Safety and Efficacy of Oral Cannabidiol in Treating Parkinson’s Psychosis Focus of Phase 2 Study in UK


A planned Phase 2 clinical trial will assess the safety and effectiveness of pharmaceutical-grade cannabidiol (CBD) in treating Parkinson’s-related psychosis.

The trial was announced by Parkinson’s UK, which is investing £1.2 million (roughly $1.5 million) to support the study, due to begin enrolling patients in 2020.

Psychosis is estimated to affect more than half of people with Parkinson’s disease. It is characterized by hallucinations (seeing, hearing, or feeling things that are not really there) and delusions (fixed beliefs that are demonstrably untrue).

These symptoms are typically managed by reducing or stopping the use of medications used to treat Parkinson’s, but such choices have the obvious drawback of arresting any benefits those treatments provide. Antipsychotics are sometimes also used, but they can carry side effects or worsen motor symptoms.

Nuplazid (pimavanserin, by Acadia Pharmaceuticals) is the only medicine currently approved to treat Parkinson’s-related psychosis in the United States; no approved therapies for this condition are available in the United Kingdom.

“Current treatments prescribed by clinicians for psychosis typically work by blocking dopamine receptors, which can increase the problems people with Parkinson’s experience with movement and other symptoms of the condition,” Sagnik Bhattacharya, a professor at King’s College London who will help lead the trial, said in the press release.

This trial will “will determine, for the first time, whether CBD can correct the abnormal functioning of the brain that is causing symptoms such as hallucinations and delusions,” Bhattacharya added.

Cannabidiol is a non-psychoactive component of the cannabis plant, meaning it is found in cannabis, but unlike tetrahydrocannabinol or THC does not induce a feeling of being “high.” CBD is currently undergoing a renaissance of interest for its potential medical uses.

“We know from a recent survey we carried out, that people with Parkinson’s would continue to use, or start using, cannabis-derived products if robust evidence became available that they are safe and effective in treating Parkinson’s symptoms,” said Arthur Roach, PhD, the director of research at Parkinson’s UK. “One of the key questions this clinical trial will address is if CBD is safe to use for Parkinson’s-related psychosis, which has never been done before.”

The two-part study will begin with a six-week pilot phase to evaluate the safety, tolerability and effectiveness of CBD in people with Parkinson’s-related psychosis. Participants will be given daily oral CBD capsules at doses up to 1 gram per day to find the optimum dose. Then, 120 patients will be randomized to treatment with either CBD or a placebo for 12 weeks.

In addition to safety, trial goals (endpoints) will include a detailed assessment of psychotic, motor and non-motor symptoms, as well as brain imaging.

“We will be assessing how safe CBD is for people with Parkinson’s, what the correct dosage is and how it is tolerated alongside the different medications someone with the condition may already be on,” Bhattacharya said. “The study will also look at the effect of CBD on other symptoms which will pave the way for scientists to investigate the potential of the compound in treating these in future studies.

“We hope that this will progress to large-scale clinical trials — the final step towards becoming a new treatment that will improve the lives of people with Parkinson’s,” Bhattacharya added.

If successful, this study “could result in a regulated cannabinoid-based medicine being prescribed and used in the clinic, as opposed to self-administration of expensive supplements that have not been monitored for their composition or effects,” Roach said.

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Mutations in Alpha-Synuclein Speed Protein Clumping in Familial Parkinson’s and Affect Severity, Study Finds

alpha-synuclein and A53T

A detailed analysis of alpha-synuclein — a key protein involved in Parkinson’s — revealed how variants of this protein change over time, allowing researchers to identify the initial stages of protein aggregation involved in early onset disease.

These findings provide new insights into how genetic mutations — especially the point mutation A53T — can contribute to familial Parkinson’s, and into understanding why this disease form manifests earlier and is often more severe than sporadic (of unknown cause) Parkinson’s.

The study, “Alpha-synuclein stepwise aggregation reveals features of an early onset mutation in Parkinson’s disease,” was published in the journal Communications Biology.

Parkinson’s is largely a sporadic disease, with 15% to 25% of all cases linked to inherited genetic mutations. One of the first genes identified as directly associated with Parkinson’s, leading to early onset disease, was the alpha-synuclein coding gene SNCA.

