MSA MEETING LEUVEN (Belgium)

Two MSA research teams at World MSA Day event @ HistArUz Leuven, Belgium Monday October 3, 2016, HistArUz, Capucijnenvoer 35, Leuven, Belgium Room: KAPEL Firstfloor.

Organized by The Belgian Patients Support Group: MSA-AMS BE

MSA Meeting: Voor Patiënten, Mantelzorgers en Familieleden HistArUZ Capucijnenvoer 35, 3000 Leuven Maandag 3 oktober 2016 Lokaal KAPEL 1ste verdiep Georganiseerd door MSA-AMS BE vzw Patiënts Support Group

PROGRAM

Organizing committee …………………………………………………………………………………………………………………… 3

Program of the Day …………………………………………………………………………………………………………………… 4

JiePie AWARD 2016 …………………………………………………………………………………………………..……………… 5

Who is Who ………………………………………………………………………………………………..………………… 6

Veerle Baekelandt ……………………………………………………..…………… 6

Janice Holton ………………….…………………………………………..…………… 7

Marc Dooms …………….……………………………………………………………… 9

Erik Tambuyzer …………………………………………….……………… 10

Ritje Schouppe-Moons …………………………………………………………… 11

Jan Jambon, …………………………………………………………… 12

Maps & Directions to the venue: …………………………………………………… …………………………………………..……………… 13 HOTEL Accomodations nearby: ………………………………………………………………………………………………..……………… 14 Sophia Fund: ………………………………………………………………………………………………..……………… 15

MSA is like the three flavoured Neapolitan ice cream. By Pam Bower – JiePie Award for MSA Awareness Ambassador 2012: ……………………………………………………………………………………………………..……… 17

MSA-AMS.be Leuven 2016

MSA organizing committee

Ritje Schouppe-Moons, Chairwomen of The Board of Directors MSA– AMS BE vzw

Dr. Veerle Baekelandt Professor faculty of medicine KU Leuven and project leader (BE Team)

Dr. Janice Holton, Director of Neuropathology Queen Square Brain Bank and project leader, (UK Team)

Marc Dooms, Senior orphan drugs specialist, Katholieke Universiteit Leuven, Belgium, and member of he Fund for rare diseases and orphan drug of the King Baudouin Foundation.

Meet and Greet:

2 MSA research teams

at World MSA Day event @ UZLeuven, Belgium

The 2 winning teams of the Sophia MSA research Grand / Fund,

will join the Meet & Greet session.

The venue at the University of Leuven - visit @ the laboratories and

an update on the on-going research.

MSA-AMS.be Leuven 2016

PROGRAM

08:45 – 09:00: Registration participants– and Welcome Coffee

Morning Chair: Erik Tambuyzer PhD

09:00 - 09:15: Opening: Ritje SCHOUPPE-MOONS, Chairwomen of The Board of Directors MSA–AMS BE vzw

Meet and Greet : 09:15 - 09:45: Dr. Veerle Bakelandt, BE Team Leader Sophia Fund

09:45 – 10:45: Prof Dr. Janice Holton, UK Team Leader Sophia Fund

10:15 - 10:45: Coffee break

10:45 – 12:00: Visit research area and Discussion, at the Fifth Floor": Laboratory for Neuroscience and Gentherapy

12:00 – 13:30: Welcome to the MSA Patients, Caregivers, and Family Members … Walking Lunch all together with the 2 teams

Afternoon Chair: Marc Dooms

13:30 - 14:00: What is MSA and latest news about European MSA Research: Update

14:00 – 14:30: Q & A: Patients / Medic

14:30 – 14:45: JiePie Award 2016

14:45 – 15:45: Guided Tour @ the Museum : One History, 1000 Stories & Rare diseases and orphan drugs: 500 years ago. "first floor" with Marc Dooms (Eng) Dominique Boogaerts (FR) (Nl)

15:45 – 16:00: Closing Remark

16:00 – 16:30: Reception

MSA-AMS.be Leuven 2016 JiePie Award 2016

JiePie Award organising committee,

Mr. Jan Jambon, Deputy Prime Minister and Minister of Security and Interior Affairs in Belgium. We are privileged that Mr. Jan Jambon is godfather from MSA-AMS BE vzw since our first JiePie Award celebration in 2011.

