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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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Neuroscientist Awarded $2.9M NIH Grant to Study and Possibly Prevent Side Effects of Levodopa

levodopa study

The National Institutes of Health has awarded a $2.9 million grant to a Feinstein Institutes for Medical Research scientist working to better understand and prevent dyskinesia, a common side effect of the levodopa used to manage motor symptoms of Parkinson’s disease.

The five-year award went to David Eidelberg, a neurologist and neuroscientist noted for his pioneering work into brain networks in states of disease.

Levodopa is widely given to Parkinson’s patients to help with stiffness and slowness of movement. Naturally found in the body, it’s the precursor of dopamine, a signaling molecule that is involved in nerve cell communication.

Often combined with other medications to reduce side effects like nausea, levodopa is carried on circulating blood to the brain. There it’s converted into dopamine, which activates dopamine receptors to improve the workings of movement control centers in the brain.

However, after about five years of daily use, most patients develop levodopa-induced dyskinesias (LID) — uncontrolled, involuntary movements that interfere with daily activities — shortly after each dose. This side effect can be disabling and problematic for long-term Parkinson’s management.

“Since levodopa is regularly used to help ease the effects of Parkinson’s disease, it is essential to understand the therapy’s full effects on the cerebral blood vessels as well as neurons,” Eidelberg, head of the Feinstein’s Center for Neurosciences in the Institute of Molecular Medicine, said in a press release. “With this research, we hope to slow down or stop the development of LID in Parkinson’s patients.”

His study is titled “Neurovascular Effects of Dopamine Replacement Therapy in Parkinson’s Disease.”

Eidelberg is internationally known for using functional brain networks as neurological disease biomarkers to aid in Parkinson’s diagnosis, disease progression monitoring, and treatment assessment. He and his team are believed to be the first to observe uncoupling of the neuronal and cerebrovascular responses to dopamine in Parkinson’s patients, a pronounced occurrence in drug-induced dyskinesias. They seek to understand the neurovascular issues that underlie these dyskinesias by charting changes over time.

“Dr. Eidelberg is a leader in Parkinson’s disease research,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes. “This further support of his work by NIH offers a new path to understand this syndrome.”

In related news, a clinical trial may soon test a potential oral treatment for levodopa-induced dyskinesia, IRLAB Therapeutics announced in a company release. It plans to open a Phase 2b/3 study in the first half of next year assessing the safety and effectiveness of its oral candidate, IRL790, in Parkinson’s patients with these dyskinesias.

A four-week Phase 1b safety and tolerability study (NCT03531060) in 15 Parkinson’s patients in Sweden reported good safety (no serious side effects) and early evidence of possible benefits (drops in scores measuring dyskinesia) in people taking IRL790 compared to those given a placebo.

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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 Awards Casma Therapeutics $370K for Innovative Therapies to Slow PD Progression

Casma Therapeutics grant

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded Casma Therapeutics $370,000 to advance an innovative class of therapies aimed at slowing disease progression.

The grant will promote the biotechnology company’s investigation into compounds that activate the calcium channel TRPML1 to accelerate autophagy — a natural cellular way of disposing of pathogens and toxic proteins. The hope is that boosting autophagy will protect and rescue damaged neurons. It’s widely believed that Parkinson’s disease (PD) results from neuronal buildup of toxic proteins.

Casma will test its novel TRPML1-activating treatments using human-induced pluripotent stem cell-derived dopaminergic neurons, which model Parkinson’s disease. The neurons will be generated by the National Human Genome Research Institute (NHGRI) from patient samples collected at the National Institutes of Health (NIH). Induced pluripotent stem cells are derived from either skin or blood cells that have been reprogrammed back into a stem cell-like state. This allows for the development of an unlimited source of any type of human cell needed for therapeutic purposes.

The study is in collaboration with co-MJFF grantee Ellen Sidransky, MD, a Parkinson’s expert and top NHGRI researcher.