It is widely accepted that alpha-synuclein is an important element that drives nerve cell death across several human neurodegenerative disorders, including Parkinson’s and dementia with Lewy bodies. Its toxic effect is, at least in part, tied to the formation of abnormal protein aggregates or clumps.

Despite available knowledge of the damaging impact alpha-synuclein clumps have on nerve cells, the process by which alpha-synuclein changes from a single protein structure into an aggregate form remains poorly understood.

Researchers at the Federal University of Rio de Janeiro (UFRJ) in Brazil conducted a series of biochemical, kinetic, and structural studies to address this gap.

They evaluated in detail the behavior of alpha-synuclein — both its normal form as well as mutated versions found in people with familial Parkinson’s — and its ability to form toxic clumps.

“The conversion from one protein stage to the other takes place slowly. The intermediate structures and the amyloid aggregates accumulate over time in the brain. So far, we don’t know which species cause the symptoms and toxicity to cells,” Guilherme A. P. de Oliveira, a professor at UFRJ and the study’s lead author, said in a press release.

“If we understand the protein species forming during the early stages of disease conversion, we can propose new therapies for disease detection before the symptoms appear.”

Results showed that the versions of alpha-synuclein carrying A53T, A30P, or E46K point mutations were able to from small aggregates (known as oligomers) at a much faster rate than a normal version of the protein.

Of note, point mutations are genetic alterations where a single nucleotide — the building blocks of DNA — is changed, inserted, or deleted from a sequence of DNA. If you think of DNA as a Lego train, a point mutation would be the same as changing, adding, or taking out a single piece. 

Next, the researchers used cutting-edge imaging techniques to visualize for the first time, in detail and over time, all the elements involved in the expansion of alpha-synuclein aggregates — their transition from early oligomers to intermediate fibrils to late filaments.

“By (…) acquiring advanced electron microscope images, we are able to better understand these wrong protein associations in their native environment and [potentially find] ways to avoid their formation,” Oliveira said.

This approach showed that the different protein versions give rise to structurally different fibrils.

The expansion of fibrils into long filaments was found to be dependent on the ability of alpha-synuclein to continue to recruit available oligomers.

Interestingly, the A53T point mutated version was able to overcome some of the limits on protein clumping imposed by the surrounding environment, and for which normal alpha-synuclein showed a sensitivity. This suggests that A53T mutations give alpha-synuclein a greater potential to promote aggregation and induce faster spreading of its toxic clumps.

“Our findings place A53T with features that may explain the early onset of familial Parkinson’s disease cases bearing this mutation,” the researchers concluded.

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We Share a Fear of Being a Burden, but We All Need Someone


“You’re not a burden. You’re a human.” –Anonymous

It’s easy to feel alone when living with a chronic illness. You may think no one else truly understands, that you’re nothing more than a burden to others — with all of your “oddities,” you no longer fit in. Those feelings can push you to believe that you are destined to being alone, which leads you toward loneliness.

If you’re not involved in a community support group for people who also have your chronic illness, you are missing out. I am not the kind of person who jumps up and heads out the door to join a support group. It took me a long time to get connected because when I was diagnosed, I was working, and my hours didn’t allow me to attend a structured group. So I found support another way.

One night while surfing the internet, I came across a site, DailyStrength. This online community of people with various diseases and issues is divided into small groups — one of these is for people with Parkinson’s disease. I clicked and joined.

At first, I was an observer. I quickly recognized that this group of people cared for each other. The sense of community was phenomenal. They understood each other in a way no one else could. They were in the same boat or rowing a similar one — venting, grieving, encouraging, supporting — like a family sharing an unspoken bond. 

I wanted to be a part of that family.

“The friend who can be silent with us in a moment of despair or confusion … who can tolerate not knowing, not curing … that is a friend who cares.”  –Henri Nouwen

The real fear of being alone is familiar to anyone with a serious illness. We fear something will happen to us when no one is around to help — for instance, falling and being unable to get up. We worry about being alone at the end of our journey with no one by our side. We fear that no one will understand what we’re going through — or will go through — and that we’ll find ourselves alone, physically and emotionally.

