Ground reality on Science, Manufacturing, Patient need and Outcome
Stem Cell Society Singapore Symposium ‘Opportunities & Challenges in Stem Cell Based Medicine’
(held 17-18 November 2015; Biopolis, Singapore)
Stem cells and its therapeutic derivatives are striving to be a first-in-class medicine. Living cells have now become the active pharmaceutical ingredients (APIs) and hold a regular share in the pharmaceutical and biotech innovation and delivery pipelines. Critically ill patients left with nearly no other options are often pinning their last hopes on infusions of these fascinating stem cells. Are these cells and the science behind them capable of delivering the promise? How close are we in realizing the immense potential of stem cells?
The Stem Cell Society Singapore Symposium 2015 set out to answer these questions. This meeting was meticulously designed to give fellow stem cell scientists and industry partners an excellent platform to understand the various key steps in delivering the stem cell promise.
The conference started on a buoyant note with speakers from Center for Commercialization of Regenerative Medicine (CCRM) and GE Healthcare emphasizing on the importance of commercialization, funding ideologies, GMP methodologies to incorporate in routine lab. This gave the scientific community a much deserved, practical foothold or grounding. The speakers from CCRM, Michael May and Kim Warren spoke about how CCRM enables commercialization of projects from their academic network by matching them with relevant partners from an industry consortia of 50 companies and how they are developing an investors network to develop and support the later stages of the projects. CCRM is currently expanding by franchising out CCRM hubs to the Asia Pacific region, bundling the intellectual properties (IPs) and eventually using the hubs to gain access to the markets. Phil Vanek from GE Healthcare gave an overview on the cell therapy manufacturing initiative which focuses on integrating the unit operations both physically and digitally, closing the manually operated ‘liquid circuity’ and transitioning towards automated systems, simulating the factory operations to estimate, evaluate and review the labor and manufacturing costs.
This will lead to better digitalization of the manufacturing processes and allow their scale up in an economically viable manner. Singapore well on its way to digitalization and during its evolution as a smart city will see a lot of these good manufacturing practices finding its way in routine bioprocess and biotech manufacturing as well.
Key points to consider before converting your lab practices to GMP mode or before embarking on a scale up operation:
1. Make sure the biology works.
2. Purity, potency and stability of raw materials, intermediates as well as final products has to be established and maintained.
3. Availability of reliable vendors, secondary suppliers, complete documentation like certificates of analysis, any new changes need to notified to the customers to keep them updated on any slight modification to the product.
4. Carefully consider which processes will have to fall under GMP mode and which can continue in a non-GMP mode according to the particular regulatory processes the products will be passed through.
5. Process is as important as the products and need to be protected by IPs.
There were a series of talks and posters driven by scientific and industry fervor showcasing some of the leading scientists both locally and internationally at the symposium.
Awards for the best poster went to Poulomi Banerjee, Manipal University; Jamie Trott, Institute of Medical Biology, A*STAR and Yi Wang, The Chinese University of Hong Kong.
The first day closed with an interesting keynote from Dr John Rasko who has a proven track record of gene and stem cell therapies and currently leads the Gene and Stem cell Therapy program at the Centenary Institute, University of Sydney, Australia.
His talk titled,’The Cell and Gene Therapy soup: Disentangling hope, realism and quackery’ gave an elaborate review on results from multiple clinical trials reported worldwide in 2014 (an excellent summary can be found in blogs by Alexey Bersenev referenced below). The current upheaval by the immune cell therapy and more recent Chimeric Antigen Receptor – T cell autologous therapy (CAR-T cells proven effective in chronic lymphocytic leukemia) have been very promising as elucidated in the number of trials involving these cells types and in their success indicated in the charts below:
A general timeline for the realization of stem cell based therapies to the clinics have been approximately 25 years and he opined that this is where the regulatory network has failed the patients as their overly stringent regulations have prevented the accelerated discovery process. Stem cell based therapy enjoys a sweet spot with a profitable economics of scale and break-even point despite the delayed return on investment.
With Japan’s recent review and amendments of the regulatory framework, pharmaceutical and biotech players across the world have joined the competition and fresh investment is being made available by the stem cell therapies to aid in the rapid advancement of stem cell based therapies. It is daunting and challenging to bring stem cells to the market but there is a promise waiting to be investigated and if proved can become disruptive and worthwhile.
1. Bersenev Alexey. Cell therapy clinical trials – 2014 report. CellTrials blog. January 22, 2015.
2. Bersenev Alexey. Results of regenerative medicine clinical studies from 2014. CellTrials blog. March 1, 2015.