Stem Cells Forum: Working Towards Clinical Application - 03 October 2012

Stem Cells Forum: Working Towards Clinical Application

The Stevenage Bioscience Catalyst, Gunnels Wood Road, Stevenage

Wednesday, 03 October 2012

This event is discussion forum, focused on clinical applications of stem cell therapy. The aim is to offer participants a chance to explore aspects with the experts during round table and one-to one discussions.

Meeting Chair: Dr Glyn Stacey, UK Stem Cell Bank, Division of Cell Biology and Imaging.

This event has CPD accreditation
On registration please submit your questions to the panel that will be asked by the chair on the day of the event

THIS EVENT IS PART OF THE 2012 EUROSCICON STEM CELL SERIES

The day before after event there is The 3rd Annual Induced Pluripotent Stem Cells: Production and Utility in Regenerative Medicine

See www.regonline.co.uk/2012londonstemcellevent for more information

9:00 – 9:30 Registration

9:30 – 9:35 Introduction by Meeting Coordinator: Dr Astrid Englezou , Euroscicon, London, UK

9:35 – 9:45 Introduction by the Chair: Glyn Stacey, UK Stem Cell Bank, Division of Cell Biology and Imaging.
9:45– 10:05 Challenges in the Delivery of Cell-based Medicines from Pluripotent Stem Cell Lines

Glyn Stacey, UK Stem Cell Bank, Division of Cell Biology and Imaging.

It is vital for those developing new cell-based medicines that they consider certain critical issues that will influence the success of their products. The suitability of the proposed cells to be used for manufacturing is fundamentally similar for products where there is one donor tissue serving many recipients or one donor tissue to one or few recipients i.e. transplantation. However, in the former case the use of in vitro expansion brings a number of additional concerns and regulatory issues that must be addressed. This presentation will address these issues and describe how some groups have worked together to address some of the key regulatory challenges and create a UK platform for delivery of seed stock of stem cell lines for clinical use.

10:05 – 10:25 Clinical grade human embryonic stem cells
Dusko Ilic, Kings College London School of Medicine, UK

hES cells are undifferentiated cells derived from an early embryo that can grow in vitro indefinitely, while retaining their capability to differentiate into specialized somatic cell types. Their use in therapy and regenerative medicine as well as in toxicity screening and drug development is widely anticipated. However, even if we pay no attention to ethical, religious and political issues that relate to hES cells, there are still a number of obstacles to be resolved before these cells can be broadly used for cell-based therapy. The talk provides an overview of the current status and the future perspective of the field from the point of view of the standard level of patient safety and efficacy for the healthcare industry.

10:25 – 10:45 GMP compliant manufacture of a 3-D airway for regenerative surgery

Edward Samuel, University College London, UK

10:45 – 11:15 Mid-morning Break and Poster Session

Please try to visit all the exhibition stands during your day at this event. Not only do our sponsors enable Euroscicon to keep the registration fees competitive, but they are also here specifically to talk to you

11:15 – 11:35 A Vision for the Future of Blindness – Stem Cells and Age-Related Macular Degeneration

Amanda-Jayne Carr, The London Project to Cure Blindness, Institute of Ophthalmology, University College London, London, UK

Age-related macular degeneration (AMD) is the leading cause of blindness in the western world and, with an increasing ageing population, the prevalence of this disease is set only to rise. AMD is associated with the degeneration of the macular region of the retina, leading to loss of high acuity central vision required for fine details tasks, such as reading and recognising faces. The underlying cause of AMD appears to stem from loss of, or defects within, the retinal pigment epithelium (RPE), a monolayer of cells found between the retina and choroid of the eye. As a single monolayer of cells associated with devastating disease, for which there are limited treatments, the RPE is an ideal tissue for stem cell therapy.

The London Project to Cure Blindness is a research programme established in 2007 with an aim to prevent blindness, restore sight and ultimately improve quality of life for AMD patients using stem cell therapy. We have generated and extensively characterised RPE derived from human embryonic stem cells and human induced pluripotent stem cells in vitro and in vivo. Having demonstrated that stem cell-derived RPE cells are capable of maintaining visual function in an animal model of retina disease, the project is poised to embark on human clinical trials using human embryonic stem cell derived RPE.

11:35 -11:55 Regulatory challenges - myths and misconceptions

Ian Rees, The Medicines and Healthcare products Regulatory Agency UK and Elaine Godfrey, The Medicines and Healthcare products Regulatory Agency UK

11:55 – 12:15 Bootstrapping across the Valley of Death

Dr. Joanna Miller, Quy Biosciences Ltd, UK

Many regenerative medicine companies are attempting to cross the Valley of Death with the ultimate goal of receiving market authorisation for their stem cell product but despite the help offered by the TSB, CATAPULT and other grant funding we are often being advised to ‘bootstrap’.

This talk will suggest an alternative method of ‘bootstrapping’ which would result in early revenue and additional proof of concept for stem cell and other regenerative medicine products.