Ms. Ritje Schouppe-Moons, Chairwomen of The Board of Directors MSA –AMS BE vzw

Ms. Pam Bower, Board of Directors - Secretary, The Multiple System Atrophy (MSA) Coalition, USA

Ms. Lesley Denys, Board Member of MSA-AMS BE vzw

Nominees for the JiePie Award 2016 for MSA Awareness Ambassador:

"I HAVE A DREAM....CURE MSA” "J'AI FAIT UN RÊVE, GUÉRIR L'AMS" “IK HEB EEN DROOM, MSA GENEZEN!“

JiePie Award 2016 for MSA Awareness Ambassador

PRESENTED TO

- Robert (Rob) TIMMERMANS, Drongen, Belgium

- Lea Koolmees – Demper, NL

MSA-AMS.be Leuven 2016 Who is Who:

Veerle Baekelandt PhD: Professor at Laboratory for Neurobiology and Gene Therapy, KU Leuven

Veerle Baekelandt, PhD, obtained a degree of Master of Romance Languages (1987), Master of Biology (1991) and a PhD in Biology (1995) at the Katholieke Universiteit Leuven. In 1992 she received a Frank Boas Fulbright scholarship for graduate study at Harvard University and became a research fellow in the Laboratory for Neuroscience Research headed by Dr. Larry Benowitz, Children’s Hospital, Harvard Medical School, Boston (1992-1993). In 1999 she joined as a post-doctoral fellow a new gene therapy project for neurodegenerative diseases, which was the start of her own research group. In 2003 she was appointed as assistant professor at the KU Leuven and in 2007 as full-time research professor (BOF-ZAP). She is now head of the Laboratory for Neurobiology and Gene Therapy. Her research focuses on disease modeling and therapy for Parkinson’s disease using viral vectors in cell culture and in vivo. The underlying rationale is that the generation of more relevant models in cells and in pre-clinical model brain will lead to a better insight into the molecular pathogenesis of PD and to the development of new therapeutic strategies and drugs.

Professor faculty of medicine KU Leuven and project leader.(Belgian team) “It is my ultimate dream that our research will contribute, even at long term, to an effective therapy for patients with MSA or Parkinson’s disease”

Research Group for Neurobiology and Gene Therapy - KU Leuven Kapucijnenvoer 33 blok i - bus 7001 – 3000 Leuven +32 (0)472 [email protected]

120.000 € (Sophia Fund)

Dr. Veerle Baekelandt, and her Team will join the meeting

MSA-AMS.be Leuven 2016 Prof Janice Holton, Honorary Consultant and Professor in Neuropathology

Director of Neuropathology Queen Square Brain Bank and project leader, (UK Team) “Multiple system atrophy is a devastating incurable disease affecting people in the prime of their lives. My research goals are to improve understanding of the disease mechanisms leading to treatment development and a positive impact on the lives of MSA patients and their caregivers”

Janice Holton, Director of Neuropathology Queen Square Brain Bank for Neurological Disorders, dept of Molecular NeuroscienceUCL Institute of Neurology, 1 Wakefield Street - London WC1N 1PJ + 44(0)[email protected] 120.000 € (Sophia Fund)

Dr. Janice Holton, Professor of Neuropathology, Dr Aoife Kiely and Dr Sandrine Wauters (UK Team) will join the meeting. Looking forward to the opportunity to visit Leuven and meet others working on MSA.

Honorary Consultant and Professor in Neuropathology Prof Holton participates in the routine work of the Division of Neuropathology reporting neurosurgical specimens as part of a team of 6 Consultant Neuropathologists and has responsibility for the adult muscle biopsy diagnostic service.

Prof Holton is actively engaged in research in the field of inflammatory muscle diseases and has recently written a book chapter on the subject. She is a founder member of the Council of the British Myology Society.

