4^th World Congress on Parkinsons & Huntington Disease August 29-30, 2018 Zurich, Switzerland

Biography:

Sebastian Knöbel is a trained Biologist and received his PhD at the RWTH Aachen University in 2006. He joined the Miltenyi R&D Department as Research Scientist in 2007.  In his current position as Manager, he heads the Media Development Group and Research Groups for pluripotent, mesenchymal stem cells generating tools and protocols for the isolation, culture and modulation of cells for research and translational applications. His current research includes many aspects of regenerative cell therapy with a focus on the manufacturing of pluripotent stem cell derived cell types.

Abstract:

Pluripotent stem cell (PSC) derived cell products hold great promise for future clinical use in a variety of indications like type i diabetes, cardiomyopathies, macular dystrophies and Parkinson’s disease. For Parkinson’s disease trials, grafted human fetal tissue has indicated utility of tissue or cell replacement therapy. However, the limited availability and ethical implications connected to using primary fetal material have inspired pluripotent stem cell (PSC) based approaches. In vitro differentiated mesencephalic dopaminergic (mesDA) progenitor cells grafted into the striatum have proven to, revert motor symptoms in pre-clinical animal models of Parkinson’s disease compensating for the loss of dopamine producing cells in the substantia nigra. Raising regulatory requirements for such advanced-therapy medicinal products (ATMPs) imply the need for standardized reagents and highly reproducible manufacturing procedures. Automation of PSC expansion, differentiation, and potential product optimization through cell sorting may contribute to successful and cost-effective innovative therapies. Using the versatile and integrated GMP cell processing platform, CliniMACS Prodigy®, we previously developed a cultivation and expansion workflow for PSCs. Now we have adapted the lab protocol for the differentiation to mesDA progenitor cells and have transferred it onto the device, for a medium-scale prototype manufacturing process within the closed system. Extrapolating the cell numbers retrieved from the protoype process would correspond to 150–250 patient doses per manufactured batch. To characterize the identity of the resulting progenitors, we have designed a concise marker panel for flow cytometry-based quality control (QC). Taken together, we have developed a method for adherent, closed-system cultivation of PSCs and differentiation to dopaminergic progenitor cells in combination with comprehensive QC assays.

Biography:

Anita Haahr—RN, MScN, PHD—is a Senior Lecturer at the School of Nursing, Aarhus, Denmark and an Adjunct Lecturer at the Department of Health, section for Nursing, Aarhus University, Denmark. The focus of her research is living and coping with life with PD from a patients and spouses perspective. She has done research within this area for more than a decade and has published several papers on the subject.

Abstract:

Parkinson’s disease (PD) is the second most common chronic progressive neurodegenerative disease affecting more than 10 million people worldwide. The main features of PD are tremor, bradykinesia, rigidity and at the later stage, postural instability. As the disease progresses, non-motor symptoms such as neuropsychiatric symptoms, depression, anxiety, cognitive challenges, sleep disturbances and fatigue may also appear. Thus, living with PD strongly affects daily living and as the disease progresses, individuals living with PD may experience the disease as limiting on a daily basis, requiring the development of individual ways of coping with the disease to maintain an active everyday life. The aim of the study was to identify coping in daily life in individuals with PD as it is described in the existing literature, ultimately to be able to describe coping patterns and challenges, in order to uncover the needs of the individual living with PD, to support their efforts in managing daily life with the disease. In this presentation, we unfold the results of a literature review performed as a meta-ethnographic metasummary and metasynthesis. A thorough literature search was conducted and adhering to the JBI guidelines, eligible studies were assessed for quality. A total of 13 articles were included in the study. Preliminary results shows the overarching motivation for coping with PD to be “striving for normalcy” and “preserving the self” from before PD. These findings will be unfolded at the presentation.

Biography:

Sana A. AlBustan is a young researcher  in the Department of Communication Disorders Sciences, College of Life Sciences, Kuwait University. She earned her Ph.D from the Department of Speech, Language and Hearing Sciences, College of Public Health and Health Professions, University of Florida. Her clinical internship and practicums were in Florida hospitals, public schools and private clinics. In May,2017, Dr. AlBustan  published “Kuwaiti Teachers' Perceptions of Voice Handicap” in the Journal of Voice . In December 2017,  she published” Towards a Standard Arabic System Usability Scale: Psychometric Evaluation using Communication Disorder App” in the International Journal of Human–Computer Interaction

Abstract:

The intended study would shed a light on Parkinson Disease (PD)in Kuwait. This study will represent the levels of awareness and knowledge among general people about PD that are currently not very well documented in Kuwait. A questionnaire will be developed  for this current study by the researcher that would consist of a structured item questionnaire that would compose of both open-ended and close-ended questions would be given to 50 males and  50 females after a committee of professionals in  the field approve the questionnaire. Questions will be devolved to address  various aspects of the  definition PD,  onset of PD, cause, treatment, and hereditariness Demographic data with participants demographic features such as age, gender, occupations, qualifications and educational level will also be considered and addressed in this research. SPSS analysis procedures will be used to examine and analyze the participants responses The study is still in its pre implementation stage progress. The researcher anticipates her results will be consistent with other researchers findings. The primary goal of this study is  to investigate the level of public awareness and knowledge of PD and facilitate the level of awareness and the services offered by governmental and private medical facilities in the Kuwait

Biography:

Jae Moon Lee completed his PhD from Duke University and Post-doctorate from Duke University School of Medicine. He is the VP of Kainos Medicine, a clinical stage Korean biotech company. He has published more than 15 papers in reputed journals.

