Published 15 July 2019 | 3 min read
With major advances in the field, there are now plenty of options — from factor concentrate to home injections, and on-demand and preventative (prophylactic) methods.¹,² Indeed, for Daniel Timmermann, Corporate Vice President of Haemophilia Research at Novo Nordisk, treatment advances mean that “with new extended half-life factor products and bypassing agents, the quality of prophylaxis has become better and the long-term prognosis for joint outcomes has improved.”
Daniel has always been motivated by improving treatment; “I have always been fascinated with the idea of employing cutting-edge science to reduce distress and make a real difference for people living with serious disease.” Indeed, using preventative measures means that people living with haemophilia can receive regular transfusions of clotting factor to stop any bleeding that happens without obvious injury.² This treatment method is now recognised as giving children the best chance of reaching adulthood with minimal damage to their joints.²
Options available today are a stark contrast to previous treatment. When Heinz went to the US in 1980 for 18 months, he had to bring 27 kilos of factor with him; “Nowadays you have these small injection devices. All of a sudden, activities became possible, which in the old days were virtually impossible.”³
These days, for seven-year-old Harry, exercise is recognised as an important aspect of treatment, and Harry’s mum Olivia encourages him to stay active to keep his joints healthy. “The more active you are, the longer you can keep your joints in a healthy state.”⁴
The challenges that science is working to address a major challenge in haemophilia is ensuring global access to adequate care and treatment. Improving access to care is a longstanding and overarching goal of Changing Haemophilia®; in fact, it is estimated that only 25% of the 400,000 strong community of people living with haemophilia worldwide receive adequate treatment.¹
For young Haemophiliacs like Harry, exercise is recognised as an important aspect of treatment.
Inhibitors also pose another considerable difficulty in haemophilia care. Inhibitors occur when the body has an immune response to treatment with clotting factor concentrates, rendering standard treatment ineffective.⁵,⁶ Ongoing efforts continue, to prevent the development of inhibitors and to establish how best to manage treatment for those who have already developed inhibitors. One major recent scientific advance in haemophilia treatment is gene therapy, which Daniel believes could change the care landscape entirely; “complete freedom-from-treatment for many patients may become a reality with a foreseeable future.”
Current methods look to bypass therapy, immune tolerance induction and immunosuppressants in the fight against inhibitors — these techniques work either by encouraging clots to form or removing inhibitors from the blood so that clotting factors can be used.⁶ Research continues efforts to find solutions, and to progress closer to the goal of helping people living with haemophilia live a full life and minimise long-term damage.
What do you believe to be the biggest challenge to haemophilia care today?