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Summary
A last-ditch strategy to beat back autoimmune disease is to destroy a patient's immune system with a blitz of chemotherapy and radiation and then perform a bone marrow transplant to, in essence, reset the immune system. The results have been mixed, but there are startling success stories. Assessing how well transplants really work is growing ever more difficult, however. Roadblocks include paltry funding—the trials lack commercial support because they're not testing new drugs—and difficulty finding patients. There's also growing evidence that stem cell transplants work best in healthier people whose disease hasn't damaged major organs. But for the most part, those aren't the patients receiving transplants: The toxicity of the treatment, uncertainty over how best to coax it to work, and tight restrictions from regulatory agencies over whom to transplant mean that many studies are restricted to the sickest of the sick—and that the therapy risks performing below its full potential.
