Drug Repurposing for Rare Diseases Conference 2019
Rick Thompson - CEO, Findacure
Becky Jenner - CEO, Rett UK & Rachael Bloom Stevenson - Executive Director, Reverse Rett
Prof. Lakshminarayan Ranganath - Clinical Director, National Alkaptonuria Centre
Xavier Paoli - Chief Commercial Officer, Pharnext & Rodolphe Hajj - Chief Pharmacology Officer, Pharnext
Logan Williams - Medical Student, University of Auckland
Pan Pantziarka - Program Director, Anticancer Fund
Catherine Lawrence - Senior Medical Advisor UK, Santhera & Janet Bloor - Parent & Duchenne Advocate
Janet also shared her story, as a parent of an adult son who has been on the Ibedone trial for the past 18 months. Janet’s son was diagnosed age 9, at which time they were told there was “no hope, no cure, dead by 20”. Janet refused to accept this and has dedicated her life to battling for her son, now 25, and other Duchenne families. As an older Duchenne patient, Janet’s son was one of those whose needs were previously ignored and Janet has advocated hard to bring him, and others, to the attention of pharmaceutical companies.
Suzanne Morris - Patient Representative, PNH
Suzanne outlived her initial life expectancy but the condition still had a significant impact on her life. She was determined to live a “normal” life, but never told anyone about her condition, partially due to the stigma of having a blood disorder during the AIDs crisis, and prejudice people received. Many years later, during an appointment at St George’s Hospital, her doctor asked her if she’d like to participate in a clinical trial, and she accepted immediately. Initially, her body completely rejected the new drug – Soliris – and she was admitted to hospital. Eventually, however, her body accustomed to it and she began to make improvements. For every single patient on the trial, it was proven that the drug had a significant positive impact on their life, which was key to it eventually being approved by the FDA and NICE. Soliris is known to be one of the most expensive drugs in the world, and Suzanne has now been on this treatment for over 15 years. Finally, Suzanne highlighted the difficulties of living with an ‘invisible condition’ and the need to focus on the mental and social effects of rare diseases.
Lightning talk programme
Joanna Segieth - Director, Biosynetix
Joanna was the first to take the five minute challenge. Her talk focused on the ways that new clinical trial designs in cancer are allowing a single drug to be trialled for multiple different conditions within the same trial. She suggested that this type of structure could be hugely beneficial for families of related rare genetic conditions.
Heather Band - Scientific Officer, BDFA
Patient groups are in a unique position to capture the patient view on life with a rare condition, and the research that is been done to improve their treatment outlook. Heather shared the experience of the Batten Disease Family Association in conducting a detailed patient survey, and how it has shaped their work.
Jabe Wilson - Consulting Director, Text and Data Analytics, Elsevier
Elsevier have been working in collaboration with US charities Cures Within Reach and Mission Cure to deliver Datathon project with the Pistoria Alliance. Here they were able to employ literature mining technology to identify and infer a number of exciting new drug repurposing ideas for chronic pancreatitis. At least one of these will be taken forward to be trialled beyond the computer this year.
Helen Springford - VP, Strategic Development, Illingworth Research
Helen’s talk looked at the experience of rare disease patients in smaller nations who are looking to take part in clinical trials. She focused on a young boy living with Duchenne in Iceland – ordinarily they would be very unlikely to be able take part in a trial, as sites would rarely be set up in Iceland; however the use of mobile research nurses within one Canadian trial meant that an Icelandic research nurse could be used, allowing a number of study visits to occur in Iceland, and thus allowing the family to take part in the trial.
Gavin Jones - Director of Rare Disease, Open Health
Gavin’s talk was inspired by attendance of Findacure’s 2018 conference as well as the European Conference on Rare Diseases. In it he outlined some of the key things that patient groups can do to form strong partnerships with the Biopharma industry in order to develop new rare disease treatments. In particular he advocated the importance of considering the impact a patient group can have along the whole development pathway, including helping to secure marketing approval and reimbursement.
With thanks to
Thank you to all of our speakers, lightning talk speakers and panellists for sharing their work and experience with us, our Photographer Arnab Ghosal, and our volunteers Gayaneh Szenkovits and Lucy McKay. Finally, thank you to our sponsors, without whom this event would not have been possible: Platinum sponsor Pharnext, Silver sponsors Sobi and Takeda, and our Bronze sponsors Alexion, LifeArc, PharmacoMedics and SOM Biotech. Our sponsors provided financial support for this event. This event was solely organised by Findacure and our sponsors did not determine the content. All funds received from pharmaceutical companies were used in accordance with the ABPI code. Please note, sponsorship does not equate to endorsement of products or services.