Blood-Brain Barrier Disruption Using Exablate Magnetic Resonance-Guided Low Intensity Focused Ultrasound With Microbubbles in Combination With T-Cell Infusion in Patients With Newly Diagnosed Diffuse Midline Glioma (LIFT)

Summary

This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for Diffuse Midline Glioma (DMG), an aggressive brain tumor with a very poor prognosis - an average one-year overall survival. This study combines blood-brain barrier (BBB) disruption (BBBD) using low-intensity focused ultrasound (LIFU) and microbubble treatment, and intravenous infusion of autologous, tumor multi-antigen associated specific cytotoxic T lymphocyte (TAA-T) therapy. The TAA-T cell investigational product in this protocol is manufactured to target Preferentially Expressed Antigen of Melanoma (PRAME), Wilms Tumor 1 (WT1), and survivin.

Conditions

• Pontine Diffuse Midline Glioma
• Diffuse Intrinsic Pontine Glioma
• Thalamic Diffuse Midline Glioma

Interventions

BIOLOGICAL

TAA-T Cell

Intravenous tumor associated antigen specific T cell (TAA-T) infusion.

DEVICE

BBB Disruption

BBB disruption using the Exablate 4000 Type 2 System with DEFINITY microbubbles.

Sponsors & Collaborators

• Focused Ultrasound Foundation

  collaborator OTHER

• Children's National Research Institute

  lead OTHER

Principal Investigators

• Luca Szalontay, MD · Children's National Research Institute

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

3 Years

Max Age

25 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2026-09-30

Primary Completion

2031-12-31

Completion

2032-12-31

FDA Drug

Yes

FDA Device

Yes