Conference Reports
4th World Stem Cells & Regenerative Medicine Congress
Royal Horseguards, London, UK, 12-14 May 2009
This year's Regenerative Medicine meeting changed from being the European Congress to the World one, causing opening speaker, Dr. Chris Mason, to joke '.and next year, the universe!'. But it was perhaps fitting given the global nature of the regenerative medicine field that this was the case. Although the Congress is still relatively small, it is, like the field of regenerative medicine itself, growing fast, with attendance this year topping 200 for the first time in its history. Accordingly, we may see a new venue for this intimate and friendly conference in 2010, with the now-familiar Royal Horseguards being filled almost to capacity with both familiar and new faces this year.
In 2009, the focus was on 'RegenMed 2.0' - the need to translate lab-based research into actual clinical benefits. This was reiterated and extended this year, with speaker Gregory Bonfiglio of Proteus Venture Partners giving an informative and interesting speech on how the funding for regenerative medicine is becoming more mature and really moving from lab to clinic. The effect of the global recession though was, as ever, evident, with Mr. Bonfiglio also stating that funds, particularly from venture capital, will be very hard to come by in the next year or so and that some companies would, regardless of the strength of their actual science, inevitably fall by the wayside in the face of this. This bad news was tempered though with the assurance that there is still money out there for this most promising of fields. The main sources of this future funding then were identified as governments and, perhaps more interestingly at least as Pharmaprojects is concerned, through the intervention of 'Big Pharma'.
This year's meeting was particularly noteworthy in two respects - firstly the increased number of talks relating not just to academic work and proof-of-principle studies, but of actual clinical results and regenerative medicine products reaching the market. The second reason to take heart from this meeting was the noticeably increased attendance by Big Pharma. Perhaps they are finally sitting up and taking notice? We hope so, as this partnership is essential to the future of at least the RegenMed industry, if not the 'classic' pharma industry as well. Although the first successes of RegenMed are relatively small, it is clear that the potential is enormous and, as many speakers and CEOs stated, this fledgling industry must learn to walk before it can run. The involvement of big Pharma at this stage only highlights that these early regenerative medicine-based companies are starting to become interesting to companies whose turnovers rate in the hundreds of billions of dollars and more; hopefully this will prove a partnership mutually beneficial to both.
Day One then, was appropriately titled 'Translating Research into Commercially Successful Therapeutics', and the first company to present on some of the therapeutic applications of stem cells was Baxter International, whose head of regenerative medicine R&D, Dr. David Amrani, provided an update on his company's CD34+ stem cell therapy for ischaemic conditions. Results from a Phase IIb trial in 167 chronic myocardial ischaemia patients who had failed standard therapies showed a clinically significant reduction in anginal episodes over six months. A two-year follow-up study is now ongoing. Dr. Amrani was followed to the stand by CEO of Osiris Therapeutics, Dr. Randal Mills. Dr. Mills focused on Osiris' allogeneic mesenchymal stem cell therapy, Prochymal. Prochymal is primarily targeted to treat graft-vs-host disease, a serious condition for which there is currently an unmet medical need. Prochymal shows astounding results in this patient population, with a 77% complete response rate observed in Phase II trials. Phase III trials are underway and a rolling BLA has been submitted. Osiris is also pursuing Prochymal in Crohn's disease, chronic obstructive pulmonary disease and acute radiation syndrome. Prochymal represents well the current thinking by many companies in the regenerative medicine field: to get on the first few rungs of the ladder by targeting a niche disease which is currently under-served by the Pharma industry.
Following on from this, Day Two was entitled 'How Regenerative Medicine will meet Unmet Medical Need' and was kicked off by Dr. Ole D Madsen from Novo Nordisk, who, unsurprisingly given his company's primary focus, gave an interesting presentation regarding the use of stem cells to treat, and possibly one day even cure, diabetes. Novo Nordisk is examining the latter of these two possibilities by developing, in collaboration with the embryonic stem cell-based company Cellartis as well as Lund University, insulin-producing cells from stem cells. Dr. Madsen's talk was appropriately followed by Dr. Ed Baetge of Novocell, whose company is taking a slightly different approach by using PEG-based encapsulation technology to protect allogeneic cells from the immune response. Novocell is planning a Phase I trial in type 1 diabetes and expects to file an IND in 2011.
