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On May 1st – 3rd, 2015, Cystic Fibrosis Canada hosted the 15th Broken Arrow Conference in Toronto. Attendees included CF researchers and healthcare professionals who shared their expertise related to the meeting’s theme, ‘Gene Discovery to Personalized Medicine’. The meeting featured Canadian and international speakers whose talks fostered helpful, open discussions among the clinical and research communities.
Dr. Lap-Chee Tsui opened the conference with an engaging recollection of the events that led to the discovery of the CF gene, from 1981, when he first arrived at The Hospital for Sick Children (SickKids) in Toronto, to 1983, when he received his first grant from the then Canadian Cystic Fibrosis Foundation, and finally to the unforgettable announcement in 1989 that the gene responsible for CF had been found. This momentous event over 25 years ago opened the door to important research to give us a better understanding of the basis of CF and the discovery of effective treatments.
The meeting continued with a presentation by Dr. Johanna Rommens, Senior Scientist at SickKids Hospital and Professor at the University of Toronto, who provided an overview of the two CFTR mutation databases and how they have contributed to, and continue to enhance, our understanding of CF.
Next, several speakers shared their expertise on different strategies to correct CFTR function. Dr. Deborah Gill from Oxford University spoke about CF gene therapy, including the results of preclinical studies and the UK Gene Therapy trial carried out in Edinburgh, Oxford, and London. Dr. Christine Bear, Co-Director of the SickKids Cystic Fibrosis Centre, and Dr. Gergely Lukacs, Professor at McGill University, discussed barriers to fixing CFTR. Dr. Bear’s presentation focused on strategies to fix CFTR with rare, disease-causing mutations while Dr. Lukacs’ talk centred on the molecular intricacies of the different CFTR mutations and how they affect the response to different CFTR-targeted drugs.
Following the discussions on strategies to correct CFTR function, Dr. Lisa Strug, Senior Scientist at SickKids Hospital, reminded us that the complex genetics of CF involve more than just the CFTR gene. It is important also to consider modifier genes to help us better understand why different individuals with CF have a different disease course and why they might respond differently to therapy.
Different CFTR mutations and, in addition to that, different modifier genes among individuals cause a variable clinical spectrum in patients. With these key concepts in mind, Dr. Elizabeth Tullis, Director of the Toronto Adult CF Clinic, discussed the atypical CF patient and Dr. Janet Rossant, Chief of Research at SickKids Hospital, highlighted the need to assess the effectiveness of new potential drugs using cells from patients.
Dr. Steven Freedman, Professor of Medicine at Harvard Medical School, provided an overview of the gastrointestinal features of CF and how they might be treated using CFTR-targeted therapies. Similarly, Dr. André Cantin, Director of the Adult CF Clinic at the Centre Hospitalier Universitaire de Sherbrooke, presented several novel anti-inflammatory therapies that, in the future, may help to treat inflammation in CF patients.
With over 1,800 published clinical trials in CF, the field is clearly very active in seeking treatments to improve the lives of those living with CF. Dr. Chris Goss, Professor of Medicine and Pediatrics at the University of Washington, and Dr. Ed McKone, Director of the European CF Patient Registry, outlined the spectrum of challenges facing CF drug trial design and explained how the abundant data available in patient registries are beneficial to the design and implementation of trials.
To close the conference, Dr. Steve Morgan, Professor of Health Policy at the University of British Columbia, delivered a thought-provoking discussion of policy and the challenges to access to medicines in Canada.
Thanks to our accomplished speakers and representatives from the CF clinical and research communities in attendance, the 15th Broken Arrow Conference was a great success and we look forward to hosting the next conference to highlight future advances in the field.
View the full presentations here.
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