Brain Tumors Decoded: A Comprehensive Guide for Patients and Caregivers – Part II

The Standard of Care for Glioblastoma: Challenges and Opportunities

GBM is the most common and aggressive malignant primary brain tumor. The standard of care, which includes surgery followed by 6 weeks of daily radiation therapy and chemotherapy with temozolomide, has remained unchanged for many years.^9,10

Patients with unmethylated GBM often experience limited benefit from standard treatment. For these individuals, molecular profiling of the tumor is critical, as it may reveal genetic or molecular targets that can inform additional treatment options. Molecular profiling is essential for every GBM patient, as it can guide discussions about clinical trials and emerging therapies that may offer additional hope.

Clinical trials are especially important for patients with unmethylated GBM, as they provide access to cutting-edge treatments that could improve outcomes. The neuro-oncology team plays a vital role in helping patients explore these options and weigh their potential benefits.

Understanding the Blood-Brain Barrier Challenge

The blood-brain barrier is a selective, protective interface that regulates the passage of substances between the bloodstream and the central nervous system.¹¹ While it shields the brain from harmful agents, it also poses a significant obstacle in the treatment of brain tumors. Its restrictive nature limits the delivery of therapeutic agents to malignant brain tissues, complicating effective treatment.

Recent research has explored various strategies to overcome this challenge, including the use of nanoparticles, focused ultrasound techniques that temporarily disrupt the barrier, and the development of drugs capable of penetrating the blood-brain barrier. Each approach aims to enhance the delivery of therapeutic agents to brain tumors, thereby improving treatment efficacy.

Despite these advancements, the blood-brain barrier remains a formidable hurdle in neuro-oncology. Ongoing research is essential to develop more effective methods for delivering treatments to brain tumors, with the goal of improving patient outcomes.

Clinical Trials: Exploring Advanced Treatment Options

Understanding when participation in a clinical trial becomes available during your brain tumor journey is crucial. Clinical trials are available at specific stages, and being aware of these time points can empower patients to make informed decisions about their current and future treatment options. Key time points for clinical trial eligibility include:

1. Before initial surgery (biopsy/resection): Some trials are designed for patients who have not yet undergone surgery and are available at the time of a new diagnosis. Participating at this stage could provide access to novel treatments that could be administered prior to or during the surgical procedure.
2. Before starting standard treatment (chemotherapy and radiation): After surgery and before beginning the typical chemotherapy and radiation regimen, certain trials are available. These studies often explore new therapies that could be more effective or have fewer side effects than the current proven standard treatments.
3. After standard treatment and before maintenance therapy: During the period after completing the initial rounds of chemotherapy and radiation but before starting any maintenance therapy, there may be trials available that focus on preventing recurrence or managing residual disease.
4. At recurrence, before additional surgery: If the tumor returns, trials are available for patients who have not yet undergone a second surgery. These studies might offer access to cutting-edge treatments aimed at controlling the tumor’s progression or involve new approaches to surgical resection.
5. After surgery for recurrence, before other therapies: Following surgery for a recurrent tumor and prior to initiating other treatments, patients may be eligible for trials designed to assess new therapies that could prevent further recurrence or manage the disease more effectively.

It’s important to note that the availability of clinical trials can vary based on individual circumstances, tumor characteristics, and evolving research. Engaging in open discussions with your healthcare team about clinical trial options at these key time points can help ensure you are aware of all potential treatment avenues.¹²

Download detailed visual representation of time points

Clinical trials offer patients access to investigational therapies that may provide new avenues for treatment (and hope) beyond the standard of care. Understanding when and how to consider clinical trial participation is crucial for making informed decisions about your healthcare.¹³

Eligibility Criteria

Eligibility for clinical trials is determined by specific criteria, including:

• Disease type and stage: Trials are often tailored to particular types and stages of disease
• Previous treatments: Prior therapies may influence eligibility, as some trials require participants to be treatment naive, while others are open to those with extensive treatment histories
  • – It is important for those with GBM to understand that exposure to Avastin or tumor treating fields like Optune, may exclude their participation from future clinical trials. Connect with a neuro-oncology navigator and your healthcare team to discuss how early treatment decisions can affect treatment options down the road
• Number of recurrences: It is important to note that some trials are open after the first recurrence only, while others are open to second, third, or more

Identifying specific genetic mutations or biomarkers can augment the number of available treatment options through clinical trials or expanded access.

• Molecular and genetic markers: Advancements in molecular profiling have enabled the development of targeted therapies. Identifying specific genetic mutations or biomarkers can augment the number of available treatment options through clinical trials or expanded access
• Overall health status: General health and the presence of other medical conditions are considered to ensure patient safety during the trial. Ask your doctor for your Karnofsky Performance Scale score, as many trials require a score of 70 or above to be eligible. The Karnofsky Performance Scale Index measures a patient’s functional ability, helping classify their level of impairment. It is used to evaluate treatment effectiveness and estimate an individual’s prognosis. In general, a lower Karnofsky score indicates greater impairment and is associated with poorer survival outcomes in serious illnesses.¹⁴

Benefits and Considerations

Participating in a clinical trial can offer several potential benefits:

• Access to innovative therapies: Gain early access to new treatments that are not yet widely available
• Contributing to research: Help advance medical knowledge and potentially benefit future patients
• Enhanced monitoring: Receive close monitoring and additional care from the clinical trial team

However, it’s important to consider:

• Unknown risks and side effects: New treatments may have unforeseen risks or side effects
• Commitment requirements: Participation may involve additional visits, tests, and adherence to specific protocols. There may be impacts to one’s home life and routine, and areas such as financial stability should be assessed prior to committing to a trial’s demands. Financial navigators play a crucial role in helping patients manage these challenges through financial impact assessments, financial resource identification, insurance navigation, and more¹⁵
• Placebo possibility: Some trials include a placebo group, in which the trial’s treatment will be the current standard of care
• Quality versus quantity: It’s imperative that patients consider how trial enrollment may affect their remaining time. Unknown side effects, travel demands, and treatment type have the potential to negatively impact remaining quality of life

Empowering Your Decision

Engaging in open discussions with your healthcare team about clinical trial options is vital. Inquiring about available trials, understanding the potential risks and benefits, and considering how participation aligns with your treatment goals are important steps. Remember, participation in a clinical trial is a personal decision that should be made based on thorough understanding and alignment with your healthcare objectives.

Click Here for Part III: Understanding the Importance of Molecular Profiling in Brain Tumors