LRMN and Cell Therapy Catapult: meeting report

Written by Alexandra Thompson

A summary of the excellent meeting hosted by LRMN and Cell Therapy Catapult

Yesterday’s meeting held three wonderful, engaging talks by experts in regenerative medicine. Sadly Dr Sven Kili was unable to present as originally planned, but Dr Natalie Mount very kindly stepped up to present instead. Below is a summary of their presentations.

The UK’s clinical pipeline of cell therapies, by Dr Natalie Mount, Cell therapy Catapult (London, UK)

The UK is a leading nation in the research and clinical translation of cell-based therapies. The Cell Therapy Catapult has compiled databases of the cell therapies in clinical trial and late preclinical development in the UK and this shows a strong and diverse portfolio, but also highlights the translational challenges faced. In this talk, Natalie gave an overview of the therapies in development in the UK, highlighted the new types of therapy now progressing to the clinic and reflected on some of the hurdles we are now addressing towards successful development, licensing and commercialisation.

Are tissue-engineered organs disruptive technology?, by Prof Martin Birchall, UCL Ear Institute (London, UK)

Conventional organ transplantation has revolutionised healthcare in the last 50 years. However, it continues to have limitations, including limited donor pools, ethical and technical issues, and the need to place patients on long-term immunosuppression, with consequent significant side effects. The advent of stem cell and cell-based therapies and organ replacement and regeneration (‘regenerative medicine’) has led to some offering this new technology as a future alternative for patients with end-stage organ and tissue failure. Some high-profile clinical successes have raised expectations, but there remains a substantial gap between what is presently possible and the holy grail of customised organ replacements for all. The talk discussed what progress has been made, and whether regenerative medicine willever become truly disruptive technology for mainstream healthcare.

Some Q&A after the talk discussed topics such as achieving research and clinician collaboration, and the likelihood that disruptive technology will ultimately use a combination of synthetic and decellularised technology.

Human embryonic stem cell-derived cardiac progenitors for cardiac repair: a translational experience, by Prof Philippe Menasché, Hôpital Europeén Georges Pompidou (Paris, France)

Philippe kindly came all the way from Paris to present to the network yesterday, so merci Philippe! He stated that the preclinical recognition, in both small and large (nonhuman primate) models, that cardiac-committed cells could repair chronically damaged hearts, led him to undertake a translational program for testing human ESC-derived cardiac progenitors in patients with advanced heart failure. The steps of this program included the expansion of a clone of pluripotent hESCs to generate a master cell bank under GMP conditions, a growth factor-induced cardiac specification, the incorporation of these cells into a fibrin scaffold, safety assessments, and extensive cytogenic and viral testing. The data collected throughout the process led to the approval by the French regulatory authorities of a first-in-man clinical trial of transplantation of these SSEA-I+ progenitors in patients with severely impaired cardiac function, who are currently being screened.

One comment I found particularly interesting was how challenging it is to find patients to enrol in the trial. Also, in the Q&A Philippe very eloquently described how he saw the future of stem cells: becoming biofactories for growth factors, meaning a cell-free cell therapy. However, much work needs to be undertaken to understand the critical factors in the secretome that provide therapeutic effect.

RegMedNet would like to thank LRMN and Cell Therapy Catapult for organising and hosting this insightful meeting. We had a wonderful time!