Treating atypical teratoid/rhabdoid tumour of the central nervous system requires a combination of aggressive medical approaches tailored to each young patient’s specific condition. This rare and fast-growing brain cancer demands immediate action and careful coordination between multiple medical specialists to give children the best possible chance.

Fighting a Rare and Aggressive Brain Cancer

When a child receives a diagnosis of atypical teratoid/rhabdoid tumour, commonly known as ATRT, families face one of the most challenging cancers affecting the brain and spinal cord. The main goal of treatment is to remove as much of the tumour as safely possible, stop the cancer cells from growing and spreading, and help the child maintain the best quality of life during and after therapy. Because ATRT grows very quickly and can spread through the fluid that surrounds the brain and spinal cord, doctors must act swiftly once the diagnosis is confirmed.^[1][2]

Treatment decisions depend heavily on several factors unique to each child. The age of the patient plays a crucial role, as younger children—especially those under three years old—face additional challenges because their developing brains are more sensitive to certain treatments like radiation. The location of the tumour within the brain or spinal cord matters significantly, as tumours in certain areas may be harder to reach surgically or may affect critical functions like breathing, movement, or vision. Whether the cancer has spread to other parts of the central nervous system also shapes the treatment plan, as metastatic disease (cancer that has spread) requires more intensive therapy.^[2][7]

The treatment journey typically involves multiple approaches used together rather than relying on a single method. Medical teams create personalized plans that may include surgery, powerful anti-cancer medications, radiation in certain cases, and ongoing monitoring to watch for any signs that the cancer might return. Families work closely with pediatric oncologists, neurosurgeons, radiation specialists, and other healthcare professionals who understand the unique needs of children with this rare cancer.^[6][10]

Standard Treatment Approaches

The first and most important step in treating ATRT is surgical removal of the tumour. A specialized brain surgeon, called a neurosurgeon, performs an operation to take out as much of the cancer as possible without damaging surrounding healthy brain tissue. Complete removal is the goal, but this is not always achievable depending on where the tumour has grown. Tumours located near critical brain structures that control vital functions like breathing or heart rate may be impossible to remove entirely without causing serious harm. Even when complete removal seems impossible, surgeons still attempt to remove as much tumour tissue as they can, because the amount of cancer left behind directly affects how well other treatments will work and the child’s overall chances of survival.^[6][10]

After surgery, intensive chemotherapy becomes the next crucial treatment phase. Chemotherapy refers to powerful medications designed to kill rapidly dividing cancer cells. Since ATRT cells grow and divide very quickly, these drugs target this characteristic. However, other fast-growing cells in the body, such as those in hair follicles, the digestive system, and blood-forming tissues, can also be affected, which explains many of the side effects children experience during treatment.^[6][7]

The chemotherapy regimen for ATRT typically involves multiple different drugs used in combination. Medical teams have found that using several medications together, each working in a different way to attack cancer cells, produces better results than using just one drug. These treatment protocols require children to receive chemotherapy in cycles—periods of treatment followed by rest periods that allow the body to recover. The entire course of chemotherapy can last many months, with children often needing to stay in the hospital during intensive treatment phases.^[10][12]

Radiation therapy uses high-energy beams to destroy cancer cells and is often part of the treatment plan for ATRT. However, its use in very young children remains controversial and carefully considered. Radiation can damage the developing brain, potentially causing learning disabilities, growth problems, hormone imbalances, and other long-term effects. For this reason, doctors try to avoid or delay radiation in children younger than three years old whenever possible. When the cancer has spread or cannot be completely removed by surgery, and the child is old enough, radiation therapy may be directed at the entire brain and spinal cord or focused on specific areas where tumour remains.^[7][10]

The decision to use radiation involves weighing the immediate need to control an aggressive cancer against the long-term effects on a developing child. Some treatment protocols use very intensive chemotherapy in the youngest children to try to avoid or delay radiation, while accepting that chemotherapy itself has significant side effects. These difficult decisions require extensive discussions between medical teams and families, considering the individual child’s situation and the specific characteristics of their tumour.^[10][12]

Treatment for ATRT is not standardized across all hospitals and may vary based on available clinical trials and institutional experience. However, the general approach involves aggressive surgical removal followed by intensive chemotherapy, with radiation added when appropriate based on age and disease extent. The entire treatment course typically lasts many months to over a year, requiring tremendous resilience from both the child and their family. Even after completing initial treatment, children need regular follow-up with brain imaging and physical examinations to watch for any signs that the cancer might return.^[7][12]

Innovative Approaches in Clinical Trials

Because standard treatments for ATRT still leave many children facing poor outcomes, researchers are actively testing new therapies in clinical trials—carefully designed studies that evaluate experimental treatments. These trials offer hope for better outcomes by exploring completely new ways to attack ATRT cells while potentially causing fewer long-term side effects than current approaches. Families may want to discuss with their medical team whether their child might be eligible to participate in any ongoing trials.^[12]