I have always been a “giver.” And I know it’s hard for some givers to receive. It feels awkward, unfamiliar, and selfish. If you are a giver, you will know of the joy and the blessing that you receive when you give. So when we refuse to be the receiver, we are taking that giver’s blessing from others.

Do you need someone? 

Do you need someone to talk to, care, or understand? Would you like to have someone to sit and cry with you? Let them know. No one can read your mind. Trust me on this.

Many of us who have a chronic illness share a fear of being a burden to someone else. If you have Parkinson’s, you will have to accept help from someone, somewhere, somehow, at some time. 

My story of being humbled

In my earlier days of Parkinson’s, I found it increasingly difficult to tie my shoes. To have someone tie them for me when I was in my early 40s was a little humiliating to me. 

I knew that it could be worse, but I felt like a kindergartener having her mommy tie her shoes because she couldn’t figure it out for herself. 

I disliked asking my husband to tie my shoes. It felt like an inconvenience, a burden with a capital “B.”

Having my shoes tied humbled me and also prompted me to find footwear that I could slip on by myself. Is it still an issue of pride? Perhaps. Or maybe I merely want to hold on to my independence for as long as possible.

“Life’s challenges are not supposed to paralyze you, they’re supposed to help you discover who you are.”Bernice Johnson Reagon

Don’t allow yourself to be paralyzed and without friends. Put on those shoes, even if someone else has to tie them for you. Then get up and go out. Make a new friend or call an old one. You’ll feel better for it.


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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Researchers Using Real-time Brain Mapping to Search for Therapeutic Targets in PD

brain mapping

Using its brain mapping platform, Inscopix will team up with researchers at the Broad Institute of MIT and Harvard to investigate how changes in brain activity alter the functioning of nerve cells. The goal is to identify new therapeutic targets for Parkinson’s disease.

The collaboration will be led by Evan Macosko, MD, PhD, of the Broad Institute’s Macosko Lab, an expert in single-cell transcriptomics — a next-generation sequencing approach that assesses how gene activity changes in a single cell.

This research builds on a previous study that used Inscopix’s miniature microscope, called nVoke, which allows simultaneous imaging and manipulation of nerve cells’ circuit dynamics in real time. The technology allows researchers to image cell activity for months with single-cell resolution in a living animal.

The previous study, “Diametric neural ensemble dynamics in parkinsonian and dyskinetic states,” published in Nature, used nVoke to identify alterations in neural activity patterns in brain circuits that regulate movement in a Parkinson’s mouse model. This model mimics the human disease by gradually losing dopaminergic neurons — a hallmark of Parkinson’s.

Dopaminergic neurons release the neurotransmitter dopamine — a chemical substance produced in response to nerve signals that allow nerve cells to communicate. In Parkinson’s disease, dopamine-producing neurons are mainly lost in a brain region known as the substantia nigra, which plays a key role in reward and movement.

Now, the researchers will investigate whether the observed changes in neuronal activity in the Parkinson’s mouse model can be correlated to changes in the expression of genes detected in single cells, which may have occurred as a result of the loss of dopamine. Gene expression is the process by which information in a gene is synthesized to create a working product, like a protein.

By analyzing neuronal activity and gene activity, the researchers hope to gain further insights into the mechanisms of Parkinson’s disease. This may allow the identification of new, cell-type specific therapeutic targets.

According to Inscopix, real-time mapping of neural activity in brain circuits has been shown to more accurately predict the efficacy of a therapy to work on the brain, when compared with analyzing animal behavior.

“Single-cell transcriptomics and brain mapping have each demonstrated the potential to increase our understanding of neurological conditions, such as Parkinson’s disease, and bringing them together could help us make even greater strides forward,” Kunal Ghosh, CEO of Inscopix, said in a press release.

“We look forward to expanding our research into PD [Parkinson’s disease] with the Macosko Lab, with the goal of paving the way for therapeutic programs that can be de-risked at earlier stages of development,” Ghosh added.

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$20M Grant Awarded for Research into Imaging Protein Misfolding in Parkinson’s

brain scans and tracers

The National Institute of Neurological Disorders and Stroke (NINDS) has awarded a five-year, $20 million grant to researchers looking for a way to image misfolded proteins in the brains of people with Parkinson’s and other neurodegenerative diseases, which could greatly advance diagnosis and disease monitoring.

