New Large-Scale Data Portal Will Promote Parkinson’s Treatment Development

data portal

The Accelerating Medicines Partnership (AMP) for Parkinson’s (PD) has opened a data portal with de-identified information from 4,298 Parkinson’s patients and healthy control subjects for use by scientists seeking new treatments for the progressive neurodegenerative disease.

With unprecedented access to a data pool of this scale, investigators now can examine intricate data sets and conduct full-scale genomic analyses.

“AMP PD is a true example of the whole being greater than the sum of its parts,” said Walter Koroshetz, MD, director of the National Institute of Neurological Disorders and Stroke (NINDS), in a press release. “The combination of many data sets could allow researchers greater power to analyze potential biomarkers for Parkinson’s disease. This effort follows other AMP programs which have the shared goal of changing the way we go about the business of studying disease.”

Launched in 2014, the AMP is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), multiple biopharmaceutical and life sciences companies, and non-profit organizations. Its goal is to transform the current model for developing new diagnostics and therapies by collaboratively identifying and validating promising biological treatment targets. The overarching mission is to develop new diagnostics and therapies relatively faster and at less cost.

Initial projects included Alzheimer’s disease, Type 2 diabetes, and rheumatoid arthritis, and lupus.

Last January, the AMP project on PD was launched. Managed by the Foundation of the National Institutes of Health (FNIH), the project includes the NIH, FDA, the Michael J. Fox Foundation (MJFF) for Parkinson’s Research, Celgene, Verily Life Sciences, Pfizer, Sanofi and GSK.

This project’s aim is to speed therapy development by providing the expertise and support necessary to learn which biomarkers demonstrate the most promise for predicting PD and disease progression. Biomarkers are molecular disease indicators.

“One important part of this platform is that, in addition to providing a place for storing complex data, we are also providing the tools to analyze that data within the platform itself,” said Debra Babcock, MD, PhD, NINDS program director and co-chair of the AMP PD steering committee. “In this way, we are bringing scientists to the data, which will increase opportunities for collaboration.”

Data in the officially named AMP PD Knowledge Portal was collected through the MJFF, NINDS and several other programs, studies and institutions. It includes information from samples of DNA, RNA, plasma, and cerebrospinal fluid, which is the liquid that surrounds the brain and spinal cord. The portal also offers a platform that can assimilate additional types and sources of data. For example, there is an upcoming study involving proteomics, the large-scale study of proteins.

With the longitudinal data in the portal, scientists can study patients’ information throughout the disease course. And, the data have been harmonized, allowing for comparison of information from different programs, and providing best practices for how to incorporate into the platform data from the PD community.

“The AMP model has provided a unique platform for bringing together diverse patient cohorts, advances in technology and scientific expertise to study Parkinson’s disease on a scale that has not been attempted before,” said David Wholley, senior vice president, research partnerships, FNIH. “With the AMP PD Knowledge Portal, we are helping the scientific community worldwide to fast-track discoveries that we hope will ultimately help Parkinson’s disease patients and their families.”

Scientists may visit this site to apply for access to the knowledge portal and interact with the data set.

Globally, roughly 7 to 10 million individuals have Parkinson’s, the second most common neurodegenerative disorder after Alzheimer’s disease.

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Collaborators Awarded Bachmann-Strauss Prize for Excellence in Dystonia Research

research grants

Investigative collaborators from the University of Alabama at Birmingham (UAB) and the University of Rome Tor Vergata in Italy have been awarded the fifth annual Bachmann-Strauss Prize for Excellence in Dystonia Research.

The Michael J. Fox Foundation (MJFF) for Parkinson’s Research presented the awards recently to UAB’s David G. Standaert, MD, PhD, and John N. Whitaker professor and chairman of neurology, and to the University of Rome Tor Vergata’s Antonio Pisani, MD, PhD, associate professor of neurology.

The award is for significant contributions to dystonia research, and comes with an unrestricted grant of $100,000 to support further joint research. Also meant to motivate the next generation of investigators, the award is part of a partnership announced in 2014 between the MJFF and the Bachmann-Strauss Dystonia & Parkinson Foundation (BSDPF).