There were two main topics which were a recurring theme of this year's Congress, namely the questions regarding the use of embryonic vs induced pluripotent adult stem cells and the autologous vs allogeneic models of stem cell therapy. The next presentation, from Eduardo Bravo, CEO of Cellerix, touched on both of these, beginning with its lead therapy Ontaril. Ontaril is an autologus cell product, based on expanded mesenchymal adult stem cells and is initially under development for perianal fistulas in patients with and without inflammatory bowel disease (IBD), including Crohn's disease. Ontaril is currently in Phase III trials in these indications and filings and launch are expected in 2010 for non-IBD perianal fistula and a year tor two later for fistula associated with IBD. Cellerix is also pursuing an allogeneic version of Ontaril, under the code Cx-601, and this is currently in a Phase I/IIa trial in Spain. Allogeneic 'off-the-shelf' versions of stem cell therapies hold clear advantages over autologous types, primarily in the distribution and business models that must be used to market any such product, with many in the industry maintaining that the autologous model is simply not viable. Of course, many others believe that it is and this will undoubtedly be a major issue to resolve within the next few years of stem cell development. Mr. Bravo also showed very positive results from a Phase II trial of its chimaeric skin product Cx-501 in epidermolysis bullosa as well as preclinical data with Cx-611, which is targeted against inflammatory conditions.
Following a highly interesting panel session entitled 'A Big Pharma Perspective on Future Commercialization of Stem Cell Therapeutics', we heard from Dr. Lynn Allen-Hoffman, CEO and CSO of a company new to Pharmaprojects, Stratatech. Its lead product Stratagraft, consisting of Stratatech's proprietary NIKS (Near-diploid Immortalized Keratinocytes that form Skin) cells, for wound healing, has completed a Phase I/IIa trial in which it proved safe and efficacious. Taking this premise a step further, Dr. Hoffman also introduced their second-generation version, Expressgraft, which is similar to Stratagraft with the difference that the cells have been genetically modified using non-viral vectors to overexpress endogenous human anti-bacterial, anti-protease or pro-angiogenic factors. This novel technology has been positively tested in a mouse model, using cells overexpressing the naturally-occurring antibacterial cathelicidin, and a Phase I trial is now under preparation.
The penultimate speaker of Day Two was Dr. Ram Mandalam of Cellerant Therapeutics, a company focused on the regulation of the haematopoietic system. Their myeloid progenitor-derived product, CLT-008 has entered a clinical trial in the US in patients with haematological malignancies. In addition to this, Cellerant intends to pursue CLT-008 for chemotherapy-induced neutropaenia and acute radiation syndrome. Interestingly, Cellerant is also using its knowledge of stem cells to target cancerous stem cells using antibody therapies. Dr. Mandalam reported that they have identified seven antigens linked to leukaemic stem cells and that preclinical candidates against these targets should be identified by the end of the year. Although the antibody program will be pursued in-house, Cellerant also intends to out-license small molecule candidates against these targets.
This year's Congress certainly built on the success of last year to deliver a highly informative and yet friendly affair, and amongst the familiar faces it was gratifying to see new attendees as well, particularly with respect to those representatives of big Pharma who will undoubtedly prove essential to really translating stem cell science from the bench to the clinic. Along with the science of course, it was also the business side of this process which was a central theme to this year's meeting and again, this can only prove good for the industry. The Stem Cells and Regenerative Medicine Congress, rather like the science as well as business it represents, goes from strength to strength and we already look forward to next year's event.
Thomas Stirzaker
Editorial Analyst
The 5th World Stem Cells & Regenerative Medicine Congress will be held in London, UK, 12-13 May 2010.