Parkinson’s disease is thought to be a proteinopathy — a condition caused by proteins in the brain folding improperly, which sets off a chain reaction of misfolding in other proteins, eventually forming clumps and damaging the brain. Specifically, Parkinson’s is characterized by clumps of the protein alpha-synuclein.

Alzheimer’s disease is another proteinopathy, characterized by clumps of beta-amyloid. But there’s a crucial difference between the two in terms of how they are diagnosed and managed.

Brains can be imaged using a positron emission tomography (PET) scan, a technique in which a radioactive dye called a tracer is injected into the body. The tracer then binds to specific proteins, allowing clumps of these proteins to be visible on the scan. Although PET scans have been able to image beta-amyloid plaques for nearly a decade, the technology to visualize clumps of alpha-synuclein doesn’t yet exist.

The NINDS grant hopes to foster the development of a PET tracer that will bind to alpha-synuclein, as well as another tracer that will bind to 4R tau, a protein with important roles in frontotemporal degeneration and progressive supranuclear palsy.

This will be done using computers to find promising chemical formulations, then synthesizing and testing them. Although straightforward in theory, actually finding a molecule that can safely and specifically bind to these proteins is akin to finding a needle in a haystack, the researchers said. Hence, the importance of beginning with computer simulations.

“Finding a needle in a haystack is much easier when you have a machine made to find needles,” Andrew Siderowf, MD, a professor at the University of Pennsylvania and study leader, said in a press release.

The effort will also be led by researchers at Washington University-St. Louis, the University of Pittsburgh, the University of California-San Francisco, and Yale University.

Finding such a dye could allow screening for early detection of Parkinson’s disease before symptoms manifest. It could be used as an objective marker of an investigative treatment’s effectiveness in clinical trials.

“Currently, when testing new drugs for Parkinson’s, assessing the patient’s clinical symptoms is the only way to measure whether or not the treatment is working, but clinical features evolve very gradually,” Siderowf said. “Having an imaging biomarker that is sensitive to changes in a Parkinson’s pathology could greatly accelerate drug development.”

Robert H. Mach, PhD, a professor at the University of Pennsylvania and study co-investigator, summarized the researchers’ goal: “At the end of five years, we hope to have a radioactive tracer that will be able to detect Parkinson’s early on and provide detailed information about the disease’s progression, which is critical for discovering and testing new treatments.”

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Letting Go Is Not ‘Forever Gone’

letting go

“Letting go” is a constant theme with Parkinson’s disease. What used to be easy is now challenging. Gone are my days of hiking for miles or spending hours in the gardens digging, hauling, lifting. Those times when 24 hours of project immersion got me through complex problem-solving and four college diplomas are over. I can’t do it the same way anymore. Giving up these expectations of myself has not been easy, and the process of letting go always presents itself at sanctuary’s door. It is never entirely gone.

Psychology Today columnist Judith Sills, PhD, explains that we tend to get stuck in our past, but by letting go we can move forward. “It’s an axiom of psychology that we are some recombination of all of our yesterdays. To move forward wisely, we are therefore often urged to look back. But there’s a point where appreciation and analysis of the past become gum on your psychological shoe. It sticks you in place, impedes forward motion, and, like gum, it doesn’t just disappear on its own. You need to do some scraping.”

American poet and philosopher Ralph Waldo Emerson said, “A foolish consistency is the hobgoblin of little minds.” When you can’t let go, you are haunted by the hobgoblin. If you let go and have nothing to replace it, the hobgoblin will rush to fill the void. Sanctuary holds safety and sacredness in place of the void allowing the possibility of well-being to unfold.

Letting go is learning to live with the bad things that happen — not by eradicating memory, but by shifting attention and perception. In my quest to let go and accommodate chronic Parkinson’s symptoms, I turn to sanctuary. I know when I am using sanctuary appropriately because I run smack into resistance. It is extremely hard to let go of old habits, old scars, and old voices playing on old tapes. The path of letting go is full of detours and wrong turns. I’m always learning more about how to let go. It is a process, and it’s never done.

