The 6th Annual Dublin Cystinosis Workshop 2020 was held on line on 16 July 2020
VIRTUAL MEETING
1145-1905 IRISH LOCAL TIME
The Dublin Cystinosis Workshop is an interactive forum for researchers to share new knowledge, spark new ideas and explore new research approaches aimed at better understanding and treating the ultra-rare disease of cystinosis
THIS IS A CLOSED WORKSHOP
PARTICIPATION IS BY INVITATION ONLY OR VIA THE CALL FOR ABSTRACTS
THE 6TH ANNUAL DUBLIN CYSTINOSIS WORKSHOP 2020 IS GOING ON LINE ON 16 JULY 2020
As in other years, the Virtual 6th Annual Dublin Cystinosis Workshop 2020 is a scientifically focused workshop that brings together internationally recognised researchers and clinicians from Ireland, the UK, Europe, North America and New Zealand to share the most recent advances in their respective research endeavours and to look at new ways to develop our understanding of cystinosis as a disease and its treatment.
The Dublin Cystinosis Workshop is the only such scientific workshop in Europe focused on cystinosis.
The aim of the workshop is to share new research developments in the area of cystinosis and to stimulate discussions and collaborations that can convert new ideas into real research that moves the cystinosis research field forward.
Topics presented at the workshop will cover the spectrum of basic through to translational research.
In particular, researchers who have been funded by or have applied to Cystinosis Ireland for funding are encouraged to present their research at the workshop.
Early stage researchers and PhD students are strongly encouraged to present their research at the workshop.
As it is a closed meeting (no industry) and participation is by invitation only, researchers are free to present their latest scientific work to colleagues spanning a wide range of cross-cutting disciplines from basic through to translational research.
This year, the workshop will take the format of include presentations from a number of keynote speakers and a series of shorter research communications presenting latest cystinosis research findings and including a number of “speed presentations” aimed at introducing new researchers and/or new cystinosis research topics.
Full details of the Workshop Agenda are available here
WHO IS THE DUBLIN CYSTINOSIS WORKSHOP FOR?
The workshop is open to all scientists and clinicians working in the field of cystinosis and in related areas of science.
Our previous in-person workshops consisted of approximately 30 scientists/clinicians in attendance. We aim to keep the Virtual Workshop small also to enable discussion, collaboration and sharing but we expect that our numbers virtually to be a little higher but still limited. , all of whom are very active in the field of cystinosis research.
Those working in the field of cystinosis are very active in this disease area. However, we also are keen to raise awareness of cystinosis research among researchers with relevant expertise but who are not currently involved in cystinosis research.
We strongly encourage early stage researchers and PhD students to participate.
The Virtual 6th Annual Dublin Cystinosis Workshop 2020 is an interactive forum for researchers to share new knowledge, spark new ideas and explore new research approaches aimed at better understanding and treating the ultra-rare disease of cystinosis.
Topics presented at the workshop will cover the spectrum of basic through to translational research.
This year, the workshop takes the format of a number of keynote speakers and a series of short research communications in which researchers present their latest cystinosis research findings.
This year’s keynote speakers are:
- Dr Rik Gijsbers – Associate Professor, Faculty of Medicine and Head of the Subdivision Viral Vector Technology & Gene Therapy, KU Leuven, Belgium
- Dr Manoe Janssen – Senior Researcher, Experimental Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, The Netherlands
- Professor Michael Taggart – Chair of Reproductive Sciences, Cardiovascular Research Centre, Newcastle University, UK
- Dr Maya Doyle – Associate Professor of Social Work, School of Health Sciences & Clinical Assistant Professor, Frank H. Netter MD School of Medicine, Quinnipiac University, USA with contributions from members of the Adult Leadership Advisory Board – Ms Karen Gledhill and Ms Cheryl Simeons
- Professor Francesco Emma MD – Head, Division of Nephrology and Dialysis, Ospedale Pediatrico Bambino Gesù, Rome, Italy
As it is a closed meeting (no industry) and participation is by invitation only, researchers are free to present their latest scientific work to colleagues spanning a wide range of cross-cutting disciplines from basic through to translational research.
CALL FOR ABSTRACTS TO PRESENT AT THE 6TH ANNUAL DUBLIN CYSTINOSIS WORKSHOP 2020 ON 16 JULY 2020
Please note the call for abstracts is now closed.