“Drs. Pisani and Standaert have made significant strides in plotting the cellular dysfunction that leads to dystonia,” said Bonnie Strauss, BSDPF founder, MJFF board member, and a dystonia patient, in a press release. “This team has laid the groundwork for development and testing of new therapies to help those living with dystonia.”

A generally poorly understood movement disorder characterized by painful, protracted muscle contractions that cause abnormal movements and postures, dystonia is both a distinct disorder and a common symptom of Parkinson’s disease. Dystonia symptoms usually begin in one body region — such as the neck, face, vocal cords, arm, or leg — then may spread to other areas.

Having co-authored their first published work in 2006, Standaert and Pisani have collaborated on nine research papers detailing dystonia’s pathology. The investigators, who are also practicing clinicians, have examined the role and relationship of neurotransmitter activity in dystonia, and have profiled a dopamine imbalance and acetylcholine activity in a condition type that usually begins in adolescence.

“We are honored to receive the prestigious Bachmann-Strauss Prize,” Pisani said. “It means we are working in the right direction for our findings to be recognized with such an award, and that we can continue building knowledge toward new treatments and cures.”

Added Standaert: “This work with Antonio is a very productive collaboration that has been an engine for discovery. There is a tremendous need for more research in the field, and more researchers. I hope this recognition from Bachmann-Strauss and MJFF will demonstrate to young investigators that there is a support and encouragement for research in dystonia and will attract more of them to this field.”

The researchers will use award funds to collaboratively study multiple models of a genetic form of dystonia to better understand the mutation’s effect.

The Bachmann-Strauss prize is intended to honor current research, an individual’s past body of work, or both.

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U.S. Defense Department Funds Research Into Parkinson’s and Exercise

The U.S. Defense Department has awarded researchers from Northeast Ohio Medical University (NOMU) and Michigan State University (MSU) about $1 million each to study the effect of exercise at different stages of Parkinson’s disease.

The three-year grant went to longtime collaborators Sheila Fleming, PhD, an assistant professor of pharmaceutical sciences at NOMU and Caryl Sortwell, PhD, a translational neuroscience professor at MSU.

“It’s a major award for both of us,” Fleming said in a news release. “We had been working together for many years because our interest and work are very complementary. Ultimately, it’s about a $2-million grant. She gets half and I get half.”

Fleming, who was awarded $954,566 by the U.S. Army Medical Research Acquisition Activity, said she will work on behavioral aspects of the study while Sortwell will handle pathological events.

The project, titled “Exercise Effects on Synuclein Aggregation, Neuroinflammation and Neurodegeneration,” will analyze the impact of exercise in an optimized preclinical disease model. That will include examining mechanisms associated with the central characterization of Parkinson’s — the buildup of toxic alpha-synuclein aggregates, neuroinflammation, and expression of certain molecules in the brain called trophic factors.

Using a progressive Parkinson’s disease animal model, Sortwell is charting the course and development over time of pathological events in the brain. Fleming is examining how the pathological occurrences relate to changes in motor and non-motor symptoms. Together, the researchers are examining the impact of introducing exercise at different stages of Parkinson’s progression.

Most scientists studying exercise in Parkinson’s have used what are called toxin models, which solely target the dopamine system, Fleming said. The chemical dopamine acts as a neurotransmitter and is essential in sending messages from the brain to direct muscle movement and coordination. As more dopamine-producing neurons die, dopamine levels slowly and progressively decrease until patients are unable to control normal movements.

But those models have issues related to reproducibility and a lack of understanding of the biological properties of alpha-synuclein pathology. Fleming and Sortwell are using a newer model supported by the Michael J. Fox Foundation called PFF — for pre-formed synthetic fibrils — to elucidate mechanisms of alpha-synuclein-induced pathology. In this model, fibrils are injected into animals, and researchers track the appearance of symptoms.

Studies have already shown the likelihood that alpha-synuclein clumping begins in the back of the brain and proceeds to the front, a pathology that may be related to non-motor Parkinson’s symptoms such as depression, anxiety, reduced sense of smell, and cognitive impairment.

