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Standard of Care for Brain Cancer Treatment

Standard of care refers to the treatments and approaches currently considered the most effective and widely accepted for managing a specific type and stage of brain cancer.

child in hospital

What is Standard of Care?

Standard of care represents the minimum level of care that would be provided based on current clinical guidelines, research evidence, and expert consensus.

The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology lay out the recognised standards for cancer treatment. The guidelines are developed and updated by 61 individual panels comprising more than 1,700 clinicians and oncology researchers from the 32 NCCN member institutions.

These guidelines are used not only in the United States but have been translated into multiple languages and are considered the standard of care in many countries around the globe, including Australia. These guidelines provide evidence-based, consensus-driven treatment recommendations to ensure that all patients receive the preventive, diagnostic, therapeutic, and supportive services that are needed to increase the chance of a positive outcome.

Adult Brain Cancers

Glioblastoma (GBM)

Glioblastoma (GBM) is the most common malignant primary brain tumour in adults, accounting for more than half of all malignant brain tumours in Australia.

Current Standard of Care for Glioblastoma

Since 2005, the standard of care has followed the Stupp protocol, which typically includes surgery to remove the tumour, then radiotherapy combined with and followed by chemotherapy using temozolomide. This approach has been shown to modestly improve median overall survival to approximately 14.6 months compared to radiotherapy alone.

Despite decades of research, this standard has remained largely unchanged. While some post-Stupp era studies suggest incremental gains in survival, these are likely due to broader systemic improvements rather than new therapeutic breakthroughs.

Progress Beyond the Current Standard

Current research is exploring avenues such as targeted therapies, immunotherapies, and novel drug delivery strategies. Early-phase trials are investigating combinations of chemoradiation with molecularly guided agents, aiming to overcome resistance mechanisms and improve survival outcomes. However, no new treatment has yet supplanted the Stupp protocol as standard of care.

Low-Grade Gliomas (LGG)

Low-grade gliomas (LGGs) are a heterogeneous group of slow-growing primary brain tumours with unpredictable behaviour, especially at recurrence.

Current Standard of Care for Low-Grade Gliomas

Surgery is the key first step, with outcomes improving when more of the tumour can be safely removed. In low-risk grade 2 cases (typically patients aged 40 or younger), when smaller tumours are fully removed, regular MRI monitoring may be an appropriate way to monitor for any changes.

For older patients or those with residual disease, adjuvant radiotherapy and chemotherapy are recommended. Chemotherapy, particularly with PCV or temozolomide, further improves outcomes when combined with radiotherapy, as shown in RTOG 9802 (Buckner et al. 2016).

Emerging Therapies Under Investigation

Management approaches are also evolving with the introduction of novel therapies, such as the IDH inhibitor vorasidenib. Early findings suggest a cautious but growing integration of vorasidenib into clinical care for patients with low-grade glioma. This reflects a shift toward treatment that target specific tumour changes.

Paediatric Brain Cancers

DIPG/DMG

Diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), is the leading cause of brain tumour-related death in children.
In Australia, 20–30 children are diagnosed annually, with a median survival of less than 12 months.

Current Standard of Care for DIPG/DMG

The current standard of care is focal radiotherapy, which may delay progression and alleviate symptoms but is not curative.

Tumours nearly always recur, and surgery is usually deemed unsafe due to the tumour’s location in the brainstem. To date, no chemotherapy, immunotherapy, or targeted drug has been shown to increase survival.

Treatment is further complicated by limited drug delivery across the blood-brain barrier and intrinsic tumour resistance mechanisms.

The Future of Treatment for DIPG/DMG

Early-phase clinical trials such as PNOC-022, evaluating agents like ONC201 and paxalisib, reflect cautious steps toward incorporating targeted therapies into clinical care in Australia.

Medulloblastoma

Medulloblastoma is a highly aggressive malignant tumour of the cerebellum and the most common malignant brain tumour in children. Although it can also occur in adults, medulloblastoma is a brain cancer that occurs most commonly in children under the age of 10 (in 70-80% of cases), with 20-30 Australian children diagnosed each year.