Cystinosis Ireland invites researchers of any aspect of cystinosis research to submit abstracts for consideration by the scientific committee to given an oral presentation of their latest research findings at the upcoming Virtual 6th Annual Dublin Cystinosis Workshop 2020.
The deadline for abstract submissions is Friday 12th June 2020.
Abstracts should be submitted by email to [email protected] with “DCW2020 Abstract Submission” included in the subject line.
Please note that abstracts must adhere to the submission guidelines (see abstract submission guidelines below) and that late submissions cannot be accepted.
Researchers submitting abstracts must be able to commit to the Cystinosis Ireland timetable (see timetable below) which requires speakers to submit their presentation slides in advance and to do a trial run of the presentation using the zoom conference facilities. This is to ensure the smooth running of the conference on the 16 July 2020 and that researchers keep to the strict presentation time limits.
A maximum of two abstracts may be submitted by any individual researcher.
WHO SHOULD APPLY?
The Scientific Committee of the 6th Annual Dublin Cystinosis Workshop 2020 invites all researchers with an interest in cystinosis research to submit an abstract on any research topic relevant to the understanding, cure, treatment or care management of cystinosis for consideration to speak at the Virtual 6th Annual Dublin Cystinosis Workshop 2020.
In particular, Cystinosis Ireland strongly encourages researchers who have received, or have applied for, research funding from Cystinosis Ireland either through its Seedcorn funding scheme or through the MRCG-HRB/HRCI-HRB Joint Funding Schemes to submit an abstract.
Early stage postdoctoral researchers and PhD candidates and researchers that are new to the field of cystinosis are particularly encouraged to submit an abstract.
Researchers of selected abstracts will be asked to give a 7 minute oral presentation on their research, with 3 minutes for questions.
PRIZES
The Professor Roz Anderson Memorial Prize for the best 7 minute oral presentation.
The winner will win a €200 cash prize as well as an invitation to present their research at the next Dublin Cystinosis Workshop in 2021.
The Professor Roz Anderson Memorial Prize is awarded by Cystinosis Ireland in memory of the late Professor Roz Anderson, University of Sunderland, who passed away in 2018. Professor Anderson was an exceptional researcher who dedicated her life to finding a cure for cystinosis. Her research legacy continues on under the leadership of Professor Herbie Newell.
ABSTRACT SUBMISSION GUIDELINES
Abstracts should be submitted as a word document and structured as follows:
- A descriptive title in Bolded Times New Roman 12 point.
- The initials and surname of the author(s) without titles or degrees should appear below the title.
- The department(s), institution(s) and city(ies)/town(s) should be listed next.
- The abstract should be of the informative type containing five paragraphs addressing the following headings:
- Lay Summary
- Purpose/Objectives of Study and a Brief Background Statement
- Methods
- Results
- Conclusions/Discussion including the potential impact(s) of the results for people living with cystinosis.
- The overall abstract must not exceed 500 words – excluding the title, authors(s) name(s), address(es), and any references.
- The text should be single spaced.
- The font should be Times New Roman 12 Point.
- The abstract may include no more than one table or figure.
- When using abbreviations, spell out the full name on first mention. Avoid excessive use of abbreviations.
- Up to two references can be included.
- Potential conflicts of interest must be disclosed.
You may submit a maximum of two abstracts.
As this is a closed workshop, you are encouraged to include unpublished work.
TIMETABLE
- CALL FOR ABSTRACTS
- ABSTRACT SUBMISSION DEADLINE
- COMMUNICATION OF SELECTION DECISIONS TO RESEARCHERS
- SUBMISSION OF PRESENTATION SLIDES TO CYSTINOSIS IRELAND ORGANISERS
- TRIAL RUNS FO PRESENTATIONS ON ZOOM (SPECIFIC TIMES TO BE AGREED WITH RESEARCHERS)
- FRIDAY 29 MAY 2020
- FRIDAY 12 JUNE 2020
- FRIDAY 19 JUNE 2020
- TUESDAY 7 JULY 2020
- 9-10 JULY AND 13-14 JULY 2020
Researchers submitting abstracts must be able to commit to the timetable to ensure the smooth running of the virtual workshop.
Speakers will be asked to provide their mobile phone numbers to be included in a WhatsApp group to facilitate fast and efficient communication between the speakers and organisers on the day of the workshop.
All speakers must login to the zoom meeting a minimum of 15 minutes in advance of their speaking time (tbc) and text the host via zoom chat or the WhatsApp group to inform us that they are on line.