Writing on Psych Central, John M. Grohol identifies some key steps in the “letting go” process:

  1. Decide to let it go.
  2. Express your pain — and your responsibility.
  3. Stop being the victim and blaming others.
  4. Focus on the present — the here and now — and joy.
  5. Forgive others and yourself.

Throughout our lives, much of our self-identity is defined by what we do rather than who we are. Strip away the things that we could do, and we feel naked without the career clothes we used to wear. Social conversation often turns to, “What do you do for a living?” I want to reply, “I’m just trying to survive.” People who still see me as the person I was can’t see my struggle with letting go that drains my energy and creates overwhelming fatigue.

Family, friends, and some medical providers often do not fully understand how letting go carves away the substance of identity, whittling it down to a splinter. The following quote sums it up for me: “Those who mind don’t matter and those who matter don’t mind.” It results in more loss thrown on a plate already overflowing with dead bones.

Letting go occurs for me on many levels, affecting my sensations, emotions, thoughts, and pain. Sanctuary is not merely a place to “feel good.” It gives me the strength and calmness to face my demons, mourn losses, move forward into the future, and find peace with myself and those around me. Letting go is not losing entire memories even when they’re interwoven with the hard times. Letting go is not forever gone. It remains at sanctuary’s door opening the possibility of well-being.



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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Genetic Parkinson’s More Common Than Thought, Global Survey Reveals

Genetic PD survey

Cases of inherited Parkinson’s disease may be more frequent than previously reported, results from an online global survey suggest.

The survey, which was conducted by members of The Michael J. Fox Foundation Global Genetic Parkinson’s Study Group (MJFF-GGPSG), also revealed the willingness of investigators to share clinical information on their patients that could be useful to conduct broader and more inclusive studies.

These findings suggest it is necessary to improve the way investigators communicate and assess clinical data. It also highlights the need of new integrative research approaches that can empower teams to enhance the understanding and recognition of genetic mutations contributing to the development of Parkinson’s disease. This could be an important step to improve early diagnosis and define preventive strategies.

The results, “Identifying genetic Parkinson’s disease patients worldwide: Exploiting novel ways of team science,” were presented as a scientific poster during the International Congress of Parkinson’s Disease and Movement Disorders in Nice, France.

“This initiative is of high relevance because it is becoming increasingly clear that even relatively common diseases like Parkinson’s disease are highly etiologically heterogeneous syndromes and that progress towards early diagnosis and causative treatments will depend on the identification of sufficient numbers of well-defined subgroups,” Thomas Gasser, MD, said in a press release. Gasser is director of the department of neurodegeneration at Hertie Institute for Clinical Brain Research, in Tuebingen, Germany.

“This will only be possible by collaborations at a very large, preferably worldwide scale,” he said.

The survey, which was conducted in 2018, was designed to evaluate the availability of demographic, clinical, genetic, and additional data of patients with genetic Parkinson’s disease. It included cases caused by SNCA, LRRK2, VPS35, PRKN, PINK1, PARK7, and GBA mutations.

MJFF-GGPSG researchers addressed the survey to 336 investigators who were selected based on articles that had been published about the subject and were represented at the Movement Disorder Society Genetic mutation database (MDSGene), and through the Genetic Epidemiology of Parkinson’s disease (GEoPD) consortium.

Of the 336 investigators invited to participate in the survey, 162 (48%) responded, 98% of whom indicated interest in further collaboration.

“The overwhelmingly positive response and willingness to collaborate impressively highlight the relevance and power of team science,” the researchers wrote.

Researchers reported information from a total of 8,453 Parkinson’s patients with genetic mutations; more than nine different ethnicities were followed at 103 international sites across 43 countries.

Overall, mutations in the SNCA, VPS35, PINK1, and PARK7 genes were present in 3% (263 patients), 0.4% (35 patients), 3% (260 patients) and 0.3% (29 patients) of this patient population.

The most commonly affected genes were LRRK2, GBA, and PRKN, with mutations present in 38% (3,182 patients), 37% (3,154 patients), and 18% (1,530 patients).

These frequencies are particularly significant given that they represent a threefold higher number of patients with mutations associated with Parkinson’s disease when compared to the cases reported in the literature.