Fleming and Sortwell will look at the effect of exercise on both non-motor and motor symptoms (such as problems walking) to determine what symptoms manifest and in what order. This information could lead to earlier diagnoses, and provide a non-pharmacological, low-cost therapeutic strategy for patients, including veterans.

Currently, Fleming said, by the time individuals first seek help for symptoms, they have typically already lost at least half their dopamine neurons.

“Slowing the progression of the disease could have a huge benefit, especially since patients aren’t usually diagnosed until between 50 and 60 years of age,” she said. “So, if you could slow it, that could have a potentially huge impact on the quality of life of patients.”

The scientists presented their project in October at the annual meeting of the Society for Neuroscience.

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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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MJFF Announces $10M Challenge to Develop Critical Research Tool for Parkinson’s

MJFF challenge

With the aim of opening entirely new avenues in Parkinson’s disease research and care, The Michael J. Fox Foundation (MJFF) has announced a $10 million contest to drive development of an imaging tracer that would be used to see alpha-synuclein protein in the living brain.

Called the “Ken Griffin Alpha-synuclein Imaging Competition,” the project will award $8.5 million to up to three investigative teams. After two years, the team that makes the most progress will receive an additional $1.5 million to continue. The application deadline is Jan. 17, 2020.

Development of such a critical and, so far, elusive research tool would be a game changer for Parkinson’s. Of the roughly 10 million patients globally, nearly all have accumulations of the protein in their brains. Investigators believe these alpha-synuclein masses negatively affect cells and cause disease symptoms. The problem is the clumps are visible only after patients die, through post-mortem studies.

The competition to produce technology for the living brain is largely funded by a $7.5 million gift from Ken Griffin, founder and CEO of the investment company Citadel.

“Providing researchers and clinicians with the ability to detect and monitor disease would be revolutionary for the field and, most importantly, for patients,” said Todd Sherer, PhD, MJFF CEO, in a press release. “Ken Griffin’s gift invigorates research toward this important tool, which will make a meaningful impact in the lives of everyone touched by Parkinson’s.”

The MJFF has been at the fore of efforts to develop a positron-emission tomography (PET) tracer. It has sponsored independent research and organized a consortium around it. Three years ago, the MJFF announced it would award $2 million to the first team to reveal clinical proof of a tracer and share it with researchers at large. That challenge continues.

Meanwhile, at the organization’s 2019 PD Therapeutics Conference on Oct. 15, biotechnology company AC Immune will present  findings from its MJFF-supported tracer study.

The Ken Griffin award announcement is expected by April. The MJFF is encouraging applications from multidisciplinary teams, and is particularly interested in collaborations between academic and industry groups that have access to diverse compound libraries.

“The Michael J. Fox Foundation has led the charge in advancing ground-breaking research in this field over the past 20 years,” Griffin said. “I hope this partnership with the Foundation will bring us closer to a cure for the millions of people living with Parkinson’s disease.”

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Fox Foundation Offers Guide to Help with Parkinson’s Cognitive Symptoms

cognition and Parkinson's

The Michael J. Fox Foundation (MJFF) is offering a new guide to help people better understand Parkinson’s cognitive symptoms and ways of dealing with them.

The 33-page guide, “Navigating Cognitive Changes in Parkinson’s Disease,” was developed by the MJFF’s Patient Council — which includes caregivers, social workers, and physicians specialized in movement disorders and cognitive function — working together with Parkinson’s patients, their families, and their clinicians.

An hourlong webinar discussing topics addressed in the guide and including a question period is set for Sept. 19 at noon ET. Those interested can register here.

“The goal of this guide is to encourage people with Parkinson’s and their loved ones to learn more about cognitive changes and to take action — whether that’s opening a discussion to lessen fear and improve care or practicing healthy habits to boost brain health,” Rachel Dolhun, the guide’s author, and MJFF’s vice president of medical communications and a movement disorder specialist, said in a press release.

While a diagnosis of Parkinson’s disease is based on characteristic motor symptoms like tremor, slowness of movement, and rigidity, cognitive impairment is a significant non-motor manifestation of the disease.