SECURITY
Please note that this is a closed meeting and attendance is by invitation only. Every effort will be made to ensure confidentiality and a ‘waiting room’ facility will be in place to ensure that only people that are invited are allowed to join the workshop. The workshop will not be recorded by the organisers. However, while we will do our best to ensure security, online events are by their nature not 100% secure and this should be taken into consideration when speakers are preparing their presentations.
REGISTRATION FOR THE VIRTUAL 6TH ANNUAL DUBLIN CYSTINOSIS WORKSHOP 2020
An Eventbrite registration page has been set up to allow researchers to register. Zoom login details and passwords will be sent to registered researchers prior to the workshop date.
Participation is by invitation only. Researchers who wish to attend but have not received an invitation directly from Cystinosis Ireland may register using the Eventbrite registration page.
If you do not have a link to the eventbrite page, please email [email protected] in order to be provided with a link to that page.
All researchers must identify the research institution and/or research lab at which they work.
VIRTUAL 6TH ANNUAL DUBLIN CYSTINOSIS WORKSHOP 2020 SCIENTIFIC ADVISORY AND ORGANISING COMMITTEE
Dr Paddy Harrison – University College Cork – DCW 2020 Scientific Chairperson
Professor Atif Awan – Consultant Paediatric Nephrologists, Temple Street Children’s University Hospital, Dublin – Medical Organiser Paediatric
Professor Elena Levtchenko – Head of Pediatric Nephrology at UZ Leuven, Belgium
Dr Jennifer Hollywood – University of Auckland, New Zealand
Dr Thomas J McDonald – Director, Drug Product EMEA/APAC External Manufacturing, Bristol Myers Squibb – Cystinosis Ireland Executive Committee Member
Dr Achim Treumann – Director, Analytical Formulation Sciences and Site Head at KBI Biopharma BV – Cystinosis Ireland Scientific Advisor
Ms Anne Marie O’Dowd – Chairperson, Cystinosis Network Europe and Cystinosis Ireland Executive Committee Member
Dr Ruth Davis – Research Manager, Cystinosis Ireland
Ms Denise Dunne – Operations Manager, Cystinosis Ireland
Virtual 6th Annual Dublin Cystinosis Workshop 2020
Workshop Agenda
Please note that all times are given as Irish Local Time – Please check your time zone for the correct times
11:45 am – 12:00 pm – Welcome Address
by Dr Patrick Harrison, UCC, Professor Elena Levtchenko, UZ Leuven and Ms Anne Marie O’Dowd, Cystinosis Ireland.
Session One – Chair Dr Patrick Harrison
12:00 pm – 12:25 pm – Keynote Speaker – Dr Rik Gijsbers – Associate Professor, Faculty of Medicine and Head of the Subdivision Viral Vector Technology & Gene Therapy, KU Leuven, Belgium
Title of Presentation: Viral Vectors for Gene Therapy: repurposing viruses to study and cure genetic disorders
12:25 pm – 12:55 pm – Short Research Communications
- Dr Jennifer Hollywood, Department of Molecular Medicine and Pathology, The University of Auckland, New Zealand – mTor inhibition in Cystinotic iPSC’s and a new rat model to study Cystinosis
- Dries David, Department of Pharmaceutical and Pharmacological Sciences, Laboratory for Viral Vector Technology and Gene Therapy, KU Leuven, Belgium – First steps towards a kidney directed gene therapeutic approach for the lysosomal storage disease cystinosis
- Amer Jamalpoor, Faculty of Science, Department of Pharmaceutical Sciences, Division of Pharmacology, Utrecht University, The Netherlands – Cysteamine-bicalutamide combination treatment restores α-ketoglutarate and corrects proximal tubule phenotype in nephropathic cystinosis
12:55 pm – 1:10 pm – Q&A and Discussion
1:10 pm – 1:15 pm – 5 MIN COMFORT BREAK
Session Two – Chair Professor Elena Levtchenko
1:15 pm – 1:40 pm – Keynote Speaker – Dr Manoe Janssen, Assistant Professor, Department of Pharmaceutical Sciences, Utrecht University, The Netherlands
Title of Presentation: Exploring new therapy options for cystinosis.
