Three recent papers and questions for clinical translation
It's not easy to assess the quality of science from press releases, three of which caught my eye this month. All of them described the use of stem cells for neurodegenerative applications. All of them looked interesting, but I wanted to be particularly careful because of their potential for translation to human patients as well as their company connections. I asked Phil Schwartz, a neural stem cell expert at Children's Hospital of Orange County, California, to help me understand the gaps between study and therapy, and then I asked authors from each paper to respond. The papers were published in Biomaterials1, The Journal of Comparative Neurology2 and Cell Transplantation3.
Some of Schwartz's skepticism about this research can be applied to any translational paper. A sample of his concerns:
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It's hard to make any conclusions about what kind of engraftment you're going to get or how effective it is going to be in a short-term experiment.
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This is the basic issue of all transplantation studies: you really have to know what your disease is and what your target should be.
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Although the studies have shown that their techniques are feasible and safe, they have not shown that the treatments have any kind of efficacy, and they don't have the proper controls to do so.
In all of these accounts, Schwartz's comments are transcribed from an interview. Unless otherwise noted, author comments are e-mailed replies to my e-mailed questions.
In-depth look at the Biomaterials paper: Stem cell–studded scaffolding fills holes left by simulated strokes in rat brains
In-depth look at the The Journal of Comparative Neurology paper: Human motor neurons engraft in a rat model of Lou Gehrig's disease
In-depth look at the Cell Transplantation paper: Eight spinal cord injury patients report improvement after surgery that included infusions of their own bone marrow stem cells
References
Bible, E. et al. The support of neural stem cells transplanted into stroke-induced brain cavities by PLGA particles. Biomaterials 10.1016/j.biomaterials.2009.02.012 (published online 9 March 2009)
Xu, L., Ryugo, D.F., Pongstaporn, T., Johe, K. & Koliatsos, V.E. Human neural stem cell grafts in the spinal cord of SOD1 transgenic rats: differentiation and structural integration into the segmental motor circuitry. J. Comp. Neurol. 514, 297–309 (2009)
Geffner, L.F. et al. Administration of autologous bone marrow stem cells into spinal cord injury patients via multiple routes is safe and improves their quality of life: comprehensive case studies. Cell Transplant 17, 1277–1293 (2008)
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Baker, M. Stem cells and neurodegenerative disease: cool science and scepticism. Nat Rep Stem Cells (2009). https://doi.org/10.1038/stemcells.2009.54
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DOI: https://doi.org/10.1038/stemcells.2009.54