MSA-AMS.be Leuven 2016

Queen Square Brain Bank

The Queen Square Brain Bank (QSBB) houses one of the largest neurodegenerative brain tissue resources in the UK. This unique collection consists of cases of Parkinson’s disease, hereditary forms of parkinsonism and cases presenting with an atypical parkinsonian syndrome such as progressive supranuclear palsy, corticobasal degeneration and multiple system atrophy. Since 2002 a prospective collection of brain tissue from well-documented early onset and familial dementia cases has been added to the portfolio. Approximately 90 cases are received each year and all are subject to an extensive diagnostic process and a full report is issued by either Dr Holton or Professor Revesz. Following this, cases are made available to researchers for study. Dr Holton is actively involved in neuropathological research in the fields of multiple system atrophy, Parkinson’s disease and dystonia.

National Committees Prof Holton is an active member of the British Neuropathological Society and has held the offices of Treasurer and Chair of the Education and Meetings Committee. In the latter role she was responsible for the organization of the annual meeting of the Society including the preparation of the scientific programme. She also organized two summer schools on behalf of the Society.

Editorial commitments Prof Holton has been a member of the editorial board of Neuropathology and Applied Neurobiology, the journal of the British Neuropathological Society since 2006 and has acted as an Executive Editor since January 2012

MSA-AMS.be Leuven 2016

Marc Dooms : Lecture: Rare diseases and orphan drugs: 500 years ago

Mr. Dooms (Pharm D) is Senior Orphan Drug Pharmacist at the University Hospitals Leuven. He is compounding/dispensing pharmacist in First in Men Randomized Clinical Trials. He has been a member of the Belgian Order of Pharmaceutical Sciences and the Flemish Society of Hospital Pharmacists (VZA) since 1975 and the Belgian representative to the European Union of Experts in Rare Diseases with frequent collaboration with Orphanet, the European Society of Clinical Pharmacy, and the American Society of Health Care Pharmacists, among others, since 2000.

Marc Dooms received his pharmacist diploma at the Catholic University of Leuven, where he graduated in 1973. In 1974, he was pharmacy teaching studies certificated at the Catholic University of Leuven, and in 1975 pursued an internship in Clinical Pharmacy at St John's University and Columbia University, New York, with a special interest in orphan drugs, compounding of topical therapy and First in Men randomized clinical trials.

Mr. Dooms is founding member of the Belgian National Board on Orphan Drugs within the King Baudouin Foundation and is a graduate-level teacher in dermatological compounding, drug adherence, and orphan drugs, since 2010. He is also teaching pharmaceutical technicians at Leuven Syntra School. He has co-authored a number of publications on orphan drugs.

MSA-AMS.be Leuven 2016 Erik Tambuyzer, PhD - Morning Chair

Dr Tambuyzer has been an innovator in medical products development since the early 80’s. In 1985, he co-founded and managed Innogenetics NV/SA, a biotechnology company later acquired by Solvay Pharma, subsequently acquired by Abbott and then Fujirebio. In 1992, he joined Genzyme Corp. as its Vice-President Europe, Diagnostics and Genetics. From 1996, as Genzyme’s Senior Vice-President, Corporate Affairs Europe, Dr Tambuyzer was the industry point person in the discussions with the European Commission and the Parliament on the European Orphan Medicinal Products Regulation. Erik was a founding Board member and Vice-Chairperson of EPPOSI, the European Platform for Patients’ Organizations, Science & Industry. He was a founding Board member and Chair of EuropaBio, the European Association for Bio-industries and set up and chaired their Ethics Working Group and Rare Diseases and Orphan Drugs Task Force (across industry associations); he received the first EuropaBio Award of Merit in 2011. For 6 years, Dr. Tambuyzer has been the Chair of the Center for Medical Innovation (CMI), a non-profit foundation in Flanders, Belgium, working on translational biomedical research and biobanking. He is a member of the Belgian national advisory committee on rare diseases and orphan drugs, a Fund within the King Baudouin Foundation which he co-founded in 2006. Currently he is co-founder and Chair of the unique international organization BioPontis Alliance for Rare Diseases (http://biopontisalliance.org ), a non-profit whose mission is to identify and fund, according to strict selection criteria, the most promising science in some rare neurological diseases. Biopontis industry and regulatory experts lead the selected preclinical therapy discovery & development programs, to transform the science into potential therapies. The first program has been started with VIB/University of Antwerp on Charcot- Marie-Tooth disease.