1:40 pm – 2:10 pm – Short Research Communications
- Dr Shu-Dong Zhang, Northern Ireland Centre for Stratified Medicine, Ulster University, Northern Ireland – Gene expression connectivity mapping approach to drug repurposing for cystinosis
- Fatima Tokhmafshan, McGill University Health Centre, Montreal, Canada – A new nonsense mutant mouse model for cystinosis
- Dr Koenraad Veys, Department of Pediatrics, Pediatric Nephrology, UZ Leuven, Belgium – Impact of early initiation of cysteamine on renal and extra-renal outcomes in cystinosis: a retrospective international multicentric cohort study in cystinosis siblings
2:10 pm – 2:25 pm – Q&A and Discussion
2:25 pm – 2:45 pm – 20 MIN BREAK
Session Three – Chair Dr Patrick Harrison
2:45 pm – 3:10 pm – Keynote Speaker – Professor Michael Taggart – Chair of Reproductive Sciences, Cardiovascular Research Centre, Newcastle University, UK
Title of Presentation: Multimorbidity and cystinosis: what about the cardiovascular system?
3:10 pm – 3:40 pm – Short Research Communications
- Dr Aude Servais, Nephrology and Transplantation, Necker hospital, APHP, Paris, France – RaDiCo-ECYSCO, a European cystinosis cohort
- Dr Brendan Keating (on behalf of the International Genetics & Translational Research in Transplantation Network – iGeneTRAiN), Penn Transplant Institute, University of Pennsylvania, Philadelphia, PA, USA – The logistics of Genome-wide Genotyping and Whole Exome Sequencing of all available Cystinosis Patients Globally: The Why, the How, and the Who
- Annika Ewert, Department of Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Children’s Hospital, Hannover, Germany – Bone and mineral metabolism in children with nephropathic cystinosis compared to other CKD entities
3:40 pm – 3:55 pm – Q&A and Discussion
3:55 pm – 4:00 pm – 5 MIN COMFORT BREAK
Session Four – Chair Professor Elena Levtchenko
4:00 pm – 4:25 pm – Keynote Speaker – Professor Maya Doyle, Associate Professor of Social Work, School of Health Sciences & Clinical Assistant Professor, Frank H. Netter MD School of Medicine, Quinnipiac University, USA – including contributions from members of the Adult Leadership Advisory Board (ALAB) – Ms Cheryl Simeons and Ms Karen Gledhill
Title of Presentation: Advancing Adult Leadership in Cystinosis Advocacy and Support – see poster for this presentation here
4:25 pm – 4:40 pm – Anne Marie O’Dowd, Director of Cystinosis Ireland; Chair Cystinosis Network Europe & Worldwide Cystinosis Community Advisory Board
Title of Presentation: The importance of Public and Patient Involvement throughout the research process and its impact on funding decisions
4:40 pm – 4:55 pm – Q&A and Discussion
4:55 pm – 5:15 pm – 20 MIN BREAK
Session Five – Chair Dr Patrick Harrison
5:15 pm – 5:40 pm – Keynote Speaker – Professor Francesco Emma MD – Head of the Department of Pediatric Subspecialties, Ospedale Pediatrico Bambino Gesù, Rome, Italy
Title of Presentation: Understanding and searching for new therapies in nephropathic cystinosis
5:40 pm – 6:10 pm – Short Research Communications
- Dr Swastika Sur, Department of Surgery, University of California San Francisco, USA – Identification of Specific Vacuolar-ATPases by Gene Expression Profiling in renal cells and testis of Nephropathic Cystinosis
- Meisha Khan, Department of Pediatrics, Division of Genetics, University of California, San Diego, USA – Macrophage tunnelling nanotube-mediated HSPC transplantation therapy for the lysosomal storage disorder cystinosis remains effective in preclinical mouse study following Shpk elimination
- Professor Justine Bacchetta, Centre de Référence des Maladies Rénales Rares, Centre de Référence des Maladies Rares du Calcium et du Phosphore, Hôpital Femme Mère Enfant, Bron, France – Osteoclasts obtained from patients with nephropathic cystinosis with residual cystinosin activity display a peculiar sensitivity to cysteamine
6:10 pm – 6:25 pm – Q&A and Discussion
6:25 pm – 6:30 pm – 5 MIN COMFORT BREAK
Session Six – Chair Dr Patrick Harrison
6:30 pm – 6:50 pm – General Workshop Discussion
6:50 pm – 7:05 pm – Wrap up, and Award of the Professor Roz Anderson Memorial Prize for Best Short Research Communication