Current Standard of Care for Medulloblastoma

Conventional treatment, for both standard and high-risk patients, involves a combination of maximal resection surgery, craniospinal irradiation (CSI), and cytotoxic chemotherapy (CT). This combination achieves long-term OS in 60–80% of patients but often at the expense of devastating long-term toxicities.

The Future of Treatment for Medulloblastoma

A study compiled by Prof. Bryan Day at QIMR Berghofer’s Sid Faithfull Brain Cancer Laboratory demonstrated that the novel drug CT-179, when used in combination with standard radiation therapy, can cross the blood-brain barrier and penetrate the tumour. It prolonged survival in a range of preclinical medulloblastoma models, delayed recurrence of the disease, and increased the effectiveness of radiotherapy.

Brain Cancer Standard of Care FAQs

Brain cancer treatment is not the same for every person. A treatment plan can depend on the type of brain tumour, whether it is benign or malignant, where it is located in the central nervous system, how quickly it is growing, and whether it is affecting surrounding brain tissue.

Doctors may also consider the person’s age, symptoms, overall health, previous treatment, scan results, and pathological diagnosis. This is why two people with brain cancer may be given different treatment options, even if their diagnoses sound similar.

Surgery may be considered when the tumour can be accessed safely, and removing part or all of it may help with diagnosis, symptoms, or further treatment. For some brain tumours, maximal surgical resection or complete resection may be part of standard care.

In other cases, surgery may not be safe because of where the tumour is growing. Surgeons need to balance tumour removal with the need to protect healthy brain tissue and important brain functions. Tools such as magnetic resonance imaging, functional MRI, advanced surgical planning, or awake craniotomy may be used in some cases to help guide treatment decisions.

Radiation therapy uses carefully targeted radiation to damage or destroy cancer cells. External beam radiation therapy is one of the common forms used to treat brain tumours.

The aim is to treat the tumour or the area where tumour cells may remain, while limiting radiation exposure to healthy cells and surrounding brain tissue as much as possible. Radiation therapy side effects can vary depending on the area treated, the dose, the person’s age, and their overall health.

If patients experience tumour progression after treatment, the medical team may review scan results, symptoms, previous therapies, and the person’s quality of life before recommending the next step. Further treatment may include more radiation treatment, chemotherapy medicines, targeted therapy drugs, surgery, supportive care, or a clinical trial, depending on the tumour type and the individual situation.

For recurrent or progressive GBM, treatment decisions can be especially complex. Some therapies may aim to slow tumour growth, relieve symptoms, manage intracranial pressure, or support quality of life.

Targeted therapy and immunotherapy are being studied across several central nervous system cancers, but they are not standard care for every type of brain cancer. Targeted therapy drugs are designed to act on specific changes in cancer cells, while immunotherapy aims to help the immune system recognise or attack cancer cells.

Some approaches, including monoclonal antibody therapies, may be used or investigated in specific situations. Other treatments are still being tested through clinical trials, including phase II trials. Your medical oncology team can explain whether any of these options may be relevant to your diagnosis.

Clinical trials help researchers test new ways to diagnose, treat, or manage brain cancer. They may study new chemotherapy drugs, targeted therapy drugs, radiation treatment approaches, drug combinations, imaging methods, or ways to improve quality of life and manage symptoms.

A clinical trial is not automatically better than standard treatment, and it may not be suitable for everyone. Some trials are designed for newly diagnosed patients, while others focus on recurrent or progressive tumours. Your treating team can help you understand the possible risks, benefits, and eligibility criteria before you decide whether to take part.

Disclaimer: : All Cure Brain Cancer Foundation website content is created and published online for informational purposes only. It is not intended to be a substitute for professional medical advice, diagnosis or treatment. You should seek your own medical advice from your doctor or other qualified health professionals.

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