Patients, partnerships and pioneering science are at the heart of developing advanced therapies in the next wave of innovation to provide transformative treatments and cures, according to latest developments reported at the Better Science, Better Health: Advanced Therapies – Opportunities and Challenges conference (14 November, London, UK).
At the event, hosted by ABPI member Takeda and supported by the ABPI, academics, policy makers and medicine developers from around the world discussed the changing global landscape for the biopharmaceutical sector and shared insights on how EU biotech can adapt and succeed.
Lord James O'Shaughnessy, Parliamentary Under-Secretary of State for Health (Lords)
Underlining the potential for advanced therapies to transform patient care in the near future, Peter Goodfellow, Scientific Advisor, Abingworth, UK, "I believe we will be able to replace all the parts of the human body over the next 20 years."
He added, "We are so lucky to be the generation able to take biology to pieces and understand how biology works. By understanding the biology we can create next generation therapies." He noted that it was essential to demonstrate efficacy and to deliver value with these new therapies, and to explain new scientific advances to the general public: "We need to remember as scientists to engage with the public."
Partnerships are a key strategy in Takeda's commitment to achieving the mission of the company's Regenerative Medicine Unit to become an industry leader in the field by bringing definitive therapies to patients with life-threatening disease, explained Seigo Izumo, Senior Vice President for Regenerative Medicine with Takeda Pharmaceuticals.
These include a unique academic-industry collaboration, the Takeda-CiRA Joint Programme, based in the Shonan Research Center, Fujisawa, Japan, where more than 100 scientists from academia and Takeda are working together on projects using induced pluripotent stem cell (iPSC) technologies.
Projects underway include a drug discovery programme with iPSC-derived neuronal cells aiming to help patients with amyotrophic lateral sclerosis (ALS) to recover motor function and suppress neuronal degeneration and a programme with T-cells with the objective of achieving tumour regression or cure.
"We want to change the future of medicine using iPS cells. T-CiRA aims to enter clinical trials within three to four years, and submit approval applications within nine years," Dr Izumo told the meeting. An Open Innovation Hub at the Research Center is supporting further research partnerships.
Lord James O'Shaughnessy, Parliamentary Under-Secretary of State for Health (Lords) with the UK Government, emphasised the government's continued support for the life sciences, in particular for advanced therapies, and for international scientific collaboration.
He said, "The potential to deliver huge leaps forward for huge patient benefit, in some cases cure, makes advanced therapies one of the most exciting areas of medicine. These opportunities and challenge can best be tackled through continued and deepened collaboration."
Speakers from research and industry organisations, regulators and biotech companies shared challenges and solutions relating to the complexity and global nature of developing advanced therapies. Delegates agreed that a proactive approach, such as the Sakigake strategy in Japan promoting research and early practical application of innovative products such as regenerative medicines, is essential to support development and commercialisation in the field.
Magda Papadaki, from the Association for the British Pharmaceutical Industry, UK, considered that comprehensive strategies are needed to provide a swift and viable route to market for these innovative products, together with a long-term regulatory mechanism that takes account of their major differences compared to traditional small molecule medicines.
Progress has been rapid, with at least 53 developers of advanced therapies now working in the UK and more than 50% growth in UK clinical trials since 2013. "But we need to all work together to ensure that we can progress the commercialisation of advanced therapies and turn a cottage industry into a robust and growing market. We need integrated development, regulation and commercialisation for advanced medicines," she concluded.