“Inducing the natural process of autophagy to clear toxic proteins is a promising new approach to treating Parkinson’s,” Daniel Ory, MD, Casma senior vice president, translational medicine, said in a press release. “We share the foundation’s sense of urgency, and we’re eager to move our program forward.”

New discoveries are revealing the fundamental role of autophagy and lysosomal flux in maintaining cellular health, according to a Casma webpage. Lysosomes are special compartments within cells that digest and recycle different types of molecules. Research has shown that inadequate or aberrant autophagy contributes to genetic diseases, including Parkinson’s.

The company believes that restoring cellular balance may arrest or reverse disease progression.

”Funding therapies against emerging targets such as TRPML1 is a crucial plank in our effort to find a cure for Parkinson’s,” said Liliana Menalled, PhD, MJFF senior associate director of research programs. “We are committed to leaving no stone unturned as we pursue therapies that will help the more than 6 million people worldwide living with this disease.”

The Parkinson’s Foundation, which says approximately 60,000 Americans are newly diagnosed each year, puts the global figure even higher, estimating that more than 10 million people live with the disease.

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Scientist Lands $5M Grant to Study Link Between Pesticides, Smell and Parkinson’s

pesticides and Parkinson's

The National Institutes of Health (NIH) has granted a Michigan State University scientist $5 million to study a possible link between pesticides, a diminished sense of smell, and early symptoms of diseases such as Parkinson’s among older farmers.

The investigator is Honglei Chen, MD, PhD, a professor of epidemiology whose research focuses on neurodegenerative disorders. His primary scientific interests include environmental and genetic risk factors for Parkinson’s disease.

“Our battle against Alzheimer’s and Parkinson’s may depend on early disease identification and intervention, and poor olfaction [sense of smell] has been identified as an early warning for these diseases,” he said in a press release.

“This grant will allow us to connect the dots by identifying factors that contribute to poor olfaction among older adults, and evaluating how this sensory deficit may progress to early stages of neurodegenerative diseases,” Chen said.

Early analyses, published in the journal Environmental Health Perspectives, revealed a link between high pesticide exposure and a self-reported impaired sense of smell.

The team studied more than 11,200 farmers, of whom 16% experienced a high pesticide exposure event, over a 20-year period. At the end of the study, participants were asked if they suffered a partial-to-complete loss of sense of smell. Those who were exposed to high pesticide levels were 50% more likely to report a poor sense of smell. Importantly, an immediate washing with soap and water after a large amount of pesticide contacting the body, for example, could lower this risk.

A study published last December in the Journal of Neurology suggested that an impaired sense of smell or taste can raise an individual’s risk of developing Parkinson’s disease 2.5 times. Presently, Parkinson’s is diagnosed chiefly through assessment of motor symptoms and their severity. However, non-motor symptoms have gained attention due to their potential to predict Parkinson’s-related motor symptoms.

Chen and his team will use the grant to measure the ability to smell among roughly 2,200 farmers. After using a scratch-and-sniff method to try to identify a dozen common smells — smoke, lemon and cinnamon, for example — some 450 farmers will get a home visit from researchers who will test the farmers’ cognitive function and motor symptoms.

Researchers are using resources from the Agricultural Health Study (AHS) and its NIH scientists, along with research assistance from partners at Duke University, the University of Chicago and Penn State University.

A collaboration of the National Institute of Environmental Health Sciences (NIEHS), the National Cancer Institute (NCI), the Environmental Protection Agency (EPA) and the National Institute for Occupational Safety and Health (NIOSH), the AHS is a prospective investigation of licensed pesticide applicators from North Carolina and Iowa who were recruited for the study from 1993 to 1997.

Through 2014, after rounds of questionnaires, a telephone interview, and collecting a buccal-cell DNA sample, follow-up is ongoing to see what, if any, diseases develop among study subjects.

In addition, researchers annually link the study group to state cancer registries and vital records to monitor cancer incidence and mortality.

