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Research and innovation in the development of everolimus for oncology

David Lebwohl1, George Thomas, Heidi A Lane

  • 1Novartis Pharmaceuticals, Inc., 180 Park Avenue, Florham Park, NJ 07932, USA +1 862 778 7218 ; david.lebwohl@novartis.com.

Expert Opinion on Drug Discovery
|June 1, 2012
PubMed
Abstract

Insights

Everolimus, an mTOR inhibitor, shows clinical efficacy in treating various cancers, including advanced renal cell carcinoma and rare diseases. Its role in blocking disease progression offers improved patient outcomes in oncology.

Area of Science:

  • Oncology
  • Pharmacology

Background:

  • Mammalian target of rapamycin (mTOR) hyperactivity is implicated in numerous diseases.
  • Inhibiting the mTOR pathway can halt disease progression and enhance patient outcomes.
  • Everolimus, an mTOR inhibitor, is approved for transplant rejection and oncology.

Purpose of the Study:

  • To review the development of everolimus as an oncology therapy.
  • To discuss its approval for advanced renal cell carcinoma (RCC) and SEGAs in tuberous sclerosis.
  • To explore its use in other cancers and rare diseases.

Main Methods:

  • PubMed search for 'everolimus or rapamycin' and 'cancer'.
  • Manual review of bibliographies from retrieved articles.
  • Inclusion of data from major cancer congresses.

Main Results:

  • Everolimus demonstrates clinical efficacy in Phase II-III studies across multiple tumor types.
  • Effective as a monotherapy and in combination with other agents.
  • Proven utility in RCC, neuroendocrine tumors, tuberous sclerosis complex, SEGAs, lymphoma, and other cancers.

Conclusions:

  • mTOR plays a significant role in various cancers and rare diseases.
  • Everolimus is a potentially effective agent for multiple indications.
  • Supports the use of everolimus in diverse oncological and rare disease settings.

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