Aspen Neuroscience Receives $6.5M to Advance New Patient-specific Cell Therapy for Parkinson’s

Aspen Neuroscience

Aspen Neuroscience, a new biotech company, has raised $6.5 million to develop cell therapies for Parkinson’s disease using patients’ own cells.

The company was co-founded by renowned stem cell scientists Jeanne F. Loring, PhD, and Andres Bratt-Leal, PhD, and initially supported by Summit for Stem Cell, a non-profit organization that provides a variety of services for Parkinson’s patients.

Parkinson’s hallmark motor symptoms include tremor, slowness of movement (bradykinesia), stiffness (rigidity), uncontrollable movements (dyskinesia), and poor balance.

As the disease progresses, patients typically need to gradually increase their dopaminergic therapeutic dose for maximum benefit. Even after that they might sometimes experience reappearance or worsening of symptoms due to diminishing effects of dopaminergic therapy, known was “off” periods.

Importantly, dopaminergic therapy is delivered to areas of the brain other than the striatum, a key motor control region severely affected in Parkinson’s disease. Because of the therapy’s off-target behavior, patients also may experience side effects such as hallucinations or cognitive impairment.

Aspen wants to combine its expertise in stem cell biology, genomics and neurology and develop the first autologous (self) stem cell-based therapy for Parkinson’s disease.

In this type of cell therapy, a patient’s own cells (usually skin cells) are reprogrammed back into a stem cell-like state, which allows the development of an unlimited source of almost any type of human cell needed, including dopamine-producing neurons, which are those mainly affected by this disorder.

Because these cells are derived from patients, they do not carry the risk of being rejected once re-implanted, eliminating the need for immunosuppressive complementary therapies, which carry serious side effects such as infections and possibly limiting therapeutic potential.

In theory, replacing lost dopaminergic neurons with new stem cell-derived dopamine-producing ones could potentially ease or reverse motor symptoms associated with the disease.

Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson’s disease,” Howard J. Federoff, MD, PhD, Aspen’s CEO, said in a press release.

“We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist,” Federoff said.

The company’s lead product (ANPD001) is undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinson’s disease. Aspen experts also are developing a gene-editing treatment (ANPD002) for familial forms of Parkinson’s, starting with the most common genetic variant in the GBA gene, which provides instructions to make the enzyme beta-glucocerebrosidase.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32, according to the press release.

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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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Parkinson’s UK Awards Scientist £100,000 to ID Ways of Protecting Dopamine-producing Neurons

Parkinson's UK grant

A scientist at the University of Sheffield in England has been awarded a £100,000 grant by Parkinson’s UK to develop a treatment that might slow or stop the progression of Parkinson’s disease and protect brain cells.

The one-year grant, worth about $120,000, was given to Heather Mortiboys, a senior research fellow at the university’s Institute for Translational Neuroscience (SITraN), by Parkinson’s Virtual Biotech Programme, the British charity’s therapeutic development arm.

“All the clinical treatments for people living with Parkinson’s at the moment are based on easing these sometimes devastating symptoms,” Mortiboys said in a press release. “With this new funding award … we have the potential to go on to develop a drug treatment which will actively address the root cause of these symptoms to slow, or halt the progression of Parkinson’s for the first time.”

Mitochondria, power factories for cells that include dopamine-producing brain cells, don’t work as they should in people with Parkinson’s disease.  Resulting shortages in cellular energy cause neurons to fail and ultimately die, particularly dopamine neurons. Those nerve cells are responsible for movement and coordination, and rely on mitochondria to function.

In her previous work, Mortiboys developed a model of dopamine brain cells — using skin cells from patients — that allows researchers to test potential therapies. Her research team was able to grow high numbers of brain cells derived from these skin cells. They used them to identify compounds that support dopamine neurons and their mitochondrial function, and potentially lessen cell death.

With this award, Mortiboys and her team will try to pinpoint the molecules in these compounds that are of greatest benefit to mitochondria in producing the energy needed to support these brain cells. Working in collaboration with the National Institute of Health Research (NIHR) Sheffield Biomedical Research Centre, the scientists will then move the molecules into a drug discovery phase.