The AHS is believed to be the world’s largest study of farmers and their families.

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How 2 Sets Nerve Cells Interact to Control Movement Seen by Scientists Using New Tool

tool tracing movement

Using a new tool, researchers were able to see how two different sets of neurons interact in mice to control movement. They believe the method, called spectrally resolved fiber photometry, may help in unraveling what goes wrong in the brains of Parkinson’s patients and those with other disorders.

The research, “Spectrally Resolved Fiber Photometry for Multi-component Analysis of Brain Circuits,” appeared in the journal Neuron.

Progressive damage to nerve cells in the substantia nigra region of the brain lowers levels of the neurotransmitter dopamine — a chemical responsible for communication between neurons, or nerve cells — and is considered a hallmark of Parkinson’s disease.

Clinical studies in Parkinson’s patients and preclinical research in monkeys suggests that loss of dopamine causes an imbalance in the activity of two groups of neurons: the direct pathway (D1) and indirect pathway (D2). However, this hypothesis could not be confirmed experimentally due an inability to accurately distinguish between these cell types in the brain.

Using spectrally resolved fiber photometry (SRFP), a tool developed at the National Institutes of Health (NIH), researchers in an NIH office labeled D1 and D2 neurons with green and red fluorescent sensors and were then able to effectively follow how they work together in neurons of living mice.

“Our method allowed us to simultaneously measure neural activity of both pathways in a mouse as the animal performed tasks,” Guohong Cui, MD, PhD, the study’s senior author, said in a press release. “In the future, we could potentially use SRFP to measure the activity of several cell populations utilizing various colors and sensors.”

The scientists observed that when activity in D1 was stronger than in D2 neurons, the animals did a “start and go” — starting movement and moving to another location. When D2 neuronal activity was stronger, a mouse does a “start and stop” — it initiates a movement, but stops soon after.

D1 (red) and D2 (green) pathway activity seen in the striatum, part of the brain’s basal ganglia, in mice. (Photo courtesy of NIEHS)

Both movements are normal in mice and  their analysis may help predict what type of movement will be made based on the neural activity seen. Importantly, being able to trace such activity may help in understanding movement in mouse models of Parkinson’s.

“Based on these observations, we hypothesize that the direct-pathway (D1) activation serves as a movement start signal, and its magnitude determines the vigor of a movement. Meanwhile, the concurrently activated indirect pathway (D2) serves as a scalable stop signal that determines whether the initiated movement will continue or be terminated,” the researchers wrote.

Unlike current methods that cannot distinguish which neurons are generating an electrical output, “SRFP is more specific, because we can distinguish between groups of neurons and see their activity,” said Chengbo Meng, PhD, one of the study’s lead authors.

“We have developed a novel … method for simultaneous multi-color fluorescent signal measurement and unmixing from deep brain structures in vivo,” the study states. “Using this method, we show for the first time that the neural activities of two parallel … pathways are highly synchronized, and the magnitude of activation in these two pathways collaboratively determines the dynamics and fate of movement.”

In addition Parkinson’s disease, the team believes SFRP will contribute to a better understanding of Alzheimer’s, multiple sclerosis, stroke and addiction.

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Source: Parkinson's News Today

Peptron, NIH Establish Agreement to Develop SR-Exenatide for Neurodegenerative Diseases

Peptron SR-exenatide

Peptron recently obtained an exclusive worldwide license of intellectual property from the National Institute on Aging (NIA), a National Institutes of Health (NIH) center, covering the delivery and use of sustained-release (SR) exenatide in the treatment of Parkinson’s disease and other neurodegenerative disorders.

The intellectual property was developed as part of a cooperative research and development agreement between Korea-based Peptron and the NIH to create a form of exenatide that effectively crosses the blood-brain barrier and provides sustained release of the neuroprotective peptide to treat Parkinson’s and other degenerative diseases of the central nervous system.

