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Deforolimus (AP23573) a novel mTOR inhibitor in clinical development

Monica Mita1, Kamalesh Sankhala, Issam Abdel-Karim

  • 1Institute for Drug Development, Cancer Therapy and Research Center, UT Health Science Center, San Antonio, USA. mitam@uthscsa.edu

Expert Opinion on Investigational Drugs
|November 18, 2008
PubMed

Insights

Deforolimus, an analog of Rapamycin, shows promise as an anticancer drug. This mTOR inhibitor is effective against various cancers, with ongoing Phase III trials for sarcoma treatment.

Area of Science:

  • Oncology
  • Pharmacology
  • Molecular Biology

Background:

  • The mechanistic target of rapamycin (mTOR) is a key target for cancer therapy.
  • Rapamycin (RAP) was the first mTOR inhibitor, but its poor solubility and stability limit its clinical use.
  • Development of RAP analogs is crucial for effective anticancer drug delivery.

Purpose of the Study:

  • To present preclinical and clinical data on deforolimus (AP23573), a novel mTOR inhibitor.
  • To highlight deforolimus's potential as an anticancer agent, particularly for sarcoma.

Main Methods:

  • Review of preclinical studies evaluating antiproliferative activity.
  • Summary of Phase I and II clinical trial outcomes for deforolimus.
  • Discussion of ongoing Phase III clinical trials in sarcoma patients.

Main Results:

  • Deforolimus demonstrated significant antiproliferative activity in preclinical models.
  • Phase I and II trials showed encouraging results in various tumor types, including sarcoma.
  • A Phase III trial for sarcoma is currently underway, indicating promising therapeutic potential.

Conclusions:

  • Deforolimus is a promising mTOR inhibitor with demonstrated efficacy in preclinical and early clinical studies.
  • Its favorable profile supports its continued investigation as a novel anticancer therapeutic.
  • Deforolimus holds potential for treating challenging malignancies like sarcoma.

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