Participants at the 10th BIC Conference.
To keep abreast with the latest scientific and medical updates on novel technologies in haemophilia and inhibitor treatment, EHC staff and steering committee members attended in September two major scientific conferences.
The first event was two joint conferences: the 10th edition of the BIC International Conference and the third edition of the International Conference on Inhibitors in Haemophilia. Both events were chaired by Prof Flora Peyvandi, who is also the Chair of the EHC Medical Advisory Group (MAG). The events were held in Genoa, Italy ahead of the 2020 Congress of the International Society for Thrombosis and Haemostasis (ISTH) taking place in Milan and also chaired by Prof Peyvandi. Mrs Amanda Bok, EHC CEO and Mrs Kristine Jansone, EHC Community Programmes Officer attended this meeting.
The second event was the Workshop on Novel and Gene Therapies organised by the National Hemophilia Foundation (NHF) in Washington DC, United States. Mr Brian O’Mahony, EHC President, Dr Radoslaw Kaczmarek, EHC Steering Committee Member and Mr Declan Noone, EHC PARTNERS Programme Consultant, attended this event.
We will report in detail the main findings from both meetings in our next issue of the ‘Novel treatments in haemophilia and other bleeding disorders: A periodic EHC review,’ to be released next week ahead of the EHC Conference. For now, we would like to inform our members that during these meetings we received, amongst others, updates on the developments of first-generation gene therapy programmes. These updates looked at how long gene therapy may last, which candidates may have a better response to the treatment, and, how can we also treat those who may not be eligible due to pre-existing antibodies to the gene therapy vector. During the meetings, there was also a glimpse into the future of the next generation of gene therapy that could use gene editing, different modes of delivery and different cells for gene production.
The meetings also included updates on other novel non-replacement treatments looking at rebalancing haemostasis. These treatments are currently in the clinical trial and could be an alternative to treating people affected by rarer bleeding disorders.
Panel discussions at the NHF Workshop on Novel and Gene Therapies. Mr Declan Noone is second from the left.
Jim is aged 66 and has Haemophilia A with a high titer inhibitor which he developed at age 14.
Maria Elisa Mancuso (MD, PhD) is a Haematologist and works as a Senior Haematology Consultant at the Center for Thrombosis and Haemorrhagic Diseases of IRCCS Humanitas Research Hospital in Rozzano, Milan, Italy. She is Adjunct Clinical Professor at Humanitas University. She obtained a post-degree in Clinical and Experimental Haematology and a PhD in Clinical Methodology. She is involved in clinical research and has published several original articles in peer-reviewed journals a The Lancet, Blood, Journal of Thrombosis and Haemostasis, Haematologica, Thrombosis and Haemostasis, British Journal of Haematology and Haemophilia. She is reviewer for several peer-reviewed journals and member of the Editorial Board of JTH. She is a member of several scientific societies (ISTH, WFH, ASH, EAHAD, SISET, AICE) and was a medical member of the Inhibitor Working Group of the European Hemophilia Consortium. She is co-chair of the ADVANCE Study Group. She has acted also as co-chair of the Scientific and Standardization Subcommittee of ISTH on FVIII, FIX and rare bleeding disorders. She has been involved as principal and co-investigator in several clinical trials, and she takes care of both children and adults with hemophilia and other congenital bleeding disorders with a specific scientific interest in novel therapies, prophylaxis, inhibitors, and chronic hepatitis C.
As a patient with factor II deficiency, the diagnostic and treatment of rare bleeding disorders is a matter dear to my heart. My motivation to participate in the work of the ERIN committee is to improve both diagnostic and treatment for patients with rare bleeding disorders across Europe.
Economist and financial expert by profession, executive coach and trainer by passion and haemophilia advocate by every drop of my blood through my son (who has severe haemophilia A with inhibitors). Bringing a good decade of practical experience from the corporate insurance world, laser focus, growth mindset and resilience from my own experience, offering you anything I can just do, in hope that together we can make life more fulfilled for those impacted by bleeding disorders.
Amy Owen-Wyard is a Registered Mental Health Nurse. With experience working with children, young people, their families and adults with severe and enduring mental health conditions. Amy was also involved in a service improvement to provide a holistic care approach for those in general hospitals to support both their mental and physical health, whilst sharing her expertise and knowledge in mental health with the wider multidisciplinary team.
