Digital Management of Parkinson Disease: Is Technology the Future?

Tori Rodriguez, MA, LPC             

March 22, 2019

The integration of technology into medicine has expanded into various uses in patients with Parkinson disease.

With the ever-increasing integration of technology into nearly every aspect of modern life, its applications in medicine continue to expand, including in the area of Parkinson disease (PD).

A recent randomized controlled trial of 195 patients with PD, for example, found that physician care delivered via telemedicine into patients’ homes was as effective as in-person care.¹ Additional emerging research supports the benefits of other digital tools in this patient population.

In a review published online in the Journal of Parkinson’s Disease, Clint Hansen, PhD, of the Department of Neurology at Christian-Albrechts-Universität Kiel and the University Hospital Schleswig-Holstein, Campus Kiel in Kiel, Germany, and colleagues explored current and future directions regarding the use of technologies such as the electronic health record (EHR) and wearable sensors in PD management.²

Although many institutions and private practices have replaced paper-based medical records with EHRs, there are often limitations on how these are used in clinical decision making. This is likely to change significantly within the next decade, as most “countries with established health structures will then have nationwide provision of innovative EHRs with cloud storage solutions, and most of these EHRs will be patient-controlled,” Dr Hansen and colleagues noted.

In addition, they wrote, EHRs will “communicate with clinical data warehouses that consolidate hospital- and practice-derived data, with population health analytics databases that will provide information about disease risk and prevention, and with digital technology that is used by… the patient.”² They also expect that moving forward, data protection laws will become more stringent, thus reducing the risk for privacy violations and increasing patient acceptance of EHRs.

With the growing use of digital devices to provide personalized feedback on measures of health, including those worn on the wrist or contained in shoes, it is conceivable that similar tools could be designed to facilitate the continuous monitoring of disease manifestations in patients with PD. These systems “will most probably be easily and widely used and not [require] an active interaction with the user, will be approved by regulatory authorities and the costs will be covered by health insurances,” noted Dr Hansen and colleauges.²

As a template for such devices intended for application in PD, the authors pointed to continuous subcutaneous glucose monitors that are used in patients with diabetes. Other possibilities include integrated detection systems in hearing aids, glasses, and earphones to allow patient interaction with the environment; analysis of mobility data such as reaction times and multitasking ability; insertable cardiac monitors; use of a smartphone as an accelerometer platform to gauge the treatment efficacy of deep brain stimulation; and remote assessment of autonomic features and gut motility. Many of these systems should be easily individualized to each patient.

In another review published in the same journal issue, the authors propose the use of a sensor-based system to analyze gait and detect falls in patients with PD, which would include decision support based on big data sources.³ One benefit of this type of approach is the “standardization of real-life medical data derived from individual patients into longitudinal multimodal disease trajectories…[which] will allow matching and clustering of different patient trajectories for individualized prediction about the expected treatment efficacy of particular interventions,” wrote Jochen Klucken, MD, of the Department of Molecular Neurology at Friedrich-Alexander University, and colleagues, in the Journal of Parkinson’s Disease. “

Ultimately, the growing amount of medical data derived from individualized real-life treatment contexts will enable academic and industrial research to further improve objective outcomes and better tailor individual care.”

To glean further insights pertaining to the use of digital health technology in the realm of PD, Neurology Advisor spoke briefly with Alberto J. Espay, MD, MSc, professor of neurology and director and endowed chair of the James J. and Joan A. Gardner Center for Parkinson’s Disease and Movement Disorders at the University of Cincinnati Academic Health Center. Dr Espay is also chair of the Movement Disorders Society Task Force on Technology, which promotes the development of digital health systems that will achieve the goals mentioned above.⁴  

Neurology Advisor: What is a key example of how digital health technologies could be used to improve PD care?

Dr Espay: Digital health technologies have the capacity of capturing intraday fluctuations with the kind of granularity that no scales, diaries, or questionnaires can.

Neurology Advisor: How can clinicians support these developments?

Dr Espay: Clinicians can stimulate their patients’ participation in a variety of technology-integration projects through many organizations. This is a great time to participate and contribute.

