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Overview

Interstitial Infusion for Pediatric Brain Tumors

A major limitation in treating pediatric brain tumors has been the ability to achieve adequate levels of a therapeutic agent within the tumor. This limitation stems principally from the presence of the blood brain barrier (BBB), a cellular characteristic that effectively isolates the brain tissue from the blood. A major advance in the field of neurooncology has been the introduction of local delivery schemes. Local delivery, the administration of therapeutic molecules directly into the tumor, has significant advantages over conventional systemic administration. First, the ability to achieve high concentrations of an agent by bypassing the BBB is far superior. Second, by avoiding systemic administration, the majority of dose limiting toxicities seen with conventional chemotherapeutic approaches, are avoided. These features have been integrated into one particular form of local delivery called interstitial infusion or convection-enhanced delivery.Interstitial infusion has been investigated in Dr. Mark Souweidane#146;s laboratory as a potential form of therapy for children with unresectable brain tumors. Specifically, this route of delivery is being studied in the hopes of applying such a therapeutic route for children with brain stem gliomas, a form of brain stem tumor in children that currently has no effective therapy and universally is fatal. The laboratory has gained national recognition in establishing the feasibility of using interstitial infusion for treating children with brain stem gliomas. The laboratory focuses upon the safety, the distributive properties, and the radiographic correlates of interstitial delivery in the brain stem.In addition to the mechanical considerations, the laboratory is also investigating the use of a number of different therapeutic compounds. By using a variety of therapeutic compounds, the toxicity of direct interstitial infusion is being studied as part of a preclinical initiative leading toward Phase I studies in children. Conventional chemotherapeutic compounds, conjugated immunotoxins, and radiolabelled monoclonal antibodies are all in different phases of preclinical investigation. Medical students, neurosurgery residents, and postdoctoral fellows have all enthusiastically been employed in the laboratory. Their efforts have been critical to the function of the laboratory.It is the ultimate goal of this research to obtain information that will lead toward the design of clinical studies for children with diffuse pontine gliomas, a goal that would offer a novel form of treatment for children who currently have no proven alternative form of therapy.

Highlights

  1. 1
    Neuro-oncology · 2025
    MMMark M SouweidaneSSSofia Sadia HaqueJSJason S. LewisNCNai-Kong CheungKKKim Kramer
    PMID: 39969230PMCID: PMC12448826DOIFirst author18 citations
  2. 2
    Science advances · 2020
    DJDavid J PisapiaNDNadia DahmaneMMMark M Souweidane
    PMID: 32832670PMCID: PMC7439439DOISenior author25 citations
  3. 3
    World neurosurgery · 2023
    MMMark M Souweidane
    PMID: 37803686DOISenior author7 citations