More than 98% of investigators who responded to the survey noted they had demographic data on their patients, with 94% of them having age-at-onset information, and only 66% reported having information on patients’ non-motor signs.

Most investigators (85%) had DNA samples from the patients, while only 8% had cerebrospinal fluid (CSF) samples. (CSF is the liquid that surrounds the brain and spinal cord.)

“This survey is only a very first small step. If international team science is to become successful, many problems concerning standardization of patient ascertainment, data privacy and protection as well as data access and use need to be solved,” Gasser said.

“Nevertheless, the survey raises awareness of these issues and it clearly shows that the Parkinson’s disease research community is ready to begin to tackle these important issues,” he said.

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FIRE-UP PD Initiative Seeks to Increase Inclusivity in Parkinson’s Research


The Michael J. Fox Foundation (MJFF) and Massachusetts General Hospital have announced an initiative to enhance diversity in Parkinson’s disease research.

Fostering Inclusivity in Research Engagement for Underrepresented Populations in Parkinson’s Disease (FIRE-UP PD) is an MJFF-funded study that will establish and assess outreach programs in Boston, Massachusetts, Weston, Florida, Denver, Colorado, and Chicago, Illinois. Massachusetts General’s Community Access, Recruitment and Engagement (CARE) Research Center is coordinating the effort.

“Parkinson’s research has made significant strides toward better diagnostics and new treatments in past decades, but most research has included only a subset of patients with a common European ancestry,” said Sohini Chowdhury, MJFF deputy CEO, in a press release.

“Imagine where we would be with a more holistic view of the disease. This program aims to broaden the vital partnership between researchers and the people living with Parkinson’s, each and every one,” Chowdhury said.

Jonathan Jackson, the CARE Research Center’s founding director and FIRE-UP PD principal investigator, said that because PD is such a varied disease and affects each person differently, both in terms of symptoms and disease progression, research inclusivity is key.

“When we include people from all backgrounds in Parkinson’s research, we better understand the disease itself, improving our chances at finding treatments that work for everyone. FIRE-UP PD is unique in its attention to diversity in Parkinson’s research and its application of community-based methods across all geographic regions,” he said.

Four academic centers will develop community-centered interventions to produce culturally sensitive messaging and resources that educate and engage around Parkinson’s research. The sites and programs include:

Boston Medical Center: Researchers will partner with community health centers to engage Boston’s Haitian and African American communities by using educational tools emphasizing the importance of Parkinson’s research  and diagnosis.

Cleveland Clinic in Weston, Florida: The focus at this site is southern Florida’s Hispanic communities, and engagement through educational seminars and collaboration with area support groups.

University of Colorado: Through a method called Boot Camp Translation, which recruits healthcare professionals and community members to translate medical information for local populations, investigators will address health literacy in Hispanic and lower-income populations.

Northwestern University, Chicago: A stakeholder partnership of patients, community leaders, caregivers and physicians will conduct focus groups and create community-specific educational toolkits for Hispanic, African American and lower-income residents.

In addition to enhancing disease awareness and fostering trust in Parkinson’s  research participation, the programs hope to promote enrollment in MJFF’s Fox Insight, an online clinical study aimed at building a large, diverse group  of Parkinson’s patients and age-matched control volunteers to gain insight into the disease’s experience, genetics and variability.

To that end, sites in Minneapolis, Minnesota, San Francisco, California, Chicago and Kirkland, Washington will offer Fox Insight materials exclusively in their clinics in order to compare conventional outreach methods with those of the intervention sites.

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Potential Treatment to Prevent Toxic Protein Clumping Enters Phase 1 Trial, Yumanity Announces

YTX-773 Phase 1 trial

Yumanity Therapeutics announced the start of a Phase 1 clinical study in healthy volunteers that will assess the safety and tolerability of YTX-7739, a potential disease-modifying therapy for Parkinson’s disease.

Trial results are expected to be announced in the first quarter of 2020.

YTX-7739 is designed to cross the blood-brain-barrier — a semipermeable membrane that protects the brain and spinal cord from the external environment — to inhibit the activity in the brain of an enzyme called stearoyl-CoA desaturase (SCD) .

