BBB Disruption + T-Cell Infusion for Diffuse Midline Glioma
(LIFT Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment for Diffuse Midline Glioma (DMG), an aggressive brain tumor. The study tests a combination of two approaches: using sound waves to temporarily open the blood-brain barrier with Low Intensity Focused Ultrasound and Microbubbles, and infusing special immune cells called TAA-T Cells to target the tumor. The trial seeks patients aged 3 to 25 with newly diagnosed pontine or thalamic DMG who have completed radiation therapy. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.
What prior data suggests that this combination therapy is safe for treating Diffuse Midline Glioma?
Previous studies have found the focused ultrasound technique used to open the blood-brain barrier (BBB) to be safe. This method helps deliver drugs to the brain without causing significant harm or severe side effects. Patients did not experience any serious negative effects, even with repeated treatments.
For TAA-T cell therapy, a type of immune cell treatment, similar therapies have also proven safe. These treatments are already being tested for other aggressive brain tumors and have generally been well-tolerated by patients.
This current study is in an early phase, primarily focusing on testing safety. While promising evidence exists from similar treatments, researchers are still carefully studying the complete safety of this combination therapy.12345Why are researchers excited about this trial's treatments?
Researchers are excited about TAA-T Cell treatment for diffuse midline glioma because it uses the body's own immune system to fight the cancer. Unlike most treatments that rely on chemotherapy or radiation, TAA-T Cell therapy involves infusing patients with specially modified T-cells that can target and destroy cancer cells more precisely. This approach could lead to fewer side effects and more effective targeting of the tumor, offering hope for improved outcomes in a condition that has been challenging to treat with conventional methods.
What evidence suggests that this trial's treatments could be effective for Diffuse Midline Glioma?
This trial will explore two separate treatment strategies for diffuse midline glioma (DMG). Research has shown that focused ultrasound (FUS) with microbubbles can safely and effectively open the blood-brain barrier (BBB), aiding treatments in reaching the brain more easily. This method holds promise for improving drug delivery in aggressive brain tumors like DMG.
Meanwhile, TAA-T cell therapy, another treatment option in this trial, uses T cells, a type of immune cell, to locate and attack tumor cells by recognizing specific proteins on them. Early studies indicate that this method has potential in shrinking tumors and improving symptoms in similar conditions. Both strategies offer new hope for treating DMG, a condition with very limited treatment options.12346Who Is on the Research Team?
Luca Szalontay, MD
Principal Investigator
Children's National Research Institute
Are You a Good Fit for This Trial?
This trial is for children and young adults (ages 3–25) who have just finished radiation therapy for newly diagnosed diffuse midline glioma in the pons or thalamus. Patients must be able to safely receive focused ultrasound treatment, as determined by a neurosurgeon.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Cycle 1
LIFT therapy consisting of blood-brain barrier disruption using low-intensity focused ultrasound with microbubbles, followed by TAA-T cell infusion. Safety monitoring for 28 to 70 days post-treatment.
Treatment Cycle 2
Second cycle of LIFT therapy with similar procedures and safety monitoring as Cycle 1, contingent on TAA-T cell availability and participant's clinical status.
Treatment Cycle 3
Third cycle of LIFT therapy with similar procedures and safety monitoring as previous cycles, contingent on TAA-T cell availability and participant's clinical status.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including assessment of adverse events and treatment response.
What Are the Treatments Tested in This Trial?
Interventions
- BBB Disruption using Low Intensity Focused Ultrasound with Microbubbles
- TAA-T Cell
Trial Overview
The study tests if it's safe and possible to use focused ultrasound with microbubbles to temporarily open the blood-brain barrier, followed by an infusion of specially prepared T-cells designed to attack brain tumor cells. All participants get both treatments.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Participants will be patients, ages 3 to 25 years, with newly diagnosed pontine DMG (Group A) who have undergone irradiation as part of their upfront therapy. Participants must not have received prior conventional or investigational chemotherapy, targeted therapy, or immunotherapy, except bevacizumab. Biopsy is not required.
Participants will be patients, ages 3 to 25 years, with newly diagnosed thalamic DMG (Group B) who have undergone irradiation as part of their upfront therapy. With the exception of temozolomide administered concurrently with radiation and bevacizumab, participants must not have received prior conventional or investigational chemotherapy, targeted therapy, or immunotherapy. Biopsy is required.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's National Research Institute
Lead Sponsor
Focused Ultrasound Foundation
Collaborator
Citations
Targeting Diffuse Midline Gliomas: The Promise of Focused ...
Focused ultrasound (FUS) with microbubbles has recently emerged as an innovative and exciting technology that non-invasively permeabilizes the BBB in a small ...
2.
fusfoundation.org
fusfoundation.org/posts/glioblastoma-clinical-trial-focused-ultrasound-blood-brain-barrier-opening-is-safe-provides-possible-survival-benefit/Focused Ultrasound Blood-Brain Barrier Opening is Safe ...
Data from a proof-of-concept study show that it is safe to use focused ultrasound blood-brain barrier opening before chemotherapy in patients ...
Blood-brain barrier opening with neuronavigation-guided ...
Focused ultrasound (FUS)–mediated blood-brain barrier (BBB) opening with microbubbles is an emerging technology that enables drug delivery ...
Study of Focused Ultrasound (FUS) with DEFINITY ...
The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known ...
Focused ultrasound-mediated blood–brain barrier opening is ...
Focused ultrasound (FUS) with microbubbles can transiently and non-invasively disrupt the BBB to enhance drug delivery.
6.
ctv.veeva.com
ctv.veeva.com/study/blood-brain-barrier-disruption-using-exablate-magnetic-resonance-guided-low-intensity-focused-ultrasBlood-Brain Barrier Disruption Using Exablate Magnetic ...
This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for Diffuse Midline Glioma (DMG), ...
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