There will be a variety of ongoing studies aimed at developing a new version of the diary in electronic format for the digital age, integrated with mobile health technologies (“e-diary/tracker”). This is being coordinated through the Task Force on Technology of the International Parkinson’s and Movement Disorders Society. Efforts such as these are ongoing or will soon occur in many countries in the world.

Neurology Advisor: What should be next steps in terms of research or development in this area?

Dr Espay: The future development of a unifying platform for integrating and synchronizing the data from multiple technologies, one of the goals of the Movement Disorders Society Technology Task Force, could provide the most comprehensive picture of patients’ function than anything we currently have.

Neurology Advisor: Would you like to note any additional takeaways on the topic?

Dr Espay: Technology offers us the means to improving the ends of health care delivery. In the future it will weave seamlessly into patients’ lives to enhance the capture of data, help anticipate periods in which changes are needed, and empower patients to apply strategies to enhance their quality of life.

References

1. Beck CA, Beran DB, Biglan KM, et al. National randomized controlled trial of virtual house calls for Parkinson disease. Neurology. 2017;89(11):1152-1161.

2. Hansen C, Sanchez-Ferro A, Maetzler W. How mobile health technology and electronic health records will change care of patients with Parkinson’s disease. J Parkinsons Dis. 2018;8(Suppl 1):S41-S45.

3. Klucken J, Krüger R, Schmidt P, Bloem BR. Management of Parkinson’s disease 20 years from now: towards digital health pathways. J Parkinsons Dis. 2018;8(Suppl 1):S85-S94.

4. Espay AJ, Bonato P, Nahab FB, et al. Technology in Parkinson’s disease: challenges and opportunities. Mov Disord. 2016;31(9):1272-1282.

This article originally appeared on Neurology Advisor

https://www.medicalbag.com/home/more/tech-talk/digital-management-of-parkinson-disease-is-technology-the-future/

A Simple Skin Swab Could Soon Identify People at Risk for Parkinson's

 BY JAKE ROSSEN            MARCH 22, 2019

More than 200 years have passed since physician James Parkinson first identified the degenerative neurological disorder that bears his name. Over five million people worldwide suffer from Parkinson’s disease, a neurological condition characterized by muscle tremors and other symptoms. Diagnosis is based on those symptoms rather than blood tests, brain imaging, or any other laboratory evidence.

Now, science may be close to a simple and non-invasive method for diagnosing the disease based on a waxy substance called sebum, which people secrete through their skin. And it’s thanks to a woman with the unique ability to sniff out differences in the sebum of those with Parkinson's—years before a diagnosis can be made.

The Guardian describes how researchers at the University of Manchester partnered with a nurse named Joy Milne, a "super smeller" who can detect a unique odor emanating from Parkinson's patients that is unnoticeable to most people. Working with Tilo Kunath, a neurobiologist at Edinburgh University, Milne and the researchers pinpointed the strongest odor coming from the patients' upper backs, where sebum-emitting pores are concentrated.

For a new study in the journal ACS Central Science, the researchers analyzed skin swabs from 64 Parkinson's and non-Parkinson's subjects and found that three substances—eicosane, hippuric acid, and octadecanal—were present in higher concentrations in the Parkinson’s patients. One substance, perillic aldehyde, was lower. Milne confirmed that these swabs bore the distinct, musky odor associated with Parkinson’s patients.

Researchers also found no difference between patients who took drugs to control symptoms and those who did not, meaning that drug metabolites had no influence on the odor or compounds.

The next step will be to swab a a much larger cohort of Parkinson’s patients and healthy volunteers to see if the results are consistent and reliable. If these compounds are able to accurately identify Parkinson’s, researchers are optimistic that it could lead to earlier diagnosis and more effective interventions.

http://mentalfloss.com/article/577724/parkinsons-disease-diagnosis-could-be-possible-with-skin-test

How I Deal with Chronic Pain in Parkinson’s

  By Frank Church · March 21, 2019

A large number of aging adults (65+ years of age) and many of us with Parkinson’s are dealing with chronic pain. However, chronic pain is not a mandatory part of normal adult aging. You control how you manage chronic pain. Here is what I do, and herein is my basic philosophy. I can summarize it in one word: proactive.

