The focus of the 1st ATSM was on Advanced Therapies, one of the most recent and significant advances in medicine. They include cell, gene and tissue engineering therapies to not only treat disease symptoms, but to potentially provide a cure for otherwise incurable chronic diseases. Advanced Therapies, also known as “living drugs”, are a disruptive medical innovation, i.e. an innovation that is changing healthcare and the way drugs are developed and used in clinical routines.
Prof Hans-Dieter Volk opened proceedings with an inspiring presentation on the aims and vision of the large-scale research initiative RESTORE. RESTORE aims to accelerate the availability of “living drugs” to all those in need by supporting collaboration throughout the transdisciplinary network required during the entire drug development trajectory, from inception to implementation and by helping to overcome regulatory, scientific and technological roadblocks that stand in the way of such a trailblazing change.
On day 1, we heard how the knowledge gleaned from foundational research is being used to identify new targets and indications for the development of novel Advanced Therapies. We also heard about new developments in the preclinical testing of Advanced Therapies, such as organ-on-a-chip systems, which are more suitable than traditional animal models. The third part of the day was dedicated to innovation in manufacturing technologies and how it may disrupt the entire field of Advanced Therapies and pave the way for reproducible, high-grade “living drug” products using current manufacturing structures.
We were truly honoured to have Nobel Prize winner Ada Yonath open day 2. She talked about her research into the ribosome and the opportunities her findings have provided for overcoming antibiotic resistance. Her talk on the translation of findings from foundational research into meaningful healthcare products bridged the topics discussed on day 1 to those discussed on day 2, bringing Advanced Therapies into the clinic. We heard about the unique regulatory challenges associated with authorising Advanced Therapies and the new reimbursement models required to pay for them. The issues of ethics, big data and public engagement surrounding Advanced Therapies were covered in a special focus session, which highlighted how machine learning could be applied to clinical development how ethics can be used to advance research and about the dangers of the dissemination of inaccurate information to the public.
Across the two days, a stunning array of topics, visionary research approaches and success stories in Advanced Therapy development were discussed, as well as the challenges that their widespread implementation still faces. Bringing together the whole of the Advanced Therapies community is part of what RESTORE is about. It was truly motivational to see and hear from so many people sharing a common goal: to ensure that ATMPs are implemented as a standard of care across Europe and beyond. We are grateful to everyone who took part and made the 1st ATSM a success!