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Diffuse Midline Glioma, H3 K27M-Mutant Treatment

Diffuse midline glioma (DMG), H3 K27M-altered, is a type of high-grade glioma that is found in the midline structures of the brain (thalamus, brainstem, pons) or spinal cord. It is more commonly known as diffuse intrinsic pontine glioma (DIPG) when it is located in the pons.

DMGs are highly aggressive tumors that are currently incurable. Due to the poor prognosis, we at St. Jude are committed to identifying novel therapies to improve this outcome.

Diffuse midline glioma, H3 K27M-mutant, causes

Sometimes people with certain genetic disorders that are passed down (inherited) are at an increased risk of developing DMGs. These inherited disorders include Li-Fraumeni Syndrome (LFS) and constitutional mismatch repair deficiency syndrome (CMMRD). But in most cases, DMGs develop without an underlying genetic predisposition.

Treatment of Diffuse midline glioma, H3 K27M-mutant

Unlike other high-grade gliomas, surgeons are unable to remove DMG because the tumor invades a part of the brain that controls many important body functions. Some of these functions are crucial for survival. But surgical biopsy (collecting a tissue sample) can be done safely. A biopsy can help the medical team determine the diagnosis and identify molecular characteristics of each tumor.

The standard and most effective treatment for DMG is radiation therapy.

  • Radiation therapy uses high-energy x-rays or other types of radiation to kill cancer cells and stop them from growing. Radiation works for a short time, but it unfortunately does not cure patients with DIPG.
  • Other treatments, including chemotherapy, targeted therapy, and cellular therapy, are being evaluated through clinical trials.

Diffuse midline glioma, H3 K27M-mutant, clinical trials

Patients with H3 K27M-altered DMG take part in clinical trials that use the most advanced tools for diagnosis and treatment. Our goal is to give each child the best treatment with the lowest number of side effects.

Our doctors and scientists work together to find better ways to treat patients. In many cases, new treatments are being developed right here on the St. Jude campus.

We also take part in trials developed by the Children’s Oncology Group (COG) and the Pediatric Brain Tumor Consortium (PBTC). Many St. Jude staff members play key roles in developing these studies. They are large studies carried out at centers across the United States. 

Learn more about clinical research at St. Jude.

Loc3CAR: B7-H3-Specific CAR T-Cell Therapy for Children with Primary CNS Tumors

Study goal:

The purpose of this study is to find: 1) the largest dose of Loc3CAR T cells that is safe to give patients with B7-H3–positive brain tumors; 2) the side effects of Loc3CAR T cells; 3) the effect Loc3CAR T cells have on brain tumors. This trial will help scientists better understand how the immune system fights this kind of tumor. What we learn from this study could help us create better treatments.


Up to 21 years old

PEPN2111: Phase 1/2 Trial of CBL0137 for Relapsed or Refractory Solid Tumors and Lymphoma

Study goal:

The main purpose of this study is to find the highest, safest dose of CBL0137 that can be given to children, teens and young adults with solid tumors or lymphoma.


12 months to 21 years old with diagnosis of relapsed or refractory solid tumor or lymphoma (including CNS tumors) or progressive or recurrent DIPG or other H3 K27M-mutant diffuse midline gliomas previously treated with radiation therapy; 12 months to 30 years old with diagnosis of relapsed or refractory osteosarcoma

Diffuse midline glioma, H3 K27M-mutant, care at St. Jude

St. Jude provides the highest quality of care for patients with DMG:

  • Our clinic brings together medical experts and specially trained staff to care for your child. We also work with brain surgeons (neurosurgeons) at Le Bonheur Children’s Hospital.
  • We have a special team that helps understand the makeup of the tumor. This team includes doctors and scientists who study:
    • Cancer cells
    • How the cancer happened
    • Current medicines and treatments to kill cancer cells
    • What makes cancer cells grow and what new medicines can stop them
    • New medicines and other therapies to improve how treatments work
  • Our brain tumor clinical trials have led to the clinical use of better therapies in children such as lower dose therapies, targeted therapy, and proton therapy. These treatments kill cancer cells while sparing healthy cells and may lessen the severity of side effects that affect brain function after treatment for some types of brain tumors.
  • Along with the treatment studies constantly being developed at St. Jude, the staff also treats patients on studies developed by the Children’s Oncology Group, the Pediatric Brain Tumor Consortium, and the Pacific Pediatric Neuro-Oncology Consortium. Many St. Jude staff members play key roles in developing these studies. They are large trials carried out at centers across the United States.
  • St. Jude offers a dedicated team of specialists to meet the needs of children with brain tumors, including:

More reasons to choose St. Jude for care include:

  • We are consistently ranked among the best childhood cancer centers in the nation by US News & World Report.
  • At St. Jude, we have created an environment where children can be children and families can be together.
  • St. Jude patients and families never pay for services authorized by St. Jude.
  • We lead more clinical trials for childhood cancer than any other hospital in the U.S.
  • St. Jude is the only National Cancer Institute–designated Comprehensive Cancer Center just for children. A Comprehensive Cancer Center meets rigorous standards for research that develops new and better approaches to prevent, diagnose, and treat cancer.
  • The nurse-to-patient ratio at St. Jude is about 1:3 in hematology and oncology and 1:1 in the Intensive Care Unit.
  • Patients may be able to get expert, compassionate care and treatment closer to their homes at one of our St. Jude Affiliate clinics.

Get more information about the Brain Tumor Treatment program at St. Jude.

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Seeking treatment at St. Jude

Patients accepted to St. Jude must have a disease we treat and must be referred by a physician or other qualified medical professional. We accept most patients based on their ability to enroll in an open clinical trial.

How to seek treatment

Contact us

You can start the referral process by letting your physician know you would like a second opinion from St. Jude. They can contact our 24-hour beeper service or contact our brain tumor coordinator. You may also contact the brain tumor coordinator directly with questions.

Learn more about the types of brain tumors we treat at St. Jude and our brain tumor eligibility requirements.

Physician / Patient Referral Office

Call: 1-888-226-4343 (toll-free) or 901-595-4055 (local)  | Email:
Fax: 901-595-4011 | 24-hour pager: 1-800-349-4334

Contact the Brain Tumor Team directly:

Submit brain tumor contact form

Contact the Surgery Team directly:


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