Among the 6 million people estimated to be living with Parkinson’s worldwide, 40% are likely to develop dementia and 25% may develop milder cognitive changes. “These symptoms are, unfortunately, also some of the least talked about,” Dolhun wrote.

The guide explores different aspects of cognitive function and their roles in daily life, such as executive function, memory, language, attention, and visual-spatial skills. It provides several strategies to help patients and their families keep track of possible cognitive changes, which can be expressed differently in each patient.

It also encourages patients to practice activities known to be good for the body, and thought to also boost brain health. These include exercising regularly, eating a healthy and balanced diet, training the brain with “mind games” or crosswords, getting enough sleep, reducing stress, being socially active, and getting involved with the Parkinson’s or local community.

A large part of the guide is dedicated to dementia, one of the most concerning potential symptoms of Parkinson’s for many patients and their families.

It starts by clarifying what defines dementia, the differences between three causes of dementia — Alzheimer’s disease, Parkinson’s disease dementia (PDD) and dementia with Lewy bodies (DLB) — and current treatment options for this condition.

PDD and DLB share the presence of abnormal protein clumps in the brain known as Lewy bodies, as well as several symptoms. Currently, DLB diagnosis is distinguished from PDD if dementia occurs before or during the first year of motor symptoms related to Parkinson’s.

The guide also provides strategies to work through Parkinson’s cognitive changes and dementia, including: finding a physician with expertise on cognitive changes, being patient and flexible with oneself and one’s difficulties, communicating in an open and honest way, learning as much as one can about the disease, asking for and accepting help from others, joining supportive groups, and participating in clinical research.

Another section offers tips to help caregivers and relatives manage a patient’s hallucinations and delusions, which can be the most difficult manifestation of dementia.

Finally, the guide shares some of the latest information in ongoing research into disease-related dementia.

“The Patient Council welcomed the opportunity to provide input on this new guide in order to offer families the knowledge and resources to talk about a sensitive topic that’s sometimes inadequately addressed at home or even in the doctor’s office,” said Dave Iverson, a founding member of MJFF’s Patient Council and a broadcast journalist.

“More resources, research, and collaboration can help us address the complex issue of cognitive decline and support people who experience it,” said Todd Sherer, MJFF’s CEO.

The free guide, and a complementary video, are available for download here. The Sept. 19 webinar features Dolhun and other disease experts, and members of the Parkinson’s community.

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Parkinson’s Advocates Set to Lobby US Lawmakers for Greater Research Support, Patient Assistance

Parkinson's Policy Forum

Joined by leaders in Parkinson’s (PD) research and public policy, some 150 patients and advocates from across the nation will gather in Washington, D.C., on Monday for the 2019 Parkinson’s Policy Forum.

The annual advocacy event, taking place Sept. 9–10, is presented this year by the Parkinson’s Foundation and The Michael J. Fox Foundation for Parkinson’s Research (MJFF), with support from nine other organizations. With the aim of improving the impact of policy on PD research and care, the forum offers an opportunity for those living with Parkinson’s to not only connect, but to share their journey with elected officials.

”Policy and research must work hand-in-hand to speed scientific breakthroughs for patients and their families,” Todd Sherer, PhD, the Fox foundation’s CEO, said in a news release. “The Parkinson’s Policy Forum brings the community together to advocate for government policies that support research and care. Its goal is to improve quality of life for everyone impacted by the disease today.”

Forum attendees — mostly patients and care partners — will spend a full day with foundation and its scientific and policy experts brushing up on effective advocacy, and exploring ways to help shape their message. They’re off to Capitol Hill the next day, where advocates will apprise legislators of the ways in which policy can help — or hinder — Parkinson’s research and care. They’ll focus specifically on the economic burden of PD, as well as recent legislative moves to limit out-of-pocket prescription medicine costs for Medicare patients, and improve access to mental health services.

The nonprofit organizations said it’s vital that advocates and policymakers seize on current momentum in Parkinson’s, particularly in research and clinical drug testing. By next year, it is estimated that nearly 1 million U.S. residents will be living with the progressive disease.