“There is an urgent need for treatments to protect the nerve cells that become damaged in patients with Parkinson’s disease, which will have a crucial impact in slowing the progression of the condition and improving the quality of life” said Pamela Shaw, director of SITraN and and the university’s new Neuroscience Institute.

Potential treatments identified through this process will be further developed through a partnership with the NIHR Biomedical Research Centre at the Royal Hallamshire Hospital, a Sheffield teaching hospital, Shaw said, adding “[w]e are hugely grateful to Parkinson’s UK for supporting this important translational research.”

“We are delighted to partner and work with Dr Heather Mortiboys and her team at the University of Sheffield. Through our Virtual Biotech initiative, we are committed to accelerating promising and breakthrough treatments for Parkinson’s,” said Richard Morphy, drug discovery manager at Parkinson’s UK.

“This is an exciting new approach that could rescue defective mitochondria inside neurons to prevent dysfunction and degeneration of dopamine-producing brain cells,” Morphy said.

Parkinson’s UK, which invests about $4.8 million a year in work that advances potential treatments, estimates that about 148,000 people in the U.K. have this neurodegenerative disease.

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APDA Meets to Discuss Grants, Diversity in Parkinson’s Research, Support, and Care

APDA Grant Research

The American Parkinson Disease Association (APDA) recently hosted two groups of experts who assessed scientific projects vying for funding, and addressed diversity issues in Parkinson’s disease research and care.

The organization met with its scientific advisory board (SAB) to decide which grant projects it will fund for the 2019-2020 academic year. Grants are based on overall significance and field impact, appropriateness of the project’s chief investigator and scientific environment, and feasibility of the project’s proposed budget and end date. Funding decisions will be announced in August.

The APDA also hosted its first-ever Diversity in Parkinson’s Research Conference, which focused on needs surrounding the disease in diverse and under-served communities. Attendees included researchers investigating Parkinson’s in ethnic and minority populations, and clinicians who treat such patients.

Panel discussions included an overview of APDA diversity initiatives, research about biomarkers in diverse populations, disparities in Parkinson’s clinical trial enrollment, and what the field of hypertension can teach Parkinson’s investigators about access to diverse communities.

Currently, most Parkinson’s research focuses on relatively older white men, the APDA said. The organization wants to expand investigations to include more patients of varying ages, genders, races and ethnicities. It also wants more access among these groups for care, programs and services.

”APDA’s mission is to help everyone impacted by Parkinson’s disease live life to the fullest, and we mean everyone,” Leslie A. Chambers, APDA president and CEO, said in a press release.

The organization plans to establish an annual grant to support research focused on closing diversity gaps. For now, it offers an annual $50,000 post-doctoral fellowship, and multiple $75,000 research grants. The three-year $300,000 George C. Cotzias Fellowship supports early-career physician-scientists. In addition, the APDA awards its Centers for Advanced Research $100,000 each year to support PD investigations. (Visit this site for more information on APDA-funded research.)

”It’s so exciting to see the fascinating ideas outlined in the grant submissions,” said Rebecca Gilbert, MD, PhD, APDA vice president and chief scientific officer, of the current crop of proposals. “Proposed research projects included everything from ways of detecting a diagnosis of PD in the blood, to exploring ways that telemedicine can improve the lives of patients with PD. The SAB certainly had their work cut out for them and made some tough choices,” she said.

In addition to deciding who gets new grants, the SAB receives updates during annual meetings about previously funded research. During the May 16 meeting, for example, members were apprised of the latest research at the University of Alabama at Birmingham, where scientists are focused on advances in the role of brain inflammation in Parkinson’s development and progression. The SAB also heard from Washington University School of Medicine researchers studying imaging biomarkers for Parkinson’s.

David Standaert, a leading Parkinson’s researcher at the University of Alabama at Birmingham, also is the SAB’s chairman. He called the Diversity in Research Conference a “fantastic” first step toward finding answers.