MSA-AMS.be Leuven 2016

Mr. Jan Jambon, We are privileged that Mr. Jan Jambon is the godfather from MSA-AMS BE vzw since our first JiePie Award celebration in 2011

Mr. Jan Jambon, is born on 26 April 1960 in Genk and living in Brasschaat. Married and father of four children and one grandchild. He is a Flemish politician, as well as a member of the N-VA. On 11 October 2014, he became Vice-Premier and Minister of Security and Home Affairs in Belgium.

Career in the business sector, among others at IBM, SD Worx and the Bank Card Company (1984–2007) Political functions: Member of the Local Board and Deputy Mayor of Brasschaat (2007-2013) Mayor of Brasschaat (2013-2014) House of Representatives (2007-2014) Party leader at the House of Representatives (2008-2014)

Sinds 2011 is Jan Jambon een heel enthousiaste en geëngageerde peter van de MSA patiënten vereniging

Jan Jambon, is geboren op 26 april 1960 te Genk en woonachtig te Brasschaat. Gehuwd en vader van vier kinderen en één kleinzoon. Hij is Vlaams politicus, lid van de N-VA. Op 11 oktober 2014 werd hij Vice-eersteminister en minister van Veiligheid en Binnenlandse zaken

Carrière in de bedrijfswereld bij o.a. IBM, SD Worx en Bank Card Company (1984–2007) Partijpolitieke functies: Gemeenteraadslid en Schepen te Brasschaat (2007-2013) Burgemeester Brasschaat (2013-2014) Kamer van Volksvertegenwoordigers (2007-2014) Fractieleider Kamer van Volksvertegenwoordigers (2008-2014)

MSA-AMS.be Leuven 2016

Ritje SCHOUPPE-MOONS

Voorzitster Raad van Bestuur van MSA-AMS.be vzw vertegenwoordiging van de MSA patiënten vereniging in de Medische Advies Raad.

Samen met JiePie startte zij met informatie te verschaffen naar ander MSA patiënten. Een eerste nieuwsbrief zag het levenslicht op 2 februari 2008. 14 december 2008 startte ze samen met JiePie de MSA-AMS.be patiënte vereniging (feitelijke vereniging). April 2009 was de website een feit. De ervaring als jarenlange mantelzorger is de ideale basis om als een ‘professionele’ vrijwilliger de MSA patiënten en hun familie te helpen. Zij houdt ook de vinger aan de pols en zal niet nalaten alles in het werk te stellen opdat het onderzoek naar het hoe en waarom van MSA te stimuleren. Ze is mede, een van de sturende krachten achter de ‘World MSA Day’. Zij is nu voorzitster van de Raad van Bestuur van MSA-AMS.be vzw. En houd zich bezig met het dagelijks bestuur.

President Board of Directors of MSA AMS.be vzw and representing the MSA patients association within the Medical Advisory Board.

Together with JiePie she started to provide information to other MSA patients. The first newsletter was born on February 2, 2008. December 14, 2008 she started together with the MSA JiePie AMS.be patient association (factual association). April 2009 the website was a fact. The years of experience as a caregiver, is an ideal basis for a 'professional' volunteer to help the MSA patients and their families. She also keeps the finger on the pulse and will not fail to make every effort to ensure that the investigation into the ‘how and why’ of MSA is continuing. She is also one of the driving forces behind the World MSA Day. She is now President of the Board of Directors of MSA AMS.be association, and deal with the daily management.

