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Investigational drugs can save or extend lives, and seriously ill patients not able to take part in clinical trials should have access to such drugs whenever possible. In a climate of increased public pressure for this access—often termed compassionate use—five states in the US have passed so-called 'right to try' legislation. These laws are ill advised, as they are not likely to substantially increase access and have the potential to compromise the clinical trial system.
The international response to the ongoing Ebola epidemic has in many respects been more reactive than proactive. But there are changes that, if made, may shift the balance toward future readiness.
The size, speed and potential reach of the 2014 Ebola virus outbreak in West Africa presents a wake-up call to the research and pharmaceutical communities—and to federal governments—of the continuing need to invest resources in the study and cure of emerging infectious diseases.
Regenerative medicine may enable replacement of damaged or diseased tissues. But its clinical success will require deeper understanding of the basic biology of the stem cell niche and coordination between stem cell biologists and those in other fields.
Current drug approval regulations in Europe and the United States require that the treatment group demonstrates a clear benefit compared with the control group. These laws should be updated to reflect patient heterogeneity in clinical trials, and allow for approval of drugs that show efficacy in only a subset of treated patients.
Key stakeholders, journals and funders must enable the additional research and early data sharing needed to advance the adoptive T cell therapy field as a whole.
Recent major scientific advances in the ability to replace mitochondria harboring pathological DNA mutations in the germ line could soon be harnessed to tackle mitochondrial diseases. But ethical and scientific concerns require continued debate before such an approach can reach the clinic.
Last month, Juan Carlos López, who was Chief Editor of Nature Medicine for a decade, left the journal for a new position in the biopharmaceutical industry. His team looks back at his legacy and forward to what's next.
The important gains in supporting local biomedical science made by South Africa's Medical Research Council in recent years may be lost unless the country continues to fund and grow this important institution.
Reproducibility in science is a prominent topic in both lay and scientific press. But a new facet of this discussion has arisen in a recent comparison of two pharmacogenomic studies, and it calls for an evaluation of how we interpret science in the face of discrepant results.
