000 veterans, half of whom are at least 65. Globally, nearly 10 million people are living with this disease.

“VA and the Foundation are working to increase access to information about Parkinson’s disease,” John L. Lehr, president and CEO of the Parkinson’s Foundation, said in a press release. “Educating and training VA staff on disease management and therapies, along with improving veterans’ access to direct health services related to the disease is a paramount objective.”

The nonprofit organization currently offers information of help in managing Parkinson’s, with topics including diet and nutrition, emotional wellness, advice for the newly diagnosed, activities of daily livingdental health, sexual health, living alone, and talking to children the disease.

A foundation webpage addresses caring for a veteran with Parkinson’s. In addition to caregiver resources, the organization provides a guide for caregivers, information about depression and other non-movement symptoms, and tips on how to prepare for hospitalizations.

VA resources specific to care partners of patients include what caregivers need to know, and Parkinson’s research, education, and clinical centers. On the second Tuesday of each month, the agency also presents a free telephone education and support conference for veterans with Parkinson’s and their  caregivers.

Many military veterans with Parkinson’s have access to specialized medical care through the VA. Visit this foundation site or this VA site to apply for care and benefits. Here are basic eligibility requirements, and information about VA healthcare for elderly veterans.

In some instances, veterans can tie their disease to their U.S. military service. Those who served from 1962 to 1975 are at an increased risk of the progressive disease due to the military’s tactical use of the herbicide known as Agent Orange.

“This collaboration ensures veterans diagnosed with Parkinson’s have access to the information and resources they need to better manage their health,” said Robert Wilkie, VA secretary. “The joint effort enhances the multitude of services that care teams at the Veterans Health Administration (VHA) provide to veterans with Parkinson’s disease.”

Go here for VHA videos of stories from veterans who are living with Parkinson’s. The integrated healthcare system serves 9 million enrolled veterans annually.

A recent study found that U.S. veterans who have post-traumatic stress disorder, or who have experienced a traumatic brain injury, are at a two times higher risk of rapid eye movement sleep behavior disorder, a risk factor for Parkinson’s.

In addition to its work to improve patient care, and the Parkinson’s Foundation is committed to advancing research toward a cure.

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Enrollment in Phase 2 Study of Cognitive Treatment Paused for Pandemic

cognition trial on hold

Aptinyx has paused patient enrollment in its Phase 2 trial evaluating NYX-458 to treat mild cognitive impairment in people with Parkinson’s disease due to the current COVID-19 pandemic

The decision was made in light of the elevated risk this virus poses to an older patient population, and the challenges in assessing changes in cognitive skills remotely. 

“In the midst of the COVID-19 global pandemic, the safety of our employees, colleagues, and patients participating in our clinical studies remains our top priority,” Norbert Riedel, PhD, president and CEO of Aptinyx, said in a press release.

People already enrolled may continue with the trial in accordance with medical guidance, the company stated, adding that updates will come “at a future date.”

NYX-458 is an oral small molecule compound that regulates the activity of N-methyl-D-aspartate (NMDA) receptors in the brain. These receptors are essential for nerve cell communication, which occur at structures called synapses located at the junction between two nerve cells.

Parkinson’s damages dopamine-producing neurons in the brain. One of dopamine’s many functions is to regulate NMDA receptors, and its loss is believed affect their regulation and, subsequently, cognition.

By controlling NMDA receptor activity, NYX-458 may help to ease or reverse cognitive impairment associated with Parkinson’s.

The randomized and double-blind Phase 2 (NCT04148391) clinical trial is evaluate the safety and tolerability of oral NYX-458 compared to placebo. Early signs of potential benefit will also be examined. It aims to enroll 135 Parkinson’s patients with mild cognitive impairment, ages 50 to 80, at sites across the U.S.

The study, which opened in December 2019, is randomly assigning patients to a daily oral dose of 10 mg, 30 mg, or 100 mg of NYX-458, or a placebo capsule, for 12 weeks. Those who complete this part then enter a two-week follow-up period.

NYX-458’s efficacy will be measured by how it affects patients’ memory, attention, executive function, visuospatial deficits, and quality of life, the company reported previously.

This Phase 2 trial was supported by positive preclinical data in a non-human primate model of Parkinson’s disease. In these animals, NYX-458 was seen to significantly increase attention, improve cognitive flexibility, and enhance working memory as quickly as one day after the administration of a single oral dose.

Importantly, the compound did not interfere with levodopa, a standard motor symptom treatment for Parkinson’s, suggesting these two therapies might be used in combination.

NYX-458’s safety, tolerability and pharmacokinetics (the way a compound moves through the body) was investigated in a placebo-controlled Phase 1 clinical trial in 62 healthy volunteers given NYX-458 in ascending doses ranging from 10 mg to 200 mg.

Results showed both safety and tolerability in these people, including a group of elderly volunteers. No serious adverse events associated with the treatment were reported.

Patient enrollment has also been paused in other Aptinyx’s clinical trials, including Phase 2b studies of NYX-2925, an investigational therapy for chronic pain in people with  painful diabetic peripheral neuropathy (NCT04146896) and fibromyalgia (NCT04147858).

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