Emerging therapeutic targets currently under investigation for the treatment of systemic amyloidosis

Mario Nuvolone, Giampaolo Merlini

Research output: Contribution to journalReview articlepeer-review


Introduction: Systemic amyloidosis occurs when one of a growing list of circulating proteins acquires an abnormal fold, aggregates and gives rise to extracellular amyloid deposits in different body sites, leading to organ dysfunction and eventually death. Current approaches are mainly aimed at lowering the supply of the amyloidogenic precursor or at stabilizing it in a non-amyloidogenic state, thus interfering with the initial phases of amyloid formation and toxicity. Areas covered: Improved understanding of the pathophysiology is indicating novel steps and molecules that could be therapeutically targeted. Here, we will review emerging molecular targets and therapeutic approaches against the main forms of systemic amyloidosis at the early preclinical level. Expert opinion: Conspicuous efforts in drug design and drug discovery have provided an unprecedented list of potential new drugs or therapeutic strategies, from gene-based therapies to small molecules and peptides, from novel monoclonal antibodies to engineered cell-based therapies. The challenge will now be to validate and optimize the most promising candidates, cross the bridge from the preclinical phase to the clinics and identify, through innovative trials design, the safest and most effective combination therapies, striving for a better care, possibly a definitive cure for these diseases.

Original languageEnglish
Pages (from-to)1095-1110
Number of pages16
JournalExpert Opinion on Therapeutic Targets
Issue number12
Publication statusPublished - Dec 2 2017


  • AL amyloidosis
  • amyloid
  • Amyloidosis
  • ATTR amyloidosis
  • drug repurposing
  • emerging therapies
  • high throughput screening
  • molecular targets
  • structure-based drug design
  • systemic amyloidosis

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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