MSA-AMS.be Leuven 2016 "United to Defeat Multiple System Atrophy"

• The Multiple System Atrophy (MSA) Coalition (USA)

• MSA-AMS be vzw, (BE).

• The Multiple System Atrophy Trust (UK)

• MSA Shoe USA/CANADA

• EURORDIS: The Rare Disease Team for Multiple System Atrophy Community

• Lid van RaDiOrg (BE)

• Vlaams Patiëntenplatform (VL)

• http://www.goededoelen.be/organisation/?id=083144143

------Beverages will be provided throughout the day. ***IF anyone in your party requires a special diet, you must let us know specifically what your dietary requirements are when you make your reservation so we can give the food supervisor notice of the special needs.

Attendees are responsible for their own travel and hotel expenses. Registration is now open for the 2016 Conference

See more at: www.msa-ams.be / MSA DAY 2016

The conference is free to all attendees. Please consider making a donation to help us offset the cost of the conference. Suggested donation per attendee: €40. (You will receive an invoice for this) Donate Now : MSA-AMS.be vzw Sprinkhaanveldestraat, 36 - 2150 BORSBEEK (Belgium) Payments only via: Special account for research - MSA Research KBC: BE17 7380 4238 8921 BIC: KREDBEBB Stating "3/10 Leuven, + your name and number of seats."

MSA-AMS.be Leuven 2016

MAPS & DIRECTIONS TO THE VENUE:

MSA-AMS.be Leuven 2016

HOTEL ACCOMODATIONS NEARBY:

Martin's Klooster : Onze-Lieve-Vrouwstraat 18 3000 Leuven - +32 16 21 31 41 - [email protected] (http://www.martinshotels.com/en/hotel/klooster-hotel)

Martin's Klooster is a four-star hotel that's beautifully located in the historical center of Leuven, less than 30 minutes from Brussels. You can enjoy an exceptional stay in a listed building, elegantly equipped with the most modern comforts Martin's Klooster is an exceptional part of Leuven's heritage, with its steeple, porte-cochère, gable, casement windows, shutters and oaken beams. Every guest room has its own singular style and emanates serenity and elegance. But Martin's Klooster is more than just a four-star hotel. The banks of the Dijle and its outdoor cafés and beautifully laid-out gardens make for great leisurely relaxing. It's the perfect base of operations to explore Leuven: the university city, the historical sites, the rich culture and the savoir- vivre that defines it, especially when the sun is out. If you're looking for an exceptional location for your seminars and meetings, you've come to the right place. Martin's Klooster boasts a conference center and three meeting rooms with the latest in technology and all the necessary comforts.

Cube Hostel : BRUSSELSESTRAAT 110, B-3000 LEUVEN +32 16 89 45 85 - [email protected] RECEPTION OPEN 9AM - 12PM AND 3PM- 7PM - AVAILABLE 24/7 Cube is a boutique style hostel in the very heart of Leuven. Newly renovated – fully equipped – retro/vintage - for the full Leuven- experience! : ( https://www.cubehostel.be ) IBIS hotel Brusselsestraat 52, 3000 LEUVEN Tel. (+32)16293111 Gelegen in het stadscentrum.. (http://www.accorhotels.com/nl/hotel- 1457-ibis-leuven-centrum/index.shtml )

Toegang met het openbaar vervoer: De Lijn: Bus 14 tot halte Brusselsestraat Treinstation LEUVEN tot Ibis hotel: (1.20 km / 0.75 mi) Luchthaven / wagen BRUXELLES NATIONAL (25.40 km / 15.78 mi) Vanuit Brussel: volg op de E40 de borden naar "Luik, Leuven". Neem de afrit "Leuven-Hasselt-Genk" en vervolgens de afrit "Leuven". Volg de borden naar "centrum" tot u bij de afslag "Kapucijnenvoer/Brusselsestraat" komt. Ga hier naar rechts, het hotel ligt na 200 m aan de linkerkant

MSA-AMS.be Leuven 2016 Fund Sophia

Research on Multiple System Atrophy and related diseases

Fund Sophia was created to support medical scientific research on neurodegenerative diseases. Priority will be given to research projects with focus on Multiple System Atrophy (MSA) or related diseases. Subject of the call 2015:

Medical scientific research on multiple system atrophy (MSA) or related diseases characterized by parkinsonism, excluding idiopathic Parkinson’s disease.