Defining chronic pain

Recently, the World Health Assembly defined chronic pain as “persistent or recurrent pain lasting longer than three months.” Furthermore, they included optional specifiers based on pain severity (intensity, pain-related distress, and functional impairment) and psychosocial factors. From a brief review of this field, there is no clear consensus for defining chronic pain, except that it a real medical issue that mandates well-orchestrated care by physicians and other allied fields and experts in healthcare. Lynne Tillman said, “Now that I am conscious of the world of chronic pain when I see somebody walking down the street who’s having trouble, I feel a sadness for them. I notice.”

Chronic pain in Parkinson’s

I recently posted a description of chronic pain in Parkinson’s. Briefly, there are six steps that our healthcare providers may use to deal with our chronic pain, including (1) your quality-of-life matters; (2) keep a “pain diary”; (3) referral to a Physical Therapist; (4) carefully assess your dopaminergic-based drug therapy; (5) the guide called the Beers Criteria (American Geriatric Society), which helps your healthcare provider in improving medication safety in older adults; and (6) non-pharmacological intervention including different forms of exercise (especially non-weight-bearing activities) and other modalities. 

Keep in mind that you must be proactive in your approach to managing your chronic pain. Just like our Parkinson’s is an individual disorder in terms of symptoms and rate of progression, chronic pain will also be individually expressed in anatomic location, the degree of discomfort, and how it alters our quality-of-life. My approach has worked for me. Moreover, I’m happy to describe it here briefly.

Teamwork makes a difference

My Neurologist and Internist are available by e-mail to answer my questions outside of my planned visits. Additionally, I routinely seek the advice of two key healthcare experts within the Movement Disorder Group, our Social Worker and one of our Physical Therapists. Separately, I have good relationships with several other Physical Therapists and researchers in the world arena of Parkinson’s disease science and research. Thus, I believe that from what my ‘constellation of healthcare experts‘ recommend, I can make the best-informed response(s) to manage my health, and especially here, chronic pain. Phil Anselmo remarked, “You can only exist as far as your mind will allow you to exist, and I think chronic pain will stop time dead in its tracks. You feel like you’re the only one, and how unfair it is, and a million different feel-sorry-for-yourself type feelings.”

My approach to managing chronic pain

My chronic pain is primarily lower back and neck. Occasionally, I have foot and wrist/arm pain, but principally, it always includes lower back and neck. It was important to be an active participant with my healthcare team to formulate a plan.

My Neurologist and I have worked hard to optimize my dopaminergic-based drug therapy (necessary since I am still working full-time and balancing many daily aspects of a career in academic medicine). My dopaminergic-based drug therapy now includes carbidopa/levodopa and a dopamine agonist (oral and external patch).

Physical therapists gave me detailed exercises for my lower back and neck. My treatment approach was to exercise the areas as required by the PT recommendations. My maintenance plan is to use stretching and training on the regions causing the chronic pain. Again, stay engaged and go after the area(s) that are chronically in pain.

In the initial time-period following my Parkinson’s diagnosis, with daily guidance by our UNC Physical Therapy and Rehabilitation Group, I did LSVT BIG® with Dr. Diane M. Meyer. What a great set of exercises for a person-with-Parkinson’s (PwP) to use to help their body be more regular, and it helped my chronic pain. It made a difference. I still use these exercises on an occasional basis.

I then advanced to PWR!Moves® (PWR = Parkinson Wellness Recovery), a more dynamic set of exercises developed by Dr. Becky Farley. Pwr!Moves® routines allow a PwP to maintain or restore skills that deteriorate and interfere with everyday movements. Dr. Farley also was responsible for the LSVT BIG® exercise program. I try to do PWR!Moves® very frequently, and it makes a big difference for my chronic pain. I routinely get advice from the experts at the PWR!Gym in Tucson, AZ, especially Dr. Jennifer Bazan-Wigle and Dr. Farley. Deep-tissue massage has also provided some relief in my chronic lower back and neck pain. 

