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Medulloblastoma in Children

What is pediatric medulloblastoma?

Medulloblastoma is a fast-growing brain cancer that can spread through the cerebrospinal fluid to the brain and spinal cord. It is the most common malignant brain tumor in children, accounting for about 20% of all pediatric brain tumors. Medulloblastoma occurs in the cerebellum, the part of the brain that controls balance, coordination, and other complex motor functions. Specifically, the tumor is in the posterior fossa, the lower rear part of the brain. Medulloblastoma is also known as a cerebellar primitive neuroectodermal tumor (PNET). About 350 cases are diagnosed in the U.S. each year, and more than 70% of patients are under the age of 10. While it may feel like an overwhelming diagnosis, medulloblastoma typically responds well to treatment.

Causes of a medulloblastoma

Medulloblastoma tumors occur when cells develop poorly early in a child’s life. Although the exact cause is unknown, researchers have identified genetic changes that may play a role in the development and growth of this type of tumor. Medulloblastoma is associated with certain inherited conditions, including Li-Fraumeni syndrome, Gorlin syndrome, and Turcot syndrome.

Signs of a pediatric medulloblastoma

Some children may not have symptoms, but the pressure on the brain may cause drowsiness, headaches, and nausea. A child may have coordination problems, difficulty with muscle control, and changes in appetite. Other symptoms may include hearing and vision problems. A patient may tilt their head to one side. If the tumor has spread to the spinal cord, a child might experience back pain, trouble walking, and problems controlling bladder and bowel functions.

Diagnosis

When a brain tumor is suspected after a physical and neurological exam, the doctor usually schedules an MRI or a CT scan of the brain. If a tumor is discovered in the posterior fossa of the brain, an MRI of the brain and spine will be ordered to determine the anatomical features of the tumor and assess whether it has spread. The next step will be surgical resection of the tumor. The tumor will be tested after surgery to confirm the diagnosis.

   

Treatment

Surgery to remove as much of the tumor as possible is the first line of treatment for medulloblastoma. Traditional surgical treatment requires a craniotomy, during which the neurosurgeon will remove a portion of the skull to access the tumor. The neurosurgeon will use the patient’s own bone to close the opening. If the tumor is completely removed, reduced-dose radiation will likely be administered. If some of the tumor remains, a higher-dose chemotherapy will be ordered. When possible, proton therapy may be used to target the tumor while avoiding healthy tissue.

In some cases, the neurosurgeon can place a ventriculoperitoneal shunt to control increased intracranial pressure. The shunt is a plastic tube that moves the fluid creating pressure inside the brain to the abdomen, where the body absorbs it. With treatment, most children with average-risk medulloblastoma are cancer free five years after diagnosis.

Clinical Trials

In addition, a clinical trial, called Infusion of Panobinostat (MTX110) into the Fourth Ventricle or Tumor Resection Cavity in Children and Adults with Recurrent Medulloblastoma: A Pilot Study, is available.

According to the American Cancer Society, about 500 children are diagnosed every year with medulloblastoma, the most common malignant brain tumor in children. Current treatments expose children to considerable toxicity, and when tumors reoccur despite treatment, survival rates are low. Sandberg is hopeful that a novel clinical trial of MTX110, a new formulation of soluble panobinostat from Midatech Pharma, will help patients overcome the devastating disease.

The clinical trial follows a successful study he led in an animal model, which demonstrated that MTX110 can be safely infused in the fourth ventricle and can achieve drug levels dramatically higher than intravenous or oral administration of the same drug. The team at McGovern Medical School at UTHealth Houston found no neurological deficits after fourth-ventricle infusions in the preclinical study.

The pilot study, which has been approved by the U.S. Food and Drug Administration, will enroll five patients with recurrent medulloblastoma at Children’s Memorial Hermann Hospital.

For more information, visit clinicaltrials.gov/ct2/show/NCT04315064

For more information about enrolling, contact Banging Yu, MD, PhD, at bangning.yu@uth.tmc.edu or call (713) 500-7363.

What You Can Expect at UTHealth Neurosciences

Our dedicated team uses advanced technology to accurately diagnose and treat neurological diseases and conditions impacting babies and children. We work in multidisciplinary teams of specialists and pediatric neurosurgeons who share insights, leading to better treatment decision-making and outcomes, as well as lower costs and time savings. Throughout treatment, we will work closely with the doctor who referred your family to ensure a smooth transition back to your child’s regular care. While your family is with us, they will receive expert care, excellent communication, and genuine compassion.


Contact Us

At UTHealth Neurosciences, we offer patients access to specialized neurological care at clinics across the greater Houston area. To ask us a question, schedule an appointment, or learn more about us, please call (713) 486-8000, or click below to send us a message. In the event of an emergency, call 911 or go to the nearest Emergency Room.