Abstract:

Current standard of care for Parkinson’s disease is symptomatic treatments by supplementing dopamine or dopamine agonists or analogous mechanisms, and the disease modifying treatment is one of the major unmedical needs to block the progression. KM-819 is an orally active small molecule drug developed as an inhibitor for FAF1, a proapoptotic protein, targeting various degenerative diseases. It has shown superior efficacy of neuroprotection in cell models and of dopaminergic neuron protection in midbrain in various animal models of Parkinson’s disease as well as improvement of behavioral tests, suggesting this drug has potential capability of slowing or stopping the progression of the disease. It has also shown inhibition of alpha-synuclein accumulation in cells. We have completed Phase 1 clinical trial for KM-819, randomized, double-blind, placebo-controlled study. The study is divided into part A (single ascending dose) and part B (multiple ascending dose) for evaluation of safety, tolerability, and pharmacokinetics as well as various pharmacodynamics markers for KM-819 in healthy volunteers. The study results showed no drug-related serious AEs and high safety profile in human. Also, the PK study showed dose-proportional exposure with higher in elderly group, ideal for Parkinson’s drug. We are currently planning for phase 2 in patients focusing on investigation of the drug’s efficacy of slowing down or halting the progression of the disease.

Biography:

Jae Moon Lee completed his PhD from Duke University and Post-doctorate from Duke University School of Medicine. He is the VP of Kainos Medicine, a clinical stage Korean biotech company. He has published more than 15 papers in reputed journals.

Abstract:

Current standard of care for Parkinson’s disease is symptomatic treatments by supplementing dopamine or dopamine agonists or analogous mechanisms, and the disease modifying treatment is one of the major unmedical needs to block the progression. KM-819 is an orally active small molecule drug developed as an inhibitor for FAF1, a proapoptotic protein, targeting various degenerative diseases. It has shown superior efficacy of neuroprotection in cell models and of dopaminergic neuron protection in midbrain in various animal models of Parkinson’s disease as well as improvement of behavioral tests, suggesting this drug has potential capability of slowing or stopping the progression of the disease. It has also shown inhibition of alpha-synuclein accumulation in cells. We have completed Phase 1 clinical trial for KM-819, randomized, double-blind, placebo-controlled study. The study is divided into part A (single ascending dose) and part B (multiple ascending dose) for evaluation of safety, tolerability, and pharmacokinetics as well as various pharmacodynamics markers for KM-819 in healthy volunteers. The study results showed no drug-related serious AEs and high safety profile in human. Also, the PK study showed dose-proportional exposure with higher in elderly group, ideal for Parkinson’s drug. We are currently planning for phase 2 in patients focusing on investigation of the drug’s efficacy of slowing down or halting the progression of the disease.

Biography:

Abstract:

This study intends to explore the challenges faced by teachers and parents of children with epilepsy during school years. It also aims to provide useful information about the parent-teacher collaboration for the benefit of the pupils, the families and the school.

Methods: Telephone interviews were conducted based on open-ended questions, with 70 head-teachers from schools from all Greek regions. Furthermore a questionnaire was distributed to 100 Greek schools. Additionally, personal interviews took place with 91 parents of children with controlled epilepsy during their school years. The data were grouped and analyzed with the use of qualitative and quantitative analysis.

Results: 89.9% of the school staff was found to know what epilepsy is. 85.1% of the teachers were informed about pupils’ epilepsy by their parents/caregivers while 63/70 head-teachers declared that parents usually misinform the school staff about their child’s condition. 76/91 parents/caregivers personally informed only the head-teacher about their child’s condition because of fear of social stigma and bullying. 87/91 parents declared that they did not know where to seek help to cope with their child's illness apart from their doctor. 92.6% of the teachers felt insufficiently trained to deal with a seizure and 96.2% expressed the need for more formal information about epilepsy. 64/70 head-teachers and all the parents/caregivers expressed the need for inclusion of experts like social workers and nurses into the school personnel.

Conclusion: Epilepsy is a condition that affects not only the pupil with the disorder but also his/her family as well as the school staff. For the school personnel, it is very important to cooperate with the pupils’ parents/caregivers in order to handle the child’s condition properly and safely. The doctor’s guidance to the parents is crucial for their communication with the school staff.

Biography:

With a corporate finance background, after her successful temporal lobectomy in 2013 and continued recovery from psychological diagnoses, Torie has became an active international public speaker and corporate consultant regarding epilepsy, disability, mental health, diversity and inclusion.