“As experienced leaders in developing sustained-released therapeutics, the scientists at Peptron have enhanced the ability of SR-exenatide to cross the blood-brain barrier and deliver long-acting therapeutic effects of the neuroprotective peptide,” Ho-Il Choi, PhD, CEO and director of Peptron, said in a press release. “Neurodegenerative disorders, such as Parkinson’s and Alzheimer’s diseases, remain a great unmet medical need for millions of people around the world, and we are dedicated to innovating and advancing therapeutics for these life-altering diseases.”

The blood–brain barrier is a highly selective semipermeable membrane that separates circulating blood from the brain and extracellular fluid in the central nervous system, and allows the passage of water, some gases, and molecules.

However, large-molecule and most small-molecule drugs are not able to cross this barrier, making it challenging to deliver therapeutic agents to specific regions of the brain and treat most brain disorders.

SR exenatide is a glucagon-like peptide 1 (GLP-1) receptor agonist, which means it binds to the GLP-1 receptor, involved in controlling blood sugar levels, and promotes its action by enhancing insulin secretion.  GLP-1 receptors are found in the pancreas but also in the brain, and are used to treat Type 2 diabetes by stimulating insulin release.

According to the Cure Parkinson’s Trust, GLP-1 agonists are safe and well-tolerated medications that can mimic the action of human gut hormones, with a more stable profile than the actual hormones.

The systemic insulin resistance typically associated with Type 2 diabetes is also thought to somehow be associated with the onset of Parkinson’s disease, as patients often display impaired glucose tolerance — which then leads to brain insulin resistance.

Because of this link, there has been a growing interest in GLP-1 receptors and exenatide treatment for neurodegenerative diseases, and preclinical studies have shown evidence that exenatide may have beneficial disease-modifying effects.

However, delivering exenatide across the blood-brain barrier to the central nervous system is a major challenge.

Peptron is using part of the proceeds from a convertible bond that raised $24 million to establish a new manufacturing facility in Osang, Korea, equipped to produce SR-exenatide. Funds will also be used to support preparations for a Phase 2 clinical trial of SR-exenatide in Parkinson’s disease and evaluate the peptide in the treatment of Alzheimer’s disease.

“The support we have received from our NIH collaborators as well as our investors has allowed us to make significant progress in advancing a novel proprietary formulation of SR-exenatide to GMP manufacturing and Phase 2 clinical studies in Parkinson’s disease,” Choi said. “Moreover, our new facility enables us to manufacture additional sustained-release formulations of peptide and biologic drugs under GMP conditions to deliver further value in our collaborations with leading pharmaceutical and biotechnology companies.”

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Source: Parkinson's News Today

NIH awards $228,325 to Tech Startup to Develop MRI Device for Improved Diagnosis

MRI scan device

A startup developing a device for more affordable and efficient magnetic resonance imaging (MRI) scans was recently awarded a Small Business Technology Transfer (STTR) grant from the National Institutes of Health.

MR-Link, a technology company affiliated with Purdue University, will receive $228,325 to develop the device. Researchers say it will provide better imaging and promises to improve the diagnosis of Parkinson’s disease and other imaging-diagnosed diseases, while lowering costs and reducing health risks.

“This grant is validation for us that our idea is on the right track and there is a need for these kind of technologies that may help researchers to understand human physiology more accurately,” Ranajay Mandal, one of three MR-Link co-founders and a graduate student at Purdue University’s Weldon School of Biomedical Engineering, said in a press release.

Designed to be inserted into an existing MRI machine, the coin-sized device is synchronized with the MRI system to perform multiple scans at once, allowing researchers to record, stimulate, and image the brain and other organs. By incorporating electro-physiological signals from several organs, the new device promises to more effectively provide insight on a patient’s physiology.

STTR is a highly competitive program that awards federal funding to small businesses and nonprofit institutions to support scientific and technological innovation, and increase private sector commercialization of these innovations.

