As Stem Cell Therapies Move to the Clinic, Regulate for Safety, Don’t Restrict Research
A recent article published in PLoS Medicine reported that human fetal neural stem cell injections used to treat the neurodegenerative disorder ataxia telangiectasia in an Israeli boy caused a multifocal brain tumor four years after his first injection. While it is unfortunate that the stem cell treatment caused such tragic medical complications for the boy, this regrettable development should not be characterized as a deathblow to the validity or usefulness of stem cell therapies, be they neural, adult, or embryonic.
Biological treatments are not drugs—they are difficult to control, and may induce a cascade of unforeseen side effects. As stem cell therapies move into clinical trials here in the United States, it is essential that patients as well as policymakers recognize that tumorigenesis is a potential problem with all pluripotent cells regardless of their source, and that there may be biological barriers inherent in these cells that cannot be overcome.
Nevertheless, we should recognize the great potential of stem cell therapies and focus our attention not on how to restrict stem cell research and therapy development, but on how to develop proper regulations that minimize dangerous side effects and satisfy strict ethical guidelines. As with any powerful disease therapy, like chemotherapy, hormone treatments, or bone marrow transplantation, we must manage the potential deleterious outcomes of stem cell therapies but never forfeit their unique potential to save lives. The authors of the journal article agree:
Stem cell therapy and neural stem cells, in particular, provide hope to patients affected by devastating diseases. However, such innovative treatments also carry substantial risks and the potential for malignant transformation of transplanted cells has been raised . . . Our findings do not imply that the research in stem cell therapeutics should be abandoned. They do, however, suggest that extensive research into the biology of stem cells and in-depth preclinical studies, especially of safety, should be pursued in order to maximize the potential benefits of regenerative medicine while minimizing the risks.
It is important that the FDA and other regulatory agencies take a proactive approach to evaluating the safety of these new therapies, and work diligently to ensure that they are developed and applied safely and ethically. There are going to be many bumps along the road to safe and effective stem cell therapies, but the promise of regenerative medicine is far too great for us to quit now, or to take our eye off the goal of patient safety.
For more on the status of biotechnology regulation around the world, see Richard Hayes’s SP article, “An Emerging Consensus.” For the CAP/Science Progress recommendations on the regulation of stem cell research and innovation, see the report from Michael Rugnetta and Michael Peroski: “A Life Sciences Crucible.”
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