Episode 114: Gene-Based Therapies for Parkinson’s Disease
Gene-based therapies for Parkinson’s disease, while still in the developmental stage, are under active investigation. For this potential therapy, genes are engineered in a laboratory and then injected into specific parts of the brain. The genes may function either to induce cells in the brain to produce dopamine, or to code for the production of enzymes that then lead to the production of dopamine.
This episode is the second part of our conversation with Dr. Roger Barker, Professor of Clinical Neuroscience at the University of Cambridge in the United Kingdom. In our previous podcast with him, he discussed cell therapy for Parkinson’s. Here, he explains what gene-based therapy is, plans for the execution of the treatments, what symptoms they are aimed at, where the field currently stands, and how it compares to developments in cell-based therapies. As the field is still in the experimental stages, he again offers advice to people with Parkinson’s who are considering entering a clinical trial of gene-based or cell-based therapy and what they may expect in terms of symptom management and disease progression.
Released: October 5, 2021
Roger Barker, BA, MBBS, MRCP, PhD is the Professor of Clinical Neuroscience at the University of Cambridge and Consultant Neurologist at the Addenbrooke’s Hospital Cambridge. He is a PI in the MRC-Wellcome Stem Cell Institute in Cambridge and Director of the MRC funded UKRMP Stem and Engineered cell hub.
His research seeks to better define the clinical heterogeneity of two common neurodegenerative disorders of the CNS- namely Parkinson’s (PD) and Huntington’s disease (HD). This has helped him define the best way by which to take new therapies into the clinic including novel experimental therapeutics such as cell and gene therapies.
Episode 89: Retention Rates in Longer Clinical Studies
Clinical studies, studies that involve people, first use healthy people to test a drug’s safety and then use people with a disease or condition to prove that the drug works as intended. They are essential for bringing any new therapy to the public. Getting U.S. Food and Drug Administration approval for devices require rigorous studies, as well. Recruiting enough people to participate is often a long process, and for trials that may go on for a year or more, retaining people in the studies is often a challenge. People may get bored, find multiple study visits burdensome, have an adverse reaction to a drug being tested, move away, or drop out for a multitude of other reasons. If too many people discontinue the study, it will not have enough statistical ”power” to give a meaningful answer when the data are analyzed.
Christine Hunter, BSN, RN, Research Director of the Parkinson’s Disease Center and Movement Disorders Clinic at Baylor College of Medicine in Houston, a Parkinson’s Foundation Center of Excellence, describes how her center finds people with Parkinson’s disease who may want to participate in trials, what factors drive retention in trials, and ways to facilitate retention.
Released: September 8, 2020
Christine Hunter, RN, BSN is a registered nurse and has worked in ICU and CCU, charge nurse on the step-down cardiac unit, and in Quality Management for the Memorial Hospital System. She joined Baylor College of Medicine Parkinson Disease Center and Movement Disorders Clinic (PDCMDC) in 1996. She has been an active member of the Parkinson Study Group (PSG) as well as Huntington Study Group (HSG) during this time and has served in several different capacities within these groups, such as the Coordinator representative on the Executive committee, serves on the steering committee for an HD trial, served on the credentialing committee, and budget committee. She has extensive experience in Clinical Trials for all Movement Disorders. She has been the Parkinson Foundation Center Coordinator at Baylor College of Medicine since 1996 and an active member of the Task Force for the PF Mentoring and Networking Program.
When people take a prescribed drug, they rarely if ever consider how it came to be. They assume it is the right drug and will work safely as it is supposed to. But leading up to that drug being available is a long process of discovery or invention of the molecule based on knowledge of the biology it is supposed to affect, then testing in the laboratory and in animals, and several phases of testing in people to make sure that it is safe and effective. Each of these steps takes time and lots of financial investment. Even after a drug is approved by the U.S. Food and Drug Administration for sale and then put on the market, further study of the drug in a larger population than in the testing phases may be carried out. In this episode, Dr. Hubert Fernandez, Director of the Center for Neurological Restoration at the Cleveland Clinic in Ohio, a Parkinson’s Foundation Center of Excellence, describes the process of bringing a drug to market, including how many of them never make it. And he explains why people need to participate in the clinical trials and what they can expect when they do.