Understanding what causes ATRT at the molecular level has opened doors to developing targeted therapies. Scientists discovered that most ATRT tumours result from mutations in genes called SMARCB1 (also known as INI1) or SMARCA4. These genes normally produce proteins that help control when cells should grow and divide. When these genes are damaged, cells lose this control and form cancers. Importantly, researchers have identified three distinct genetic subtypes of ATRT—called AT/RT-TYR, AT/RT-SHH, and AT/RT-MYC—which may respond differently to various treatments. This knowledge has led scientists to search for drugs that specifically target the molecular problems caused by these genetic changes.^[2][12]

One promising area of research involves drugs that target epigenetic changes in ATRT cells. Epigenetics refers to chemical modifications that affect how genes are turned on or off without changing the DNA sequence itself. When SMARCB1 is lost in ATRT, it causes widespread epigenetic disruption that allows cancer cells to grow uncontrollably. Researchers are testing medications called EZH2 inhibitors and histone deacetylase (HDAC) inhibitors, which work by correcting some of these epigenetic abnormalities. These drugs essentially try to reset the abnormal gene expression patterns in ATRT cells, potentially slowing or stopping tumour growth.^[12]

Another research direction explores inhibitors of signaling pathways that ATRT cells use to survive and grow. The mTOR pathway and MEK pathway are chemical signaling systems inside cells that control growth and division. Some pre-clinical studies—research done in laboratory models before testing in patients—have shown that blocking these pathways with specific drugs can slow ATRT growth. Scientists are working to determine which ATRT subtypes might benefit most from these targeted approaches and how to best combine them with standard chemotherapy.^[12]

Some clinical trials are testing whether adjusting treatment based on how each individual tumour responds can improve outcomes. This approach, called adaptive therapy, involves regularly checking how much tumour remains during treatment and modifying the intensity or type of therapy accordingly. If a tumour is shrinking quickly, treatment might be adjusted to reduce side effects while maintaining effectiveness. If the cancer is not responding well, therapies can be intensified or changed earlier rather than waiting until the standard treatment course is completed.^[12]

Clinical trials exploring ATRT treatments are conducted at specialized pediatric cancer centers, primarily in the United States, Europe, and other developed regions where research infrastructure exists. These trials typically proceed through phases. Phase I trials primarily test whether a new treatment is safe and work to find the right dose in children. Phase II trials evaluate whether the treatment shows evidence of working against the cancer in a small group of patients. Phase III trials compare a new treatment against the current standard approach in larger groups to determine if the new therapy is truly better. Children may be eligible for different trials depending on whether they are newly diagnosed or have cancer that has returned after initial treatment.^[12]

Early results from some of these novel approaches show promise, though it is important to understand that many experimental treatments ultimately do not prove better than existing therapies. Some targeted drug trials have demonstrated that ATRT tumours can respond to these medications, with some children experiencing tumour shrinkage or stabilization. However, researchers are still learning which combinations of treatments work best, which children are most likely to benefit, and how to manage the side effects of these new drugs alongside standard chemotherapy and radiation.^[12]

Most common treatment methods

• Surgery
  • Neurosurgeons attempt to remove as much of the tumour as safely possible during an operation
  • Complete removal is the goal but may not be achievable depending on tumour location near critical brain structures
  • The extent of surgical removal affects how well other treatments work and overall survival chances
  • Biopsy during surgery confirms the ATRT diagnosis through tissue examination
• Chemotherapy
  • Intensive multi-drug regimens delivered in cycles over many months
  • Combination of different medications that each work differently to kill cancer cells
  • May cause significant side effects including infections, nausea, hair loss, and effects on organs
  • Treatment intensity and duration depend on the child’s age and how far the cancer has spread
• Radiation therapy
  • High-energy beams directed at tumour sites to destroy remaining cancer cells
  • Used cautiously in very young children due to potential effects on brain development
  • May be applied to specific tumour areas or the entire brain and spinal cord depending on disease spread
  • Often delayed or avoided in children under three years old when possible
• Targeted therapy (in clinical trials)
  • Epigenetic modifiers like EZH2 inhibitors and HDAC inhibitors that correct gene expression abnormalities
  • Drugs blocking growth signaling pathways such as mTOR inhibitors and MEK inhibitors
  • Approaches designed to specifically target the molecular problems caused by SMARCB1 or SMARCA4 gene loss
  • Currently being tested in research studies to determine effectiveness and safety
• Supportive care
  • Management of treatment side effects and complications throughout therapy
  • Regular monitoring with brain imaging and laboratory tests to assess treatment response
  • Rehabilitation services to help with physical, cognitive, and developmental challenges
  • Long-term follow-up to watch for cancer recurrence and late treatment effects