Her purposes are to enlighten the uneducated, reduce the stigma held against those with disabilities (particularly epilepsy), and hold organisations accountable for corporate social responsibility when it comes to employment.

Featuring on UK BBC London, 5Live, talkRadio, US Brain Waves Audio and Australian Noongar Radio, Torie’s BBC3 YouTube video in which she featured: “Things Not to Say to Someone with Epilepsy” has been viewed more than half a million times.

Featuring neurological professionals, patients and employers from all over the world, Torie:

• Both founded and frequently writes for both the torierobinson.com and epilepsysparks.com blogs;
• Co-founded the Cheeky Sparks epilepsy podcast;
• Filmed for NHS70
• Works with DWP (UK Government) regarding disability employment;
• Has attended meetings within UK Parliament;
• Is an Epilepsy Action Accredited Volunteer (Trainer), and;
• Has featured in Huffington Post

Torie plays the piano, enjoys communicating to overseas audiences and forever learning more about neurology and cosmology.

Born in the UK, Torie grew up in both England and Australia and has worked for international firms with staff in Europe, Australia, the US and Asia. Torie lives in London with her partner.

Abstract:

Patient in early 30s with refractory, left temporal lobe epilepsy in a setting of left hippocampal sclerosis. Increase in seizure severity and freqency over 25 years, with worsening depression. Patient working in corporate environment.

Aim:

To decrease frequency and/or severity of seizures; increase quality of life and life expectancy

Treatment:

Likely Cause of Epilepsy:

• Lengthy febrile seizure aged 6months

Past AEDs tried:

• Sodium Valproate, Levetiracetam, Phenytoin

AED side effects experienced:

• Memory function decrease, fatigue, numbness (phenytoin), mood instability (Levetiracetam)

Seizure triggers:

• Sleep deprevation, anxiety, forgetting AEDs

Further Diagnosis Related to Epilepsy/AEDs

Surgery Exploration Testing:

• Video Telemetry
• EEG
• fMRI
• Psychological evalutation/neuropsychology assessment inc IQ
• Full understanding of surgery risks and potential outcomes by patient

Surgical Procedure:

• Left Temporal Lobectomy, Feb 2013
• CSF leak followed, leading to suture of the wound

Post-operative Diagnoses Included:

• Severe depression, extreme problems with memory, and exhaustion (for 6-12months)
• Blind spot in right eye
• 5 tonic-clonic seizures post surgery, last in November 2017
• Irregular complex partial seizures (average 4/5 per year)

Current Drugs:

• Levothyroxine 50mg QD
• Lamotrigine 100mg BD
• Lacosamide 100mg BD
• Venlafaxine 150 BD
• Clobazam 10mg

Conclusion:

Status: outcome Engel class 2

Patient has experienced significant decrease in number and severity of seizures. Along with psychological therapy, patient’s quality of life is greatly improved. Despite continued taking of AEDs, and infrequent seizures, the results of surgery is considered to be a success by patient. Patient has greater focus, continues to travel and has supportive partner and friends.

Biography:

Elaine Wyllie, Professor, Cleveland Clinic Lerner College of Medicine, is a world renowned thought leader in neurology and epilepsy. Dr. Wyllie has been on the Castle Connolly list of America’s Best Doctors for many years, as well as on several other national and regional Top Doctors lists. Her many honors and awards include the highly competitive Award for Outstanding Work in Epilepsy Research from the American Epilepsy Society and Honorary Membership in the Canadian Association for Child Neurology.

Abstract:

New research in pediatric epilepsy surgery is providing opportunities to help more children than ever before.   Some of our most exciting results have been in children with early focal brain lesions and diffuse EEG abnormalities.  The diffuse findings on EEG reflect the evolution of the epilepsy as the early focal lesion, usually cerebral infarction or malformation of cortical development, interacts with the brain at different stages of development.  Infants with focal lesions tend to manifest with hypsarrhythmia, and the older children tend to manifest with slow spike wave complexes and other patterns, but in both age groups the epilepsy typically disappears when the lesion is removed.  Wyllie and colleagues studied 209 children with an early focal lesion who underwent epilepsy surgery, and found no significant difference in seizure outcome based on presence or abundance of generalized epileptiform discharges and EEG seizures.

A second exciting new opportunity for pediatric epilepsy surgery has emerged for children with bilateral abnormalities on brain MRI. Hallbook and colleagues reviewed preoperative MRIs in 110 children who underwent hemispherectomy at Cleveland Clinic, and found abnormalities on the contralateral side in 74%.  In a follow up study of 170 children who underwent hemispherectomy, Moosa and colleagues found that contralateral MRI findings had no significant impact on the frequency of seizure-free outcome among Cleveland Clinic’s highly selected cases.  The contralateral MRI abnormalities in these children, although not insignificant, were always less extensive and less prominent than those on the side of hemispherectomy. 

Research suggests that for patients of all ages, shorter epilepsy duration may positively affect postoperative seizure outcome.  By recognizing surgical opportunity and shortening the delay, we can help more children than ever before.