2. Academic Validation
  3. Brief Report: A Phase 1b/Pharmacokinetic Trial of PTC299, a Novel PostTranscriptional VEGF Inhibitor, for AIDS-Related Kaposi's Sarcoma: AIDS Malignancy Consortium Trial 059

Brief Report: A Phase 1b/Pharmacokinetic Trial of PTC299, a Novel PostTranscriptional VEGF Inhibitor, for AIDS-Related Kaposi's Sarcoma: AIDS Malignancy Consortium Trial 059

  • J Acquir Immune Defic Syndr. 2016 May 1;72(1):52-7. doi: 10.1097/QAI.0000000000000918.
Rachel A Bender Ignacio 1,  Jeannette Y Lee,  Michelle A Rudek,  Dirk P Dittmer,  Richard F Ambinder,  Susan E Krown,  AIDS Malignancy Consortium (AMC)-059 Study Team
Affiliations

Affiliation

  • 1 *Division of Allergy and Infectious Diseases, Department of Medicine, University of Washington, Seattle, WA;†Vaccine and Infectious Diseases Division, Fred Hutchinson Cancer Research Center, Seattle, WA;‡Department of Biostatistics, University of Arkansas for Medical Sciences, Little Rock, AR;§The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD;‖Department of Microbiology and Immunology, University of North Carolina School of Medicine, Chapel Hill, NC;¶Lineberger Comprehensive Cancer Center, University of North Carolina School of Medicine, Chapel Hill, NC; and#Division of Medical Oncology, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
PMID: 26689971 DOI: 10.1097/QAI.0000000000000918
Abstract

Vascular endothelial growth factor (VEGF) plays an important role in Kaposi's sarcoma (KS). We administered PTC299, a post-transcriptional inhibitor of pathogenic VEGF, to persons with HIV-related KS. Seventeen participants received 3 different doses of PTC299. Adverse events typically observed with VEGF inhibition were absent. Three participants had partial tumor responses and 11 had stable disease. There were no differences in exposure to PTC299 by antiretroviral regimen. Serum VEGF, but not KS-associated herpesvirus DNA, decreased on treatment. Given redundancies in the VEGF feedback loop, future trials should consider combining PTC299 with agents that inhibit different pathways implicated in KS and KS-associated herpesvirus proliferation.

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