”People with Parkinson’s disease and their families need legislative action with increased federal research funding to expand access to care that will help patients live healthier lives,” said John Lehr, CEO of the Parkinson’s Foundation. “The Parkinson’s Foundation leverages our nationwide network of healthcare professionals and research advocates to mobilize around PD community policy priorities. To better serve people with Parkinson’s and their families we must ensure they continue to have their voice heard by lawmakers.”

Visit this site for more forum information.

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Fox Foundation Awards $5M to Support Genetic Studies of Parkinson’s in Africa, Asia and India

Fox Foundation grant

With the overarching goal of helping scientists develop and test targeted therapies in Parkinson’s (PD), The Michael J. Fox Foundation (MJFF) is awarding $5 million in grants to three teams conducting genetic studies in African, East Asian and Indian populations.

The funding seeks to broaden these studies in order to better understand the role of genetics in PD onset and progression, and to expand treatment options for patients globally. Historically, the majority of research has focused on people of European descent. The grants will enable genetic testing of samples from more than 30,000 people.

“While the field has made significant strides in genetic research, we know we have more to learn about the changes in DNA that lead to Parkinson’s disease and impact its progression,” Brian Fiske, PhD, MJFF senior vice president of research programs, said a news release. “This is an all-star initiative with world-class geneticists, clinic networks and study volunteers coming together to paint a global picture of Parkinson’s and work toward cures for everyone.”

Since researchers discovered the first genetic mutation linked to PD in 1997, more than 80 others have been identified. Scientists are studying the cellular impact of these mutations, associated with about 15 percent of PD cases, in order to better understand Parkinson’s and possible ways of treating it.

Potential therapies aimed at proteins including LRRK2, one of the most commonly known genetic causes of Parkinson’s, are in clinical trials. Work like this is what the global Parkinson’s genetics program hopes to build upon. The non-profit foundation has long backed genetic studies, and diversity and inclusivity in clinical investigations.

Grants under this global program — with support from the Edmond J. Safra Foundation, a long-time partner of the Fox Foundation — will go to the following projects:

“Parkinson’s is a global issue, and we are grateful to The Michael J. Fox Foundation for fostering representation in research,” said Njideka Okubadejo, a professor of research at the University of Lagos in Nigeria. “We hope this partnership results in greater understanding of disease causes and contributors, and leads to new treatments for people living in Africa and beyond.”

Parkinson’s is the second most common age-related neurodegenerative disorder (after Alzheimer’s), and estimated to affect 7 to 10 million people worldwide.

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MJFF Nationwide Event Series ‘Parkinson’s IQ + You’ Kicks Off This Fall

Parkinson's IQ + You

A new nationwide event series aims to empower people with Parkinson’s (PD) and their caregivers to manage the disease, learn about research participation, and plug into local resources.

Called “Parkinson’s IQ + You,” the effort is presented by The Michael J. Fox Foundation (MJFF). The events seek to support PD families in learning more about all facets of the disease — what a comprehensive care team looks like, how to achieve optimal communication between patients and physicians, the latest in treatments, and the benefits of joining clinical trials.

Kick-off events are slated this year for Atlanta on Sept. 14, and southern California, in Anaheim, on Dec. 14. More sessions will come next year, including one in Phoenix on Jan. 11. Registration is free. This year’s events will be hosted by Larry Gifford, a patient and host of the “When life gives you Parkinson’s” weekly podcast.

“Between its symptoms and progression, Parkinson’s can present unique challenges for those of us who live with the disease, but also for our loved ones who support us,” John L. Lipp, a writer, nonprofit executive director, and Parkinson’s patient, said in a news release.

“I often say, ‘I can’t control my Parkinson’s, but I can control how I respond to it.’ Parkinson’s IQ + You will offer people in the community, including me and my husband, an opportunity to learn from other patients and families and from experts in the field, so that we can turn education today into action tomorrow,” Lipp said.

Crafted to support care partners and people with Alzheimer’s at every disease stage, the series will feature a variety of panel participants, including patients and their caregivers, to reflect the community’s diversity and the disorder’s variability. The organizers note that PD symptoms and progression vary significantly, making each Parkinson’s experience unique.