Released: August 25, 2020
Hubert H. Fernandez, MD, is Professor of Medicine (Neurology) at the Cleveland Clinic Lerner College of Medicine, Case Western Reserve University and the Head of Movement Disorders under the Center for Neurological Restoration at Cleveland Clinic in Cleveland, Ohio.
Dr Fernandez received both his BS in Biology and MD degree in the Philippines. He completed his internship in internal medicine at University of Pennsylvania/Pennsylvania Hospital in Philadelphia, Pennsylvania; his residency in neurology at Boston University Medical Center in Massachusetts; and his fellowship in movement disorders at Brown University in Rhode Island.
Dr. Fernandez is an internationally recognized expert in movement disorders who has been voted one of the Best Doctors in America by his peers. After completing his medical training, he joined the faculty of Brown University School of Medicine as Assistant Professor of the Department of Clinical Neurosciences and served as Associate Director of the Movement Disorders Unit and Neurological Director of its Functional Neurosurgical Program. In 2003, Dr Fernandez relocated to the University of Florida, where he eventually became Director of the Clinical Research Unit for Neurological and Psychiatric Disorders, Vice Chair of Academic Affairs, and Professor of Neurology prior to joining Cleveland Clinic. An active and productive researcher, he has initiated or participated in over 50 clinical trials and has published his findings in well over 300 articles and abstracts on Parkinson’s disease, cervical dystonia, blepharospasm, and other movement disorders. He has nearly 40 published book chapters and books to his credit, and has served on the editorial board of Movement Disorders and is currently an editorial board member of the American Journal of Clinical Neurology, European Neurological Journal, and Clinical Neuropharmacology.
Dr Fernandez is a fellow of the American Academy of Neurology, and a member of the American Neurological Association. He is currently elected as a Councilor for the AAN Movement Disorders Section, Executive Committee Member of the Parkinson Study Group and Dystonia Study Group; and is also an Executive Board Member of the World Neurology Foundation. He has served as President of the Florida Society of Neurology, and is the current Co-Medical Editor of the Movement Disorders Society Web site. Recently, he has been elected the Co-Chair of the Parkinson Study Group for a 6 year term.
Neuro Talk: Why Are More People Getting Diagnosed with Parkinson’s?
The number of people diagnosed with Parkinson’s disease (PD) in the U.S. recently increased by 50%, according to a new study. What do these results mean for the PD community?
In this Neuro Talk, Parkinson's Foundation Chief Scientific Officer James Beck, PhD, shares results from this groundbreaking study on Parkinson’s disease incidence. Dr. Beck also highlights how the new incidence number will impact PD research.
Episode 86: Personalized Medicine: The Voice of the Patient
Personalized medicine has garnered a lot of attention over the past decade. Usually it means determining the factors for each person that affect their health, their diseases, and potentially their treatments. Some examples are biomarkers that are found in their blood, their genetic make-up, diet and nutrition, behaviors, and environment. One example is the Parkinson’s Foundation’s PD GENEration initiative that offers free genetic testing and counseling for people with Parkinson’s disease (PD) to determine what genes and gene variants affect the course of their disease and response to treatments.
But despite all the scientific advances that allow these forms of personalized medicine, one crucial aspect of personalized medicine is the voice of the patient, both in each person’s encounters with the medical system and treatment team, as well as to inform the kinds of research that should be done and how to design and perform them. Dr. Bas Bloem, a professor of movement disorder neurology at Radboud University Medical Center in Nijmegen, the Netherlands, a Parkinson’s Foundation Center of Excellence, discusses how people with PD want to be heard and how a new definition of health may best put people’s disease into the overall context of their lives.