Neural Reset Therapy® or NRT is a specialized form of therapy. NRT applies the laws of physiology to approach the affected body area directly. The nervous system controls muscles, and the trained NRT therapist resets the nervous system to release a tissue from excessive tension. With this reset, NRT renews the area, and the pain is reduced. It truly works. I am fortunate to have access to an NRT-trained medical massage therapist (Kristi Vloedman). 

The final form of treatment I receive is by chiropractic care. At first, I was skeptical of this type of treatment. However, I have found much relief in my back and neck by routine visits to my chiropractors in Chapel Hill. A chiropractor focuses treatment of neuromuscular disorders, usually by manipulation of the spine. I am fortunate to have two phenomenal chiropractors (Dr. Shannon Bigbie and Dr. Avery Garrabrant at http://www.ignitechapelhill.com/) because they use a wide range of techniques and technology to treat both my back and neck pain, and they stress education and awareness of your physical condition.

To recap, I use several different types of treatment plans and this uses several healthcare providers and experts to manage my Parkinson’s and the chronic pain involved in this complex neurodegenerative disorder. Likely, in the absence of chronic pain, I might still have followed this treatment plan to deal with my disease. 

The reality of chronic pain in Parkinson’s

In a recent study, Rana et al. found that PwP had significantly more pain than their age-match controls without Parkinson’s. Additionally, they reported that PwP with symptoms of depression had significantly higher levels of pain compared to control patients without depression. 

Your chronic pain in the presence of Parkinson’s is real. Cheryl Richardson said, “If I were somebody dealing with chronic pain, I would see it as a challenge to manage my mind; even knowing that the effect of my thoughts is not only affecting my experience, but it is absolutely affecting the state of my health.” You need to manage chronic pain carefully because it’s part of your life-equation for quality-of-life. Use your experts to make a plan, try it, be proactive. If it is not working for you, revise the plan, but stay engaged. The goal is reducing your chronic pain; please keep working on it.

https://parkinsonsdisease.net/living/dealing-chronic-pain/

The Death of Self: A Casualty of Chronic Disease

MARCH 22, 2019 Dr. C     BY DR. C

Death, the “D” in the CHRONDI Creed, refers more to the death of our self-identity than it does to physical death. As we endure the long battle with a chronic disease and deal with a gradual progression in symptoms, a loss of function occurs. I touch upon the stealing away of bits and pieces of both physical and mental function in my column about the “disease thief.”

The disease thief robs us of so many of the ways by which we know ourselves. It is a death of the self that is a casualty of chronic disease. The death of self needs to be addressed with as much mindfulness as any other part of the creed for total health to be maintained at the highest level possible.

There is no manual for navigating through the death of self. I was educated in many ways to be prepared for it. And yet when it happened, I was shocked by the severity of its effects. Parkinson’s disease gradually took from me those things I identified as belonging to myself, things I would pull out of my pocket when someone asked, “What do you do?”

Following is a list of things that were stolen from me, roughly in chronological order:

• Field mineral specimen collecting (since I was a teenager)
• Professional field geologist
• Hiking and exploring rugged terrain
• Clinical counseling work
• Professor of counseling and geology

The time and money spent on four college degrees are behind all the years of experience expressed in the above list. Now all are casualties of a chronic disease. It is the death of self.

Looking in the mirror, past the gray hair and crevasses of age, deep into multicolored eyes, I found nothing that I remembered as me. The self I once knew was gone — dead! I was sitting in a void in a life without meaning, with nothing of familiarity.

From my clinical work, I knew that people get lost when this happens. It can be quite difficult to find the way back. I also knew something about this journey from mystical teachings, but knowing and living through it personally are two different things. Somehow, I had to find my way out. I had to heal from the death of self.

We can apply stages of grief to healing from the death of self. As I mentioned in the disease thief column, we should use terror management should as needed. And it is important to have a support network through the process, including peers, family, and technology. Additionally, the CHRONDI Creed can be used to help with healing, particularly the “I” in Identity. I will cover that in the next column.

