Drug discovery in focal and segmental glomerulosclerosis

Nick Pullen, Alessia Fornoni

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations


Despite the high medical burden experienced by patients with focal segmental glomerulosclerosis, the etiology of the condition remains largely unknown. Focal segmental glomerulosclerosis is highly heterogeneous in clinical and morphologic manifestations. While this presents challenges for the development of new treatments, research investments over the last 2 decades have yielded a surfeit of potential avenues for therapeutic intervention. The development of many of those ideas and concepts into new therapies, however, has been very disappointing. Here, we describe some of the factors that have potentially contributed to the poor translational performance from this research investment, including the confidence we ascribe to a target, the conduct of experimental studies, and the availability of selective reagents to test hypotheses. We will discuss the significance of genetic and systems traits as well as other methods for reducing bias. We will analyze the limitations of a successful drug development. We will use specific examples hoping that these will guide a consensus for investment and drive greater translational quality. We hope that this substrate will serve to exemplify the tremendous opportunity for intervention as well as facilitate greater collaborative effort between industry, academia, and private foundations in promoting appropriate validation of these targets. Only then will we have achieved our goal for curative therapies for this devastating disease.

Original languageEnglish (US)
Pages (from-to)1211-1220
Number of pages10
JournalKidney international
Issue number6
StatePublished - 2016


  • focal segmental glomerulosclerosis
  • podocyte
  • proteinuria

ASJC Scopus subject areas

  • Nephrology


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