Jury composition:

1. Chairman

· Prof. Alain Maertens de Noordhout , CHR La Citadelle Liège

2. Members

· Prof Teus Van Laar Fac. Medische Wetenschappen UMCG, dienst Neurologie

· Prof. Niall Quinn Emeritus Professor of Clinical Neurology - University College London (UCL)

· Prof. Francois Tison Centre Hospitalier Universitaire de Bordeaux

· Prof. Alberto Albanese, MD Professor of Neurology and Head Neurologist of the National Neurological Hospital Carlo Besta Milano

· Ritje Schouppe-Moons Chairwoman of The Board of Directors MSA-AMS.be vzw

MSA-AMS.be Leuven 2016

Mr. chairman, Dear members of the jury,

May I thank you again for your very valuable contribution to the evaluation of the projects introduced in the framework of the first call for MSA-research of the Fund Sophia. Please find herewith the minutes of the jury meeting that took place on last 9th November.

I’m happy to tell you that the members of the Fund Steering Group have decided to support two projects instead of one single project:

Project E007: Modeling the role of α-synuclein strains in the pathogenesis of multiple system atrophy - Veerle Baekelandt, KU Leuven (B) Score 8,60 Project E009: Understanding selective regional neuronal and cellular vulnerability in multiple system atrophy - Janice Holton, UCL Institute of Neurology, London (UK) Score 8,60

Each project will receive a grant of € 120.000 An intermediary report and a final report will be asked.

The FSG of the Fund Sophia will decide in 2018 if a new call has to be launched, or if the expert teams will be supported with a new grant so that they can continue their research.

Don’t hesitate to contact us for further information. With our kindest regards,

Annemie T’Seyen | Project manager King Baudouin Foundation | Rue Brederodestraat 21, B-1000 Brussels

MSA-AMS.be Leuven 2016 MSA is like the three flavoured Neapolitan ice cream.

I thought of this analogy many years ago to describe MSA,

Everyone with MSA gets a scoop of the MSA ice cream just as everyone at a birthday party gets a scoop of Neapolitan ice cream in their dish. If you were at the party and looked at your dish and at the dish of the person next to you, you would notice that there is not the exact same amount of vanilla, chocolate and strawberry in both of the dishes.

In someone with Multiple System Atrophy, if their symptoms are mainly ataxia, slurred speech or difficulty swallowing they have mostly vanilla ice cream in their dish. If their symptoms are mainly tremors and slowness of movement they have mostly chocolate ice cream in their dish. If their symptoms are mainly Orthostatic hypotension or urinary incontinence or urinary retention or sexual dysfunction they have mostly strawberry ice cream in their dish. Everyone needs to have some strawberry symptoms to have possible MSA. On top of this they need to have some vanilla and some chocolate symptoms too. Note that they may have a whole lot of vanilla and a tiny bit of chocolate. If so they are called MSA-C (cerebellar). If they have a whole lot of chocolate and a tiny bit of vanilla they are called MSA-P (parkinsonism). People with MSA have a changing bowl of ice cream though ... their vanilla and chocolate and strawberry amounts increase over time as they develop more symptoms from the cerebellar or the parkinsons or the autonomic sides. ... but this will vary from person to person. This is because the disease is spreading throughout the brain. It does not spread exactly the same way in every person. I hope this helps you understand MSA a bit better.

Take care, Pam Bower Board of Directors – Secretary The Multiple System Atrophy (MSA) Coalition 9935-D Rea Road, #212 Charlotte NC 28277 United States

Meervoudig Systeem Atrofie - Atrophie Multi-Systématisée - Multiple System Atrophy

MSA-AMS.be Leuven 2016

NOTES:

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3 MSA-AMS.be vzw – Leuven 2016