Out of more than 1,000 applications received from startups throughout the U.S. for Phase 1 STTR grants, only 169 were funded. MR-Link is the only one to receive funding from the state of Indiana, out of 32 that applied.

“We will use this funding to further develop our device and software into a user-friendly system, so that MR-Link can begin to distribute its beta testing units to MRI researchers,” said co-founder Nishant Babaria, a graduate student at the Purdue School of Electrical and Computer Engineering. “We hope to also use the money to enrich our research team with new professionals to help us package the software and hardware.”

MR-Link is reaching out to research facilities first before moving into the clinical market.

“We are open to partnerships with other laboratories and device manufacturers so we could soon deliver devices to more people and to benefit their research and to hopefully soon deliver to clinicians for them to better treat patients,” said co-founder Zhongming Liu, PhD, an assistant professor of electrical and computer engineering and biomedical engineering at Purdue.

The researchers are presenting the device at the upcoming International Society for Magnetic Resonance in Medicine, June 16-21, 2018, in Paris.

MR-Link is opening offices in the Purdue Research Park in West Lafayette, Indiana, the largest university-affiliated business incubation complex in the country.

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Source: Parkinson's News Today

Six Parkinson’s Community Leaders Receive Advocacy Awards

Parkinson's Advocacy Awards

Six Parkinson’s community leaders have received 2018 Advocacy Awards for their efforts to push policies that benefit patients, families, caregivers and others.

Two of the winners were recognized for their role in establishing the Accelerating Medicines Partnership Program, which in January 2018 announced a new Parkinson’s disease focus.

Representatives of the Parkinson’s Foundation and the Michael J. Fox Foundation presented the awards during the Parkinson’s Policy Forum.

The winners are:

  • Charles Brown of Massachusetts, who received the Social Media Advocacy Award.
  • Kevin Mansfield, Oregon, the Year-round Advocacy Award.
  • Leslie Peters, Colorado, the Milly Kondracke Award for Outstanding Advocacy.
  • Dr. Francis Collins, the Morris K. Udall Award for Public Service.
  • Dr. Walter Koroshetz, the Morris K. Udall Award for Public Service.
  • Gus Bilirakis, Florida, the Morris K. Udall Award for Public Service.

Collins, Koroshetz and Bilirakis received the Udall Award for their commitment to improving public policy. The award is named for Morris K. Udall, a member of Congress for three decades who died of Parkinson’s in 1998.

Udall, who was a candidate for the Democratic nomination for president in 1976, infused politics with a sense of humor, grace and dignity. He was diagnosed with Parkinson’s in 1980.

Collins, the director of the National Institutes of Health (NIH) since 2009, was one of those responsible for setting up the Accelerating Medicines Partnership Program. The goal of the pioneering, multi-pronged, public-private partnership program is to accelerate the development of new Parkinson’s treatments.

The NIH is leading the five-year collaboration, which includes government, the pharmaceutical industry, life sciences companies, and non-profit organizations. The program’s initial focus is identify biomarkers of Parkinson’s progression and assessing their potential as therapy targets.

“Advancing treatments for Parkinson’s disease is hampered by insufficient understanding of biological networks,” Collins said in a press release. “Drugs aimed at seemingly promising therapeutic targets fail in clinical trials. By combining our expertise and resources [the partners] hope to increase our collective odds of success in accelerating the development of effective treatments for a million Americans who suffer from this debilitating disease.”

The program will work with several Parkinson’s organizations, including The Michael J. Fox Foundation for Parkinson’s Research, the Foundation for the National Institutes of Health and the NIH’s National Institute of Neurological Disorders and Stroke (NINDS).

Koroshetz, one of the other Udall Award winners, has been the NINDS director since 2015. He was instrumental in establishing the Accelerating Medicines program in Parkinson’s disease. Koroshetz also oversees the eight Morris K. Udall Centers of Excellence for Parkinson’s Disease Research nationwide.

Bilirakis, the other Udall Award recipient, is a congressman representing the 12th district of Florida who is serving his sixth term in the House.