Released: July 28, 2020
Professor Bas Bloem is a consultant neurologist at the Department of Neurology, Radboud University Medical Centre, Nijmegen, The Netherlands. He received his medical degree, with honours, at Leiden University Medical Centre in 1993 and obtained his PhD degree in 1994. He trained as a neurologist between 1994 and 2000, also at Leiden University Medical Centre. He received additional training as a movement disorders specialist during fellowships at The Parkinson's Institute, Sunnyvale, California, and at the Institute of Neurology, Queen Square, London. In September 2008, he was appointed professor of neurology, with movement disorders as special area of interest.
Professor Bloem is on the editorial board for several national and international journals and has published over 750 publications, including more than 640 peer-reviewed international papers. This includes a series of large clinical trials, all of which were published in high-end scientific journals. He has also supervised 48 successfully completed PhD dissertations. His H-index is 74 (Publons) / 86 (Research Gate) / 97 (Google Scholar).
Professor Bloem is past president of the International Society for Gait and Postural Research. He recently became an Officer (secretary-elect) for the International Parkinson and Movement Disorder Society. From 2009 until September 2017, he was part of the board of ZonMw (The Netherlands Organisation for Health Research and Development). In 2011, he was elected the National Healthcare Hero by the Dutch Ministry of Health and Citizen of the Year for the city of Nijmegen in 2012. Since 2017, he has served on the Executive Scientific Advisory Board of The Michael J Fox Foundation for Parkinson’s Research. In 2018, he was elected as member of the “Koninklijke Hollandsche Maatschappij der Wetenschappen” (the Royal Holland Society of Sciences and Humanities), the oldest scientific society in the Netherlands. In 2018, he won the Tom Isaacs award as a recognition of his longstanding achievements in the field of Parkinson’s disease.
Also in 2018, he was elected as member of the Academia Europaea. In 2019, he was elected as Fellow of the Royal College of Physicians of Edinburgh. In 2020, he was elected as member of the Royal Netherlands Academy of Arts and Sciences. Also in 2020, he became co-Editor in Chief of the Journal of Parkinson’s Disease.
In 2002, Professor Bloem founded and became director of the Radboudumc Centre of Expertise for Parkinson & Movement Disorders, which was recognised from 2005 onwards as a centre of excellence for Parkinson’s disease. Together with Dr Marten Munneke, he also developed ParkinsonNet, an innovative healthcare concept that now consists of 70 professional networks for Parkinson’s disease patients, covering all of The Netherlands (www.parkinsonnet.nl).
Because of the evidence-based quality improvement and significant cost reduction, ParkinsonNet has received multiple awards, including the Best Pearl for Healthcare Innovation prize in 2011. In 2015, ParkinsonNet was awarded with the Value-Based Health Care Prize.
Professor Bloem has two main research interests: cerebral compensatory mechanisms, especially in the field of gait and balance; and healthcare innovation, aiming to develop and scientifically evaluate patient-centred collaborative care. He also values the publication of remarkable observations in single patients.
Expert Briefing: Can We Put the Brakes on Parkinson's Disease Progression?
April 6, 2022
Discovering the right treatments, interventions and support can enhance your quality of life with Parkinson’s disease (PD), but can it slow progression? Learn what the latest research is saying about slowing Parkinson’s progression including the most promising treatment and day-to-day adjustments. This program will also review how care and treatment strategies are adapted based on lessons learned from the Parkinson’s community.
Joash Lazarus, MD
Multiple Sclerosis Center of Atlanta
Videos & Webinars
Neuro Talk: Propelling a Future of New Parkinson’s Drugs Through Research
We know that research is an essential part of finding a cure for Parkinson’s disease (PD), but how can we speed up the process? In our latest Neuro Talk, Parkinson's Foundation Chief Scientific Officer James Beck, PhD, discusses the Venture Philanthropy Fund — our newest research-driven investment fund. Dr. Beck explains why Parkinson’s research is underfunded and shares how the Venture Philanthropy Fund is working to accelerate the breakthroughs in treatments that people with PD need today.