”Together, I think we can do great things to make both our research and services more inclusive and accessible,” he said.

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APDA Awards More Than $1.7 Million to Parkinson’s Disease Researchers

APDA grants

Hoping to lure new researchers to the Parkinson’s disease field and to support others’ new and innovative ideas, the American Parkinson Disease Association (APDA) has awarded more than $1.7 million in fellowships, research grants, and funding for APDA Centers for Advanced Research.

 The awards include a highly competitive George C. Cotzias Fellowship, two post-doctoral fellowships, 11 research grants, and eight APDA Centers for Advanced Research. The centers support front-line research into the cause, treatment, and eventual cure of Parkinson’s, according to a press release.

Applications are reviewed annually by the APDA’s Scientific Advisory Board (SAB). During that meeting, board members also decide the kind of research they want to invest in that year.

Among other things, the association’s support this year will give insight into the disease’s new biomarkers, and examine how and why Parkinson’s affects genders and some ethnic groups differently, said Rebecca Gilbert, MD, PhD, APDA’s vice president and chief scientific officer.

“APDA is funding an exciting array of projects this year,” she said.

In the past, research projects have produced important pilot data, according to the release, resulting in lucrative research grants from the National Institutes of Health and other funding entities.

“It is vital to accelerate research and support translational ideas that can lead to new treatments for those people living with [Parkinson’s disease],” said David G. Standaert, MD, PhD, John N. Whitaker professor and chair of neurology at the University of Alabama at Birmingham‘s School of Medicine, and SAB chairman. “APDA’s SAB is focused on investing in the best science to support that important mission.”

This year’s George C. Cotzias Fellowship went to emerging physician-scientist Aasef Shaikh, PhD, of the Cleveland VA Medical Center and Case Western Reserve University. The three-year award is designed to fund a long-term Parkinson’s project.

Shaikh’s project is aimed at learning how Parkinson’s affects the part of the brain that maintains gait and balance.

Learn more about the awardees and browse all APDA-funded research here. Researchers and physicians interested in applying for APDA funding can visit here for 2019-2020 opportunities.

APDA’s Centers for Advanced Research fund expansive Parkinson’s research programs. These programs include novice researchers, fellowship and early-stage discovery programs, and advanced clinical translation. Through a rigorous application process, two new centers were chosen this year.

 “The work being done as a result of the grants in this new funding cycle will have an incredible impact on the world of [Parkinson’s disease],” said Leslie A. Chambers, APDA’s president and CEO.

In its 57-year history, APDA has raised more than $177 million to provide patient services, increase public awareness of Parkinson’s, support its research, and ultimately end the disease.

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Michael J. Fox Foundation Offering $7.5M in Funding for Parkinson’s Research Projects

MJFF research funding

The Michael J. Fox Foundation is continuing to support the advancement of Parkinson’s disease research by investing up to $7.5 million in projects proposed by investigators, the nonprofit recently announced.

Funding will be considered for projects aimed at the following four areas: exploring new biological targets for therapeutic development; identifying biomarkers to objectively measure disease progression; testing potential new treatments; and determining preventive factors.

“Our core goals are to better understand, measure, treat and — most significantly — prevent Parkinson’s disease,” Todd Sherer, PhD, the foundation’s CEO, said in a press release. “We ask scientists to bring us their promising, innovative projects in these areas to move the needle closer to cures and better quality of life for people with this disease today.”

Scientists can submit their proposals through Sept. 26, with funds anticipated in May 2019.

The foundation has divided each of the research areas into four programs for which investigators can apply for grants.

To find new biological targets of the disease, the Target Advancement Program will focus on the identification of proteins and pathways that are involved in the onset and progression of Parkinson’s.

Finding these targets will not only shed light on neurodegeneration and motor and non-motor dysfunction and symptoms, but it will also enable the development of potential new therapies to slow or stop disease progression.

The Improved Biomarkers and Clinical Outcome Measures program is intended to identify biomarkers associated with the disease that can be effectively quantified using objective tests, and accelerate the development of new therapies. These tests are expected to improve diagnosis, track disease progression, and monitor treatment response.

