Interview with NeoStem CEO Robin Smith Part 2: VSEL, ES cells, and iPS cells

I recently did a Q&A interview with NeoStem CEO Dr. Robin Smith. I posted Part 1 of that interview a few days ago here. Now we have part two focused on VSEL, ES cells, and iPS cells.

PK: I frequently have readers of my blog ask questions about VSEL. They seem puzzled and unsure of what to think about these cells and their potential clinical use. Can you tell us in a nutshell what they are, why NeoStem is making such a big bet on them, and why it seems that surprisingly few stem cell scientists work on them?

Smith: Research by a group headed by Dr. Mariusz Ratajczak, M.D., Ph.D., head of the Stem Cell Biology Program at the James Graham Brown Cancer Center at the University of Louisville, co-inventor of VSEL^TM Technology and a member of the NeoStem Scientific Advisory Board, provides compelling evidence that bone marrow contains a heterogeneous population of stem cells that have properties similar to those of embryonic stem cells and hence are referred to as very small embryonic-like stem cells or VSELs^TM. This finding opens the possibility of capturing many of the key advantages associated with embryonic stem cells without the ethical or moral dilemmas and without some of the potential negative biological effects associated with stem cells of embryonic derivation.

Since 2007, NeoStem has been engaged in research and development of new therapies based on VSEL^TM Technology, with the University of Louisville Research Foundation and other academic partners. VSEL^TM Technology offers the potential to go beyond the paracrine effect, yielding cells that actually differentiate into the target tissue and create true cellular regeneration. Our studies to date suggest that human VSELs have the potential to differentiate into cells of each of the three germ lineages in animal models.

NeoStem and our collaborators have shown that VSELs are present in the bone marrow and other organs of humans, and can be mobilized from the bone marrow into the peripheral blood and collected by apheresis. It has been established that these human VSELs are comparable to those described by Dr. Ratajczak. The possibility of autologous VSEL treatments is yet another huge potential benefit to this unique population of adult stem cells.

Recently, NeoStem and its partners at the University of Michigan have demonstrated that VSELs can regenerate bone in a mouse model. NeoStem is seeing progress in animal models of wound healing and further intends to begin the first human clinical trial for VSEL Technology (for periodontitis) in 2013. NeoStem holds a worldwide exclusive license to VSEL^TM Technology and continues to focus on developing these cells into therapies in a cost-effective manner.

VSEL Technology is just one asset in NeoStem’s portfolio, and it is a preclinical asset, so I think our cell “bets” are actually well diversified. The field of scientists looking at these cells goes beyond NeoStem’s affiliates. According to Stanford University’s HighWire press, over 100 articles and abstracts exploring the range of potential of Very Small Embryonic Like (VSELs) cells have been published since 2006. It is also important to note that not all those studying these cells use the descriptor “VSEL”, which was coined by Dr. Ratajczak. A number of articles talk about adult pluripotent and multipotent stem cells that, as described, may well be “VSELs”.

We acknowledge that the final chapter describing the full potential of VSELs is yet to be written, and that controversy exists at this time regarding how that final verse will read.  However, we are proud of our efforts, and those of our collaborators, to get to the truth about these cells, and to apply rigorous scientific method to write the script.  Through the diligent efforts of our team and of our collaborators, as well as other investigators in the field, we continue to develop the required laboratory methods and have shown sufficient preliminary evidence in various in vivo animal models, to earn financial support from both the National Institute of Health and the Department of Defense to initiate clinical studies in humans to further advance the field of knowledge around these cells with data.

We believe that these cells may hold the potential to provide regenerative healing where current therapies fail.  And for this reason we remain excited about our journey with them, and are committed to moving this process forward through in a rigorous, ethical, and scientific way.

PK: Are you personally opposed to embryonic stem cell research? Can you see a unique, meaningful role for embryonic stem cell research and potential clinical applications in specific areas that might not be workable using adult stem cells only? For example, what about the hESC-based clinical trials by ACT for macular degeneration?

Smith: Paul, as we can all agree, the topic of embryonic stem cell science draws great passion from all corners of the cell therapy arena, and in particular, amongst patients and patient advocates.  However, the ‘for’ or ‘against’ terrain often divides us, triggering arguments that can slow collaboration and cures.  Suffice it to say, stem cell research is an incredibly exciting field that holds the promise to treat many serious diseases and debilitating medical conditions. I think we can all agree that the human body provides the most elegant solutions, wherever the cells and great health discoveries will continue to be made.  We are all looking forward to progress on the trials and data from them including the Mesoblast Phase 3 CHF trial, Aastrom’s  Phase 3 CLI trial, Baxter’s  Phase 3 CMI trial, NeoStem’s  Phase 2 AMI trial as well as ACT’s trial for macular degeneration. In the end, we are all fighting for the very same patients who stand to benefit most from safe cellular therapies.

PK: Any thoughts on iPS cells and/or transdifferentiation technologies? Any interest from NeoStem in those technologies?

Smith: NeoStem is not currently investigating induced pluripotent stem cell technologies or transdifferentiation, as our expertise has been in the use of autologous adult stem cells through our background in VSEL™ Technology and CD34 cells through Amorcyte, as well as T cells through Athelos. PCT has worked with cells as diverse as dendritic cells, HSCs, MSCs, T cells, B cells, macrophages, CD34 selected cells, keratinocytes, fibrolasts, porcine islets, and adherent neural stem cells. While we feel iPS cells and transdifferentiation technologies are valid and important fields of inquiry, they just aren’t part of NeoStem’s strategy at this time.