Each full-day event will feature interactive programming, as well as an exposition of local resources. Called Parkinson’s Partner Expo, this portion will include representatives from movement disorder centers and patient advocacy organizations, speech and physical therapists, and clinical trial teams.

There also will be panels and sessions on topics such as understanding Parkinson’s and living well with it. During a related forum called “If I Knew Then What I Know Now,” a patient and caregiver will share experiences and strategies. There also will be a session on “Building Your Care Team.”

To help people with Parkison’s and their caregivers understand the advantages of seeing a specialist, what to expect during visits, and how to optimize each one, each event will feature a demonstration of what a typical appointment might be.

“Patients are the force driving our research priorities and our urgent mission to end Parkinson’s,” said Todd Sherer, PhD, the foundation’s CEO. “The foundation is committed to ensuring all people with Parkinson’s, and their families, are empowered to make decisions about treatment and care. And we want to work side by side in partnership with patients to advance Parkinson’s research.”

The series includes complimentary meals, and is funded by Acadia Pharmaceuticals, with additional support from MJFF biotech and pharmaceutical partners.

“The Parkinson’s IQ + You program has been uniquely designed to bring much-needed support and information to the Parkinson’s community,” said Rob Kaper, MD, Acadia’s senior vice president and global head of medical affairs.

“We look forward to this national series of events across the country, which will offer attendees the opportunity to interact with healthcare experts and other people with Parkinson’s and their families, and ask important questions about disease management,” he said.

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MJFF Assists Nitrome Biosciences’ Pursuit of Parkinson’s Therapies

Nitrome Bioscience

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded Nitrome Biosciences a Target Advancement grant to further the company’s development of therapies targeting Parkinson’s disease.

Specifically, the grant will be used to further Nitrome’s biological studies of a new Parkinson’s drug target.

The therapies are aimed at inhibiting a newly identified enzyme the company calls synuclein nitrase. The company said the enzyme causes or accelerates the nitration — a type of chemical modification caused by cellular stress — and aggregation of alpha-synuclein, a hallmark of Parkinson’s disease. At length, Nitrome will test whether an impeded enzyme can slow or stop Parkinson’s progression.

“We’re immensely grateful to MJFF for awarding this grant to Nitrome. This provides not only critically needed support but also shows interest in our unique approach to Parkinson’s disease drug development,” Irene Griswold-Prenner, PhD, said in a press release. Griswold-Prenner is Nitrome’s founder and CEO. “Additionally, the close connection with MJFF personnel provides important feedback as well as access to information, disease models and reagents.”

MJFF associate director of research programs, Luis M. Oliveira, PhD, calls the alpha-synuclein pathway a “compelling” target for development of Parkinson’s treatments.

“We are glad to support this study to mediate pathology and advance toward treatments that slow or stop disease progression,” he said.

Accumulation of alpha-synuclein, the chief component of Parkinson’s disease Lewy bodies, is found not only in the brain, but in the peripheral autonomic nervous system, which ultimately affects breathing and digestion.

A modified — nitrated — form of the protein can be found in salivary gland tissue of Parkinson’s patients. Because nitrated alpha-synuclein exists in early stages of PD, it could be a promising disease biomarker.

While starting with Parkinson’s, the company plans to use its discovery of the newly identified enzyme class to develop disease-modifying compounds for other neurodegenerative disorders, plus diabetes, heart disease and cancer.

The MJFF is the world’s largest nonprofit funder of Parkinson’s investigations. Its Target Advancement Program focuses on the identification of proteins and pathways involved in the onset and progression of the disease. Typically, such grants are for 12 to 18 months and are valued at up to $150,000. The amount of this grant was undisclosed.

“Highly nitrated and misfolded proteins play important roles in multiple neurodegenerative diseases, including Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis,” said Ephraim Heller, chairman of Nitrome’s board of directors. “Nitrome will deploy its platform technology to develop therapies for multiple diseases involving enzymatic protein nitration.”

According to the MJFF, nearly one million U.S. residents will be living with Parkinson’s by 2020. The disease affects 10 million individuals globally.

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