The Therapeutic Pipeline program seeks to develop new therapies that can change the course of the disease and improve treatment beyond the current standards of care. Existing treatments alleviate symptoms, but do not address several aspects of the disease, and can result in serious side effects.

A fourth program is centered on Parkinson’s prevention. By using epidemiological factors, such as lifestyle behaviors (e.g. diet and exercise), medication taken or other types of treatments, the foundation hopes to identify which factors can decrease the risk of Parkinson’s disease.

Each of the four programs will receive a total of $1.5 million of the grant money, with the exception of the Therapeutic Pipeline program, which will receive $3 million to fund preclinical and clinical studies.

Applications for project submissions are now open. Both academic and industry scientists worldwide are invited to apply.

The foundation will host a webinar at 12 p.m. EST Sept. 6 to review the goals of the programs, explain the funding process, and answer applicant questions. For more information and to register for the webinar, visit here.

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Neuraly Raises $36M to Advance Potential Parkinson’s Therapy into Clinical Testing

NLY01 and Neuraly financing round

Neuraly announced it has raised $36 million to advance its lead candidate NLY01 into clinical testing as a potential disease-modifying agent for Parkinson’s disease, and to develop other investigative compounds for this and similar neurodegenerative disorders.

The company, a Johns Hopkins University startup, expects to start a clinical trial of NLY01 this year.

“Currently, there aren’t any treatments that reverse, stop, or even slow neurodegeneration in diseases like Parkinson’s and Alzheimer’s. The treatments that do exist – all symptomatic – provide only temporary improvement in motor and cognitive function, but even these become less effective over time,” Seulki Lee, PhD, chairman, founder, and CEO of Neuraly, said in a press release. “We believe that the science supports NLY01 as a potential disease-modifying therapy capable of slowing the progression of disease.”

NLY01, developed by researchers at Johns Hopkins School of Medicine, binds to a specific type of receptors called glucagon-like peptide-1 receptors (GLP-1R), which are found on pancreas cells.

The receptor is best known for its role in insulin signaling, and has been studied as a means to treat diabetes type 2. But studies also suggest that GLP-1 mimicking compounds — which activate GLP-1R — can penetrate into the brain, where they seem to exert neuroprotective functions.

In preclinical studies in mice models of Parkinson’s, NLY01 was seen to prevent nerve cell damage by binding to microglia cells, which are involved in immune responses to infection or injury in the brain. In Parkinson’s patients, microglia cells are often overactive, damaging nerve cells.

Because similar drugs are already approved to treat diabetes — such as Byetta (exenatide) and Victoza (liraglutide) — researchers expect that further work will show NLY01 has a similar and well-tolerated safety profile.

With the $36 million raised in a Series A financing round, Neuraly is planning to soon begin a Phase 1 trial of NLY01, and to add other neuroprotective candidates to its treatment pipeline.

“We expect NLY01 to be a pioneering treatment for Parkinson’s with low development risks as we have seen unprecedented efficacy in pre-clinical models and well-characterized safety profiles in a similar class of molecules,” said Viktor Roschke, PhD, chief scientific officer of Neuraly. “We look forward to initiating NLY01 into clinical trials later this year.”

As part of the funding agreement, John Ku, executive vice president of Smilegate Investment; Phillip Jung, associate at Maryland Venture Fund; Junghee Lim, executive managing director of InterVest; and Keele Park, CEO of Magna Investment, will join Lee and Roschke on Neuraly’s board of directors.

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Researchers Receive Funding to Study New Gene Linked to Parkinson’s Disease

gene linked to Parkinson's

A multidisciplinary research team at Purdue University in Indiana and the University of Bordeaux in France was awarded a $107,000 grant by the The Michael J. Fox Foundation for Parkinson’s Research to study the neuroprotective ability of a newly discovered gene associated with Parkinson’s disease.

The gene was discovered by Jean-Christophe Rochet, PhD, a professor of medicinal chemistry and molecular pharmacology at Purdue, when he and Min Zhang, PhD, a professor of statistics at Purdue, identified a list of genes related to Parkinson’s by analyzing several data sets obtained from one of the National Institutes of Health-Designated Data Repositories.