Episode 25: Meet the High School Student Who Is Changing Parkinson's Disease Diagnosis
There is no single, definitive test for Parkinson’s disease (PD). The diagnosis is made by an expert clinician who asks questions about a person’s health and medical history and observes their movement. But an enterprising high school student is working on a system that analyzes movements of facial muscles to make an early diagnosis and track Parkinson’s progression. Erin Smith of Shawnee Mission West High School in the Kansas City, Kansas area adapted a real-time facial expression recognition system to detect “facial masking,” a common Parkinson’s symptom caused by stiff facial muscles. Her system, called FacePrint, uses a web camera or smartphone to analyze facial movements and compare them to a database of people with and without Parkinson’s.
Released: March 27, 2018
Ms. Smith is the founder of FacePrint, a novel, telemedicine diagnostic tool for Parkinson’s disease. FacePrint uses facial recognition software and machine learning algorithms to detect early-stage differences in facial muscle movements. FacePrint has won numerous awards, including first prize in the Twitter #BUILTBYGIRLS Challenge. Her work has been featured in Forbes, Fortune and Seventeen Magazine. Ms. Smith has won top awards for her research at several international science competitions, including the Intel International Science and Engineering Fair and the International BioGENEius Challenge. She loves participating in hackathons and is the co-founder of KC STEMinists, which teaches middle and high school students how to use computer science to address societal issues. Ms. Smith is a senior at Shawnee Mission High School near Kansas City, Kansas. As she pursues higher education and beyond, she wants to continue to develop innovative healthcare technologies by combining her interests in neuroscience and computer science, transforming the way diseases are diagnosed and treated.
Episode 24: Donate Your Brain for Parkinson’s Research
Despite great advances in genetics and molecular biology, many aspects of research on diseases affecting the brain, including Parkinson’s, still depend on actual human brain tissue for study. How do researchers get these brains for study? They need you to become a brain donor! It is important for people with neurological diseases to donate their brains after death, but healthy brains are also needed for comparison. Brain retrieval must occur very quickly to be useful, so advance planning is essential. Tish Hevel, founder and CEO of the Brain Donation Project, explains how to become a brain donor, and she dispels some misperceptions about the subject.
Released: February 27, 2018
Ms. Hevel is founder and CEO of The Brain Donor Project, an innovative not-for-profit that exclusively supports the NeuroBioBank of the National Institutes of Health (NIH) by raising awareness of the critical need for donated brain tissue and by simplifying the process to donate. Since its launch in late 2016, more than 1,300 people have signed up to become brain donors, representing all 50 states and more than 60 categories of brain diseases and disorders. Ms. Hevel began her career as a journalist, managing television newsrooms in Cincinnati, Dayton and Detroit. She later developed a communications consultancy providing strategy, content development, media coaching and relations, executive briefings, speech writing and other PR services. Her work included a month-long assignment in London on behalf of a worldwide sponsor at the London 2012 Olympic Games. She also served as Chief Communications Officer for a 42-county region of the American Red Cross. She is a mother of two sons and one dog and lives in Liberty Township, Ohio.
Episode 113: Cell-Based Therapies for Parkinson’s Disease
Researchers have been investigating cell-based therapies for treating Parkinson’s disease (PD) for some time now, using a variety of materials and methods. Two approaches have been to implant dopamine-producing cells or to induce cells already in the brain to become dopamine-producers. We asked Dr. Roger Barker, Professor of Clinical Neuroscience at the University of Cambridge in the United Kingdom, to summarize what has been learned so far and based on that, to give his perspective on where the field may be headed. The field is still in the experimental stages, and he cautions about what people with PD should ask when considering entering a clinical trial of cell-based therapy and what they may expect in terms of symptom management and disease progression.
Released: September 21, 2021
Roger Barker, BA, MBBS, MRCP, PhD is the Professor of Clinical Neuroscience at the University of Cambridge and Consultant Neurologist at the Addenbrooke’s Hospital Cambridge. He is a PI in the MRC-Wellcome Stem Cell Institute in Cambridge and Director of the MRC funded UKRMP Stem and Engineered cell hub.
His research seeks to better define the clinical heterogeneity of two common neurodegenerative disorders of the CNS- namely Parkinson’s (PD) and Huntington’s disease (HD). This has helped him define the best way by which to take new therapies into the clinic including novel experimental therapeutics such as cell and gene therapies.
