8 Years After Prop 71: Industry Perspectives On CIRM
Earlier this week I had the opportunity to testify before a Review of the California Institute for Regenerative Medicine (CIRM) at the Institute of Medicine, to provide some perspective on the team at CIRM, their diligent efforts to advance the cause of stem cell research, and some of the 'blind spots,' from an industry standpoint, of CIRM's strategic plan. Below is the text of my statement to the committee:
April 10, 2012
Testimony of Michael D. West, Ph.D.
President & CEO BioTime, Inc.
Before the Institute of Medicine, Committee on a Review of the California Institute for Regenerative Medicine (CIRM)
Mr. Chairman and members of the Committee, my name is Michael D. West and I am the President and Chief Executive Officer of BioTime, Inc., a biotechnology company based in Alameda, California. A copy of my curriculum vitae is presented in attached Appendix A. I have been asked to comment on the effectiveness of CIRM, in particular to offer constructive criticisms from the perspective of industry. Let me begin by thanking you for the opportunity to speak on the subject of regenerative medicine. There are few areas of human endeavor with the potential to improve human health than this emerging field of medicine. And California’s leadership role as a means of accelerating the development of new cures is a model for the world. Therefore, given the importance of the effort in California, I will not hesitate to offer criticism in the confidence that it will be viewed in a constructive manner.
First, I must state unequivocally that I and many other scientists in the field of stem cell research are great admirers of the team at CIRM and its diligent efforts to advance the cause. Managing the shear volume of their workload is nothing less than heroic. Nevertheless, there is always room for improvement, and I would like to point out what in my opinion are blind spots in CIRMs strategic plan, that could use improvement.
The first blind spot relates to the manner in which new human therapeutic products are developed in the United States. To put it simply, stem cell research by itself will not lead to cures. Research and DEVELOPMENT leads to cures. In my opinion, if CIRM fails to deliver on its goal to deliver cures, it will not be a result of internal governance issues. Instead, it will be a result of inefficient capital allocation. A graphic way of visualizing my point is to say that CIRM has historically funded primarily research, and little product development, i.e. large “R” little “d”. Approximately 5% of CIRM’s expenditures have been allocated to biotechnology and health science entities whose expertise is product development, and 95% has been allocated to nonprofit institutions in the state for basic research. Human therapeutic product development in the United States requires a very intense and expensive process for approval that is primarily focused on development side of the equation. In this respect, therapeutic approvals differ significantly from the discovery and development of silicon-based technologies that have been so successfully commercialized in California.
Sometimes analogies help clarify an otherwise abstract considerations. So let me offer one. Imagine that instead of being a proposition to promote stem cell product development, Proposition 71 was instituted to promote computer technology, e.g. new laptops and smartphones where none previously existed. In the event that 95% of the funds were allocated to prestigious microelectronics research facilities such as the Paul Allen Center at Stanford University, and only 5% of the funds allocated to commercial entities such as Apple Computer and Microsoft, we might easily imagine that Steve Jobs would shut down his computer business shifting to his Pixar investment, and Bill Gates would move to Seattle. This helps us understand the reasons for Geron, the pioneer in commercializing embryonic stem cell-based therapies, shutting down its stem cell business, and Advanced Cell Technology shutting down its California operations moving all operations to Massachusetts. Let me add that these latter real-world events not only represented lost opportunity, but a loss of jobs in California (a net decrease in commercial employees) and a tragic loss of science from discarded programs, reagents, and knowhow.
Another potential blind spot relates to the research side of the equation. Human embryonic stem cells and related induced pluripotent stem cells are promising because to their ability to differentiate into all of the complex cell types in the human body. However, this is also their greatest challenge. They make ALL of the cell types in the human body. This logically leads to the manufacturing conundrum of how do we manufacture purified and identified cell types when thousands of diverse cell types emerge from these cultures. BioTime has benefitted from a $4.7 million grant from CIRM to further the development of ACTCellerate(TM), a novel manufacturing technology allowing the scalable manufacture of over 200 diverse highly purified cell types. This advance has highlighted the urgent need for a “Rand McNally road atlas” for this complex branching tree from pluripotent stem cells. Currently, for all the thousands of cell types that emerge from pluripotent stem cells, little to no information is available in an organized form for the scientific community to help identify the cells (i.e. where the scientist is on the road from pluripotent stem cells to the final desired cell type). As a result, to meet the rigorous standards of the FDA in regard to purity and identity, companies have found themselves paddling upstream against the very difficult challenge of identifying the cells contaminating their potential products. CIRM could provide a critically valuable research function by building an online database that for the first time laid out a roadmap of the cells of human development, their molecular markers, CD antigens, and other markers. Similar to the foundational impact that the mapping of the genome had on science and medicine, the mapping of the “embryome” would lay a broad and effective foundation for subsequent product development worldwide for decades to come (Regen. Med. 2007, 2(4):329-333).
Lastly, a third potential blind spot relates to a possible scenario where CIRM eventually ceases to fund programs in the State. In anticipation of the possibility of this event, there should be plan of transition to avert the possibility of a massive loss of effort, reagents, and trained personnel. Again, a logical solution would be expanded funding in the latter years of CIRM to support the California regenerative medicine industry with the goal of building an “economic engine” to continue the translation of CIRM-funded research into the clinic. In summary, I believe the citizens of the State of California as well as local biotechnology companies appreciate the dedication of the CIRM team, and look forward to finding a path to accelerate these new life-saving therapies to the people in need and thereby fulfill the historic mission of the California Institute for Regenerative Medicine.