“While some of the genes on the list were already known, Chris [Rochet] found an interesting gene that has not been reported to be directly associated with Parkinson’s disease yet,” Zhang said in a press release.

The gene, called NFE2L1, produces a protein that controls other genes involved in the survival and maturation of dopaminergic neurons.

Parkinson’s disease is caused by the impairment or death of these dopamine-producing nerve cells, or neurons, in a region of the brain called the substantia nigra, which controls the body’s balance and movement.

“NFE2L1 levels are reduced in dopaminergic neurons in the brains of Parkinson’s disease patients,” Rochet said. “We recently found in a large-scale genomic study that a minor allele of NFE2L1 can lower Parkinson’s risk. These observations imply that neuron death in Parkinson’s disease may result in part from a loss of the neuroprotective action of NFE2L1.”

Researchers hypothesize that increasing the production of the NFE2L1 protein in rodent models of Parkinson’s disease will reduce nerve cell death.

The team then intends to screen for compounds that can increase NF2L1 levels in the brain, either by stimulating its production or by blocking its degradation by the proteasome, a protein complex that destroys unnecessary or damaged proteins.

This research will not only shed light on the protein’s ability to reduce neurotoxicity, but the team also hopes it will aid in the development of Parkinson’s therapies that target NFE2L1 levels in the brain.

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Foundations Announce Latest Centers for Training Parkinson’s Specialists

Parkinson's specialists

The Michael J. Fox Foundation (MJFF) and the Edmond J. Safra Philanthropic Foundation recently awarded five new medical centers with funding to train clinician-researchers working with Parkinson’s disease and other movement disorders.

The five centers, awarded at the fourth round of fellowship funding, will receive financial assistance through a program, the Edmond J. Safra Fellowship in Movement Disorders, designed to have 20 new movement disorder specialists graduated by 2021.

The fellowship was launched in 2014 by the two foundations and it awards funding annually to five international academic medical centers to train one new movement disorder clinician-researcher (respectively) for two years.

The latest centers awarded are: Emory University, in Atlanta, Georgia; Northwestern University, in Chicago, Illinois; Radboud University, in Nijmegen, The Netherlands; University of Lübeck, in Lübeck, Germany, and University of Pennsylvania, in Philadelphia, Pennsylvania.

Each institution must now identify a fellow who will begin two years of training in July 2019. Fellows work directly with movement disorder specialists who serve as mentors to learn the skills necessary for a career as a clinician-researcher.

“This program signals our commitment to Parkinson’s research and care, and we’re honored to collaborate with our longtime partner and supporter the Edmond J. Safra Foundation,” Todd Sherer, PhD, CEO of the MJFF, said in a press release. “Building an international network of movement disorder specialists is critical to driving research momentum and better addressing the considerable care needs of those living with Parkinson’s.”

The foundations announced the five winners at the New York City “Fellowship Symposium Day,” an event created to unite fellows and mentors to share research progress.

The special day was attended by 25 Edmond J. Safra fellows and fellowship directors and by Lily Safra herself, chairwoman of the Edmond J. Safra Foundation and MJFF Board member since 2001.

Graduating fellows in the Class of 2018 talked about their experience throughout the day and at a special luncheon, also attended by Ms. Safra.

“This program is serving a critical need,” said Safra. “More expertly trained movement disorder specialists means more doctors to care for people with Parkinson’s and lead research toward better therapies and a cure,” she said.

The Edmond J. Safra fellowship program addresses the growing need of being able to see a movement disorder specialist. These physicians, who are small in numbers, combine training in diagnosing and treating patients, with knowledge and experience in balancing complex medication regimens, allowing for  integration of the latest therapies.

When also trained as researchers, movement disorder specialists can use insights from their patients to inform studies toward improved understanding of disease and treatments.

With Parkinson’s becoming a growing problem — 12 million people are estimated to be affected worldwide by 2040 — care and continued research for this population is critical.

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