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Michael J. Fox Foundation Funds Search for Parkinson’s Urine Biomarkers

urine biomarkers, Parkinson's

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) is funding work at Tymora Analytical Operations that screens for protein biomarkers in urine to help detect the neurodegenerative disease earlier.

Tymora, a research and development laboratory company affiliated with Purdue University, uses the EVtrap approach, or Extracellular Vesicles Total Recovery and Purification. This approach analyzes urine samples to find disease biomarkers such as proteins and their versions known as phosphoproteins — proteins that have phosphate groups attached.

More than 100 samples provided by MJFF will be analyzed to look for phosphoproteins found in people with Parkinson’s.

“This award will support our analysis work to find new urine biomarkers, which can lead to early Parkinson’s disease diagnoses and treatments,” Anton Iliuk, PhD, Tymora’s chief technology officer, said in a press release written by Chris Adam.

Iliuk noted that the lack of specific tests to identify Parkinson’s often leads to reviews of medical histories and to neurological and physical tests that can be inconclusive. “Our approach,” he said, “uses a simple urine test, often already performed during regular exams.”

Parkinson’s is typically diagnosed at late stages, when patients are already experiencing symptoms. That reduces treatment effectiveness.

“Diagnosing Parkinson’s at an early stage with our method would give doctors a greater opportunity to successfully treat the condition,” Iliuk said.

According to Tymora, the technology enables the identification of more than 1,200 unique proteins from only 0.2mL of urine. It also allows the identification of up to 1,000 unique phosphoproteins from 10mL of urine, as described in a 2018 study. The team at Tymora previously had demonstrated the feasibility of high-throughput identification of phosphoproteins in tiny vesicles — called exosomes — found in plasma, which contain unique cargo if released by diseased cells.

EVtrap, says Tymora, is the optimal technique to develop urine-derived exosome phosphoproteins for more accurate disease profiling and better treatments.

Most of the company’s technology was developed at Purdue by W. Andy Tao, PhD, Tymora’s chief scientific officer. Tao is a professor of biochemistry at Purdue’s College of Agriculture and received the 2017 Outstanding Commercialization Award for Purdue faculty.

The EVtrap technology aligns with the university’s Giant Leaps celebration of its global advancements in health, which is part of Purdue’s 150th anniversary.

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Fox Foundation Awards AC Immune New Grant to Advance Alpha-Synuclein Imaging Agent

Grant award

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded AC Immune a new grant to further the development of tracer compounds for Parkinson’s disease (PD).

Specifically, this award continues MJFF support for AC Immune’s alpha-synuclein positron-emission tomography (PET) tracer program, aiming for an accurate imaging agent of the alpha-synuclein protein clumps in nerve cells of the brain that are thought to underlie Parkinson’s development and progression.

Positron-emission tomography (PET) is a non-invasive imaging technique that enables the visualization of the metabolic processes in the body. A PET tracer that is alpha-synuclein specific would allow scientists to study the distribution and alterations of these toxic clumps as the disease progresses.

AC Immune researchers has identified several PET tracer compounds with high affinity and selectivity to alpha-synuclein deposits. They did so by screening the company’s library of small molecules for suitable alpha-synuclein PET tracer candidates.

This “molecular collection,” also known as the Morphomer platform, enables the identification of a new class of low molecular weight compounds. This platform, in turn, allows for the generation of small molecules — called morphomers — that specifically bind to misfolded proteins, working to break up the neurotoxic clusters and prevent protein aggregation.

Importantly, these molecules can reach the brains of non-human primates, adding to their potential as a central nervous system tracer.

A lead alpha-synuclein PET tracer candidate, ACI-3024, entered a Phase 1 clinical trial of its ability to capture pathological alpha-synuclein in neurodegenerative diseases like Parkinson’s. The study is assessing the safety, tolerability, and interactions between the body and ACI-3024 (pharmacokinetics and pharmacodynamics) in healthy volunteers.

Jan Stöhr, PhD, head of Non-Alzheimer’s Disease Proteinopathies at AC Immune, will give an oral presentation about this alpha-synuclein PET tracer program at the Fox Foundation’s 13th Annual PD Therapeutics Conference set for Oct. 15 in New York City.

“We are very proud to be working together with MJFF on our a-syn [alpha-synuclein] PET tracer program, which offers patients the potential for earlier diagnosis of PD and facilitates the development … of imaging agents capable of earlier detection and disease monitoring, as well as the development of a broad pipeline of effective therapeutic candidates focused on the prevention and treatment,” Andrea Pfeifer, PhD, CEO of AC Immune, said in a news release.

The Fox Foundation first began supporting AC Immune’s program for alpha-synuclein-specific tracer compounds in 2015. If the program is successful, it could offer a first imaging agent capable of accurately identifying and monitoring Parkinson’s progression.