Have you experienced the “death of self” while battling a chronic disease? What progress have you made?

***

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.

https://parkinsonsnewstoday.com/2019/03/22/death-self-casualty-chronic-disease-chrondi-creed-identity/

Grant Will Enable Pharmacologist to Expand Gaucher Disease Research to Parkinson’s

 MARCH 22, 2019     BY MARY CHAPMAN 

As it sometimes happens in rare-disease research, a prospective therapy for one disorder has potential to help another. So it is that, at Temple University, molecular pharmacologist Marlene Jacobson, PhD, has been awarded a joint grant to explore how a discovery could affect Parkinson’s disease (PD) patients.

Specifically, The Michael J. Fox Foundation (MJFF) and The Silverstein Foundation for Parkinson’s with GBA want Jacobson to further study compounds that she found could reverse Gaucher disease Type 2, the most severe form of the lysosomal storage disorder caused by deficiencies in the glucocerbrosidase (GCase) protein due to mutations in the GBA1 gene. 

Some 10 percent of PD patients carry the same mutation, making it the most common genetic disease risk. GCase allows brain cells to clear debris via lysosomes, which are special compartments within cells that digest and recycle different types of molecules. Patients with reductions in GCase activity can’t effectively remove debris, which scientists widely hypothesize is toxic to brain cells.

“I came to Temple because I wanted to make a difference in patients’ lives,” said Jacobson in a press release. “I wanted to use my drug discovery skills and apply them to help patients who are underserved.”

An associate professor of pharmacodynamics in Temple’s Department of Pharmaceutical Sciences, and associate director of its Moulder Center for Drug Discovery Research, Jacobson will expand her Gaucher research to include PD patient cells that have the GBA1 mutation. She joined Temple six years ago after 24 years in research and drug discovery for Merck Research Laboratories.

”The key is we have the full disease environment in the cell,” said Jacobson. “We’re very fortunate to have access to these patient cells, and we treat them with respect as they provide an advantage in demonstrating a compound’s potential to reverse a disease state.”

Parkinson’s patients with the GBA mutation develop the disease at an earlier age, she said, and their cognition symptoms are worse relative to patients without the mutation.

”The link between mutations in Gaucher disease and Parkinson’s disease suggests a common disrupted mechanism or pathway. We can translate the compounds we have found to improve Gaucher and Parkinson’s.”

Currently, there is no effective treatment for Gaucher disease Type 2. In Parkinson’s, available therapies are focused on raising dopamine levels in patients’ brains, or on controlling the symptoms themselves.

“This work is so exciting and so primed for discovery,” Jacobson said, adding that the pharmaceutical industry is increasingly looking to academic investigators for breakthrough ideas leading to new treatments.

The amount of the grant was not disclosed.

The MJFF aims to find a cure for Parkinson’s through an aggressively funded research agenda, and to help develop improved therapies for current patients. The Silverstein Foundation is focused on finding ways to prevent the onset of Parkinson’s in GBA-mutation carriers.

https://parkinsonsnewstoday.com/2019/03/22/temple-pharmacologist-grant-expand-research-parkinsons/

Motor Dysfunction Among the Predictive Markers of Parkinson’s for People with Sleep Disorder, Study Reports

 MARCH 22, 2019 BY JOSE MARQUES LOPES, PHD 

Mild cognitive impairment, motor and olfactory deficits, and erectile dysfunction are among the markers able to predict the development of Parkinson’s and associated disorders in people with rapid eye movement sleep behavior disorder, according to a large study.

The research was published in the article, “Risk and predictors of dementia and parkinsonism in idiopathic REM sleep behaviour disorder: a multicentre study,” in the journal Brain.

Disorders characterized by the aggregation of alpha-synuclein — such as Parkinson’s, dementia with Lewy bodies, and multiple system atrophy  — may all have an early period of more than 10 years that’s characterized by signs of neurodegeneration, but without full clinical disease.

