What side effects are associated with this type of intervention?

Common treatments for brain tumors include chemotherapy agents like doxorubicin and innovative techniques such as focused ultrasound blood-brain barrier disruption. Doxorubicin can cause side effects such as alopecia (hair loss), myelosuppression (decreased bone marrow activity), and cardiotoxicity (heart damage). Focused ultrasound blood-brain barrier disruption, while still under investigation, may lead to side effects like headaches, scalp pain, and potential neurocognitive effects. Patients should discuss these potential side effects with their healthcare provider to make informed treatment decisions.

What prior FDA approvals exist?

FDA approvals for the treatment of brain tumors have included various chemotherapy agents and innovative delivery methods. Temozolomide, an oral alkylating agent, has been widely used for glioblastoma and other high-grade gliomas. Additionally, bevacizumab, an anti-angiogenic drug, has been approved for recurrent glioblastoma. Innovative approaches such as the use of focused ultrasound to disrupt the blood-brain barrier are being studied to enhance the delivery of chemotherapeutic agents like doxorubicin. These methods aim to improve drug penetration into the brain, potentially increasing the efficacy of treatments for brain tumors.

What other types of research exist?

Promising novel alternatives for brain tumor treatment in 2024 include: 1) Bevacizumab, an antiangiogenic therapy, which has shown efficacy in reducing tumor growth and improving hearing in NF2-related vestibular schwannomas. 2) Tucatinib, capecitabine, and trastuzumab combination, which has demonstrated improved progression-free survival and overall survival in HER2-positive metastatic breast cancer with brain metastases. 3) Immune checkpoint inhibitors such as nivolumab, which have shown promising response rates in neuroendocrine carcinoma of the lung and may be applicable to brain tumors. 4) Temozolomide, an oral alkylating agent, which has been effective in treating high-grade gliomas and can penetrate the blood-brain barrier.

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Anti-tumor antibiotic

Focused Ultrasound + Chemotherapy for Pediatric Brain Tumor

Phase 1 & 2

Recruiting

Research Sponsored by InSightec

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new way to treat children with a difficult-to-treat brain tumor called DIPG. It uses a device to temporarily open the brain's protective barrier so that a cancer drug, Doxorubicin, can enter and target the tumor. The goal is to see if this method is safe and effective. Doxorubicin is a well-known chemotherapy drug, but its effectiveness in treating brain tumors like DIPG has been limited due to the blood-brain barrier.

Who is the study for?

This trial is for pediatric patients aged 5-21 with DIPG who have completed radiation therapy within the last 4-12 weeks and show no signs of necrosis or hemorrhage. They must be neurologically stable, not on increasing steroids, and haven't had brain surgery in the past two weeks. Exclusions include hypertension, contraindications to Doxorubicin, metastatic disease, bleeding disorders, vasculopathy, immunosuppression (except certain steroids), active seizures despite treatment, known sensitivities to specific contrast agents or drugs like Avastin.

What is being tested?

The study tests the safety and effectiveness of disrupting the blood-brain barrier using Exablate Focused Ultrasound (Model 4000 Type2.0/2.1) combined with Doxorubicin chemotherapy for treating pediatric DIPG—a type of brain tumor.

What are the potential side effects?

Potential side effects may include those related to Doxorubicin such as hair loss, nausea/vomiting, mouth sores; heart damage; low blood counts leading to infection risk; and those from focused ultrasound like discomfort at the target site or headaches due to BBB disruption.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse Events

Secondary study objectives

Blood Brain Barrier Disruption (BBBD)

Other study objectives

Progression Free Survival (PFS)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Blood Brain Barrier Disruption (BBBD)Experimental Treatment2 Interventions

Exablate MR Guided Focused Ultrasound for Blood Brain Barrier Disruption with Doxorubicin for treating pediatric patients with DIPG

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Doxorubicin

2012

Completed Phase 3

~8030

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Focused ultrasound blood-brain barrier (BBB) disruption, such as with the Exablate Model 4000, uses targeted ultrasound waves to temporarily open the BBB, allowing for enhanced delivery of therapeutic agents directly to the brain tumor. This is crucial because the BBB typically prevents many drugs from reaching brain tissue. Doxorubicin, a chemotherapy agent, works by intercalating DNA strands, thereby inhibiting the replication of cancer cells and inducing apoptosis. For brain tumor patients, combining these treatments can potentially increase the efficacy of chemotherapy by ensuring higher concentrations of the drug reach the tumor site, which may improve treatment outcomes and reduce systemic side effects.