Trends in Regenerative Medicine Late Stage Clinical Development

late stage clinical development stem cells (ed uthman )Late stage clinical development was the flavour of the afternoon on Day One of the World Stem Cells and Regenerative Medicine Congress Europe 2013. Dolores Baksh, Director of R&D at Organogenesis spoke on "Improving product development efficiency using scientific, risk-based approaches". Key to her talk was the implementation of Quality by Design (QbD) principles to give a systematic, scientific, risk-based approach for product development. In the design and development of a product, a company needs to define a quality target product profile (QTPP) – a desired product performance – to lead the development process, something which can evolve throughout the development process. Antonio Lee, Associate Director, Business Development, MEDIPOST, gave a presentation on "Cord blood cells for Alzheimer's disease and bronchopulmonary disease (BPD). He outlined the use of umbilical cord blood for mesenchymal stem cells, and talked us through three MEDIPOST products:

  • CARTISTEM – for degenerative osteoarthritis.
  • PNEUMOSTEM – for BPD (this is an orphan indication). Phase I complete. Mechanism of action is paracrine.
  • NEUROSTEM – for Alzheimer's disease. Phase I complete. Mechanism of action is paracrine.

Yael Margolin, President and CEO of Gamida Cell, gave a presentation wonderfully titled "The Gamida Cell chronicles: diary of a clinical adventure". She took us on a journey through Gameda Cell's TEPA (copper chelator) and NAM (nicotinamide) technology. She explained that as CD34+ cell culture, they differentiate. However, using TEPA, the cells proliferate but undergo less differentation. The umbilical stem cells in the company's StemEx product are expanded using the copper chelator technology. The company's NAM technology can be used to increase quantity and quality of stem cells in culture, and preserve the functionality of expanded cells. She described the use of the NAM technology in their NiCord pilot study, and NiCord in haematological malignancy (Phase I/II completed) and sickle cell disease (Phase I/II ongoing).

Roland Gordon-Beresford, RA and IP Senior Director, Cardio3 BioSciences, gave a presentation on "Balancing financial contrains and regulatory requirements within a clinical development plan for cell therapies". With more patients surviving myocardial infarctions, more patients are now suffering from heart failure. Roland gave a brief case study of C3BS and how they develop regeneative therapies for the treatment of cardiac diseases. He spoke about the C-CURE multicenter Phase II trial, and also the development of C-CATHez –  an intra-myocardial injection catheter to increase cardiac retention of cells and ease of use in the Phase III trial. Phase III is to focus on moderate and severe NYHA Class III and IV patients.

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