AC Immune is also working to develop oral small molecule alpha-synuclein inhibitors, and anti-alpha-synuclein antibodies to treat Parkinson’s and related diseases.

The grant amount was not released.

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Feedback from Parkinson’s Patients Can Help Improve Trial Recruitment, Retention, Study Says

clinical trial feedback

Getting the perspective of Parkinson’s disease patients on their motivations and experiences participating in clinical trials can help increase recruitment and retention of future studies, a report suggests.

Although preventable, one of the most common reasons Phase 2 or 3 trials fail is because not enough patients participate. This could be due to inadequate recruitment or because patients drop out during the trial.

The report, “Recruitment and Retention in Clinical Trials of Deep Brain Stimulation in Early-Stage Parkinson’s Disease: Past Experiences and Future Considerations,” published in the Journal of Parkinson’s Diseaseevaluated patient responses after a pilot study on the safety and tolerability of subthalamic (STN) deep brain stimulation (DBS) for the treatment of early-stage Parkinson’s disease.

DBS is commonly used to treat patients with advanced Parkinson’s who no longer respond to available medications and are unable to adequately manage their symptoms.

The U.S. Food and Drug Administration also recently expanded the use of DBS — which requires surgery to implant a device to stimulate targeted regions of the brain — to patients with mid-stage disease who also respond poorly to standard medications.

Vanderbilt University in Nashville, Tennessee, completed a clinical trial (NCT00282152) that included 30 patients, ages 50 to 75, with early-stage Parkinson’s disease. The participants were randomized to receive either optimal drug therapy (ODT) or ODT plus STN-DBS and followed for two years.

At the end of this pilot study, participants were asked to complete a survey regarding their experiences participating in the trial. Their responses were compared with those from an independent survey that used Fox Insight, an online clinical study platform established by The Michael J. Fox Foundation for Parkinson’s Research, to survey early-stage Parkinson’s patients who were considering possibly participating in a trial for DBS.

The pilot trial was specifically designed to collect preliminary safety and tolerability data necessary to conduct an FDA-approved Phase 3 trial (IDE#G050016) to investigate the hypothesis that DBS in patients with early Parkinson’s can slow disease progression.

Importantly, patient experiences were expected to inform the feasibility of scaling up recruitment for the future trial.

Results revealed that the primary motivation for participating in a clinical trial was the desire to advance medical research, cited by 85% of trial respondents. This was followed by a desire for the best medical treatment, for 70%, and for 59%, a desire to learn more about Parkinson’s disease. Similarly, altruistic motivations were also seen in the Fox Insight survey.

The most frequently listed fear that could impact trial participation was surgery-related concerns, in 44% of participants. Although 37% of the pilot study participants did not report any burdens, the most common burdens associated with clinical trials were financial commitments such as time off work and traveling costs (30%), as well as some of the therapeutic interventions such as neuropsychological testing (26%), and the weeklong therapeutic washout periods — without medication (26%).

The pilot study implemented a rigorous informed consent progress aimed at educating potential participants on their role in the study. Most participants responded positively to this approach. Providing sufficient education at the start of the trial may decrease the drop-out rate. In fact, only 3% of participants did not complete the study. As a result, this approach of informed consent will be used in the upcoming Phase 3 trial, according to the authors.

Getting patient perspectives is a new strategy that could improve clinical trial recruitment and retention. Whereas most clinical trials were solely designed by physicians, now, as the authors suggest, by asking patients about their experiences and including their feedback in trial development, “the focus shifts away from the physician and toward the patient.”

“By understanding the motivations and barriers to trial participation of past and potential subjects, we attempt to predict feasibility of recruitment in the future pivotal trial,” the authors wrote. “The fundamental similarities of these two cohorts of patients with early-stage [Parkinson’s disease] suggest that the planned multicenter, pivotal trial will experience similarly successful recruitment and retention as the single-center pilot trial.”

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Source: Parkinson's News Today

‘PATH to PD’ Program Seeks to Deepen Understanding of Parkinson’s Onset, Progression

Michael J. Fox Foundation

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will fund a new program to investigate the pathogenesis of Parkinson’s disease called “PATH to PD.”

The two-year program includes three research teams that will collaborate to investigate risks associated with genetics, environment and aging, while working with a common framework to gain a better understanding of the onset and progression of Parkinson’s.

Each PATH to PD-funded project will receive $2 million.

“The vast diversity of pathways implicated in Parkinson’s pathology to date indicates that multiple physiological routes can lead to PD, and these routes may intersect or be temporally dependent,” Todd Sherer, PhD, CEO of MJFF, said in a press release. “Through PATH to PD, our Foundation aims to encourage researchers to bring a holistic new approach to bear on refining today’s understanding of what Parkinson’s is — so that we can better strategize how to slow or stop the disease.”