Unlike most markers of this early — or prodromal — period, rapid eye movement sleep behavior disorder (RBD) has been specifically linked to the development of synuclein-related diseases. RBD of no known cause, or idiopathic RBD (iRBD), occurs in approximately 1% of people older than 60, and usually converts to Parkinson’s or related disorders over a decade or more. This means that 1% of the elderly population have an identifiable but often undetected early-stage neurodegenerative syndrome.

As most studies on predictors of these parkinsonism diseases had been single-center, a team at The Neuro — Montreal Neurological Institute and Hospital — and the Montreal General Hospital of the McGill University Health Centre combined the research experience of 24 centers in North America, Europe, Seoul, and Sydney, which participated in the International RBD Study Group, to measure the risk of developing such disorders and test 21 potential predictors.

At the beginning of the study, a total of 1,280 participants (average age of 66.3 years, and 82.5% men) with iRBD but without parkinsonism or dementia underwent a variety of tests to assess sleep disturbances, motor function, cognition, depression, anxiety, olfaction, and autonomic function. The patients were then followed for up to 19 years. According to the team, this was “the largest study ever performed in iRBD.”

Over a mean period of 4.6 years, 352 patients (28%, with a mean age of 67.6 years) acquired an overt neurodegenerative syndrome, which corresponded to an annual rate of 6.25%. The risk of developing such diseases progressively increased from 10.6% after two years to 73.5% after 12 years. Among these 352 patients, 199 first showed signs of parkinsonism, while 153 developed dementia first.

Then the analysis revealed that motor dysfunction — as assessed through different measures — olfactory deficit, mild cognitive impairment, erectile dysfunction, an abnormal dopamine transporter (DAT) scan, color vision impairment, constipation, REM sleep without muscle atonia (reduced strength), and older age significantly predicted neurodegenerative disease development.

DAT is responsible for the uptake of dopamine — the neurotransmitter found in lower levels in people with Parkinson’s — into nerve cells.

In contrast, sex, insomnia, daytime sleepiness, restless legs syndrome, sleep apnea, urinary dysfunction, and depression or anxiety were not significant predictors.

Only those predictive markers that tested cognition and quantitative motor function differentiated the people who first developed dementia from those first showing signs of parkinsonism. These assessments of quantitative motor function were simple office-based tests that took less than five minutes.

“Clearly these are strong candidates for selecting patients for future neuroprotective trials, and could even obviate the need for sophisticated imaging techniques,” the investigators wrote.

“We confirmed a very high risk of (Parkinson’s) in people with REM sleep disorder and found several strong predictors of this progression,” Ron Postuma, the study’s lead author, said in a press release. “As new disease-modifying treatments are being developed for (Parkinson’s) and related diseases, these patients are ideal candidates for neuroprotective trials.”

A separate analysis estimated that 366 patients per experimental group would need to be recruited into a two-year trial for a therapy to reduce in half the incidence of RBD converting to parkinsonism or dementia. Increasing the trial duration or assuming a greater reduction in disease development led to lower estimates for the number of patients required. Also, this analysis showed that using different predictive markers to classify patients would significantly alter the number of patients required for clinical trials.

“Of course, exact sample size calculations will depend on the specifics of a clinical trial; nevertheless, the fact that 24 centers combined to produce these estimates can provide some confidence for trial planners that sample sizes will be representative of the global experience,” the study stated. “Notably, the total sample size for a future neuroprotective trial is less than the number of participants who were recruited to this study.”

https://parkinsonsnewstoday.com/2019/03/22/motor-dysfunction-among-predictive-markers-of-parkinsons-in-people-with-sleep-disorder-study-reports/

Exosomes help track effectiveness of experimental Parkinson's disease drug

March 21, 2019

Exosomes—nanoscale particles that are produced by all types of cells and circulate in the bloodstream—have proven to be reliable markers for the effectiveness of an experimental drug for Parkinson’s disease (PD) in a new study. The research, jointly led by investigators from the NIA Intramural Research Program and colleagues from the United Kingdom, was published in the Jan. 14 issue of JAMA Neurology.

