Evidence map›Paper›PMID 22402322›Full record

ArticleGene therapy2013

Neural stem cell-mediated CE/CPT-11 enzyme/prodrug therapy in transgenic mouse model of intracerebellar medulloblastoma.

M Gutova, G M Shackleford, V Khankaldyyan, K A Herrmann, X-H Shi, K Mittelholtz, Y Abramyants, M S Blanchard, S U Kim, A J Annala and 6 more

Open access · bronzeAbstract read
In one paragraph

Article in Gene therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed
2.9field-weighted citation impact, top 9% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

27 citing papers in PubMed, 45 citations in OpenAlex.

  1. Engineered tumor-tropic mesenchymal stem cells as targeted therapeutic delivery systems for refractory Ovarian cancer.Journal of controlled release : official journal of the Controlled Release Society · 2026
    Article
  2. Review
  3. Article
  4. Review
  5. Article
  6. Article
  7. Bioengineered adipose-derived stem cells for targeted enzyme-prodrug therapy of ovarian cancer intraperitoneal metastasis.Journal of controlled release : official journal of the Controlled Release Society · 2019
    Article
  8. Article
  9. Review
  10. Article
  11. Article
  12. Article
  13. Neural Stem Cell-Based Anticancer Gene Therapy: A First-in-Human Study in Recurrent High-Grade Glioma Patients.Clinical cancer research : an official journal of the American Association for Cancer Research · 2017
    Article
  14. Article
  15. Article
  16. Article
  17. Article
  18. Engineering Stem Cells for Biomedical Applications.Advanced healthcare materials · 2016
    Review
  19. Article
  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 6 institutions in 3 countries.

M GutovaDepartment of Neurosciences, Beckman Research Institute of the City of Hope, Duarte, CA 91010-3000, USA. mgutova@coh.org
G M Shackleford
V Khankaldyyan
K A Herrmann
X-H Shi
K Mittelholtz
Y Abramyants
M S Blanchard
S U Kim
A J Annala
J Najbauer
T W Synold
M D'Apuzzo
M E Barish
R A Moats
K S Aboody
Beckman Research InstituteChildren's Hospital of Los Angeles · USCity of Hope · USChung-Ang University · KRCity Of Hope National Medical Center · USUniversity of British Columbia Hospital · CA

Funding

Transgenic Mouse FacilityP30CA033572 · NCI · CITY OF HOPE/BECKMAN RESEARCH INSTITUTE · PI STANLEY R. HAMILTON · 1985 to 2026
$86.3M
NCI NIH HHS P30 CA033572NCI NIH HHS P30 CA33572
6 · The paper itself

Abstract

Medulloblastoma is a heterogeneous diffuse neoplasm that can be highly disseminated, and is the most common malignant childhood brain tumor. Although multimodal treatments have improved survival rates for patients with medulloblastoma, these tumors are associated with high morbidity and mortality. New treatment strategies are urgently needed to improve cure rates and, importantly, to spare normal brain tissue from neurotoxicity and patients from life-long cognitive and functional deficits associated with current therapies. In numerous preclinical brain tumor models, neural stem cells (NSCs) have shown great promise as delivery vehicles for therapeutic genes. Here, we have used an established, genetically modified human NSC line (HB1.F3.CD) to deliver carboxylesterase (CE) to cerebellar tumor foci and locally activate the prodrug camptothecin-11 (CPT-11) (Irinotecan) to the potent topoisomerase I inhibitor SN-38. HB1.F3.CD NSC tumor tropism, intratumoral distribution and therapeutic efficacy were investigated in clinically relevant experimental models. Magnetic resonance imaging was used for in vivo tracking of iron nanoparticle-labeled NSCs, and to assess the therapeutic efficacy of CE-expressing HB1.F3.CD cells. As compared with controls, a significant decrease in tumor growth rate was seen in mice that received both NSCs and CPT-11 as their treatment regimen. Thus, this study provides proof-of-concept for NSC-mediated CE/CPT-11 treatment of medulloblastoma, and serves as a foundation for further studies toward potential clinical application.

Indexed as

Genetic TherapyAnimalsAntineoplastic Agents, PhytogenicCamptothecinCarboxylesteraseCell Line, TumorCerebellar NeoplasmsGene Transfer TechniquesHumansIrinotecanMedulloblastomaMiceMice, NudeMice, TransgenicNeural Stem CellsProdrugsAntineoplastic Agents, PhytogenicCamptothecinCarboxylesteraseIrinotecanProdrugs

Identifiers

PMID22402322
PMCPMC4149468
OpenAlexW2090841339

What OpenQuestion holds

Textmetadata
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Read underepoch 390

Registered trials

None linked

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.