Parkinson’s is not fully understood by scientists. Even though it is known the disease stems from gene-environment interactions as we age, the emerging picture of Parkinson’s is that of a vast, interwoven network culminating in a disease that varies greatly in cause, rate of progression, symptomology and treatment response.

All three teams will collaborate to build a common framework linking mechanisms through which genetics, environment and aging-associated risk and causal factors may lead to Parkinson’s disease.

University of Pittsburgh researchers, led by J. Timothy Greenamyre, MD, PhD, will seek out links between environmental and genetic triggers of the disease. Researchers will look into the mechanisms that lead neurotoxins to cause neurodegeneration and how these pathways are involved in interactions with genetic factors such as LRRK2 (leucine-rich repeat kinase 2), the leading genetic cause of the disease.

National Institutes of Health researchers, led by Andrew Singleton, PhD, will map the genetic effects in Parkinson’s by growing nerve cells from induced pluripotent stem cells, and map how various genetic alterations lead to the molecular and cellular changes associated with Parkinson’s.

Northwestern University researchers, led by D. James Surmeier, PhD, will investigate the link between aging and Parkinson’s. Using advanced gene-editing techniques, they will look at how cellular aging and related DNA and mitochondrial damage contribute to neurodegeneration in rodent and human cells.

These efforts have the common goal of developing a disease-modifying treatment for Parkinson’s disease, so that it can stop or slow disease progression.

For disease-modifying treatments to work best, however, they should target key underlying disease-causing pathways, which need to be identified and isolated first.

Some cases of Parkinson’s occur due to genetic factors, known as familial Parkinson’s disease. However, familial Parkinson’s is only a small fraction of all diagnoses of the disease. Informed guesses lean toward  environmental factors, including heavy metal or pesticide exposure, since aging still accounts for the greatest risk factor underlying this disease.

The fact that there is no identifiable cause of immediate contamination, researchers suspect this might be due to a lifetime accumulation of otherwise minor compound exposures that eventually lead to the development of the disease or contribute to a “switch” in Parkinson’s risk genes. Perhaps the process occurs naturally as medicine allows us to live longer, or perhaps it’s a combination of both.

“With Parkinson’s prevalence expected to double by 2040 to nearly 13 million people worldwide, our Foundation believes it is our obligation to continue building on current research momentum to eradicate this disease once and for all,” Sherer added.

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Source: Parkinson's News Today

10 Organizations That Support Parkinson’s Disease Patients and Their Families

If you’re struggling with Parkinson’s disease or know someone who is, it’s incredibly helpful to have a list of organizations that can support you or your loved one along the way. The following is not an exhaustive list, but hopefully it will prove to be a helpful resource.

1. National Parkinson’s Foundation helps patients actively enjoy life through expert care and treatment research.

2. American Parkinson’s Disease Association provides support, education, and research to help patients and caregivers live a fuller life.

3. Michael J. Fox Foundation is helping to raise money for much-needed research to help find a cure. From ways you can get involved to a great blog chock-full of Parkinson’s-related information, the foundation is a wonderful resource.

MORE: Seven things to know about deep brain stimulation for Parkinson’s disease

4. Parkinson’s Disease Foundation provides information including news about the disease, information about upcoming events, insight into the latest research, and education about what to expect.

5. European Parkinson’s Disease Association advocates for the rights and needs of patients and their families.

6. The Davis Phinney Foundation is committed to supporting research programs that help deliver inspiration, information and tools that will enable people living with Parkinson’s to have more control in managing their disease.

MORE: Is blurred vision an early symptom of Parkinson’s disease?

7. The Parkinson Alliance is the umbrella organization for the Parkinson’s Unity Walk which takes place every spring in New York City. They also sponsor Team Parkinson, a fundraising racing event. You can find current news and information about research projects on their site.

8. Partners in Parkinson’s has a program that connects patients and caregivers to an advocate who will listen and offer advice and support at no cost. This is just one of the services you can find through Partners in Parkinson’s.

9. Caring.com has a support group for caregivers, family and friends of those with Parkinson’s disease. No subject is off-limits in this forum.

10. The National Parkinson Foundation has a site specifically geared to caregivers. They help carers navigate the emotional, financial, and physical challenges they may face caring for someone with Parkinson’s.

Having resources that help keep you informed, and offer support and encouragement to patients and their loved ones is so important. These are just 10 of the hundreds of organizations out there that are available to you. You can search online or visit your local library for more.

MORE: 11 facts about Parkinson’s disease you may not know

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.

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Source: Parkinson's News Today