Publications

262 publications

Funding

13 grants

Active

3 grants
  • KP
    Low-intensity focused ultrasound for targeted release of liposomal doxorubicin in pediatric diffuse midline glioma.
    National Institute of Neurological Disorders and Stroke · 2026–2028
    R03EB040376-01
  • Sub-PI
    Pediatric Early Phase Clinical Trials Network (PEP-CTN)
    National Cancer Institute · 2026–2026 · via Children's Oncology Group
    UM1CA228823-07
  • PI
    Addressing Disparity in Pediatric Brain Tumor Clinical Trials Enrollment
    St. Baldrick's Foundation · 2023–2025
    SBF 1184936
Completed grants (10)
  • PI
    Integration of serial CSF cell-free DNA sampling as a liquid biopsy for near real-time monitoring of disease state in diffuse midline glioma patients
    St. Baldrick's Foundation · 2022–2025
    SAP-21-127-01-SBF-ACS
  • KP
    Targeting CAFs to sensitize irradiated pHGG to EGFR blockade
    St. Baldrick's Foundation · 2022–2024
    SBF 222633-01
  • PI
    WCM Souweidane laboratory and MacroGenics collaborative translational efforts with using MGC018 (anti-B7H3 Antibody-Drug Conjugate)
    MacroGenics · 2019–2020Contract
    SRA 194531-01
  • PI
    Alex's Lemonade Stand Foundation award for Rachel Yan, 2020
    Alex's Lemonade Stand Foundation · 2020–2020
    ALSF 203943-01
  • Co-I
    Multifunctional Nanofiber for Convection-Enhanced Delivery of Theranostics to Diffuse Intrinsic Pontine Glioma
    U.S. Department of Defense · 2017–2020
    W81XWH-17-1-0518
  • PI
    Image Guided Diffuse Intrinsic Pontine Glioma Drug Delivery and Design
    Alex's Lemonade Stand Foundation · 2016–2018
    ALSF 180578-2
  • KP
    Modulation of Convection Enhanced Delivery (CED) Distribution Using Focused Ultrasound (FUS)
    Alex's Lemonade Stand Foundation · 2017–2018Training
    ALSF 17091347-01
  • KP
    Image Guided Design and Delivery of DIPG Drugs and Nanoparticles
    Matthew Larson Foundation for Pediatric Brain Tumors, The · 2016–2018
    MLF 170786-EWOF
  • KP
    Targeting Neuroendocrine (Androgen and Aromatase) Pathways for the Treatment of Pediatric Pontine Gliomas
    Alex's Lemonade Stand Foundation · 2017–2017Training
    ALSF 17091493-01
  • PI
    Assessing Regional Tumor Response After Convection Enhanced Delivery (CED) for Diffuse Intrinsic Pontine Glioma
    Lyla Nsouli Foundation for Children's Brain Cancer Research · 2012–2016
    LNF CBCR 13020159-01

Clinical research

5 studies

Active

3 studies
  • PI
    Characterizing Rare Pineal Region Tumors: A Retrospective Study of PPTIDs and PTPRs with Molecular Insights
    Weill Medical College of Cornell University · OPEN TO ACCRUAL (as of 2026)
  • PI
    Assessing the Role of Middle Meningeal Artery Embolization in the Management of Pediatric Traumatic Extradural Hematoma - BRANYplus Transfer
    Weill Medical College of Cornell University · OPEN TO ACCRUAL (as of 2022)
  • PI
    Identification of Genetic Mutations for Indication of Survival and Treatment Response in Pediatric Thalamic Glioma Patients
    Columbia University · OPEN TO ACCRUAL (as of 2021)
Completed & closed trials (2)
  • PI
    Liquid Biopsy for Longitudinal Monitoring in Diffuse Midline Glioma (DMG) Patients
    Weill Medical College of Cornell University · CLOSED TO ACCRUAL (as of 2025)
  • PI
    Intra-Arterial (IA) Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma (ACPP) and Choroid Plexus Carcinoma (CPC) Prior to Second-Look Surgery
    PHASE1 · Weill Medical College of Cornell University · CLOSED TO ACCRUAL (as of 2024)
    Atypical Choroid Plexus Papilloma; Choroid Plexus Carcinoma
    NCT04994977

News mentions

3 mentionsView all WCM news ↗
  • Trial Shows Targeted Treatment for Childhood Brain Tumor is Safe
    April 3, 2025

    A Phase I clinical trial demonstrated that a targeted approach to treating a deadly brain tumor in children called diffuse intrinsic pontine glioma (DIPG) is safe, reported Weill Cornell Medicine and Memorial Sloan Kettering Cancer Center investigators.

  • Faculty Members Honored with Distinguished Awards
    May 16, 2024

    Weill Cornell Medicine faculty members and alumni were honored with awards for their exemplary achievements in research, education and patient care during the institution’s commencement activities on May 15 and 16.

  • In Brief: Surgery May be Avoided for Children with Rare Skull Tumor
    June 30, 2022

    Surgery can usually be avoided in children with a rare tumor of the skull, based on new research by investigators from Weill Cornell Medicine, NewYork-Presbyterian, Nicklaus Children’s Hospital, the University of Texas Southwestern, the University of British Columbia and McGovern Medical School.

Media highlights

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Mentoring

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