He is a long-time member and co-chair of the Congressional Caucus on Parkinson’s Disease. He also serves on the Health Subcommittee of the House Energy and Commerce Committee, which oversees the U.S. Food and Drug Administration. The FDA will also be a key partner in the Accelerating Medicines program, providing regulatory guidance for drug development.

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Source: Parkinson's News Today

New Parkinson’s Collaboration Is Exactly What’s Needed, Editorial Says

Lancet editorial

So much Parkinson’s-related biomarker information has been collected under various research projects that sorting through it for additional clues to the disease poses a challenge to scientists.

The scope of the information makes a collaborative research effort paramount — and a U.S. government-sponsored initiative is meeting that need, according to an editorial in the journal The Lancet Neurology. The initiative is also focusing on therapy development.

It praised the Accelerating Medicines Partnership on Parkinson’s disease as the most ambitious effort so far to find a cure for Parkinson’s.

Led by the Foundation for the National Institutes of Health (NIH), the collaboration includes the US Food and Drug Administration (FDA), five biopharmaceutical and life science companies, and one non-profit foundation.

The aim of the partnership, formed on Jan. 30, 2018, is to discover new therapies for the disease plus new biomarkers that can predict its progression and whether treatments are working.

Scientists have collected a lot of Parkinson’s-related biosamples under programs such as the US National Institute of Neurological Disorders and Stroke (NINDS) Parkinson’s Disease Biomarkers Program (PDPB), the Michael J Fox Foundation Parkinson’s Progression Markers Initiative (PPMI) and BioFIND study, and the Harvard Biomarkers Study.

Samples include cerebrospinal fluid (CSP), RNA, blood plasma and DNA samples from more than 3,000 patients and more than 1,700 healthy controls.

“These initiatives have highlighted a shared interest between public and private organisations, and the data being generated present an opportunity to drive biomarker development more intensively than to date for translation into more effective therapies,” the editorial said.

The biosample information could help scientists identify new biomarkers, but its huge scope has posed a challenge to both researchers and companies.

Despite major investments, companies have yet to come up with disease-modifying Parkinson’s therapies — those can delay progression or relapses. Collaboration may be the way to achieve breakthroughs.

The Accelerating Medicines Partnership researchers will be digging into the large amounts of data to identify the most promising biomarkers of disease progression, which can then be used to validate new treatments.

Importantly, both the data and analyses will be shared with the broader research community in a platform called the AMP PD Knowledge Portal.

The portal will also incorporate data from two ongoing Phase 3 clinical trials in patients with early Parkinson’s disease. One is the Study of Urate Elevation in Parkinson’s disease (SURE-PD3; NCT02642393) and the other the Efficacy of Isradipine in Early Parkinson Disease (STEADY-PD3; NCT02168842).

In addition, the portal will enable the rest of the research community to participate in the collaborative effort and share their results.

“Sharing results from analyses of Phase 3 data via the knowledge portal will provide the opportunity to conduct analyses on a scale that could not be performed by a single partner alone,” according to the editorial.

The program will have three stages. The first, expected to end within 18 months, will create the working groups, the knowledge portal, and further analyze RNA from patients to identify potential biomarkers.

The second stage, expected to take three years, will pursue additional large-scale biomarker discovery to categorize patients, monitor their disease progression, and predict their outcomes. It will also identify Parkinson’s biomarkers that are common among cerebrospinal fluid, plasma, and brain tissue.

The last stage will validate the most promising biomarkers.

NIH will manage the partnership with GlaxoSmithKline, the Michael J Fox Foundation, NINDS, Pfizer, Sanofi, Celgene, Verily Life Sciences and the FDA, and coordinate the pledged investment of $24 million over five years.

“Bringing together the collective capabilities and resources across public and private sectors offers the best opportunity to identify and address challenges in early-stage drug development and thus the best opportunity for finding a cure,” the editorial concluded.

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Source: Parkinson's News Today