12:15 – 12:30 Working Lunch and Poster Session

Please collect your lunch and take it to your discussion table

This is also a good time to fill out your feedback forms

12:30 – 13:45 Discussion Group Sessions (sessions 1 – 3) Round table discussion groups (20 minutes each) will be held throughout the afternoon

• Delegates will rotate so that they may participate in all the discussion tables
• All delegates will also be allocated a session for visiting the exhibition stands
• Where appropriate delegates will be able to bring their samples to the discussions
• See end of agenda for description of discussion tables

13:45 – 14:15 Chemical Screens for regenerative medicine

Dr Davide Danovi, Progenitor Labs, UK

Progenitor Labs is a new drug discovery company launched to isolate chemicals acting on specific progenitors of the human body. These cells emerge from stem cells and give rise to different cell types. Because they are transient in nature, they are quite difficult to isolate with current technologies. Progenitor Labs derives progenitors from stem cells using innovative stem cell technologies and uses chemical screenings to isolate therapeutics targeting endogenous progenitor cells of the human body for specific indications.

14:15 – 15:30 Discussion Group Sessions (sessions 4 – 6)

15:30 – 16:30 Question and Answer Session

This session will include summing up of the discussion tables and questions submitted both prior to the meeting and throughout the day

16:30 Chairman’s Summing Up and Feedback Prize Draw

Discussion tables

Table A: Glyn Stacey

Table B: Dusko Ilic

Table C: Ian Rees and Elaine Godfrey

Table D: Edward Samuel

Table E: Amanda-Jayne Carr

Table F: Joanna Miller

About the Chair
Glyn Stacey, Head of the Division of Cell Biology and Imaging at NIBSC and Director for the UK Stem Cell Bank.
His scientific background has been in microbiology and cancer research. From 1989-1998 he worked at Porton Down, UK, where he developed cell banking procedures and the development of cells for manufacture of medical products and cell-based diagnostic assays. At NIBSC he has developed a broad remit relating to the quality and safety of new biological medicines and therapies based on the use of human and animal cells. He has also acted as an advisor to the UK Department of Health and the World Health Organization. He coordinates the International Stem Cell Banking Initiative funded by a consortium of funding agencies from 20 countries. He has recently overseen the establishment of a new and expanded GMP facility for banking stem cell lines. He has published numerous scientific papers and books on cell banking and quality control.

About the Speakers
Dusko Ilic obtained his MD degree and BSci in Molecular Biology at the University of Belgrade, PhD at the Tokyo University, Japan, and postdoctoral training at the University of California in San Francisco. Before joining King’s College School of Medicine in London as a Senior Lecturer in Stem Cell Science, he held positions of Adjunct Associate Professor at the University of California San Francisco, Consultant at the Veteran Affairs Medical Center, San Francisco, and the Director of R&D at StemLifeLine, a California-licensed stem cell company. His current research interest lies in human embryonic stem (hES) cells, induced pluripotent stem (iPS) cells, cancer stem cells, and regenerative medicine.

Amanda-Jayne Carr obtained a Dual Honours BSc in Biology and Biochemistry at Keele University in 1998 and PhD in Circadian Rhythms from the University of Manchester in 2002. She then moved to the Anatomy and Developmental Biology Department in University College London to study the effects of light on circadian rhythms in embryonic cells. In 2006 she moved to the UCL Institute of Ophthalmology, a world leader in eye research, where she examined the potential for human RPE cells to transdifferentiate into retinal cells. Amanda joined the London Project to Cure Blindness in 2007. The aim of the London Project is to develop of cellular therapeutic for the treatment of age-related macular degeneration. Amanda has been involved in the initial basic science, developing protocols to derive RPE from human embryonic stem cells and assays to characterise the resulting cells in terms of morphology and function. She is now working with induced pluripotent stem (iPS) cells and developing protocols to derive patient-specific RPE cells from patients with retinal diseases.
Joanna Miller is a biochemist who has specialised in control of cell differention. She worked in Research for Glaxo for 10 years and is now the Science Director of Quy Biosciences Ltd, a stem cell company trading as VetCell.

Davide Danovi works as principal scientist in Progenitor Labs, a biotechnology company founded to isolate chemicals for regenerative medicine using innovative stem cell technology. He was previously Research Associate at University College London and University of Cambridge in the groups of Prof. Austin Smith and Dr. Steve Pollard.. He developed a live image based high content screening platform to isolate drugs specifically targeting brain tumour stem cells. He trained as an MD and had his PhD at the European Institute of Oncology, one of the biggest comprehensive cancer center in Europe where he demonstrated the causative role of the HdmX protein in human cancer.

Keywords: Clinical Trial Partnerships, stem cells, banking,manufacturing,scale-up,GMP, regenerative,IPS, ATMPs, somatic, stem cells, constructs, human embryonic stem cells, clinical grade, cGMP, macular degeneration, retinal pigment epithelium, RPE, AMD, Clinical development, Valley of Death, Bootstrapping, Early revenue, Proof of concept, progenitors, chemical screening, high content