Evidence map›Paper›PMID 32089640›Full record

ArticleSarcoma2020

Targeting Refractory Sarcomas and Malignant Peripheral Nerve Sheath Tumors in a Phase I/II Study of Sirolimus in Combination with Ganetespib (SARC023).

AeRang Kim, Yao Lu, Scott H Okuno, Denise Reinke, Ophélia Maertens, John Perentesis, Mitali Basu, Pamela L Wolters, Thomas De Raedt, Sant Chawla and 6 more

Registry-linked trialOpen access · hybridAbstract read
In one paragraph

Article in Sarcoma, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02008877 (A Phase I/II Trial of Ganetespib in Combination With the mTOR Inhibitor Sirolimus for Patients With Recurrent or Refractory Sarcomas Including Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors), which is not on this map. Cited by 42 papers.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed
3.5field-weighted citation impact, top 5% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02008877 phase1 / phase2completednot on this map

A Phase I/II Trial of Ganetespib in Combination With the mTOR Inhibitor Sirolimus for Patients With Recurrent or Refractory Sarcomas Including Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors

TypeinterventionalSponsorSarcoma Alliance for Research through CollaborationRan2013 to 2018Enrolled20ConditionsMalignant Peripheral Nerve Sheath Tumors (MPNST), SarcomaArmsganetespib, Sirolimus
3 · Its place in the literature

Who cites it

42 citing papers in PubMed, 66 citations in OpenAlex.

  1. SARC031: A Phase II Trial of Selumetinib and Sirolimus for Patients with Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors (MPNST).Clinical cancer research : an official journal of the American Association for Cancer Research · 2026
    Trial
  2. Trial
  3. Review
  4. Article
  5. Review
  6. Review
  7. Review
  8. Article
  9. Triple Combination of MEK, BET, and CDK Inhibitors Significantly Reduces Human Malignant Peripheral Nerve Sheath Tumors in Mouse Models.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025
    Article
  10. Review
  11. Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Review
  17. Contemporary Approach to Neurofibromatosis Type 1-Associated Malignant Peripheral Nerve Sheath Tumors.American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting · 2024
    Review
  18. Article
  19. Review
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors at 10 institutions in 1 country.

AeRang KimChildren's National Medical Center, 111 Michigan Ave., NW, Washington, DC 20010, USA.ORCID https://orcid.org/0000-0002-7813-5568
Yao LuSARC Statistics, Weill Cornell Medicine Healthcare and Policy Research, 602 East 67 Street, New York, NY 10065, USA.
Scott H OkunoMayo Clinic, 200 First St., SW, Rochester, MN 55905, USA.ORCID https://orcid.org/0000-0002-1297-6946
Denise ReinkeSARC, 24 Frank Lloyd Wright Drive, Ann Arbor, MI 48105, USA.
Ophélia MaertensChildren's Hospital of Philadelphia, Univeristy of Pennsylvania, 3501 Civic Center Boulevard, 19104 Philadelphia, PA, USA.
John PerentesisCincinnati Children's Hospital & Uinviersity of Cincinnati, 3333 Burnet Ave., Cincinnati, OH 45229, USA.
Mitali BasuCincinnati Children's Hospital & Uinviersity of Cincinnati, 3333 Burnet Ave., Cincinnati, OH 45229, USA.
Pamela L WoltersPediatric Oncology Branch, National Cancer Institute, 10 Center Drive, Bethesda, MD 20892, USA.
Thomas De RaedtChildren's Hospital of Philadelphia, Univeristy of Pennsylvania, 3501 Civic Center Boulevard, 19104 Philadelphia, PA, USA.
Sant ChawlaSarcoma Oncology Center, 2811 Wilshire Blvd, Santa Monica, CA 90403, USA.
Rashmi ChughUniversity of Michigan, 1500 E. Medical Center Dr., SPC 5912, Ann Arbor, MI 48109, USA.
Brian A Van TineWashington University in St. Louis, 660 S Euclid Ave., St. Louis, MO 63110, USA.
Geraldine O'SullivanNational Cancer Institute, Developmental Therapeutics Clinic, Division of Cancer Treatment and Diagnosis, Bethesda, MD 20892, USA.
Alice ChenNational Cancer Institute, Developmental Therapeutics Clinic, Division of Cancer Treatment and Diagnosis, Bethesda, MD 20892, USA.
Karen CichowskiChildren's Hospital of Philadelphia, Univeristy of Pennsylvania, 3501 Civic Center Boulevard, 19104 Philadelphia, PA, USA.
Brigitte C WidemannPediatric Oncology Branch, National Cancer Institute, 10 Center Drive, Bethesda, MD 20892, USA.ORCID https://orcid.org/0000-0002-9198-7175
National Cancer Institute · USChildren's Hospital of Philadelphia · USCincinnati Children's Hospital Medical Center · USChildren's National · USCornell University · USMayo Clinic · USSarcoma Alliance for Research through Collaboration · USSarcoma Oncology Center · USUniversity of Michigan · USWashington University in St. Louis · US

Funding

Pediatric Oncology Branch Clinical Care and EducationZIEBC011516 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI GLOD, JOHN · 2013 to 2025
$69.1M
Therapies for patients with rare tumors and genetic tumor predispositionZIASC010354 · NCI · DIVISION OF CLINICAL SCIENCES - NCI · PI WIDEMANN, BRIGITTE · 2009 to 2025
$19.2M
Pediatric Oncology Branch Behvioral Science CoreZIDBC011517 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI GLOD, JOHN · 2013 to 2025
$15.6M
6 · The paper itself

Abstract

purposeMalignant peripheral nerve sheath tumors (MPNSTs) are aggressive soft tissue sarcomas. Combining Hsp90 inhibitors to enhance endoplasmic reticulum stress with mTOR inhibition results in dramatic MPNST shrinkage in a genetically engineered MPNST mouse model. Ganetespib is an injectable potent small molecule inhibitor of Hsp90. Sirolimus is an oral mTOR inhibitor. We sought to determine the safety, tolerability, and recommended dose of ganetespib and sirolimus in patients with refractory sarcomas and assess clinical benefits in patients with unresectable/refractory MPNSTs.

resultsTwenty patients were enrolled (10 per phase). Toxicities were manageable; most frequent non-DLTs were diarrhea, elevated liver transaminases, and fatigue. The recommended dose of ganetespib was 200 mg/m

conclusionDespite promising preclinical rationale and tolerability of the combination therapy, no responses were observed, and the study did not meet parameters for further evaluation in MPNSTs. This trial was registered with (NCT02008877).

Identifiers

PMID32089640
PMCPMC7013290
OpenAlexW3004051267

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.