This enzyme is known to play a key role in the production of certain fat molecules, called unsaturated fatty acids, that mediate the neurotoxic effects of alpha-synuclein protein accumulation — a key constituent of Lewy bodies, the toxic protein clumps that are a Parkinson’s hallmark.

In cell and animal disease models, the investigational medicine was shown to protect neurons against alpha-synuclein-derived toxicity and improve their survival.

“We advanced YTX-7739, an orally-active SCD inhibitor, into clinical development because of recent evidence established at Yumanity Therapeutics demonstrating its promise to protect cells from a-synuclein toxicity,” said Kenneth Rhodes, PhD, the company’s chief scientific officer, said in a press release.

“We look forward to fully characterizing the potential clinical use of YTX-7739, which is clearly differentiated from currently available Parkinson’s disease therapies that only address the symptoms, not the underlying causes.”

The Phase 1 trial is expected to enroll about 40 healthy volunteers, who will be randomly assigned to increasing doses of oral YTX-7739 or dose-matching oral placebo. Collected data will assess YTX-7739’s safety and tolerability, as well as its stability and metabolization (pharmacokinetics) inside the body: essentially, how the body affects a medicine.

If results are promising, Yumanity plans to advance YTX-7739 into a Phase 1b proof-of-concept clinical trial in patients, possibly in the second half of 2020.

“This Phase 1 trial will provide important validation for the broad application of our technology to help address arguably the most important therapeutic challenges of our time,” like that of “protect[ing] cells from neurodegeneration,” said Richard Peters, MD, PhD, Yumanity’s CEO.

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MIT Scientists Building Artificial Gut to Study Bacteria’s Influence on Parkinson’s, Other Diseases

gut microbiome study

MIT Lincoln Laboratory researchers are developing an artificial gut to study how the human microbiome — the trillions of microorganisms and their genetic material that live within our body, and are as unique to a person as fingerprints — influences the onset and progression of diseases linked to changes in gut bacterial constitution, such as Parkinson’s disease.

The so-called “gut-brain axis” is a highly complex and interactive network between the gut and the brain, composed of endocrine (hormonal), immunological, and neural mediators. Dysregulation of “cross-talk” within this axis has been associated with metabolic syndrome, depression, anxiety and autism, as well as to neurodegenerative diseases like Parkinson’s, and Alzheimer’s.

By manipulating the gut microbiome in Parkinson’s patients, researchers could study its effects on neurodegenerative processes.

“Until now, no one has been able to culture a microbiome sample and maintain it,” David Walsh, a PhD with the Biological and Chemical Technologies Group at MIT who led the prototype device’s development and fabrication, said in a university news story by Anne McGovern. Further refinements are still being made.

“The question from the mechanical side is, how do you emulate the colon?” said Todd Thorsen, PhD, the project’s principal investigator and an assistant professor with the MIT group.

“Bacteria in the colon occupy lots of ecological niches,” Thorsen added. This means that all bacteria living in the colon have organism-specific demands for survival, including nutritional and environmental requirements. For instance, some are oxygen-dependent and others not.

To mimic the intestinal microenvironment, Lincoln Laboratory investigators are developing an easily accessible and cost-effective platform made of permeable silicon rubber and other plastics, like polystyrene. Importantly, in this “artificial gut,” scientists can regulate oxygen and mucus concentrations within microculture chambers, modeling the human colon. Because it can be easily replicated, it might also be of use to others studying the gut microbiome, and the impact of disease or treatment on it.

“If we can maintain a culture, we can do things like add toxins and therapeutics to see how they change the culture over time,” Walsh said. Such an ability could move research a step closer to tackling real-world problems, including bacterial resistance.

Using gut microbiome samples from Parkinson’s patients and healthy people, the scientists plan to use their device to study intestinal bacteria’s influence on the neurodegenerative processes seen in Parkinson’s.

Experiments are expected to begin soon, in collaboration with researchers at the University of Alabama at Birmingham, Northeastern University, and the University of California at San Francisco.

The team also plans to build a tube-shaped origami-like gut that rolls up during assembly to simulate the colon and the surrounding vascularized tissue, and to develop modeling software to predict how different bacterial communities change over time.

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