Induced pluripotent stem cells (iPSCs) from inherited erythromelalgia (IEM) study participants and healthy donors differentiating into sensory neurons.SCIENCE TRANSLATIONAL MEDICINE, L. CAO ET AL.Pain can be tough to take, and it’s also difficult to study: rodent models for pain do not necessarily translate to human pain conditions and expression of disease-causing mutations in cell lines may not precisely mimic the physiology of human pain disorders. Now, researchers have developed a new way to test pain—and, potentially, other sensory-targeting medications. Edward Stevens and James Bilsland of the Pfizer’s U.K.-based neuroscience and pain research units and their colleagues have shown that induced pluripotent stem cells (iPSCs) derived from blood samples of patients with a pain disorder can be used to create sensory neurons that recapitulate the disease phenotype. Testing a novel pain inhibitor on the patient-derived, iPSC-based neurons, the researchers recapitulated the sensitivity to the drug seen in the corresponding patients in a clinical trial.
The team’s results, published this week (April 20) in Science Translational Medicine, suggest that such a stem cell-based approach may be useful to study nerve dysfunction. “We hope this approach will have wide application to many pain states and translate to other therapeutic areas,” Stevens wrote in an email to The Scientist.
“This is an interesting and important foundational study,” Paul Knoepfler, a stem cell researcher at the University of California, Davis, who was not involved in the work, wrote in an email. “In a single study, going all the way from reprogramming cells to testing a drug on neurons made from those cells and finally to testing it on patients seems very unique.”
“What is new here is using iPSC-derived neurons as a tool ...