SMA Europe organised the first European SMA Research Congress, which took place in Krakow, Poland, between 25th & 27th January 2018.
Our first congress was a resounding success. 432 people, from 37 countries, participated. The majority were researchers (39%), followed by clinicians (30%), industry (13%) and patient representatives (12%).
The programme included 35 oral presentations across 7 topics, 75 posters as well as workshops and sessions with pharmaceutical companies who have programmes for SMA. We were delighted to be able to offer a Best Investigator Award, which went to PhD student Eva Janzen (Institute of Human Genetics, Cologne, Germany) as well as 3 best poster prizes, which went to Dr. Sukrat Arya (Oxford University, UK), Dr. Agnes Poirier (Roche) and Linda Lowes (Nationwide Children’s Hospital, USA).
Professor Dr. Brunhilde Wirth and Eva Janzen (Institute of Human Genetics, Cologne, Germany)
Professor Dr. Brunhilde Wirth and Dr. Sukrat Arya (Oxford University, UK)
Professor Dr. Brunhilde Wirth and Dr. Linda Lowes of Nationwide Children's Hospital, USA)
Professor Dr. Brunhilde Wirth with Nikolai Naryshkin (PTC Therapeutics), on behalf of Dr. Agnes Poirier (Roche)
Several main themes emerged during the congress, which will have high priority in SMA research in the years to come.
Pre-clinical research is focusing on gaining a better understanding of the changes in cellular mechanisms that lead to SMA and, specifically, on the function of the SMN protein. Research over the past decade has shown that tissues and cells other than the motor neurons are also affected in SMA, although not as severely. The importance of these so-called non-motor symptoms of SMA was subject of significant debate during the congress. As increasing numbers of patients are now receiving Spinraza treatment –that specifically improves the function of motor neurons– it is possible that, in Spinraza-treated patients, non-motor symptoms become more pronounced. Thus, the extent and importance of non-motor symptoms in SMA will be the subject of further research. Moreover, many cellular mechanisms are changed in SMA because levels of the SMN protein are low. In fact, so many cellular mechanisms have now been identified to play a role in SMA that it becomes increasingly complicated to understand which mechanisms are crucial to maintain healthy motor neurons. Although different researchers have different ideas and views about this, there was much agreement that better understanding on mechanistic links between apparently separate cellular pathways and how to prioritise the involvement of specific cellular mechanisms in SMA should be a main focus of research in years to come.
The clinical sessions of the congress focused on Spinraza and other therapies that are still under development. We learned that promising progress on therapies other than Spinraza is being made, highlighting the possibility that further treatments might become available in the near future. However, as more details become available on the response of patients to therapies, a number of issues and challenges have come-up which will need to be addressed in research over the coming years. For example, past clinical trials used relatively crude measures to determine the effect of novel therapies, such as survival and reaching motor milestones such as sitting unaided. However, it has become increasingly clear that, for some patients, the benefit from therapies such as Spinraza can be quite subtle –yet still meaningful. Therefore, sensitive and unbiased measures, such as MRI scans, are likely to become more important to follow disease progression in SMA patients. In addition, several speakers addressed the current complexity of providing Spinraza to an increasingly large group of patients. The challenging logistics and complex delivery of Spinraza suggests there will be a role for other, less invasive SMA treatments in the clinic in the coming years.
The Jagiellonian University, Krakow, Poland.
Our wonderful Tripp Company team who made everything possible!
SMA Europe President Marie-Christine Ouillade delivering her welcome speech.
Fundacja SMA & SMA Europe member Kacper Rucinski introducing Poland and thanking everyone.
Dr. Maria Jędrzejowska, neurologist.
Professor Dr. rer.nat. Brunhilde Wirth opening the Congress!
Key note speaker and SMA Europe Scientific Advisory Board member Professor Arthur Burghes of Ohio State University, USA.
Session Chair Dr. Livio Pellizzoni of Columbia University, New York, USA.
Dr. Gabriella Viero of the Institute of Biophysics in Trento, Italy.
Singapore University PhD student Shermaine Tay.
Professor Michael Sendtner of the University of Wursburg, Germany.
Dr. Ewout Groen of the University of Edinburgh, UK.
Session Chair, Professor Charlotte Sumner of the John Hopkins School of Medicine, USA.
Dr. Melissa Bowerman of the University of Keele in the UK.
Dr. Melissa Bowerman of Keele University in the UK.
Dr. Rashmi Kothary of the Ottawa Research Institute in Canada.
Professor Simon Parson of Aberdeen University talking to us about heart defects in severe SMA models.
Prof. Dr. rer. nat. Brunhilde Wirth of the Institute of Human Genetics in Cologne, Germany and Chair of SMA Europe' Scientific Advisory Board.
Insitute of Human Genetics PhD student Eva Janzen.
Dr. Rocio Tejero of the University of Seville, Spain.
Dr. Lyndsay Murray, Lecturer at the University of Edinburgh in the UK.
SMA Europe Scientific Advisory Board member Dr. Stefania Corti of the University of Milan, Italy.
Session Chair Dr. Chris Lorson of the University of Missouri, USA.
SMA Europe grant-holder Dr. Laura Torres-Benito of the Insitute of Human Genetics in Cologne, Germany.
SMA Europe grant-holder Dr. Niko Hensel of Hannover Medical School in Germany.
Dr. Jamuna Selvakumaran of the Royal Holloway, University of London, UK.
Dr. Laurent Servais of i-Motion, Paris, France and SMA Europe Scientific Advisory Board member.
Professor Richard Finkel, Nemours Children's Hospital USA and SMA Europe Scientific Advisory Board member.
Professor Eduardo Tizzano, Vall d'Hebron Hospital, Barcelona, Spain and SMA Europe Scientific Advisory Board member.
Dr. James Sleigh of University College London, UK, reading posters.
Biogen panel Meeting
Eva Janzen and SMA Europe grant-holder Dr. Niko Hensel of Hannover Medical School, Germany.
The Edinburgh contingency! Ines Boehm, Nikki Yu-Ting Huang and Dinja van der Hoorn.
One of the poster sessions
Our poster: Results of a survey carried-out across Europe to ascertain disease impact on the general well-being & the therapeutic expectations of European Type II & III SMA patients.
Professor Michael Sendtner of the University of Wursburg, Germany and Professor Kevin Talbot of the University of Oxford, UK and also vice-Chair of SMA Europe's Scientific Advisory Board.
SMA Europe Board Anna Wittchen
Former SMA Europe President Casimir Knight with Michelle Levene of Novartis.
Dr. Ewout Groen (Edinburgh University) and Karin Meijboom, DPhil student at the University of Oxford.
Fundacja SMA volunteer Maria Kozminska and Fundacja SMA President & SMA Europe Board member Kacper Rucinski.
SMA Europe Scientific Advisory Board member Dr. Stefania Corti (middle).
Beata Żuchowicz of Tripp, SMA Europe President Marie-Christine Ouillade and SMA Europe Coordinator Vanessa Christie-Brown.
Sukrat Arya and Kevin Talbot deep in conversation.
Professor Francesco Muntoni, University College London.
Our Scientific Advisory Board and three of our session Chairs.