Exosomes originating from neurons carry an information-rich cellular cargo and can cross the blood-brain barrier. Several previous studies have demonstrated that it’s possible to track key disease-related proteins within this exosome cargo and that this technique can successfully differentiate between healthy control samples and those from patients with Alzheimer’s or Parkinson’s.

In this study, the NIA team examined neuronal exosomes found in blood serum samples from 60 participants in the Exenatide-PD trial, which was conducted in the United Kingdom. Exenatide, an experimental drug of a type commonly used to treat diabetes, has shown promise for improving motor function and potentially protecting against PD, and other neurological diseases.

Patients with PD who received regular exenatide injections showed higher levels of active forms of brain insulin signaling proteins and related mechanisms in their exosomes over time compared with their baseline measurements and those of participants who received a placebo. The patients on exenatide also showed improvements in motor function tests for PD compared to those in the control group, and these improvements were found to correspond to changes in exosome proteins.

This study confirms the value of exosomes as an additional analytical tool to show target engagement in clinical trials. The investigators are optimistic about future work to examine how exosomes could help speed up validation in clinical trials of potential new drugs for PD, Alzheimer’s disease, and other neurological disorders.

Reference: Athauda D, et al. Utility of neuronal-derived exosomes to examine molecular mechanisms that affect motor function in patients with Parkinson disease: A secondary analysis of the Exenatide-PD trial. JAMA Neurology. 2019 Jan 14. doi: 10.1001/jamaneurol.2018.4304.

https://www.nia.nih.gov/news/exosomes-help-track-effectiveness-experimental-parkinsons-disease-drug

Many of the Rare diseases and their treatments are set to become cheaper.

Mar 21, 2019  By Ananya Mandal, MD

 Ananya Mandal, MD

Under Australia's Pharmaceutical Benefits Scheme (PBS), soon many of the rare diseases and their treatments are set to become cheaper.

Brentuximab vedotin for CTCL patients

Patients with cutaneous T-cell lymphomas (CTCL) may soon be able to get access to brentuximab vedotin (sold as adcetris) under the Pharmaceutical Benefits Scheme (PBS). An additional $19 million a year has been added to the scheme.

CTCL is diagnosed in around 150 to 200 persons annually and is the result of a cancer in the T cells of the skin. The initial symptoms are similar to eczema with itchy rash like lesions and may go undiagnosed. It commonly affects males between ages of 40 and 60 years.

In these patients a combined treatment with brentuximab vedotin and chemotherapy can kill the specific cells. The treatment can cost up to $300,000 a year and this news about availability of the therapy under the scheme is a welcome one says Associate Professor Joel Rhee, Chair of the RACGP Cancer and Palliative Care Specific Interests. He said, “GPs have an important role in the assessment and diagnosis of cancers, including rare types such as CTCL, which is often diagnosed through a skin biopsy. GPs should familiarise themselves with immunotherapy, especially their side effects and potential complications, as they become an integral part of cancer care.” Under the PBS the prices of brentuximab vedotin will come down to $40.30 per script, or $6.50 with a concession card.

Health Minister Greg Hunt in a statement said, “It has the potential to save and protect lives.”

Safinamide for Parkinson’s disease patients and Riluzole for motor neurone disease patients

Patients with Parkinson’s disease would soon get an easier access to safinamide (sold as xadago). This drug works by increasing the levels of dopamine in the brains of these patients. At present patients are paying over $1400 annually for treatment with the disease.

Riluzole (sold as teglutik) is helpful in patients with amyotrophic lateral sclerosis, a form of motor neurone disease. This is a severely debilitating disease leading to degeneration of muscles. The drug presently costs around $2900 annually to patients.

Safinamide and riluzole, under the PBS would now be available for $40.30 per script, or $6.50 with a concession card.

The drugs under the scheme would be accessible from 1st April this year say the officials.

https://www.news-medical.net/news/20190321/New-drugs-on-PBS-for-Parkinsons-MND-and-Cutaneous-T-cell-lymphoma.aspx