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Two Children’s Minnesota neuro-oncologists join the nation’s top investigators in medulloblastoma clinical trial

An exciting phase two clinical trial led by St. Jude Children’s Research Hospital neuro-oncologists Giles Robinson, MD, and Aditi Bagchi, MD, PhD, recently launched at Children’s Minnesota. Anne Bendel, MD, director of the pediatric neuro-oncology program, and Maggie Skrypek, MD, pediatric neuro-oncologist at Children’s Minnesota, are investigators in the study and will play key roles in managing the trial, completing the study, and preparing the results for publication.

Anne Bendel, MD, director of the pediatric neuro-oncology program
Anne Bendel, MD, director of the pediatric neuro-oncology program at Children's Minnesota
Maggie Skrypek, MD, pediatric neuro-oncologist at Children’s Minnesota
Maggie Skrypek, MD, pediatric neuro-oncologist at Children’s Minnesota

New research through the phase two trial SJiMB21 (NCT05535166) has introduced a new understanding of the molecular underpinnings of medulloblastoma, allowing physicians to predict which group of patients fare well with less intensive treatment regimens. Infants with low molecular risk will be offered low-intensity therapy to protect them from the long-term side effects of cancer-directed therapy and maintain a very good survival rate; the most intensive therapy will be reserved for a small percentage of patients who exhibit the highest molecular risk and need it for the cure.  

About SJiMB21

The study uses newly acquired knowledge about the disease to test whether treating patients according to their molecular groups and sub-groups will improve outcomes. A new tool for diagnosis, called “Methylation,” will detect marks on the patient’s DNA to determine their molecular groups and subgroups. Treatment will be guided according to these findings.   

Molecular profiles help limit cytotoxic exposure

Medulloblastoma is best understood not as a single disease but as one that can be divided into four distinct molecular groups with unique dependencies and outcomes: WNT, SHH, G3, and G4. Infants and young children primarily belong to SHH and G3/G4.

More than two-thirds of infants and young children with medulloblastoma belong to the SHH group. SHH medulloblastoma infants can be further divided into two molecular subgroups: SHH-1 and SHH-2. These two molecular subgroups respond differently to therapy.

On SJiMB21, infants with SHH-2 medulloblastoma will be treated with low-intensity systemic chemotherapy. Higher doses of chemotherapy will be delivered to those with SHH-1 medulloblastoma. This kind of granular tailoring of therapy is unique to SJiMB21 and helps limit exposure to unnecessary cytotoxic therapies.

Results from previous studies have shown that infants and young children with G3 (20%) and G4 (5%) medulloblastoma cannot be cured without radiation. On SJiMB21, physicians will give kids in early childhood a chance to develop in areas such as vocabulary, motor skills, and brain function until the child reaches the age of 3. They will then use craniospinal irradiation and adapt the dosage according to the patient’s molecular risk features and response to chemotherapy.

SJiMB21 embeds supportive care measures in its protocol, helping physicians detect possible late effects early on. Pediatric practitioners monitor children’s thinking, learning, social, emotional, and physical function in the trial. They evaluate the need for interventions and promote the healthy development of these children. Such a trial, and those that follow in its path, will maximize survival, and reduce the long-term toxicities of treatment.  

SJiMB21 at Children’s Minnesota

The small size of the infant medulloblastoma population pool makes it difficult to achieve a rapid advancement of cures. To accelerate progress on brain tumor research and care, the St. Jude Brain Tumor Program has chosen to collaborate with premier institutions in the United States, Australia, and New Zealand, including Children’s Minnesota.  

Patient referrals

The SJiMB21 trial is open and is currently recruiting participants. To refer a patient, contact Children’s Minnesota Physician Access at 866-755-2121.

Eligibility criteria: 

  • All patients with newly diagnosed medulloblastoma between birth and 3 years old.  
  •  A select group of low-risk patients with newly diagnosed medulloblastoma between 3 and 5 years old. 
  • Newly diagnosed medulloblastoma. 
  • Has received no previous radiotherapy, chemotherapy, or other brain tumor-directed therapy other than corticosteroid therapy and surgery.

Exclusion criteria:  

  • Central nervous system (CNS) embryonal tumor other than medulloblastoma. For example, patients with atypical teratoid/rhabdoid tumor, PNET, pineoblastoma, ependymoma, and ETMR are excluded.  
  • Prior treatment for medulloblastoma except surgery and corticosteroids.  
  • Actively receiving any other investigational agents directed to the tumor/cancer.  
  • Other clinically significant medical disorders, such as serious infections or significant cardiac, pulmonary, hepatic, psychiatric, or other organ dysfunction that could compromise a patient’s ability to tolerate protocol therapy or would interfere with the study procedures or results.  

About Children’s Minnesota Research Institute

The Children’s Minnesota Research Institute is dedicated to pushing the boundaries of pediatric research. Our kid experts are constantly exploring new avenues for diagnosing and treating childhood illnesses. Visit our Research Institute for more information.

About the neurooncology program at Children’s Minnesota

Children’s Minnesota is an established leader in pediatric hematology-oncology. Our clinicians provide a portal of access to experimental therapies for infants, children and young adults and have achieved international recognition for their leadership in pediatric medicine.  

The Children’s Minnesota central nervous system (CNS) tumor program is one of the largest pediatric programs in the region. We provide access to the most current national and international treatment protocols using chemotherapy, radiation, and innovative therapeutics. Our multidisciplinary team of kid experts includes pediatric specialists from a wide variety of disciplines, including neurooncology, neurosurgery, neurology, neuroradiology, endocrinology, radiation oncology, anesthesia, pain management, psychology, pharmacy, physical therapy, nutrition, and others. Learn more about the CNS program here. 

Alexandra Rothstein