Outlook in 2016

Lysosomal storage disorders (LSDs) highlight the diverse ways in which the failure of a single organelle can bring cells to their knees. Most are rare and poorly understood, making the development of therapies a daunting task.

Most rare diseases lack even one approved treatment. Regulators have tried to encourage drug development, but advocacy groups are having to fight to get the research done.

Two competing hypotheses about Parkinson's and Gaucher's diseases need to be knitted together, say Anthony H. Futerman and John Hardy.

In the 1980s, bone-marrow transplants transformed the lives of children with fatal lysosomal diseases. Researchers are now working on another revolutionary treatment.

It is now feasible to test babies for several lysosomal storage disorders, but this goes against the gold standard for screening that was established nearly 50 years ago. The ethical issues raised are forcing a rethink of the way that newborns are screened.

Treatments that can cross the blood–brain barrier are needed if doctors are to treat the devastating neurological symptoms of many lysosomal storage disorders.

Problems with the lysosome cause more than just lysosomal storage disorders. This crucial cellular component has a surprising role in several common and complex conditions.

Kidney cancer is being detected earlier thanks to modern diagnostic techniques, meaning more patients receive treatment.

Advanced tumours may have met their match with new drugs, but why have these treatments proved ineffective at stopping early-stage tumours from coming back?

Obese people have a higher incidence of kidney cancer, but are also more likely to survive the disease. Is the 'obesity paradox' real or an artefact of how studies are conducted?

We need to focus on five main areas to make real progress in the treatment of kidney cancer, says Robert J. Motzer.

Immunotherapies such as checkpoint inhibitors unleash the body's ability to defend itself against kidney cancer.

Alcohol intake boosts the risk of cancers of the liver, breast and colon, but it seems to reduce the risk of kidney cancer.

We need to combine epidemiology and exposures research to fulfil the potential of precision medicine, say John Leppert and Chirag Patel.

The first medical interventions were often individualized but ineffective, because doctors lacked an understanding of disease biology. As medicine became more scientific, physicians started grouping patients by disease. Now, genetic insights let doctors consider their patients' unique make-up when prescribing treatments.

Precision oncology has not been shown to work, and perhaps it never will, says Vinay Prasad.

Living with a rare disease but no concrete diagnosis can be difficult. Genetic sequencing may finally provide a solution.