Stem cells given go-ahead for clinical trial
Preclinical evidence supports taking a stem cell-based treatment for life-threatening infections forward into human clinical trials.
Most attempts to transform exciting findings from laboratories into clinical treatments are unsuccessful. One reason for this may be the failure to consider all of the laboratory work that has been performed before deciding to test a treatment on patients for the first time. In particular, negative findings (that suggest that a potential new treatment is ineffective) may be overlooked.
Stem cells are cells with the potential to develop into any one of the various kinds of cell that make up the body. These cells may also help to treat life-threatening infections, but this has not been tested in human patients. However, the effectiveness of stem cell treatments has been tested in animals that act as models of human infection.
Before deciding to begin a clinical trial of stem cell therapy for life-threatening infections, Manoj Lalu and colleagues performed an exhaustive search to find all the studies in which stem cells were used to treat animal models of infection. Combining the results of all of these studies using particular analysis techniques revealed that stem cell therapy increased the survival of these animals overall. These positive effects were seen over a range of different experimental conditions (for example, when treating the animals with different doses of stem cells, or giving the doses at different times).
Lalu and colleagues also identified some limitations with most of the laboratory studies that had tested stem cell therapy for infections. Many of the studies used animal models that may not be the best representations of humans with severe infection. In addition, many of the scientists did not report that they had used methods (such as randomization) that would generate the most confidence in their results. Despite these limitations, there was a lot of consistency in the reported results.
Overall, the results support the decision to proceed to a clinical trial that tests the effectiveness of stem cells for treating infections in people. More generally, Lalu and colleagues’ analysis demonstrates a way of considering all laboratory evidence before deciding to proceed to a first clinical trial in humans. This may help researchers to identify promising therapies to further develop, and also to identify potential failures before they are tested in patients.
To find out more
Read the eLife research paper on which this eLife digest is based: “Evaluating mesenchymal stem cell therapy for sepsis with preclinical meta-analyses prior to initiating a first-in-human trial”(November 17, 2016).