Element Z (Stem Cell-based Therapies for Clinical Use)

Element Z translates discoveries to the clinic as highlighted by groundbreaking technologies and treatments at Stanford University. Remarkably, in approving Proposition 71, the citizens of California sought to use state funding to promote research that may provide novel cell-based cures, or knowledge from the biochemistry and genetics of purified stem cells to produce treatments for common and uncommon diseases that afflict our citizens. Stanford University and the SoM are renowned for their excellence in basic science, fundamental discovery and innovation. The critical groundwork for extending these strengths into translational and clinical research began six years ago. As noted above, under the leadership of Dean Philip Pizzo, the SoM began a strategic analysis and redefinition of its basic and educational structures in 2001, with the goal of defining and implementing a new core mission: Translating Discoveries. This mission aims to promote the translation of discoveries from the laboratory to the bedside into the community and then back again from the community and bedside to the academic laboratory in a highly iterative process. These changes are transforming Stanford into a leader in the new discipline of Clinical and Translational Science. SIM1 targets stem cell biology as the first outgrowth of an ever-increasing effort that highlights our commitment to translational research. SoM faculty members are currently engaged in 930 projects involving human subjects.

We believe that Stanford University, the SoM, and the SISCB/RM are uniquely positioned to lead the way for stem cell research in the areas of development and clinical research, and Lokey Stem Cell (SIM1) nucleates the underlying discovery process that will feed this pipeline. Perhaps the most important question in evaluating “Element Z” at Stanford is to ask about our track record of accomplishments in the spectrum from fundamental discovery to clinical translation. In short, Stanford is a world leader in these areas. Table 2 below lists a highly select group of our groundbreaking technologies and treatments that have their roots at the Medical Center.

Table 2.  Selected highlights of groundbreaking technologies and treatments at Stanford


First development of high energy (6MeV) linear accelerators for cancer therapy (with Varian)


Discovery that mutations in a single gene cause the most common type of skin cancer


First significant cures of a previously incurable cancer, Hodgkin’s Disease


First use of purified HSC in clinical trails


First isolation of any adult stem cell, mouse HSC


First use of gene profiling to predict cancer outcomes


First isolation of any homing receptor, the separate lymphocyte homing receptors for lymph nodes, peyers patches and mucosa in mouse and man


First isolation of human brain stem cells(with Stem Cells, Inc)


First isolation of human HSC (with SyStemix, Inc)


Discovery of molecular mechanisms that prevent regeneration in the CNS


First use of a monoclonal antibody therapy (rituximab) for human cancer


First to show MRI patterns to show stroke response patterns


Development of microarray technology


First demonstration of chemical genetics to rescue a birth defect in mice

To further support Stanford’s leadership nationally in innovation, the School of Medicine has 3 of 17 living Stanford Nobel Laureates, 135 members of the National Academy of Sciences, and 51 members of the Institute of Medicine. Finally, in a national measure of pioneering health research, Stanford University faculty members have received 9 out of 46 National Institutes of Health Pioneer Awards (more than any other Institution in the US). The contributions and efforts of our faculty were lauded in the CIRM critiques of Part I of this proposal. The reviewers noted in Element Z that this institution has pioneered efforts to translate stem cell therapies to the clinic.”

Interdisciplinary collaborations distinguish Stanford and fuel our discovery engine. Based on our track record and proven accomplishments, we are confident that we will achieve our goals in all elements. Our combined efforts build on various Schools of Stanford University, including Business, Law, Humanities and Science, and Medicine, and occur in alignment with principles guided by the Stanford Center for Biomedical Ethics (SCBE) and the internationally-recognized Program in Stem Cells and Society. Each of these programmatic elements and key resources are located within a few minutes’ walk from SIM1. Indeed, our highly interdisciplinary collaborative approach is one of our hallmarks. Our Part I review noted, “Several reviewers felt this collective expertise in the area of stem cell biology is unique to this university.”

In addition, we have established a model of deep collaboration in situ rather than at a distance, which we believe will accelerate translational and clinical research. At our current offsite facility we have piloted a new and highly successful method of expanding faculty involvement in collaborative stem cell research. Approximately 20% of the benches are reserved for collaborations between specific faculty and fellows. Initially collaborations have been in the cancer stem cell arena, with clinical physician scientists and basic scientists each contributing expertise, and usually one fellow each, to study a specific problem, such as the isolation of a variety of human cancer stem cells. We have reserved up to 60 such benches in SIM1 to make sure that translation is carried out by resident scientific groups and those allied to solve a particular problem, regardless of their home space and department location. The use of these collaborative benches will be extended to our collaborators outside Stanford as well.

Finally, we are establishing a formal doctoral degree-granting program to train the next generation of leaders in stem cell research and augment our expanding training program in stem cell biology and regenerative medicine. The current highly-successful CIRM scholars program trains predoctoral, postdoctoral and clinical fellows, MD medical scholars with a concentration in preclinical research, undergraduate students, and visiting scholars.  

The strategic scientific agenda outlined above is guided by the SISCB/RM Steering Committee, a group of senior faculty leaders in the SoM, who have shaped the vision and established critical parameters for stem cell research in each of the X, Y, and Z elements, including: new faculty recruitment efforts, core service needs, expansion of resources and research space, space allocation in SIM1, and fundraising. This committee, with the help and input from faculty across the entire Stanford campus, has played a central role in defining SIM1 design criteria and will continue to guide the SISCB/RM as we move forward. The SISCB/RM Steering Committee comprises: Philip Beachy, Steve Galli, Jonathan Berek, Karl Blume, Robert Negrin, Richard Myers, Michael Clarke, Michael Cleary, Mark Davis, Ronald Levy, Michael Longaker, Beverly Mitchell, William Mobley, Theo Palmer, Roeland Nusse, Renee Reijo Pera, Lucy Shapiro, Dean Philip Pizzo, and Irving Weissman. The chair of the Steering Committee is appointed by the Dean of the SoM for renewable 5-year terms. Irving Weissman, Director of the SISCB/RM, is Steering Committee chair. We are also guided by an external advisory committee consisting of Austin Smith (Cambridge University), Rudolf Jaenisch (MIT/Whitehead Institute), Rob Krumlauf (Stowers Institute), and Doug Melton (Harvard University) that will meet annually to advise the Director.

With the design and building of SIM1, as outlined below, we believe we can catalyze the transition from vision to reality and enhance our movement of studies from the basic and preclinical stage to clinical trials. Our history bears record to that possibility.

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