News and events News Breakthrough for haemophilia A patients Perth Blood Institute has participated in a ground-breaking clinical trial which has uncovered a new life-changing treatment for people with haemophilia. With the help of one brave West Australian who agreed to participate in the trial, the drug was found to provide widespread relief for patients who are at risk of life-threatening bleeding events and joint damage. As a result, the Hon Greg Hunt MP, Minister for Health, recently announced that the commonwealth and state governments have all agreed to fund the drug Hemlibra, which will be available free-of-charge to eligible people with haemophilia across Australia, through the National Blood Authority. Consultant Haematologist Professor Ross Baker, Founding Director of PBI, says the innovative treatment is a huge success story for science, the brave people who participate in clinical trials, and also for the continuation of best-practice treatment for future generations, especially for children with haemophilia. “This new drug transforms haemophilia care and attests to the huge benefits of laboratory and clinical training of haematologists which enables clinicians to understand and personalise treatment for people with bleeding disorders,” Professor Baker says. Hemlibra is a total game changer for patients with haemophilia A who can go on to live more normal lifestyles, including being able to take part in activities they may never have had the confidence to pursue because of living with their condition. “It’s a wonderful result for everyone involved with the trial at PBI and provides new hope for adults and children with haemophilia right across Australia who will benefit immensely from this new treatment. A simple injection under the skin every two to four weeks is all that is required.” Cockburn resident John Wyatt was deemed suitable to take part in the clinical trial at PBI, having experienced continuous life-affecting health issues as a result of being born with haemophilia, which saw him undertake long stays in hospital in acute pain, particularly as a young man. “With three grandchildren who also have haemophilia A, I was committed to participating in any trial that could potentially lead to life-changing treatments,” says Mr Wyatt, who has been under Professor Baker’s care for more than 25 years. “I have taken part in several trials, but the last one, in 2017, for Hemlibra was the game changer, and for the first time in over 50 years I have had no bleeding issues, in fact not even a bruise. “Being able to participate in Perth Blood Institute’s clinical trial program has literally changed my life. It’s been wonderful.” The drug Hemlibra was designed to mimic the missing function of Factor VIII, which patients with haemophilia A lack. As a result, it provides the bridge for restoring normal blood clotting processes and provides relief for patients who would previously suffer life-threatening bleeding events and joint damage, which even occurred with the best previous clotting factor replacement treatment. This agent has since advanced to be approved by the Therapeutic Goods Administration in Australia, and regular supply of Hemlibra is expected to be available by December 2020. PBI is a charitable, not-for-profit, haematological research institute. Based in West Perth, the organisation undertakes research into blood related disorders, delivers blood related clinical trials and provides an education service to the community and professionals on blood conditions. The organisation is focused on providing outstanding patient care to people diagnosed with blood conditions – including blood cancers, bleeding and clotting disorders – through an ethical and sustainable approach.